MPS IIIAPhase 1/2NCT04088734
Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease
A Phase 1/2 study testing ABO-102 in people with MPS IIIA and Sanfilippo Syndrome, run by Ultragenyx Pharmaceutical Inc at 3 sites in 3 countries.
Trial summary
Open-label, clinical trial of scAAV9.U1a.hSGSH injected intravenously through a peripheral limb vein Read more about this study
- Who is it for?
- Ages 2 to 18 years
- Conditions the study is for
- MPS IIIA, Sanfilippo Syndrome, Sanfilippo A
- What is being tested?
- ABO-102
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 3 sitesin 3 countries
- Study dates
- 18 Sep 2019to 10 Mar 2022
- Phase
- Phase 1/2
- 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 2 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosis of MPS IIIA confirmed by the following methods:
- No detectable or significantly reduced SGSH enzyme activity by leukocyte assay and
- Genomic DNA analysis demonstrating homozygous or compound heterozygous mutations in the SGSH gene
- Cognitive Development Quotient (DQ) lower than 60 (calculated by Bayley Scales of Infant and Toddler Development - Third Edition)
- Must be ambulatory, though may receive assistance with ambulation
- Age range of 2 years up to 18 years (excluded)
Who cannot join
- Inability to participate in the clinical evaluation as determined by Principal Investigator
- Identification of two nonsense or null variants on genetic testing of the SGSH gene
- At least one S298P mutation in the SGSH gene
- Has evidence of an attenuated phenotype of MPS IIIA
- Presence of a concomitant medical condition that precludes lumbar puncture or use of anesthetics
- Active viral infection based on clinical observations
Show all 23Show fewer
- Concomitant illness or requirement for chronic drug treatment that in the opinion of the PI creates unnecessary risks for gene transfer, or precludes the child from participating in the protocol assessments and follow up
- Participants with total anti-AAV9 antibody titers greater than or equal to 1:100 as determined by ELISA binding immunoassay
- Participants with a positive response for the ELISPOT for T-cell responses to AAV9
- Serology consistent with exposure to HIV, or serology consistent with active hepatitis B or C infection
- Bleeding disorder or any other medical condition or circumstance in which a lumbar puncture (for collection of CSF) is contraindicated according to local institutional policy
- Visual or hearing impairment sufficient to preclude cooperation with neurodevelopmental testing
- Any item (braces, etc.) which would exclude the participant from being able to undergo MRI according to local institutional policy
- Any other situation that precludes the participant from undergoing procedures required in this study
- Participants with cardiomyopathy or significant congenital heart abnormalities
- The presence of significant non-MPS IlIA related CNS impairment or behavioral disturbances that would confound the scientific rigor or interpretation of results of the study
- Abnormal laboratory values Grade 2 or higher as defined in CTCAE v4.03 for GGT, total bilirubin (except in subjects diagnosed with Gilbert's syndrome), creatinine, hemoglobin, WBC count, platelet count, PT and aPTT
- Female participant who is pregnant or demonstrates a positive urine or beta-hCG result at screening assessment (if applicable)
- Any vaccination with viral attenuated vaccines less than 30 days prior to the scheduled date of treatment (and use of prednisolone)
- Previous treatment by Haematopoietic Stem Cell transplantation
- Previous participation in a gene/cell therapy or ERT clinical trial
- Participants who are anticipated to undergo a procedure involving anesthesia within 6 months post- drug administration
- Dysphagia present at Grade 3 or higher, as defined in CTCAE v4.03
Trial locations and contacts
3 sites in 3 countries.
3 sites in 3 countries
United States1 site
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
Australia1 site
Adelaide Women's and Children's Hospital
North Adelaide, South Australia, 5006
Spain1 site
Hospital Clínico Universitario de Santiago
Santiago de Compostela, 15706
About this study
Official title: A Phase I/II Open Label, Single-dose, Gene Transfer Study of scAAV9.U1a.hSGSH (ABO-102) in Patients With Middle and Advanced Phases of MPS IIIA Disease
Open-label, clinical trial of scAAV9.U1a.hSGSH injected intravenously through a peripheral limb vein
Read the detailed description
This is an open-label, single dose clinical trial. All participants will receive 3 X 10^13 vg/kg of ABO-102 delivered one time through a venous catheter inserted into a peripheral limb vein. The target population includes MPS IIIA participants with a DQ lower than 60 in middle and advanced phases of the disease. Similar numbers of MPS IIIA participants with age equivalent above and below 18 months of age will be enrolled to ensure a representation of middle and advanced phases of the disease.
