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NCT04091737 · ClinicalTrials.gov registry record · Phase 1
CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease
A Phase 1 study, sponsored by CSL Behring.
- Terminated
- Registry status
- Phase 1
- Development phase
- 1
- Enrollment target
NCT04091737: Clinical Trial Phase 1 study, sponsored by CSL Behring.
NCT04091737 is a Phase 1 study that was terminated before completion, run by CSL Behring. The registered enrollment target is 1 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (98% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04091737, a Phase 1 study, was terminated before completion, sponsored by CSL Behring.
- TERMINATED
- Registry status
- Phase 1
- Development phase
- 1 participants
- Enrollment target
Study Summary
This is a phase 1 pilot study of CSL200 in adult subjects with severe sickle cell disease. The primary objectives of this study are to evaluate the safety of the following: collection of CD34+ hematopoietic stem / progenitor cells by apheresis after mobilization with plerixafor, reduced intensity conditioning with melphalan, and administration of CSL200.
Primary Outcome
Adverse event (AE) is defined as any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. Serious adverse event (SAE) is defined as any untoward medical occurrence that at any dose results in death, is life-threatening, requires in-patient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability or incapaci
Interventions
- BIOLOGICAL Autologous enriched CD34+ cell fraction that contains CD34+ cells transduced with lentiviral vector encoding human γ-globinG16D and short-hairpin RNA734
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 1 participants |
| Start Date | 2019-10-02 |
| Est. Completion | 2021-05-05 |
| Phase | Phase 1 |
Why NCT04091737 stopped before completion
NCT04091737 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 1 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (98% lower).
The record links to 0 conditions, and to 1 intervention - of which Autologous enriched CD34+ cell fraction that contains CD34+ cells transduced with lentiviral vector encoding human γ-globinG16D and short-hairpin RNA734 is the first listed.
NCT04091737 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT04091737 about?
NCT04091737 is a clinical study titled "CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease". This is a phase 1 pilot study of CSL200 in adult subjects with severe sickle cell disease. The primary objectives of this study are to evaluate the safety of the following: collection of CD34+ hematopoietic stem / progenitor cells by apheresis after mobilization with plerixafor, reduced intensity co...
What is the current status of trial NCT04091737?
This trial is currently terminated. It is a Phase 1 study. The enrollment target is 1 participants. The study started on 2019-10-02. Estimated completion is 2021-05-05.
What interventions are being tested in trial NCT04091737?
The interventions under investigation include: Autologous enriched CD34+ cell fraction that contains CD34+ cells transduced with lentiviral vector encoding human γ-globinG16D and short-hairpin RNA734 (BIOLOGICAL).
Who is sponsoring clinical trial NCT04091737?
This trial is sponsored by CSL Behring, which has 71 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT04091737's enrollment target sits among peer trials
1 2001st of 2000 the lowest 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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