Metachromatic Leukodystrophy (MLD)Phase 1NCT00418561
Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
A Phase 1 study testing rhASA - Dose Level 1, rhASA - Dose Level 2 and rhASA - Dose Level 3 in people with Metachromatic Leukodystrophy (MLD), run by Shire at 1 site in Denmark.
Trial summary
Objectives: The overall objective is to evaluate the safety, efficacy and pharmacokinetics (PK) of rhASA treatment in patients with late infantile MLD. Methodology: This is a single center, open-label study of patients with late infantile MLD. Read more about this study
- Who is it for?
- Ages 1 year to 5 years
- Conditions the study is for
- Metachromatic Leukodystrophy (MLD)
- What is being tested?
- rhASA - Dose Level 1, rhASA - Dose Level 2 and rhASA - Dose Level 3
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 1 siteDenmark
- Study dates
- 22 Jan 2007to 27 Mar 2008
- Phase
- Phase 1
- 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 1 year to 5 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Subject's legally authorized guardian(s) must provide signed, informed consent prior to performing any study-related activities (Trial-related activities are any procedures that would not have been performed during normal management of the subject).
- The patient must have a confirmed diagnosis of MLD as defined by:
- ASA activity < 10 nmol/h/mg in leukocytes Presence of elevated sulfatide in urine
- The patient must have a confirmed nerve conduction velocity < 2 standard deviations (from the appropriate age level)
- The patient must have a residual level of voluntary function (as judged by the investigator), including presence of residual cognitive function (attention, executive and visual functions) as well as the presence of residual voluntary motor function in one upper or lower limb as a minimum.
- The patient must have an age at the time of screening ≥ 1 year and < 6 years
Show all 9Show fewer
- The patient must have had onset of symptoms before the age of 4 years
- The subject and his/her guardian(s) must have the ability to comply with the clinical protocol
- The patients' medical record must document that the legal guardian(s) has had independent counselling or a consultation regarding stem cell transplantation in order to assure that the guardian(s) is fully informed regarding the risks and benefits of this alternative
Who cannot join
- Patients will be excluded from this study if they do not meet the specific inclusion criteria, or if any of the following criteria apply:
- Lack of voluntary function
- Presence of severe pseudo-bulbar signs (weakness and disco-ordination of tongue and swallowing muscles leading to severe difficulty with swallowing)
- Spasticity so severe to inhibit transportation
- Known multiple sulfatase deficiency
- Presence of major congenital abnormality
Show all 13Show fewer
- Presence of known chromosomal abnormality and syndromes affecting psychomotor development
- History of stem cell transplantation
- Presence of known clinically significant cardiovascular, hepatic, pulmonary or renal disease or other medical condition
- Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the trial
- Use of any investigational product within 30 days prior to study enrolment or currently enrolled in another study which involves clinical investigations
- Received ERT with rhASA from any source
- Planned or anticipated initiation of antispastic treatment after trial initiation
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Single Center, Open-label, Non-randomized, Uncontrolled, Multiple-dose, Dose Escalation Study of the Safety, Pharmacokinetics and Efficacy of Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
Objectives: The overall objective is to evaluate the safety, efficacy and pharmacokinetics (PK) of rhASA treatment in patients with late infantile MLD.
Methodology: This is a single center, open-label study of patients with late infantile MLD. Twelve patients will be enrolled in this study receiving a total of thirteen intravenous infusions of Metazym. One infusion will be given every other week for a period of half a year. After the half year the subjects will continue treatment every other week until safety data is available. Safety (AE/SAE) will be monitored at every visit during this period.
Read the detailed description
Test product, dose, mode of administration, batch No.: The lowest dose level will be evaluated as a single dose of 25 U/kg. The three upper dose levels will be evaluated as repeated doses. Patients in each cohort will receive one dose of enzyme every other week for a period of eight weeks, a total of five doses. Dosing will be performed as follows: Cohort 1: 25 U/kg as a single dose - hereafter 50 U/kg; Cohort 2: 100 U/kg; Cohort 3: 200 U/kg. Patients receiving the lowest dose as a single dose will receive the next dose level as a repeated dose. After twenty six weeks the subjects will continue treatment every other week until safety data is available. Safety (AE/SAE) will be monitored at every visit during this period. The dose will be adjusted monthly to account for changes in body weight. The infusion length will be dependent on the dose. Doses of 25 U/kg, 50 U/kg and 100 U/kg will be diluted in 50 ml isotonic sodium chloride and infused over 30 minutes. Infusion of 200 U/kg will be administered in the same manner except for an infusion time of 60 minutes.
