Sphingomyelin LipidosisPhase 2NCT02004704
A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
A Phase 2 study testing GZ402665 in people with Sphingomyelin Lipidosis, run by Genzyme, a Sanofi Company at 9 sites in 7 countries.
Trial summary
The primary objective of this study was to obtain data regarding the safety of olipudase alfa in participants with acid sphingomyelinase deficiency (ASMD) who were exposed to long term treatment with olipudase alfa. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Sphingomyelin Lipidosis
- What is being tested?
- GZ402665
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 9 sitesin 7 countries
- Study dates
- 4 Dec 2013to 6 Sep 2023
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- The participant completed the treatment period of a previous study of olipudase alfa with an acceptable safety profile in the opinion of the investigator and sponsor.
- The participant and/or the participant's parent(s)/legal guardian(s) was willing and able to provide signed written informed consent.
- The participant who is female and of childbearing potential must have had a negative urine pregnancy test for beta human chorionic gonadotropin (β HCG).
- Female participants of childbearing potential and sexually mature male participants must have been willing to practice true abstinence in line with their preferred and usual lifestyle or use 2 acceptable effective methods of contraception up to 15 days following their last dose of study drug.
Who cannot join
- The participant had any new condition or worsening of an existing condition which in the opinion of the investigator would make the participant unsuitable for enrollment, or could interfere with the participation or completion the study.
- The participant, in the opinion of the investigator, was unable to adhere to the requirements of the study.
- The participant was unwilling or unable to abstain from the use of alcohol for 1 day prior to and 3 days after each olipudase alfa infusion for the duration of the treatment period.
- The participant was unwilling or unable to avoid, for 10 days before and 3 days after liver biopsies, medications or herbal supplements that are potentially hepatotoxic (only participants who previously participated in the DFI13412 study).
- The participant required medication(s) that may decrease olipudase alfa activity (eg, fluoxetine, chlorpromazine; tricyclic antidepressants [eg, imipramine, desipramine]).
- The above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
Trial locations and contacts
9 sites in 7 countries.
9 sites in 7 countries
Italy2 sites
Investigational Site Number 380002
Sassari, 07100
Investigational Site Number 380001
Udine, 33100
United Kingdom2 sites
Investigational Site Number 826001
London, WC1N 3JZ
Investigational Site Number 826002
Manchester, M13 9WL
United States1 site
Investigational Site Number 840001
New York, New York, 10029-6574
About this study
Official title: A Long-Term Study to Assess the Ongoing Safety and Efficacy of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
The primary objective of this study was to obtain data regarding the safety of olipudase alfa in participants with acid sphingomyelinase deficiency (ASMD) who were exposed to long term treatment with olipudase alfa.
The secondary objectives of this study were to obtain data regarding the efficacy of olipudase alfa and to characterize olipudase alfa pharmacodynamics (PD) and pharmacokinetics (PK) following long-term administration.
Read the detailed description
LTS13632 is a multicenter, nonrandomized, open-label, long-term extension study of participants with ASMD who have previously participated in a study of olipudase alfa. (DFI13803 for pediatric participants and DFI13412 for adult participants).
The maximum study duration per participant was 9 years or until olipudase alfa becomes commercially accessible. Notwithstanding the above, every pediatric participant were treated in LTS13632 study for at least 3 years to comply with the requirements agreed in the olipudase alfa Pediatric Investigational Plan.
- Conditions
- Sphingomyelin Lipidosis
- Being tested
- GZ402665
- Medical subject headings
- Niemann-Pick Disease, Type A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GZ402665Drug
Also called Olipudase alfa
Pharmaceutical form: Powder for concentrate for solution for infusion
Route of administration: intravenous infusion
| Study group | Role | What they receive |
|---|---|---|
| GZ402665 | Experimental | GZ402665 administered intravenously once every 2 weeks at the dose each participant was receiving at the end of their previous olipudase alfa study, for 9 years or until olipudase alfa becomes commercially accessible, whichever comes first, unless the participant decides to enter another olipudase alfa clinical trial within the 9-year period prior to when olipudase alfa is commercially accessible. Receives: Drug: GZ402665 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Treatment Emergent Serious Adverse Events (TESAEs) and Adverse Events of Special Interest (AESIs) Except Infusion-Associated Reactions (IARs)
An AE: Any untoward medical occurrence in participant or clinical investigation participant administered with pharmaceutical product, which did not necessarily have to have causal relationship with study treatment. SAEs: Any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalization/prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was congenital anomaly/birth defect, was medically important event. TEAEs: AEs that started during on-treatment period-either in this study or in original study which were ongoing at the time the participant signed the written informed consent. An AESI: AE (serious or nonserious) of scientific and medical concern specific to sponsor's product or program, for which ongoing monitoring and rapid communication by investigator to sponsor was appropriate.
Measured: From the signature of informed consent up to 9 years or until olipudase alfa was commercially accessible, whichever came first
Number of Participants With IARs
Protocol-defined IARs were AEs that occurred during the infusion or within up to 24 hours after the start of infusion and were considered as related or possibly related to the study treatment by the investigator or the sponsor. Events occurring greater than or equal to (>=) 24 hours after the start of an infusion might have been judged an IAR at the discretion of the investigator or sponsor. Algorithm-defined IARs were all AEs that started between the start of infusion and the end of infusion plus 24 hours, irrespective of the perceived relation with study treatment.
Measured: From the signature of informed consent up to 9 years or until olipudase alfa was commercially accessible, whichever came first
For Adults: Number of Participants With Abnormality in Complete Physical Examinations Including Abbreviated Physical Exam
A complete physical examination included assessment of the participant's general appearance, general neurological status, skin, head, eyes, ears, nose, throat, lymph nodes, heart, lungs, abdomen, and extremities/joints. Shift from Baseline was monitored. An abbreviated physical exam (general appearance only) was only performed pre- and post-infusion for those who did not do complete exam.
