A Phase 3 interventional study of Laronidase and Antihistamine in Mucopolysaccharidosis Type 1, sponsored by Cinnagen. Completed at 4 sites in Iran, Islamic Republic of. Open to participants aged 5 Years to 18 Years. Per ClinicalTrials.gov, last updated 2024-08-06.
Sponsored by Cinnagen · Phase 3, Interventional, and Treatment
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Among 16 studies testing laronidase in Mucopolysaccharidosis I — none open to participants now, none under way, none of them phase 3. laronidase · Mucopolysaccharidosis I.
Among the 16 studies testing laronidase in Mucopolysaccharidosis I, this one is the 7th largest by enrollment, is the only one that started in 2022, is one of 3 in phase 3, and was the 16th registered.
74 studies on the registry are indexed under Mucopolysaccharidosis I; 7 are open to participants now.
Browse Mucopolysaccharidosis I studies →None of the 16 studies testing laronidase in Mucopolysaccharidosis I is open now. The open studies under Mucopolysaccharidosis I, anywhere →
laronidase: first FDA approval 2003 (ALDURAZYME), first EMA approval 2003. What it is approved for, and how studies give it, is on its page →
Of the 6 Mucopolysaccharidosis I studies recruiting now, 3 set no upper age limit and 3 stop at 75 or younger; 5 take children under 18; none take healthy volunteers.
This study: 5 Years to 18 Years · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
16 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 5 · Phase 2 3 · Phase 3 3 · Phase 4 4
First in 2001; this study’s year is marked. 1 started in 2022.
This study's enrollment of 12 is below the median of 14 across 47 interventional studies indexed under Mucopolysaccharidosis I.
Median enrollment 12 across the 16 interventional studies. Of the 16 that completed or were terminated, 9 (56%) have results posted.
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9 of 16 have an industry lead sponsor. The 5 most frequent:
Cinnagen is the lead sponsor of 21 studies on the registry; 1 is open to participants now.
Cinnagen has 1 study filed under Mucopolysaccharidosis I — fewer than ten, so there is no comparison to draw.
None of the studies under way reaches primary completion within a year.
Among the 16 studies testing laronidase in Mucopolysaccharidosis I, from their registry updates:
Nothing recorded among them since 2026-09-25.
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The purpose of this phase III study is to assess the efficacy and safety of YW17 produced by CinnaGen Company compared to Aldurazyme® in mucopolysaccharidosis type I (MPS I) patients.
All patients receive Aldurazyme® for 12 weeks, followed by YW17 for another 12 weeks.
The primary outcome is the assessment of the maintenance of the mean uGAG levels at the end of each medication administration. The secondary outcomes are the assessment of 6-minute walking test (6MWT), predicted forced vital capacity (FVC), enzyme activity assay, and adverse events (AEs).
This is a phase III, single-sequence, cross-over study to assess the efficacy and safety of YW17 produced by CinnaGen Company in comparison with Aldurazyme® in MPS I patients.
All patients receive 0.58 mg/kg of Aldurazyme® for 12 weeks and then receive 0.58 mg/kg of YW17 for another 12 weeks.
Premedication with antipyretics and/or antihistamines is administered for all patients one hour before the infusion.
The primary outcome is to compare the mean uGAG levels at weeks 8, 10, and 12 (related to Aldurazyme®) with the mean uGAG levels at weeks 20, 22, and 24 (related to YW17).
The secondary outcomes, including 6MWT and FVC are assessed at the beginning and the end of each medication administration. Enzyme activity is assessed at the end of each medication administration. Safety assessments are performed during the study.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Allogeneic and autologous transplant
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
YW17 (2.9 mg/5 mL) produced by CinnaGen Company, is administered 0.58 mg/kg weekly.
Biological: Laronidase · Drug: Antihistamine · Drug: Antipyretic
Aldurazyme® (2.9 mg/5 mL), is administered 0.58 mg/kg weekly.
Biological: Laronidase · Drug: Antihistamine · Drug: Antipyretic
Laronidase as Aldurazyme® (the first 12 weeks) or YW17 (the second 12 weeks) is administered.
An antihistamine is administered one hour before the infusion.
An antipyretic is administered one hour before the infusion.
mean uGAG
urinary glycosaminoglycan
Time frame: Baseline, weeks 8, 10, 12, 20, 22, and 24
mean 6MWT
6-minute walking test
Time frame: Baseline, week 12, week 24
mean predicted FVC
Forced vital capacity
Time frame: Baseline, week 12, week 24
mean enzyme activity level
Enzyme activity assay
Time frame: Weeks 11 and 23
Number of participants with adverse events
All reported AEs are classified based on the Medical Dictionary for Regulatory Activities (MedDRA) terms and are graded according to the Common Terminology Criteria for Adverse Events (CTCAE) v5.0.
Time frame: During the study period (screening visit up to week 24)
Plan to share: No
No publications or documents are linked to this record.
Ran from Sep 2022 to primary completion in Aug 2023, 11 months.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Aug 2024. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Laronidase
Cinnagen