Duration of treatment: Half a year (26 weeks)
- Conditions
- Metachromatic Leukodystrophy (MLD)
- Being tested
- rhASA - Dose Level 1, rhASA - Dose Level 2, rhASA - Dose Level 3
- Keywords
- rhASA, MLD, Metazym
- Medical subject headings
- Leukodystrophy, Metachromatic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
rhASA - Dose Level 1Biological
Also called HGT-1111, metazym
Intravenous infusion 25 U/kg as a single dose - hereafter 50 U/kg every other week for 26 weeks
rhASA - Dose Level 2Biological
Also called HGT-1111, metazym
Intravenous infusion 100 U/kg every other week for 26 weeks
rhASA - Dose Level 3Biological
Also called HGT-1111, metazym
Intravenous infusion 200 U/kg every other week for 26 weeks
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | Metazym (Recombinant human arylsulfatase A (rhASA)): 25 U/kg as a single dose - hereafter 50 U/kg Receives: Biological: rhASA - Dose Level 1 |
| Cohort 2 | Experimental | 100 U/kg Metazym (Recombinant human arylsulfatase A (rhASA)) Receives: Biological: rhASA - Dose Level 2 |
| Cohort 3 | Experimental | 200 U/kg Metazym (Recombinant human arylsulfatase A (rhASA)) Receives: Biological: rhASA - Dose Level 3 |
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)
An adverse event (AE) is any untoward, undesired, unplanned clinical event in the form of signs, symptoms, disease, or laboratory or physiological observations occurring in a participant, participating in a clinical study with study drug, regardless of causal relationship. TEAEs were AEs occurred after study drug administration that were absent before treatment or that worsened relative to pre-treatment state, up to Week 28 until evaluation (when last cohort had 26-week evaluation and data management performed within 4 weeks) completed.
Measured: From study drug administration up to Week 28
Change From Baseline in Gross Motor Function Measure (GMFM) at Week 26
GMFM was measured using GMFM-88 item scores and summed to calculate a total GMFM-88 score. For each GMFM-88 item, the score was between 0 (minimal) to 3 (maximum). The total GMFM-88 score was between 0 (minimal) and 264 (maximum). The decrease in GMFM score over time indicates worsening of disease over time. Relative change from baseline at Week 26 was calculated as percentage change from baseline divided by the age-difference in months between first and last visit. Adjusted mean and 95 percent (%) confidence intervals were reported.
Measured: Baseline, Week 26
Change From Baseline in Cerebrospinal Fluid (CSF) Sulfatide at Week 26
Relative change from baseline at Week 26 was calculated as percentage change from baseline divided by the age-difference in months between first and last visit. Adjusted mean and 95 percent (%) confidence intervals were reported.
Measured: Baseline, Week 26
Number of Participants With Shift From Baseline to Week 26 in Sulfatide Levels in Urine
Number of participants with shifts between negative (value=0) and positive (value=1) values in urine sulfatide levels from baseline at Week 26 is reported.
Measured: Baseline up to Week 26
Change From Baseline in Mullen's Scales of Early Learning at Week 26
Mullen's Scales of Early Learning is used to assess performance and learning ability in young children. The scale consisted of 144 items that had specific scoring criteria for each item. The scores were converted to T-scores with a decrease in score indicating worsening of disease. Relative change from baseline at Week 26 was calculated as percentage change from baseline divided by the age-difference in months between first and last visit. Adjusted mean and 95 percent (%) confidence intervals were reported.