Measured: Baseline (Day 1 of original study), Month 78
For Pediatrics: Number of Participants With Abnormality in Complete Physical Examinations Including Abbreviated Physical Exam
A complete physical examination included assessment of the participant's general appearance, skin, head, eyes, ears, nose, throat, lymph nodes, heart, lungs, abdomen, and extremities/joints. Shift from Baseline was monitored. An abbreviated physical exam (general appearance only) was only performed pre- and post-infusion for those who did not do complete exam.
Measured: Baseline (Day 1 of original study), Month 84
For Adults: Number of Participants With Abnormality in Extended Neurological Examinations
Extended neurological examination for adults included examination of mental status. Shift from Baseline was monitored.
Measured: Baseline (Day 1 of original study), Month 78
For Pediatrics: Number of Participants With Abnormality in Extended Neurological Examinations
The neurological examination in pediatric participants included examination of mental status, cranial nerve examination, motor examination: tone, reflexes examination, sensory examination, coordination, gait and coordination, and strength examination. Shift from Baseline was monitored.
Measured: Baseline (Day 1 of original study), Month 84
Number of Participants With Potentially Clinically Significant Abnormalities (PCSA) in Vital Signs
PCSA: Heart Rate: High: >=120 beats per minute (bpm) (adults [ad], adolescents [ado], children [chi]), >=140 bpm (early chi [ec]), >=175 bpm (infants[inf]) \& increase from baseline (IFB)>=20 bpm for all age ranges (AAR), HR Low: <=50 bpm (ad, ado, chi), <=75 bpm (ec), <=80 bpm (inf) \& decrease from baseline (DFB) >=20 bpm for AAR. Systolic blood pressure: High: >= 160 millimeters of mercury (mmHg) (ad); >=119 mmHg (ado), >=108 mmHg (chi), >=101 mmHg (ec),>=98 mmHg (inf) \& IFB >=20 mmHg for AAR. SBP Low: <=95 mmHg (ad), <=90 mmHg (ado), <= 80mm Hg (chi), <=70 mmHg (ec), <=70 mmHg (inf) \& DFB >=20 mmHg for AAR. Diastolic blood pressure: High:>110 mmHg (ad), >=78 mmHg (ado), >=72 mmHg (chi), >=59 mmHg (ec), >=54 mmHg (inf) and IFB >=10 mmHg for AAR. DBP Low:<=45 mmHg (ad), <=54 mmHg (ado), <=48 mmHg (chi), <=34mmHg (ec and inf) \& DFB>=10 mmHg for AAR.
Measured: Baseline (Day 1 of original study) up to 9 years or until olipudase alfa was commercially accessible, whichever came first
Number of Participants With PCSA in Clinical Chemistry Parameters
PCSA: Creatinine High, category 1: >=150 micromole/L (umol) (adults), >=132 umol/L or 1.5 mg/deciliter (dL) (adolescents), >=90 umol/litre (L) or 1.1 mg/dL (children), >53 umol/L or 0.6 mg/dL (early children and infants); category 2: >=30% IFB (adults). Blood Urea Nitrogen: >=17 millimole (mmol)/L (adults), >=6.4 mmol/L or 18 mg/dL (pediatrics [ped]). Glucose Low<=3.9 mmol/L and <lower limit of normal (adults), <2.7 mmol/L (ped), High: >=11.1 mmol/L (unfasted), >7 mmol/L (fasted) (adults), >=7 mmol/L (fasted after >12 hours of fast); >=10.0 mmol/L (unfasted) (ped).
Measured: Baseline (Day 1 of original study) up to 9 years or until olipudase alfa was commercially accessible, whichever came first
Number of Participants With PCSA in Liver Function Tests
PCSA: Alanine aminotransferase (ALT) >3 x upper limit of normal (ULN). Aspartate aminotransferase (AST) >3 x ULN. Alkaline phosphatase (ALP) > 1.5 x ULN. Total Bilirubin >1.5 x ULN (ad) and >1.3 x ULN (ped). ALT and total bilirubin: ALT > 3 x ULN and total bilirubin > 2 x ULN.
Measured: Baseline (Day 1 of original study) up to 9 years or until olipudase alfa was commercially accessible, whichever came first
Number of Participants With PCSA in Hematology Parameters
PCSA: White blood cells (WBC): Low: <3.0 Giga (G)/L (Non-Black [NB])/<2.0 G/L (Black [B])(ad), <4.5 G/L (ado), <5.0 G/L(chi), <3.0 G/L (ec), <4.0 G/L (inf), High: >=16.0 G/L(ad), >13.5 G/L (ado), >17.0 G/L(chi), >16 G/L (ec), >20 G/L (inf). Hemoglobin-ad: Low-1: <=115 g/dL (Male [M])/<=95 g/dL (Female [F]), 20% DFB for both, High:>=185 g/L (M)/>=165 g/L (F), Low-2: DFB>=20 g/L (ad), <10 g/dL (ado, chi, ec); <9.0 g/dL (inf); 20% DFB for all. Platelets: Low: <100 G/L (AAR) and 20% DFB and High: >=700 G/L (ad and ped).
