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NCT02060526 · ClinicalTrials.gov registry record · Phase 2
Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease
A Phase 2 study, sponsored by Shire.
- Completed
- Registry status
- Phase 2
- Development phase
- 21
- Enrollment target
NCT02060526 is a Phase 2 study that has completed, run by Shire. The registered enrollment target is 21 participants.
The verdict
NCT02060526, a Phase 2 study, has completed, sponsored by Shire.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 21 participants
- Enrollment target
Study Summary
Sanfilippo syndrome Type A, or Mucopolysaccharidosis (MPS) IIIA, is a rare lysosomal storage disease caused by deficiency of the enzyme heparan N-sulfatase (sulfamidase). In the absence of this enzyme, there is an accumulation of the glycosaminoglycan, heparan sulfate, resulting in progressive neurodegeneration. Symptoms are usually first noted in the 1st or 2nd year of life, although definitive diagnosis is often delayed, with an average age of diagnosis of 4.5 years. The disease is characterized by developmental delays initially, followed by neurological developmental arrest, then regression. These developmental deficits are typically associated with severe behavioral disturbances. Patients have a significantly reduced lifespan, with few surviving beyond the 2nd or 3rd decade. The purpose of this study is to evaluate the safety and efficacy of recombinant human heparan-N-sulfatase (rhHNS) in pediatric patients with Early Stage Mucopolysaccharidosis Type III A Disease.
Interventions
- DRUG Recombinant human heparan N-sulfatase [rhHNS]
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 21 participants |
| Start Date | 2014-02-26 |
| Est. Completion | 2016-06-01 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT02060526
The ClinicalTrials.gov registry entry for NCT02060526 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 21 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Shire, which has 245 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which Recombinant human heparan N-sulfatase [rhHNS] is the first listed.
NCT02060526 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT02060526 about?
NCT02060526 is a clinical study titled "Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease". Sanfilippo syndrome Type A, or Mucopolysaccharidosis (MPS) IIIA, is a rare lysosomal storage disease caused by deficiency of the enzyme heparan N-sulfatase (sulfamidase). In the absence of this enzyme, there is an accumulation of the glycosaminoglycan, heparan sulfate, resulting in progressive neuro...
What is the current status of trial NCT02060526?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 21 participants. The study started on 2014-02-26. Estimated completion is 2016-06-01.
What interventions are being tested in trial NCT02060526?
The interventions under investigation include: Recombinant human heparan N-sulfatase [rhHNS] (DRUG).
Who is sponsoring clinical trial NCT02060526?
This trial is sponsored by Shire, which has 245 total clinical trials registered on ClinicalTrials.gov.
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