Measured: Baseline, Week 26
Maximum Plasma Drug Concentration (Cmax) of Recombinant Human Arylsulphatase A (rhASA)
Measured: Pre-dose and post-dose at 20, 40, 90 minutes, 3, 6 and 8 hours on Day 0, 40 minutes post-dose at Week 4, Pre-dose and post-dose at 20, 40, 90 minutes, 3, 6 and 8 hours at Week 8
Arylsulfatase A (ASA) Activity in Leukocytes
Measured: Pre-dose and post-dose at 24 hours on Day 0 and at Weeks 8 and 26
Secondary outcomes (4)
Change From Baseline in Nerve Conduction Velocity at Week 26
An electrophysiological evaluation using standard electrophysiological and electromyography to measure the speed and extent of nerve conduction and units are expressed in meters per second. Abbreviations: MN=Median Nerve; PN=Peroneal Nerve; SN=Sural Nerve; Dig.=Digit; FH=fibular hemimelia; L LM=left lateral medial; R LM=right lateral medial; MC=medial collateral.
Measured: Baseline, Week 26
Number of Participants Who Had Undergone Nerve Biopsy and Had a Normal Nerve at Both Baseline and Week 26
Measured: Baseline, Week 26
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores
Loes scoring system is used to grade the demyelinating abnormalities on brain MRI. A total of 17 locations of the brain were scored from 0 (normal appearance) to 2 (dense appearance). The total score ranged from 0 to 34 with a score of 14 or greater being considered severe. Number of participants with any shift of score between 0 to 2 for each of the 17 locations (Parieto Occipital [PO]-Periventricular [P], Central [C], Subcortical [Sc]; Anterior Temporal [AT]-P, C, Sc; Frontal [F]-P, C, Sc; Corpus Callosum [CC]-Splenium [S], Genus [G]; Projection Fibers [PF]-Capsular interna [CI] ant, CI post, Brainstem [B]; Cerebellum [Cb]-Cortex, Atrophy; Basal Ganglia [BG]-BG, Thalamus [T]; Cerebral Atrophy [CA]-CA), are only reported.
Measured: Baseline up to Week 26
Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26
PEDI is used for the clinical evaluation of functional capabilities, performance and changes in functional skills in children with disabilities. It consisted of 20 items scored on a scale from 0 (total assistance) to 5 (independent). Total score ranged from 0-100 with higher scores indicating better functioning. None, child, rehab, extensive are items in 3 domains (self-care, mobility and social functioning).
Measured: Baseline, Week 26
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jan 2007 (estimated)
- Study start22 Jan 2007
- Primary completion27 Mar 2008
- Study completion27 Mar 2008
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 Metachromatic Leukodystrophy (MLD)Studies 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 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Factorial
- Masking
- None (open label)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00418561
- Sponsor's protocol ID
- rhASA-01
- Other IDs
- 2006-005341-11
- Status last verified
- Jun 2021
- First submitted
- 4 Jan 2007
- Last updated
- 25 Jun 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Dali CI, Barton NW, Farah MH, Moldovan M, Mansson JE, Nair N, Duno M, Risom L, Cao H, Pan L, Sellos-Moura M, Corse AM, Krarup C. Sulfatide levels correlate with severity of neuropathy in metachromatic leukodystrophy. Ann Clin Transl Neurol. 2015 May;2(5):518-33. doi: 10.1002/acn3.193. Epub 2015 Mar 27.
PubMed 26000324I Dali C, Groeschel S, Moldovan M, Farah MH, Krageloh-Mann I, Wasilewski M, Li J, Barton N, Krarup C. Intravenous arylsulfatase A in metachromatic leukodystrophy: a phase 1/2 study. Ann Clin Transl Neurol. 2021 Jan;8(1):66-80. doi: 10.1002/acn3.51254. Epub 2020 Dec 17.
PubMed 33332761
Study at a glance
From the registry record, last updated by the sponsor on 25 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single Center, Open-label, Non-randomized, Uncontrolled, Multiple-dose, Dose Escalation Study of the Safety, Pharmacokinetics and Efficacy of Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
- ClinicalTrials.gov ID
- NCT00418561
- Acronym / sponsor ID
- rhASA-01
- Conditions
- Metachromatic Leukodystrophy (MLD)
- Keywords
- rhASA, MLD, Metazym
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 13 participants (actual)
- Lead sponsor
- ShireIndustry (pharma, biotech, devices)
- Study start
- 22 Jan 2007 (actual)
- Primary completion
- 27 Mar 2008 (actual)
- Study completion
- 27 Mar 2008 (actual)
- First posted
- 5 Jan 2007 (estimated)
- Results
- Posted 13 Aug 2015
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Objectives: The overall objective is to evaluate the safety, efficacy and pharmacokinetics (PK) of rhASA treatment in patients with late infantile MLD.