Measured: Baseline (Day 1 of original study) up to 9 years or until olipudase alfa was commercially accessible, whichever came first
Number of Participants With PCSA in Electrocardiogram (ECG)
PCSA: Heart rate: High:>=120 bpm (ad, ado, chi), >=140 bpm (ec), >=175 bpm (inf) \& IFB>=20 bpm for AAR, Heart rate: Low: <=50 bpm (ad, ado, chi), <=75 bpm (ec), <=80 bpm (inf) \& DFB >=20 bpm for AAR. PR duration: High: >=200 milliseconds (ms) (ad) and IFB>=20 ms, >180 ms (ado), 170 ms (chi), 160 ms (ec), and 140 ms (inf). QT correction-Bazett (QTcB): Borderline absolute (category 1): 431-450 ms (ad and ado M, chi, ec and inf), 451-470 ms (ad and ado F), Prolonged-absolute (category 2): >450 ms (ad and ado M and chi, ec and inf), >470 ms (ad and ado F), Additional-absolute (category 3): >=500 ms (AAR), Borderline increase (category 4): 30-60 ms (AAR), Prolonged increase (category 5): > 60 ms (AAR).
Measured: Baseline (Day 1 of original study) up to 9 years or until olipudase alfa was commercially accessible, whichever came first
For Adults: Percent Change From Baseline in Pre-Infusion Plasma Ceramide at Month 90
Plasma samples were collected to evaluate ceramide levels for safety biomarker analysis. Baseline was defined as the last available non-missing value prior to the first infusion in original study.
Measured: Baseline (Day 1 of original study) and Month 90 (pre-infusion)
For Pediatrics: Percent Change From Baseline in Pre-Infusion Plasma Ceramide at Month 66
Plasma samples were collected to evaluate ceramide levels for safety biomarker analysis. Baseline was defined as the last available non-missing value prior to the first infusion in original study.
Measured: Baseline (Day 1 of original study) and Month 66 (pre-infusion)
Participants From Adult Study DFI13412: Number of Participants With Abnormal Liver Biopsy
Liver biopsy samples were evaluated for sphingomyelin accumulation and liver pathology in the participants who previously participated in the DFI13412 study who were at least 18 years old when they entered in this study. Sphingomyelin accumulation was quantified in liver by computer morphometry of high-resolution light microscopy images. Fibrosis grading was assessed on the Laennec scoring system, which grades the extent of fibrosis on a scale from 0 to 4 (0=none; 1=minimal; 2=mild; 3=moderate; 4=cirrhosis). Higher score indicated more severity.
Measured: Baseline (Day 1 of original study) and Month 36
Participants From Pediatric Study DFI13803: Number of Participants With Abnormalities in Liver Ultrasound Doppler
Liver ultrasound doppler was performed for pediatric participants transitioning from DFI13803 to document hepatic blood flow characteristics, principally portal vein pressure, and blood flow direction. The parameters evaluated were liver dysmorphic findings, liver surface abnormalities, mild ascites and hepatic steatosis. Liver ultrasound Doppler was performed using methods that were compatible with the standard institutional procedures of the investigational site.
Measured: Baseline (Day 1 of original study), Week 2 and Months 12, 18 and 24
Number of Participants With Anti-Drug Antibodies (ADA) Against Olipudase Alfa
Blood samples were collected for the presence of ADAs against olipudase alfa. Treatment-boosted ADA: Positive ADA status positive at baseline (pre-existing ADA) and ADA titer level anytime post-baseline significantly higher than that at baseline. Transient ADA: Treatment-induced ADA detected only at 1 sampling time point post-baseline and treatment-induced ADA detected at 2 or more sampling time points post-baseline, where first and last ADA-positive samples (irrespective of any negative samples in between) separated by period of less than 16 weeks, and participant's last sampling time point was ADA-negative. Persistent ADA response: Treatment-induced ADA detected at 2 or more sampling time points post-baseline, where first and the last ADA-positive on-treatment sample (irrespective of any negative samples in between) separated by at least 16 weeks. Treatment-induced: ADA detected in the last 2 sampling time points, irrespective of the time period in between.
Measured: Baseline (Day 1 of original study) up to 9 years or until olipudase alfa was commercially accessible, whichever came first
Secondary outcomes (11)
Percent Change From Baseline in Spleen and Liver Volumes at Month 102 for Adults and Month 84 for Pediatrics
Spleen and liver volumes were assessed by abdominal magnetic resonance imaging (MRI) collected and read centrally by a third party blinded to participant number and study visit to quantify the degree of splenomegaly and hepatomegaly at specified time points. Spleen/Liver volume in multiples of normal (MN) = Spleen/Liver volume (cubic centimeter [cm^3]) / [2xweight (kg)] where weight was the available value closest to the MRI scan date. Negative value signifies reduction of volume. Baseline was defined as the last available non-missing value prior to the first infusion in original study.
Measured: Baseline (Day 1 of original study), Month 102 for adults and Month 84 for pediatrics
Change From Baseline in Pulmonary Imaging by High Resolution Computed Tomography (HRCT) at Month 102 for Adults and Month 84 for Pediatrics
Pulmonary imaging of chest using HRCT was obtained for qualitative assessment of ground glass appearance, interstitial lung disease, pleural thickening and reticulo-nodular density. Images were collected and read centrally by a third party blinded to participant number and study visit. Lung fields were scored subjectively for degree of diffuse lung disease (infiltrative lung disease) in scale of 0-3 ranging from 0 = No disease, 1 = Mild (affecting 1% to 25% of the lung volume), 2 = Moderate (affecting 26% to 50% of the lung volume), 3 = Severe (affecting 51% to 100% of the lung volume) where higher scores indicated more severity. The score of each participant was averaged over all 4 levels and both sides of lung. Mean score across 4 levels and both lungs = (Mean score across X levels for left lung + Mean score across X levels for right lung)/2. Baseline was defined as the last available non-missing value prior to the first infusion in original study.
