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NCT03003533 · ClinicalTrials.gov registry record · Phase 1
A Gene Transfer Study for Hemophilia A
A Phase 1 study, sponsored by Spark Therapeutics.
- Completed
- Registry status
- Phase 1
- Development phase
- 25
- Enrollment target
NCT03003533: Completed Phase 1 study, sponsored by Spark Therapeutics.
NCT03003533 is a Phase 1 study that has completed, run by Spark Therapeutics. The registered enrollment target is 25 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (58% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03003533, a Phase 1 study, has completed, sponsored by Spark Therapeutics.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 25 participants
- Enrollment target
Study Summary
This clinical research study is being conducted by Spark Therapeutics, Inc. to determine the safety and efficacy of the factor VIII gene transfer treatment with SPK-8011 in individuals with hemophilia A.
Primary Outcome
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse events (SAEs) were defined as adverse events that result in death, are life-threatening, require inpatient hospitalization or prolongation of existing hospitalization, result in persistent or significant disability or incapacity, are a congenital anomaly or birth defect, or are an important medical event that jeopardiz
Interventions
- GENETIC SPK-8011
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 25 participants |
| Start Date | 2017-01-26 |
| Est. Completion | 2023-12-05 |
| Phase | Phase 1 |
What the finished NCT03003533 record still lists
NCT03003533 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 25 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (58% lower).
The record links to 0 conditions, and to 1 intervention - of which SPK-8011 is the first listed.
NCT03003533 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT03003533 about?
NCT03003533 is a clinical study titled "A Gene Transfer Study for Hemophilia A". This clinical research study is being conducted by Spark Therapeutics, Inc. to determine the safety and efficacy of the factor VIII gene transfer treatment with SPK-8011 in individuals with hemophilia A.
What is the current status of trial NCT03003533?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 25 participants. The study started on 2017-01-26. Estimated completion is 2023-12-05.
What interventions are being tested in trial NCT03003533?
The interventions under investigation include: SPK-8011 (GENETIC).
Who is sponsoring clinical trial NCT03003533?
This trial is sponsored by Spark Therapeutics, which has 13 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT03003533's enrollment target sits among peer trials
25 1495th of 2000 higher than 481 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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