Methodology: This is a single center, open-label study of patients with late infantile MLD. Twelve patients will be enrolled in this study receiving a total of thirteen intravenous infusions of Metazym. One infusion will be given every other week for a period of half a year. After the half year the subjects will continue treatment every other week until safety data is available. Safety (AE/SAE) will be monitored at every visit during this period.
Detailed description
Test product, dose, mode of administration, batch No.: The lowest dose level will be evaluated as a single dose of 25 U/kg. The three upper dose levels will be evaluated as repeated doses. Patients in each cohort will receive one dose of enzyme every other week for a period of eight weeks, a total of five doses. Dosing will be performed as follows: Cohort 1: 25 U/kg as a single dose - hereafter 50 U/kg; Cohort 2: 100 U/kg; Cohort 3: 200 U/kg. Patients receiving the lowest dose as a single dose will receive the next dose level as a repeated dose. After twenty six weeks the subjects will continue treatment every other week until safety data is available. Safety (AE/SAE) will be monitored at every visit during this period. The dose will be adjusted monthly to account for changes in body weight. The infusion length will be dependent on the dose. Doses of 25 U/kg, 50 U/kg and 100 U/kg will be diluted in 50 ml isotonic sodium chloride and infused over 30 minutes. Infusion of 200 U/kg will be administered in the same manner except for an infusion time of 60 minutes.
Duration of treatment: Half a year (26 weeks)
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Factorial
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Biological: rhASA - Dose Level 1Metazym (Recombinant human arylsulfatase A (rhASA)): 25 U/kg as a single dose - hereafter 50 U/kg |
| Cohort 2 | Experimental | Biological: rhASA - Dose Level 2100 U/kg Metazym (Recombinant human arylsulfatase A (rhASA)) |
| Cohort 3 | Experimental | Biological: rhASA - Dose Level 3200 U/kg Metazym (Recombinant human arylsulfatase A (rhASA)) |
Who may qualify
- Ages
- 1 year to 5 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (9 listed)
- Subject's legally authorized guardian(s) must provide signed, informed consent prior to performing any study-related activities (Trial-related activities are any procedures that would not have been performed during normal management of the subject).
- The patient must have a confirmed diagnosis of MLD as defined by:
- ASA activity < 10 nmol/h/mg in leukocytes Presence of elevated sulfatide in urine
- The patient must have a confirmed nerve conduction velocity < 2 standard deviations (from the appropriate age level)
- The patient must have a residual level of voluntary function (as judged by the investigator), including presence of residual cognitive function (attention, executive and visual functions) as well as the presence of residual voluntary motor function in one upper or lower limb as a minimum.
Key exclusion criteria (13 listed)
- Patients will be excluded from this study if they do not meet the specific inclusion criteria, or if any of the following criteria apply:
- Lack of voluntary function
- Presence of severe pseudo-bulbar signs (weakness and disco-ordination of tongue and swallowing muscles leading to severe difficulty with swallowing)
- Spasticity so severe to inhibit transportation
- Known multiple sulfatase deficiency
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 |
|---|---|---|
| rhASA - Dose Level 1Also: HGT-1111, metazymIntravenous infusion 25 U/kg as a single dose - hereafter 50 U/kg every other week for 26 weeks | Biological | Cohort 1 |
| rhASA - Dose Level 2Also: HGT-1111, metazymIntravenous infusion 100 U/kg every other week for 26 weeks | Biological | Cohort 2 |
| rhASA - Dose Level 3Also: HGT-1111, metazymIntravenous infusion 200 U/kg every other week for 26 weeks | Biological | Cohort 3 |
Primary outcome
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Time frame: From study drug administration up to Week 28
An adverse event (AE) is any untoward, undesired, unplanned clinical event in the form of signs, symptoms, disease, or laboratory or physiological observations occurring in a participant, participating in a clinical study with study drug, regardless of causal relationship. TEAEs were AEs occurred after study drug administration that were absent before treatment or that worsened relative to pre-treatment state, up to Week 28 until evaluation (when last cohort had 26-week evaluation and data management performed within 4 weeks) completed.