Measured: Baseline (Day 1 of original study), Month 102 for adults and Month 84 for pediatrics
Percent Change From Baseline in Percent Predicted Diffusing Capacity of Lungs For Carbon Monoxide (DLco) (Hemoglobin-Adjusted) at Month 78 for Adults and Month 84 for Pediatrics
DLco was used to measure gas exchange across the alveolocapillary membrane. Percent predicted hemoglobin-adjusted DLco was calculated as: 100 x Adjusted DLco/Predicted DLco in unit of mL co/minute (min)/mmHg where, adjusted DLco = Observed DLco (in mL co/min/mmHg) divided by Hemoglobin-adjusted factor. Baseline was defined as the last available non-missing value prior to the first infusion in original study.
Measured: Baseline (Day 1 of original study), Month 78 for adults and Month 84 for pediatrics
Percent Change From Baseline in Platelet Count at Month 90 for Adults and Month 78 for Pediatrics
Blood samples were collected at specified timepoints for the estimation of platelet count as ASMD is known to result in hematologic disorders. Baseline was defined as the average of all available values before the start of first infusion from original study
Measured: Baseline (Day 1 of original study), Month 90 for adults (pre-infusion) and Month 78 for pediatrics (pre-infusion)
Percent Change From Baseline in Low-density Lipoprotein at Month 90 for Adults and Month 66 for Pediatrics
Blood samples were collected at specified timepoints for fasting lipids as ASMD leads to progressive accumulation of lipids. Baseline was defined as the last available non-missing value prior to the first infusion in original study.
Measured: Baseline (Day 1 of original study), Month 90 for adults and Month 66 for pediatrics
Participants From Adult Study DFI13412: Change From Baseline in Brief Fatigue Inventory (BFI) Questionnaire at Month 78
The BFI involves a total of 9 items:3 of which measure BFI-fatigue severity score and 6 of which measure BFI-fatigue interference score. Score for each item measuring severity score is assessed by numerical rating scale (NRS); ranges from 0-10 from 0 (no fatigue) to 10 (worst imaginable fatigue) ultimately leading to overall severity score that ranges from 0-30. Respectively, amount that fatigue interfered with different aspects of participant's life during preceding 24 hours (general activity,mood,walking ability,normal work,relations with other people,and enjoyment of life) is captured as part of interference score.These different aspects are measured in scores ranging from 0:does not interfere and 10:completely interferes,ultimately leading to overall interference score ranging between 0-60. Higher scores in both categories mentioned below indicate worse outcome; mean is presented. Baseline was defined as last available non-missing value prior to first infusion in original study.
Measured: Baseline (Day 1 of original study) and Month 78
Participants From Adult Study DFI13412: Change From Baseline in Brief Pain Inventory-Short Form (BPI-SF) Questionnaire at Month 78
The BPI-SF is a 15-item, validated, self-administered questionnaire designed to measure participant's perceived level of pain. The BPI-SF measured the participant's intensity of pain (sensory dimension), the interference of pain in participant's life (reactive dimension), and asked participant about pain relief, pain quality, and perception of cause of pain. Scoring was based on 4 out of 8 questions in pain severity domain, and 7 questions in pain interference domain, which used numerical rating scale. Pain severity response ranged from 0 = "No pain" to 10 = "Pain as bad as you can imagine". Pain interference response ranged from 0 = "Does not interfere" to 10 = "Completely interferes". Scores were averaged and mean is presented here. Higher scores indicated greater intensity of pain. Baseline was defined as the last available non-missing value prior to the first infusion in original study.
Measured: Baseline (Day 1 of original study) and Month 78
Participants From Adult Study DFI13412: Change From Baseline in Chronic Respiratory Disease Questionnaire Self-Administered Standardized (CRQ-SAS) at Month 78
The CRQ-SAS is a validated, self-administered questionnaire designed to evaluate health related quality of life (HRQoL) in adults with chronic airflow limitation, chronic respiratory disease and cystic fibrosis. It had 20 items and evaluated 4 dimensions of respiratory impairment (dyspnea, emotional function, fatigue and participant's feeling of control over the disease [mastery]). Each domain was measured on 7-point-scale, with 8 reserved for "not done" (1: worst and 7: best). Each domain score was calculated separately. Scores for each question of each domain were added together and divided by number of questions answered in each domain. Only items answered were scored. Mean of average score of completed items is reported. Higher scores indicated better HRQoL in each domain. Baseline was defined as last available non-missing value prior to first infusion in original study.
Measured: Baseline (Day 1 of original study) and Month 78
Participants From Pediatric Study DFI13803: Change From Baseline in Pediatric Quality of Life (PedsQL) Generic Core Total Scale Score at Month 72
PedsQL included a child self-report for participants 5 to 18 years and parents' report of participants from 2 to 18 years. Child-reported: 23-item PedsQL Generic Core Scales report included 4 scales, physical functioning, emotional functioning, social functioning, and school functioning. Parent-reported: 21-item PedsQL Generic Core Scales report and included similar scales as above. Each item used a 5-point rating scale (from 0=never to 4=almost always). Items are reverse scored and linearly transformed to a 0 (almost always) -100 (never) scale. All summary/total scores were mean of specific items where higher score indicated better HRQoL. Baseline was defined as the last available non-missing value prior to the first infusion in original study.
Measured: Baseline (Day 1 of original study) and Month 72
Participants From Pediatric Study DFI13803: Change From Baseline in Difference Between Bone Age and Actual Age of Participants at Month 72
Hand X-ray was performed on participant's left hand, fingers and wrist to assess the bone age. The X-rays were collected and read centrally by a third party blinded to participant number and study visit. At each visit, difference between the bone age and actual age at that visit was calculated. Difference in age in months was calculated as bone age in months minus real age at time of assessment in months. Baseline was defined as the last available non-missing value prior to the first infusion in original study.
