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NCT04443907 · ClinicalTrials.gov registry record · Phase 1
Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD)
A Phase 1 study, sponsored by Novartis Pharmaceuticals.
- Terminated
- Registry status
- Phase 1
- Development phase
- 4
- Enrollment target
NCT04443907: Clinical Trial Phase 1 study, sponsored by Novartis Pharmaceuticals.
NCT04443907 is a Phase 1 study that was terminated before completion, run by Novartis Pharmaceuticals. The registered enrollment target is 4 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (93% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04443907, a Phase 1 study, was terminated before completion, sponsored by Novartis Pharmaceuticals.
- TERMINATED
- Registry status
- Phase 1
- Development phase
- 4 participants
- Enrollment target
Study Summary
This study evaluated a genome-edited, autologous, hematopoietic stem and progenitor cell (HSPC) product - OTQ923 to reduce the biologic activity of BCL11A, increasing fetal hemoglobin (HbF) and reducing complications of sickle cell disease.
Primary Outcome
Number of participants with adverse events (AEs) and serious adverse events (SAEs), including changes from baseline in vital signs, electrocardiograms and laboratory results qualifying and reported as AEs.
Interventions
- BIOLOGICAL OTQ923
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 4 participants |
| Start Date | 2020-08-25 |
| Est. Completion | 2025-01-06 |
| Phase | Phase 1 |
Why NCT04443907 stopped before completion
NCT04443907 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 4 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (93% lower).
The record links to 0 conditions, and to 1 intervention - of which OTQ923 is the first listed.
NCT04443907 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT04443907 about?
NCT04443907 is a clinical study titled "Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD)". This study evaluated a genome-edited, autologous, hematopoietic stem and progenitor cell (HSPC) product - OTQ923 to reduce the biologic activity of BCL11A, increasing fetal hemoglobin (HbF) and reducing complications of sickle cell disease.
What is the current status of trial NCT04443907?
This trial is currently terminated. It is a Phase 1 study. The enrollment target is 4 participants. The study started on 2020-08-25. Estimated completion is 2025-01-06.
What interventions are being tested in trial NCT04443907?
The interventions under investigation include: OTQ923 (BIOLOGICAL).
Who is sponsoring clinical trial NCT04443907?
This trial is sponsored by Novartis Pharmaceuticals, which has 1,375 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT04443907's enrollment target sits among peer trials
4 1995th of 2000 higher than 2 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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