This study was previously posted by Abeona Therapeutics, Inc and was transferred to Ultragenyx in August 2022.
- Conditions
- MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III
- Being tested
- ABO-102
- Keywords
- MPS IIIA, Sanfilippo, Gene therapy
- Medical subject headings
- Mucopolysaccharidosis III
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ABO-102Drug
Also called scAAV9.U1a.hSGSH, UX111
Single dose of ABO-102 (scAAV9.U1a.hSGSH) administered by intravenous injection through a peripheral limb vein at a dose of 3 X 10^13 vg/kg
| Study group | Role | What they receive |
|---|---|---|
| ABO-102 | Experimental | Dose of 3x10^13 vg/kg Receives: Drug: ABO-102 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence, Type and Severity of Related Treatment-Emergent Adverse Events (TEAEs) by Time Frame
An adverse event (AE) is any untoward medical occurrence or unintended change from the time informed consent form (ICF) is signed, including inter-current illness that occurs during the course of a clinical trial after treatment has started, whether considered related to treatment or not. TEAEs are those that occurred after the start of study drug. Related adverse events were categorized as possible, probable, or definitely.
Measured: From the first dose of study drug to <30 days postdose, Day 30, 60, 90, 180 and Month 12
Incidence, Type and Severity of Serious Adverse Events (SAEs) by Time Frame
An SAE is defined as any untoward medical occurrence that, at any dose:
- Results in death
- Is life threatening
- Requires inpatient hospitalization or prolongation of existing hospitalization
- Results in persistent disability/incapacity
- Is a congenital anomaly/birth defect
- Other situations such as important medical events that may not be immediately life threatening or result in death or hospitalization but may jeopardize the participant or may require medical or surgical intervention to prevent one of the other outcomes listed in the above definition.
Relationship to study drug was defined as unrelated, unlikely, possible, probable, or definitely.
Measured: From signing of informed consent through Day 60, 90, 180 and up to Day 454 (> 12 months)
Change From Baseline (BL) in Multiples of Normal of Liver and Spleen Volumes After Treatment
As Measured by Magnetic Resonance Imaging (MRI). Baseline value of multiple of normal is calculated using the baseline values of the Liver volume/Spleen Volume/Height and Weight.
- Body Surface Area (BSA) (m2)=( Height(cm) * Weight (kg)/3600)1/2.
- Normal Liver Volume=(689.9 * BSA (m)) - 24.7.
- Normal Spleen Volume (mL)=(4.6 * Weight (kg)) + 0.7.
- Liver Volume (multiples of normal)=Subject Liver Volume (mL)/Normal Liver Volume (mL).
- Spleen Volume (multiples of normal)=Subject Spleen Volume (mL)/Normal Spleen Volume (mL).