Measured: Baseline (Day 1 of original study) and Month 72
Participants From Pediatric Study DFI13803: Change From Baseline in Height Z-score at Month 72
Linear growth in pediatric participants was assessed by height Z-scores. Height Z-score, i.e., the height-for-age Z-score, is the number of standard deviations of the actual height of a child from the median height of the children of the corresponding age and sex as determined from the standard sample. A height Z-score of 0 is equal to the median and is considered normal. Negative numbers indicate values lower than the median and positive numbers indicate values higher than the median. For analysis, mean Z-score was calculated and an increase of mean height Z-score from baseline indicated an improvement on growth. Baseline was defined as the last available non-missing value prior to the first infusion in original study.
Measured: Baseline (Day 1 of original study) and Month 72
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Dec 2013 (estimated)
- Study start4 Dec 2013
- Primary completion6 Sep 2023
- Study completion6 Sep 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Sphingomyelin LipidosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- Genzyme, a Sanofi Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Sciences & Operations, Study director, Sanofi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02004704
- Sponsor's protocol ID
- LTS13632
- Other IDs
- 2013-000051-40
- Status last verified
- Jun 2024
- First submitted
- 26 Nov 2013
- Last updated
- 3 Jul 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
- Expanded access
- Available
Publications
Scarpa M, Diaz GA, Giugliani R, Jones SA, Mengel E, Guffon N, Witters P, Ganesh J, Armstrong NM, Srivastava S, Kim Y. Long-Term Safety and Clinical Outcomes With Olipudase Alfa Enzyme Replacement Therapy in Children and Adolescents With Acid Sphingomyelinase Deficiency. J Inherit Metab Dis. 2025 Sep;48(5):e70086. doi: 10.1002/jimd.70086.
PubMed 40937531Lachmann RH, Diaz GA, Wasserstein MP, Armstrong NM, Yarramaneni A, Kim Y, Kumar M. Olipudase alfa enzyme replacement therapy for acid sphingomyelinase deficiency (ASMD): sustained improvements in clinical outcomes after 6.5 years of treatment in adults. Orphanet J Rare Dis. 2023 Apr 25;18(1):94. doi: 10.1186/s13023-023-02700-x.
PubMed 37098529Thurberg BL, Diaz GA, Lachmann RH, Schiano T, Wasserstein MP, Ji AJ, Zaher A, Peterschmitt MJ. Long-term efficacy of olipudase alfa in adults with acid sphingomyelinase deficiency (ASMD): Further clearance of hepatic sphingomyelin is associated with additional improvements in pro- and anti-atherogenic lipid profiles after 42 months of treatment. Mol Genet Metab. 2020 Sep-Oct;131(1-2):245-252. doi: 10.1016/j.ymgme.2020.06.010. Epub 2020 Jun 24.
PubMed 32620536Wasserstein MP, Diaz GA, Lachmann RH, Jouvin MH, Nandy I, Ji AJ, Puga AC. Olipudase alfa for treatment of acid sphingomyelinase deficiency (ASMD): safety and efficacy in adults treated for 30 months. J Inherit Metab Dis. 2018 Sep;41(5):829-838. doi: 10.1007/s10545-017-0123-6. Epub 2018 Jan 5.
PubMed 29305734
Study at a glance
From the registry record, last updated by the sponsor on 3 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Long-Term Study to Assess the Ongoing Safety and Efficacy of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
- ClinicalTrials.gov ID
- NCT02004704
- Acronym / sponsor ID
- LTS13632
- Conditions
- Sphingomyelin Lipidosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Study start
- 4 Dec 2013 (actual)
- Primary completion
- 6 Sep 2023 (actual)
- Study completion
- 6 Sep 2023 (actual)
- First posted
- 9 Dec 2013 (estimated)
- Results
- Posted 3 Jul 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objective of this study was to obtain data regarding the safety of olipudase alfa in participants with acid sphingomyelinase deficiency (ASMD) who were exposed to long term treatment with olipudase alfa.
The secondary objectives of this study were to obtain data regarding the efficacy of olipudase alfa and to characterize olipudase alfa pharmacodynamics (PD) and pharmacokinetics (PK) following long-term administration.
Detailed description
LTS13632 is a multicenter, nonrandomized, open-label, long-term extension study of participants with ASMD who have previously participated in a study of olipudase alfa. (DFI13803 for pediatric participants and DFI13412 for adult participants).
The maximum study duration per participant was 9 years or until olipudase alfa becomes commercially accessible. Notwithstanding the above, every pediatric participant were treated in LTS13632 study for at least 3 years to comply with the requirements agreed in the olipudase alfa Pediatric Investigational Plan.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| GZ402665 | Experimental | Drug: GZ402665GZ402665 administered intravenously once every 2 weeks at the dose each participant was receiving at the end of their previous olipudase alfa study, for 9 years or until olipudase alfa becomes commercially accessible, whichever comes first, unless the participant decides to enter another olipudase alfa clinical trial within the 9-year period prior to when olipudase alfa is commercially accessible. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- The participant completed the treatment period of a previous study of olipudase alfa with an acceptable safety profile in the opinion of the investigator and sponsor.
- The participant and/or the participant's parent(s)/legal guardian(s) was willing and able to provide signed written informed consent.
- The participant who is female and of childbearing potential must have had a negative urine pregnancy test for beta human chorionic gonadotropin (β HCG).
- Female participants of childbearing potential and sexually mature male participants must have been willing to practice true abstinence in line with their preferred and usual lifestyle or use 2 acceptable effective methods of contraception up to 15 days following their last dose of study drug.
Key exclusion criteria (6 listed)
- The participant had any new condition or worsening of an existing condition which in the opinion of the investigator would make the participant unsuitable for enrollment, or could interfere with the participation or completion the study.
