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NCT04819841 · ClinicalTrials.gov registry record · Phase 1
Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease
A Phase 1 study of Sickle Cell Disease, sponsored by Kamau Therapeutics.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 15
- Enrollment target
- 4
- Study locations
NCT04819841: Recruiting Phase 1 study of Sickle Cell Disease, sponsored by Kamau Therapeutics.
NCT04819841 is a Phase 1 study of Sickle Cell Disease that is actively recruiting participants, run by Kamau Therapeutics. The registered enrollment target is 15 participants, below the 293-participant average among 213 other Sickle Cell Disease trials with a reported enrollment target (95% lower). The trial reports 4 study locations across 3 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04819841, a Phase 1 study of Sickle Cell Disease, is actively recruiting participants, sponsored by Kamau Therapeutics.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 15 participants
- Enrollment target
- 4
- Study locations
Study Summary
This study is a first-in-human, single-arm, open-label Phase I/II study of nula-cel in approximately 15 participants, diagnosed with severe Sickle Cell Disease. The primary objective is to evaluate safety of the treatment in this patient population, as well as preliminary efficacy and pharmacodynamic data.
Conditions Studied
Interventions
- GENETIC nula-cel Drug Product
Study Locations (4)
California
- Children's Hospital Los Angeles - Los Angeles
- Lucile Packard Children's Hospital - Palo Alto
Missouri
- Washington University - St Louis
Ohio
- Nationwide Children's Hospital - Columbus
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 15 participants |
| Start Date | 2021-11-15 |
| Est. Completion | 2028-12-31 |
| Phase | Phase 1 |
What NCT04819841 shows while recruiting
NCT04819841 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 15 participants, a relatively small participant target, below the 293-participant average among 213 other Sickle Cell Disease trials with a reported enrollment target (95% lower).
The record links to 1 condition, with Sickle Cell Disease appearing as the primary indexed condition, and to 1 intervention - of which nula-cel Drug Product is the first listed.
NCT04819841 reports a single indexed study location in California, Missouri, Ohio.
Frequently Asked Questions
What is clinical trial NCT04819841 about?
NCT04819841 is a clinical study titled "Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease". This study is a first-in-human, single-arm, open-label Phase I/II study of nula-cel in approximately 15 participants, diagnosed with severe Sickle Cell Disease. The primary objective is to evaluate safety of the treatment in this patient population, as well as preliminary efficacy and pharmacodynami...
What is the current status of trial NCT04819841?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 15 participants. The study started on 2021-11-15. Estimated completion is 2028-12-31.
What conditions does trial NCT04819841 study?
This clinical trial studies the following conditions: Sickle Cell Disease.
What interventions are being tested in trial NCT04819841?
The interventions under investigation include: nula-cel Drug Product (GENETIC).
Who is sponsoring clinical trial NCT04819841?
This trial is sponsored by Kamau Therapeutics, which has 1 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04819841 being conducted?
This trial has 4 study locations across California, Missouri, Ohio. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT04819841's enrollment target sits among peer trials
15 185th of 213 higher than 24 of 213 other Sickle Cell Disease trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Sickle Cell Disease 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.
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