Measured: Baseline, Day 30, 180, Month 12
Change From BL in Cerebrospinal Fluid (CSF) Heparan Sulfate Levels After Treatment
Change from baseline in CSF heparan sulfate levels after treatment
Measured: Baseline, Day 30, Day 180, Month 12
Secondary outcomes (18)
Change From Baseline in Plasma Heparan Sulfate After Treatment
Measured: Baseline, Day 30, Day 180, Month 12
Change From Baseline in Urine Glycosaminoglycans After Treatment
Measured: Baseline, Day 30, Day 180, Month 12
Change From Baseline in Urine Heparan Sulfate After Treatment
Measured: Baseline, Day 30, Day 180, Month 12
Change From Baseline in CSF N-Sulfoglucosamine Sulfohydrolase (SGSH) Enzyme Activity Levels After Treatment
Measured: Baseline, Day 30, Day 180, and Month 12
Change From Baseline in Heparan N-Sulfatase (Type A) After Treatment
Measured: Baseline, Day 30, Day 180, Month 12
Change From Baseline in Plasma SGSH After Treatment
Measured: Baseline, Day 30, Day 180, Month 12
Change From Baseline in Brain Volumes After Treatment
As measured by MRI
Measured: Baseline, 12 months
Change From Baseline in Brain Volumes After Treatment: Average Total Cortical Thickness
As measured by MRI
Measured: Baseline, 12 months
Change From Baseline in Sleep Pattern as Measured by the Modified Children's Sleep Habits Questionnaire (CSHQ) Subscore Total After Treatment
CSHQ is a caregiver-completed, 35-item questionnaire that assesses the frequency of behaviors associated with common pediatric sleep difficulties. Eight domains of sleep, including Bedtime Resistance, Sleep Onset Delay, Sleep Duration, Sleep Anxiety, Night Awakenings, Parasomnias, Sleep Disordered Breathing, and Daytime Sleepiness are assessed, producing eight individual subdomain scores and an overall CSHQ subscore total. CSHQ total score is calculated by adding all the 8 subscores, and ranges from 36 to 108. A higher score is indicative of more disturbed sleep.
Measured: Baseline, Day 180, Month 12
Change From Baseline in Pediatric Quality of Life Inventory (PedsQL™) Core Generic Scales Total Score
PedsQL is a brief measure of health-related quality of life in children. The Peds QL Generic Core Scales was used in the study, consisting of 23 items applicable for healthy school and community populations, as well as pediatric populations with acute and chronic conditions. The four scales include Physical Functioning, Emotional Functioning, Social Functioning, and School Functioning. Item scores are added together and averaged to produce a Core total score, where higher scores on a scale of 0-100 indicate better Health-related Quality of Life (HRQOL).
Measured: Baseline, Day 180, Month 12
Change From Baseline in Parent Quality of Life, Using the Parenting Stress Index, 4th Edition (PSI-4) Total Stress Raw Score
The Parenting Stress Index, 4th Edition evaluates the magnitude and type of stress in a parent/child relationship. The short form version was used in the study, consisting of 36 items divided into three domains: Parental Distress (PD), Parent-Child Dysfunctional Interaction (P-CDI), and Difficult Child (DC), which combine together to form a Total Stress raw score. Total Stress raw scores can range from 36 - 180, with higher raw scores indicating higher levels of stress.
Measured: Baseline, Day 180, Month 12
Change From Baseline in Gastrointestinal Symptoms Using the PedsQL™ Gastrointestinal (GI) Symptoms Scales Score
The PedsQL Gastrointestinal Symptoms Scale is a specific module of the PedsQL that measures gastrointestinal symptoms in patients with acute and chronic health conditions as well as healthy school and community populations. The Parent Report version was used on the study, consisting of 58 items across 10 dimensions, assessing parents' perceptions of their child's GI-specific symptoms. Item scores are added together and averaged to produce a GI symptoms scales score, where higher scores on a scale of 0-100 indicate better GI specific QOL.
Measured: Baseline, Day 180, Month 12
Change From Baseline in Parent Global Impression (PGI) Total Score
The Parent Global Impression scale evaluates all aspects of a patients' health and assesses if there has been an improvement or decline in clinical status, as reported by the parent/caregiver. This study used a modified version with symptoms relevant to the patient population in the trial. Nine symptoms were scored at each visit, using a 7-point rating scale where 3 = much better, 2 = better, 1 = slightly better, 0 = same, -1 = slightly worse, -2 = worse, and -3 = much worse. The nine symptom scores are added together to produce a PGI total score, ranging from -27 to 27.
Measured: Baseline, Day 180, Month 12
Clinical Global Impression Improvement Scale at Day 180 and Month 12
The Clinical Global Impression of Improvement scale is a brief, stand-alone assessment of the clinician's view of the patient's global functioning prior to and after initiating a study medication, specifically looking at whether the patient has demonstrated improvement or not. Assessment of improvement was scored at each visit, using a 7-point rating scale where 0 = not assessed, 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, and 7 = very much worse.