- The participant, in the opinion of the investigator, was unable to adhere to the requirements of the study.
- The participant was unwilling or unable to abstain from the use of alcohol for 1 day prior to and 3 days after each olipudase alfa infusion for the duration of the treatment period.
- The participant was unwilling or unable to avoid, for 10 days before and 3 days after liver biopsies, medications or herbal supplements that are potentially hepatotoxic (only participants who previously participated in the DFI13412 study).
- The participant required medication(s) that may decrease olipudase alfa activity (eg, fluoxetine, chlorpromazine; tricyclic antidepressants [eg, imipramine, desipramine]).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GZ402665Also: Olipudase alfaPharmaceutical form: Powder for concentrate for solution for infusion Route of administration: intravenous infusion | Drug | GZ402665 |
Primary outcome
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Treatment Emergent Serious Adverse Events (TESAEs) and Adverse Events of Special Interest (AESIs) Except Infusion-Associated Reactions (IARs)
Time frame: From the signature of informed consent up to 9 years or until olipudase alfa was commercially accessible, whichever came first
An AE: Any untoward medical occurrence in participant or clinical investigation participant administered with pharmaceutical product, which did not necessarily have to have causal relationship with study treatment. SAEs: Any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalization/prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was congenital anomaly/birth defect, was medically important event. TEAEs: AEs that started during on-treatment period-either in this study or in original study which were ongoing at the time the participant signed the written informed consent. An AESI: AE (serious or nonserious) of scientific and medical concern specific to sponsor's product or program, for which ongoing monitoring and rapid communication by investigator to sponsor was appropriate.
Number of Participants With IARs
Time frame: From the signature of informed consent up to 9 years or until olipudase alfa was commercially accessible, whichever came first
Protocol-defined IARs were AEs that occurred during the infusion or within up to 24 hours after the start of infusion and were considered as related or possibly related to the study treatment by the investigator or the sponsor. Events occurring greater than or equal to (>=) 24 hours after the start of an infusion might have been judged an IAR at the discretion of the investigator or sponsor. Algorithm-defined IARs were all AEs that started between the start of infusion and the end of infusion plus 24 hours, irrespective of the perceived relation with study treatment.
For Adults: Number of Participants With Abnormality in Complete Physical Examinations Including Abbreviated Physical Exam
Time frame: Baseline (Day 1 of original study), Month 78
A complete physical examination included assessment of the participant's general appearance, general neurological status, skin, head, eyes, ears, nose, throat, lymph nodes, heart, lungs, abdomen, and extremities/joints. Shift from Baseline was monitored. An abbreviated physical exam (general appearance only) was only performed pre- and post-infusion for those who did not do complete exam.
For Pediatrics: Number of Participants With Abnormality in Complete Physical Examinations Including Abbreviated Physical Exam
Time frame: Baseline (Day 1 of original study), Month 84
A complete physical examination included assessment of the participant's general appearance, skin, head, eyes, ears, nose, throat, lymph nodes, heart, lungs, abdomen, and extremities/joints. Shift from Baseline was monitored. An abbreviated physical exam (general appearance only) was only performed pre- and post-infusion for those who did not do complete exam.
For Adults: Number of Participants With Abnormality in Extended Neurological Examinations
Time frame: Baseline (Day 1 of original study), Month 78
Extended neurological examination for adults included examination of mental status. Shift from Baseline was monitored.
For Pediatrics: Number of Participants With Abnormality in Extended Neurological Examinations
Time frame: Baseline (Day 1 of original study), Month 84
The neurological examination in pediatric participants included examination of mental status, cranial nerve examination, motor examination: tone, reflexes examination, sensory examination, coordination, gait and coordination, and strength examination. Shift from Baseline was monitored.
Number of Participants With Potentially Clinically Significant Abnormalities (PCSA) in Vital Signs
Time frame: Baseline (Day 1 of original study) up to 9 years or until olipudase alfa was commercially accessible, whichever came first
PCSA: Heart Rate: High: >=120 beats per minute (bpm) (adults [ad], adolescents [ado], children [chi]), >=140 bpm (early chi [ec]), >=175 bpm (infants[inf]) \& increase from baseline (IFB)>=20 bpm for all age ranges (AAR), HR Low: <=50 bpm (ad, ado, chi), <=75 bpm (ec), <=80 bpm (inf) \& decrease from baseline (DFB) >=20 bpm for AAR. Systolic blood pressure: High: >= 160 millimeters of mercury (mmHg) (ad); >=119 mmHg (ado), >=108 mmHg (chi), >=101 mmHg (ec),>=98 mmHg (inf) \& IFB >=20 mmHg for AAR. SBP Low: <=95 mmHg (ad), <=90 mmHg (ado), <= 80mm Hg (chi), <=70 mmHg (ec), <=70 mmHg (inf) \& DFB >=20 mmHg for AAR. Diastolic blood pressure: High:>110 mmHg (ad), >=78 mmHg (ado), >=72 mmHg (chi), >=59 mmHg (ec), >=54 mmHg (inf) and IFB >=10 mmHg for AAR. DBP Low:<=45 mmHg (ad), <=54 mmHg (ado), <=48 mmHg (chi), <=34mmHg (ec and inf) \& DFB>=10 mmHg for AAR.
