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NCT01722526 · ClinicalTrials.gov registry record · Phase 1
Tolerability and Safety Study of Recombinant Human Acid Sphingomyelinase in Acid Sphingomyelinase Deficiency Patients
A Phase 1 study of Human Acid Sphingomyelinase Deficiency, sponsored by Genzyme, a Sanofi Company.
- Completed
- Registry status
- Phase 1
- Development phase
- 5
- Enrollment target
- 2
- Study locations
NCT01722526: Completed Phase 1 study of Human Acid Sphingomyelinase Deficiency, sponsored by Genzyme, a Sanofi Company.
NCT01722526 is a Phase 1 study of Human Acid Sphingomyelinase Deficiency that has completed, run by Genzyme, a Sanofi Company. The registered enrollment target is 5 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (92% lower). The trial reports 2 study locations across 2 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01722526, a Phase 1 study of Human Acid Sphingomyelinase Deficiency, has completed, sponsored by Genzyme, a Sanofi Company.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 5 participants
- Enrollment target
- 2
- Study locations
Study Summary
To evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic profile of rhASM in adult patients with Acid Sphingomyelinase Deficiency (ASMD) following repeated-dose administration.
Conditions Studied
Interventions
- DRUG Recombinant human acid sphingomyelinase
Study Locations (2)
New York
- Mount Sinai School of Medicine - New York
Other
- St. Mary's Hospital - Manchester
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 5 participants |
| Start Date | 2013-03 |
| Est. Completion | 2014-01 |
| Phase | Phase 1 |
What the finished NCT01722526 record still lists
NCT01722526 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 5 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (92% lower).
The record links to 1 condition, with Human Acid Sphingomyelinase Deficiency appearing as the primary indexed condition, and to 1 intervention - of which Recombinant human acid sphingomyelinase is the first listed.
NCT01722526 reports a single indexed study location in New York, Other.
Frequently Asked Questions
What is clinical trial NCT01722526 about?
NCT01722526 is a clinical study titled "Tolerability and Safety Study of Recombinant Human Acid Sphingomyelinase in Acid Sphingomyelinase Deficiency Patients". To evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic profile of rhASM in adult patients with Acid Sphingomyelinase Deficiency (ASMD) following repeated-dose administration.
What is the current status of trial NCT01722526?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 5 participants. The study started on 2013-03. Estimated completion is 2014-01.
What conditions does trial NCT01722526 study?
This clinical trial studies the following conditions: Human Acid Sphingomyelinase Deficiency.
What interventions are being tested in trial NCT01722526?
The interventions under investigation include: Recombinant human acid sphingomyelinase (DRUG).
Who is sponsoring clinical trial NCT01722526?
This trial is sponsored by Genzyme, a Sanofi Company, which has 196 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT01722526 being conducted?
This trial has 2 study locations across New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT01722526's enrollment target sits among peer trials
5 1989th of 2000 higher than 6 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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