Measured: Day 180, Month 12
Change From Baseline in Parent Symptoms Score Questionnaire
The Parent Symptoms Score Questionnaire contains 29 symptoms with an indicator of whether the symptom was present or absent.
Measured: Baseline, Day 180, Month 12
Percent Change From Baseline in Body Mass Index After Treatment
Measured: Baseline, Day 30, Day 180, Month 12
Number of Participants With Abnormalities in Standard Awake 45-Minutes-Electroencephalogram (EEG) Monitoring at Baseline and Day 180
NCS=not clinically significant CS=clinically significant
Measured: Baseline, Day 180
Change From Baseline in Vector Shedding Analysis in Plasma, Saliva, Stool and Urine
Detection of the adeno-associated Virus 9 (AAV9) viral deoxyribonucleic acid (DNA) in plasma, saliva, urine and feces was analyzed. Per protocol, data were not collected for urine at Month 12.
Measured: Baseline, Day 30, Day 180, Month 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Sep 2019
- Study start18 Sep 2019
- Primary completion10 Mar 2022
- Study completion10 Mar 2022
Why it stopped: Terminated due to lack of efficacy seen in patients with advanced MPS IIIA disease. The patients will be followed up annually for safety until five years post dosing
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 (2)Papers the sponsor has linked to this study.
- Other trials for MPS IIIAStudies of the same condition, open and under way.
- More trials run by Ultragenyx Pharmaceutical IncThe sponsor's other registered studies.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Abeona Therapeutics, Inc
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Ultragenyx Pharmaceutical Inc
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04088734
- Sponsor's protocol ID
- ABT-003
- Other IDs
- UX111-CL201, 2018-000504-42, 2018
- Status last verified
- Jul 2023
- First submitted
- 11 Sep 2019
- Last updated
- 25 Jul 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Fu H, Cataldi MP, Ware TA, Zaraspe K, Meadows AS, Murrey DA, McCarty DM. Functional correction of neurological and somatic disorders at later stages of disease in MPS IIIA mice by systemic scAAV9-hSGSH gene delivery. Mol Ther Methods Clin Dev. 2016 Jun 8;3:16036. doi: 10.1038/mtm.2016.36. eCollection 2016.
PubMed 27331076Fu H, Meadows AS, Pineda RJ, Kunkler KL, Truxal KV, McBride KL, Flanigan KM, McCarty DM. Differential Prevalence of Antibodies Against Adeno-Associated Virus in Healthy Children and Patients with Mucopolysaccharidosis III: Perspective for AAV-Mediated Gene Therapy. Hum Gene Ther Clin Dev. 2017 Dec;28(4):187-196. doi: 10.1089/humc.2017.109. Epub 2017 Oct 24.
PubMed 29064732
Study at a glance
From the registry record, last updated by the sponsor on 25 Jul 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Open Label, Single-dose, Gene Transfer Study of scAAV9.U1a.hSGSH (ABO-102) in Patients With Middle and Advanced Phases of MPS IIIA Disease
- ClinicalTrials.gov ID
- NCT04088734
- Acronym / sponsor ID
- ABT-003
- Conditions
- MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III
- Keywords
- MPS IIIA, Sanfilippo, Gene therapy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Terminated due to lack of efficacy seen in patients with advanced MPS IIIA disease. The patients will be followed up annually for safety until five years post dosing
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Ultragenyx Pharmaceutical IncIndustry (pharma, biotech, devices)
- Collaborators
- Abeona Therapeutics, Inc
- Study start
- 18 Sep 2019 (actual)
- Primary completion
- 10 Mar 2022 (actual)
- Study completion
- 10 Mar 2022 (actual)
- First posted
- 13 Sep 2019 (actual)
- Results
- Posted 25 Jul 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Open-label, clinical trial of scAAV9.U1a.hSGSH injected intravenously through a peripheral limb vein
Detailed description
This is an open-label, single dose clinical trial. All participants will receive 3 X 10^13 vg/kg of ABO-102 delivered one time through a venous catheter inserted into a peripheral limb vein. The target population includes MPS IIIA participants with a DQ lower than 60 in middle and advanced phases of the disease. Similar numbers of MPS IIIA participants with age equivalent above and below 18 months of age will be enrolled to ensure a representation of middle and advanced phases of the disease.