Number of Participants With PCSA in Clinical Chemistry Parameters
Time frame: Baseline (Day 1 of original study) up to 9 years or until olipudase alfa was commercially accessible, whichever came first
PCSA: Creatinine High, category 1: >=150 micromole/L (umol) (adults), >=132 umol/L or 1.5 mg/deciliter (dL) (adolescents), >=90 umol/litre (L) or 1.1 mg/dL (children), >53 umol/L or 0.6 mg/dL (early children and infants); category 2: >=30% IFB (adults). Blood Urea Nitrogen: >=17 millimole (mmol)/L (adults), >=6.4 mmol/L or 18 mg/dL (pediatrics [ped]). Glucose Low<=3.9 mmol/L and <lower limit of normal (adults), <2.7 mmol/L (ped), High: >=11.1 mmol/L (unfasted), >7 mmol/L (fasted) (adults), >=7 mmol/L (fasted after >12 hours of fast); >=10.0 mmol/L (unfasted) (ped).
Number of Participants With PCSA in Liver Function Tests
Time frame: Baseline (Day 1 of original study) up to 9 years or until olipudase alfa was commercially accessible, whichever came first
PCSA: Alanine aminotransferase (ALT) >3 x upper limit of normal (ULN). Aspartate aminotransferase (AST) >3 x ULN. Alkaline phosphatase (ALP) > 1.5 x ULN. Total Bilirubin >1.5 x ULN (ad) and >1.3 x ULN (ped). ALT and total bilirubin: ALT > 3 x ULN and total bilirubin > 2 x ULN.
Number of Participants With PCSA in Hematology Parameters
Time frame: Baseline (Day 1 of original study) up to 9 years or until olipudase alfa was commercially accessible, whichever came first
PCSA: White blood cells (WBC): Low: <3.0 Giga (G)/L (Non-Black [NB])/<2.0 G/L (Black [B])(ad), <4.5 G/L (ado), <5.0 G/L(chi), <3.0 G/L (ec), <4.0 G/L (inf), High: >=16.0 G/L(ad), >13.5 G/L (ado), >17.0 G/L(chi), >16 G/L (ec), >20 G/L (inf). Hemoglobin-ad: Low-1: <=115 g/dL (Male [M])/<=95 g/dL (Female [F]), 20% DFB for both, High:>=185 g/L (M)/>=165 g/L (F), Low-2: DFB>=20 g/L (ad), <10 g/dL (ado, chi, ec); <9.0 g/dL (inf); 20% DFB for all. Platelets: Low: <100 G/L (AAR) and 20% DFB and High: >=700 G/L (ad and ped).
Number of Participants With PCSA in Electrocardiogram (ECG)
Time frame: Baseline (Day 1 of original study) up to 9 years or until olipudase alfa was commercially accessible, whichever came first
PCSA: Heart rate: High:>=120 bpm (ad, ado, chi), >=140 bpm (ec), >=175 bpm (inf) \& IFB>=20 bpm for AAR, Heart rate: Low: <=50 bpm (ad, ado, chi), <=75 bpm (ec), <=80 bpm (inf) \& DFB >=20 bpm for AAR. PR duration: High: >=200 milliseconds (ms) (ad) and IFB>=20 ms, >180 ms (ado), 170 ms (chi), 160 ms (ec), and 140 ms (inf). QT correction-Bazett (QTcB): Borderline absolute (category 1): 431-450 ms (ad and ado M, chi, ec and inf), 451-470 ms (ad and ado F), Prolonged-absolute (category 2): >450 ms (ad and ado M and chi, ec and inf), >470 ms (ad and ado F), Additional-absolute (category 3): >=500 ms (AAR), Borderline increase (category 4): 30-60 ms (AAR), Prolonged increase (category 5): > 60 ms (AAR).
For Adults: Percent Change From Baseline in Pre-Infusion Plasma Ceramide at Month 90
Time frame: Baseline (Day 1 of original study) and Month 90 (pre-infusion)
Plasma samples were collected to evaluate ceramide levels for safety biomarker analysis. Baseline was defined as the last available non-missing value prior to the first infusion in original study.
For Pediatrics: Percent Change From Baseline in Pre-Infusion Plasma Ceramide at Month 66
Time frame: Baseline (Day 1 of original study) and Month 66 (pre-infusion)
Plasma samples were collected to evaluate ceramide levels for safety biomarker analysis. Baseline was defined as the last available non-missing value prior to the first infusion in original study.
Participants From Adult Study DFI13412: Number of Participants With Abnormal Liver Biopsy
Time frame: Baseline (Day 1 of original study) and Month 36
Liver biopsy samples were evaluated for sphingomyelin accumulation and liver pathology in the participants who previously participated in the DFI13412 study who were at least 18 years old when they entered in this study. Sphingomyelin accumulation was quantified in liver by computer morphometry of high-resolution light microscopy images. Fibrosis grading was assessed on the Laennec scoring system, which grades the extent of fibrosis on a scale from 0 to 4 (0=none; 1=minimal; 2=mild; 3=moderate; 4=cirrhosis). Higher score indicated more severity.
Participants From Pediatric Study DFI13803: Number of Participants With Abnormalities in Liver Ultrasound Doppler
Time frame: Baseline (Day 1 of original study), Week 2 and Months 12, 18 and 24
Liver ultrasound doppler was performed for pediatric participants transitioning from DFI13803 to document hepatic blood flow characteristics, principally portal vein pressure, and blood flow direction. The parameters evaluated were liver dysmorphic findings, liver surface abnormalities, mild ascites and hepatic steatosis. Liver ultrasound Doppler was performed using methods that were compatible with the standard institutional procedures of the investigational site.