Change From Baseline in Gross Motor Function Measure (GMFM) at Week 26
Time frame: Baseline, Week 26
GMFM was measured using GMFM-88 item scores and summed to calculate a total GMFM-88 score. For each GMFM-88 item, the score was between 0 (minimal) to 3 (maximum). The total GMFM-88 score was between 0 (minimal) and 264 (maximum). The decrease in GMFM score over time indicates worsening of disease over time. Relative change from baseline at Week 26 was calculated as percentage change from baseline divided by the age-difference in months between first and last visit. Adjusted mean and 95 percent (%) confidence intervals were reported.
Change From Baseline in Cerebrospinal Fluid (CSF) Sulfatide at Week 26
Time frame: Baseline, Week 26
Relative change from baseline at Week 26 was calculated as percentage change from baseline divided by the age-difference in months between first and last visit. Adjusted mean and 95 percent (%) confidence intervals were reported.
Number of Participants With Shift From Baseline to Week 26 in Sulfatide Levels in Urine
Time frame: Baseline up to Week 26
Number of participants with shifts between negative (value=0) and positive (value=1) values in urine sulfatide levels from baseline at Week 26 is reported.
Change From Baseline in Mullen's Scales of Early Learning at Week 26
Time frame: Baseline, Week 26
Mullen's Scales of Early Learning is used to assess performance and learning ability in young children. The scale consisted of 144 items that had specific scoring criteria for each item. The scores were converted to T-scores with a decrease in score indicating worsening of disease. Relative change from baseline at Week 26 was calculated as percentage change from baseline divided by the age-difference in months between first and last visit. Adjusted mean and 95 percent (%) confidence intervals were reported.
Maximum Plasma Drug Concentration (Cmax) of Recombinant Human Arylsulphatase A (rhASA)
Time frame: Pre-dose and post-dose at 20, 40, 90 minutes, 3, 6 and 8 hours on Day 0, 40 minutes post-dose at Week 4, Pre-dose and post-dose at 20, 40, 90 minutes, 3, 6 and 8 hours at Week 8
Arylsulfatase A (ASA) Activity in Leukocytes
Time frame: Pre-dose and post-dose at 24 hours on Day 0 and at Weeks 8 and 26
4 secondary outcomes
Change From Baseline in Nerve Conduction Velocity at Week 26
Time frame: Baseline, Week 26
Number of Participants Who Had Undergone Nerve Biopsy and Had a Normal Nerve at Both Baseline and Week 26
Time frame: Baseline, Week 26
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores
Time frame: Baseline up to Week 26
Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26
Time frame: Baseline, Week 26
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Jun 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 26000324Dali CI, Barton NW, Farah MH, Moldovan M, Mansson JE, Nair N, Duno M, Risom L, Cao H, Pan L, Sellos-Moura M, Corse AM, Krarup C. Sulfatide levels correlate with severity of neuropathy in metachromatic leukodystrophy. Ann Clin Transl Neurol. 2015 May;2(5):518-33. doi: 10.1002/acn3.193. Epub 2015 Mar 27.
- Derived from this study: PubMed 33332761I Dali C, Groeschel S, Moldovan M, Farah MH, Krageloh-Mann I, Wasilewski M, Li J, Barton N, Krarup C. Intravenous arylsulfatase A in metachromatic leukodystrophy: a phase 1/2 study. Ann Clin Transl Neurol. 2021 Jan;8(1):66-80. doi: 10.1002/acn3.51254. Epub 2020 Dec 17.
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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.
Study Director study director
Takeda
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing rhASA - Dose Level 1, rhASA - Dose Level 2 and rhASA - Dose Level 3 in people with Metachromatic Leukodystrophy (MLD), run by Shire at 1 site in Denmark.
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 9 inclusion and 13 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 25 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Denmark. 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 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.
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 13 Aug 2015). 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) (time frame: From study drug administration up to Week 28). The study lists 7 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 NCT00418561, 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 Jun 2021.