This study was previously posted by Abeona Therapeutics, Inc and was transferred to Ultragenyx in August 2022.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ABO-102 | Experimental | Drug: ABO-102Dose of 3x10^13 vg/kg |
Who may qualify
- Ages
- 2 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- Diagnosis of MPS IIIA confirmed by the following methods:
- Cognitive Development Quotient (DQ) lower than 60 (calculated by Bayley Scales of Infant and Toddler Development - Third Edition)
- Must be ambulatory, though may receive assistance with ambulation
- Age range of 2 years up to 18 years (excluded)
Key exclusion criteria (23 listed)
- Inability to participate in the clinical evaluation as determined by Principal Investigator
- Identification of two nonsense or null variants on genetic testing of the SGSH gene
- At least one S298P mutation in the SGSH gene
- Has evidence of an attenuated phenotype of MPS IIIA
- Presence of a concomitant medical condition that precludes lumbar puncture or use of anesthetics
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 |
|---|---|---|
| ABO-102Also: scAAV9.U1a.hSGSH, UX111Single dose of ABO-102 (scAAV9.U1a.hSGSH) administered by intravenous injection through a peripheral limb vein at a dose of 3 X 10^13 vg/kg | Drug | ABO-102 |
Primary outcome
Incidence, Type and Severity of Related Treatment-Emergent Adverse Events (TEAEs) by Time Frame
Time frame: From the first dose of study drug to <30 days postdose, Day 30, 60, 90, 180 and Month 12
An adverse event (AE) is any untoward medical occurrence or unintended change from the time informed consent form (ICF) is signed, including inter-current illness that occurs during the course of a clinical trial after treatment has started, whether considered related to treatment or not. TEAEs are those that occurred after the start of study drug. Related adverse events were categorized as possible, probable, or definitely.
Incidence, Type and Severity of Serious Adverse Events (SAEs) by Time Frame
Time frame: From signing of informed consent through Day 60, 90, 180 and up to Day 454 (> 12 months)
An SAE is defined as any untoward medical occurrence that, at any dose: 1. Results in death 2. Is life threatening 3. Requires inpatient hospitalization or prolongation of existing hospitalization 4. Results in persistent disability/incapacity 5. Is a congenital anomaly/birth defect 6. Other situations such as important medical events that may not be immediately life threatening or result in death or hospitalization but may jeopardize the participant or may require medical or surgical intervention to prevent one of the other outcomes listed in the above definition. Relationship to study drug was defined as unrelated, unlikely, possible, probable, or definitely.
Change From Baseline (BL) in Multiples of Normal of Liver and Spleen Volumes After Treatment
Time frame: Baseline, Day 30, 180, Month 12
As Measured by Magnetic Resonance Imaging (MRI). Baseline value of multiple of normal is calculated using the baseline values of the Liver volume/Spleen Volume/Height and Weight. * Body Surface Area (BSA) (m2)=( Height(cm) * Weight (kg)/3600)1/2. * Normal Liver Volume=(689.9 * BSA (m)) - 24.7. * Normal Spleen Volume (mL)=(4.6 * Weight (kg)) + 0.7. * Liver Volume (multiples of normal)=Subject Liver Volume (mL)/Normal Liver Volume (mL). * Spleen Volume (multiples of normal)=Subject Spleen Volume (mL)/Normal Spleen Volume (mL).