Number of Participants With Anti-Drug Antibodies (ADA) Against Olipudase Alfa
Time frame: Baseline (Day 1 of original study) up to 9 years or until olipudase alfa was commercially accessible, whichever came first
Blood samples were collected for the presence of ADAs against olipudase alfa. Treatment-boosted ADA: Positive ADA status positive at baseline (pre-existing ADA) and ADA titer level anytime post-baseline significantly higher than that at baseline. Transient ADA: Treatment-induced ADA detected only at 1 sampling time point post-baseline and treatment-induced ADA detected at 2 or more sampling time points post-baseline, where first and last ADA-positive samples (irrespective of any negative samples in between) separated by period of less than 16 weeks, and participant's last sampling time point was ADA-negative. Persistent ADA response: Treatment-induced ADA detected at 2 or more sampling time points post-baseline, where first and the last ADA-positive on-treatment sample (irrespective of any negative samples in between) separated by at least 16 weeks. Treatment-induced: ADA detected in the last 2 sampling time points, irrespective of the time period in between.
11 secondary outcomes
Percent Change From Baseline in Spleen and Liver Volumes at Month 102 for Adults and Month 84 for Pediatrics
Time frame: Baseline (Day 1 of original study), Month 102 for adults and Month 84 for pediatrics
Change From Baseline in Pulmonary Imaging by High Resolution Computed Tomography (HRCT) at Month 102 for Adults and Month 84 for Pediatrics
Time frame: Baseline (Day 1 of original study), Month 102 for adults and Month 84 for pediatrics
Percent Change From Baseline in Percent Predicted Diffusing Capacity of Lungs For Carbon Monoxide (DLco) (Hemoglobin-Adjusted) at Month 78 for Adults and Month 84 for Pediatrics
Time frame: Baseline (Day 1 of original study), Month 78 for adults and Month 84 for pediatrics
Percent Change From Baseline in Platelet Count at Month 90 for Adults and Month 78 for Pediatrics
Time frame: Baseline (Day 1 of original study), Month 90 for adults (pre-infusion) and Month 78 for pediatrics (pre-infusion)
Percent Change From Baseline in Low-density Lipoprotein at Month 90 for Adults and Month 66 for Pediatrics
Time frame: Baseline (Day 1 of original study), Month 90 for adults and Month 66 for pediatrics
Participants From Adult Study DFI13412: Change From Baseline in Brief Fatigue Inventory (BFI) Questionnaire at Month 78
Time frame: Baseline (Day 1 of original study) and Month 78
Participants From Adult Study DFI13412: Change From Baseline in Brief Pain Inventory-Short Form (BPI-SF) Questionnaire at Month 78
Time frame: Baseline (Day 1 of original study) and Month 78
Participants From Adult Study DFI13412: Change From Baseline in Chronic Respiratory Disease Questionnaire Self-Administered Standardized (CRQ-SAS) at Month 78
Time frame: Baseline (Day 1 of original study) and Month 78
Participants From Pediatric Study DFI13803: Change From Baseline in Pediatric Quality of Life (PedsQL) Generic Core Total Scale Score at Month 72
Time frame: Baseline (Day 1 of original study) and Month 72
Participants From Pediatric Study DFI13803: Change From Baseline in Difference Between Bone Age and Actual Age of Participants at Month 72
Time frame: Baseline (Day 1 of original study) and Month 72
Participants From Pediatric Study DFI13803: Change From Baseline in Height Z-score at Month 72
Time frame: Baseline (Day 1 of original study) and Month 72
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Jul 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 40937531Scarpa M, Diaz GA, Giugliani R, Jones SA, Mengel E, Guffon N, Witters P, Ganesh J, Armstrong NM, Srivastava S, Kim Y. Long-Term Safety and Clinical Outcomes With Olipudase Alfa Enzyme Replacement Therapy in Children and Adolescents With Acid Sphingomyelinase Deficiency. J Inherit Metab Dis. 2025 Sep;48(5):e70086. doi: 10.1002/jimd.70086.
- Derived from this study: PubMed 37098529Lachmann RH, Diaz GA, Wasserstein MP, Armstrong NM, Yarramaneni A, Kim Y, Kumar M. Olipudase alfa enzyme replacement therapy for acid sphingomyelinase deficiency (ASMD): sustained improvements in clinical outcomes after 6.5 years of treatment in adults. Orphanet J Rare Dis. 2023 Apr 25;18(1):94. doi: 10.1186/s13023-023-02700-x.
- Derived from this study: PubMed 32620536Thurberg BL, Diaz GA, Lachmann RH, Schiano T, Wasserstein MP, Ji AJ, Zaher A, Peterschmitt MJ. Long-term efficacy of olipudase alfa in adults with acid sphingomyelinase deficiency (ASMD): Further clearance of hepatic sphingomyelin is associated with additional improvements in pro- and anti-atherogenic lipid profiles after 42 months of treatment. Mol Genet Metab. 2020 Sep-Oct;131(1-2):245-252. doi: 10.1016/j.ymgme.2020.06.010. Epub 2020 Jun 24.
- Derived from this study: PubMed 29305734Wasserstein MP, Diaz GA, Lachmann RH, Jouvin MH, Nandy I, Ji AJ, Puga AC. Olipudase alfa for treatment of acid sphingomyelinase deficiency (ASMD): safety and efficacy in adults treated for 30 months. J Inherit Metab Dis. 2018 Sep;41(5):829-838. doi: 10.1007/s10545-017-0123-6. Epub 2018 Jan 5.
- Individual participant dataSharing planned: Yes. Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Sciences & Operations study director
Sanofi
9 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing GZ402665 in people with Sphingomyelin Lipidosis, run by Genzyme, a Sanofi Company at 9 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in Italy, United Kingdom, United States, Belgium, Brazil and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 3 Jul 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Treatment Emergent Serious Adverse Events (TESAEs) and Adverse Events of Special Interest (AESIs) Except Infusion-Associated Reactions (IARs) (time frame: From the signature of informed consent up to 9 years or until olipudase alfa was commercially accessible, whichever came first). The study lists 16 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02004704, which also lists 4 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Jul 2024.