Change From BL in Cerebrospinal Fluid (CSF) Heparan Sulfate Levels After Treatment
Time frame: Baseline, Day 30, Day 180, Month 12
Change from baseline in CSF heparan sulfate levels after treatment
18 secondary outcomes
Change From Baseline in Plasma Heparan Sulfate After Treatment
Time frame: Baseline, Day 30, Day 180, Month 12
Change From Baseline in Urine Glycosaminoglycans After Treatment
Time frame: Baseline, Day 30, Day 180, Month 12
Change From Baseline in Urine Heparan Sulfate After Treatment
Time frame: Baseline, Day 30, Day 180, Month 12
Change From Baseline in CSF N-Sulfoglucosamine Sulfohydrolase (SGSH) Enzyme Activity Levels After Treatment
Time frame: Baseline, Day 30, Day 180, and Month 12
Change From Baseline in Heparan N-Sulfatase (Type A) After Treatment
Time frame: Baseline, Day 30, Day 180, Month 12
Change From Baseline in Plasma SGSH After Treatment
Time frame: Baseline, Day 30, Day 180, Month 12
Change From Baseline in Brain Volumes After Treatment
Time frame: Baseline, 12 months
Change From Baseline in Brain Volumes After Treatment: Average Total Cortical Thickness
Time frame: Baseline, 12 months
Change From Baseline in Sleep Pattern as Measured by the Modified Children's Sleep Habits Questionnaire (CSHQ) Subscore Total After Treatment
Time frame: Baseline, Day 180, Month 12
Change From Baseline in Pediatric Quality of Life Inventory (PedsQL™) Core Generic Scales Total Score
Time frame: Baseline, Day 180, Month 12
Change From Baseline in Parent Quality of Life, Using the Parenting Stress Index, 4th Edition (PSI-4) Total Stress Raw Score
Time frame: Baseline, Day 180, Month 12
Change From Baseline in Gastrointestinal Symptoms Using the PedsQL™ Gastrointestinal (GI) Symptoms Scales Score
Time frame: Baseline, Day 180, Month 12
Change From Baseline in Parent Global Impression (PGI) Total Score
Time frame: Baseline, Day 180, Month 12
Clinical Global Impression Improvement Scale at Day 180 and Month 12
Time frame: Day 180, Month 12
Change From Baseline in Parent Symptoms Score Questionnaire
Time frame: Baseline, Day 180, Month 12
Percent Change From Baseline in Body Mass Index After Treatment
Time frame: Baseline, Day 30, Day 180, Month 12
Number of Participants With Abnormalities in Standard Awake 45-Minutes-Electroencephalogram (EEG) Monitoring at Baseline and Day 180
Time frame: Baseline, Day 180
Change From Baseline in Vector Shedding Analysis in Plasma, Saliva, Stool and Urine
Time frame: Baseline, Day 30, Day 180, Month 12
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Jul 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 27331076Fu H, Cataldi MP, Ware TA, Zaraspe K, Meadows AS, Murrey DA, McCarty DM. Functional correction of neurological and somatic disorders at later stages of disease in MPS IIIA mice by systemic scAAV9-hSGSH gene delivery. Mol Ther Methods Clin Dev. 2016 Jun 8;3:16036. doi: 10.1038/mtm.2016.36. eCollection 2016.
- Background: PubMed 29064732Fu H, Meadows AS, Pineda RJ, Kunkler KL, Truxal KV, McBride KL, Flanigan KM, McCarty DM. Differential Prevalence of Antibodies Against Adeno-Associated Virus in Healthy Children and Patients with Mucopolysaccharidosis III: Perspective for AAV-Mediated Gene Therapy. Hum Gene Ther Clin Dev. 2017 Dec;28(4):187-196. doi: 10.1089/humc.2017.109. Epub 2017 Oct 24.
- Individual participant dataSharing planned: No
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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.
Medical Director study director
Ultragenyx Pharmaceutical Inc
3 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing ABO-102 in people with MPS IIIA and Sanfilippo Syndrome, run by Ultragenyx Pharmaceutical Inc at 3 sites in 3 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 6 inclusion and 23 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: stopped early, as of the sponsor's last update on 25 Jul 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States, Australia, Spain. 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 25 Jul 2023). 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?
Incidence, Type and Severity of Related Treatment-Emergent Adverse Events (TEAEs) by Time Frame (time frame: From the first dose of study drug to <30 days postdose, Day 30, 60, 90, 180 and Month 12). The study lists 4 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 NCT04088734, which also lists 2 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 25 Jul 2023.