Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease
Recruiting
Phase 1
Phase 2
Interventional Study
Sickle Cell Disease
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 12 – 50
- Sex
- Any
- Trial phase
- Phase 1/2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 15 (estimated)
- Sponsor
- Kamau Therapeutics · Industry
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About This Trial
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.
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* ≥12 to ≤ 40 years
* Severe disease, as defined by having experienced at least one of the following SCD-related events despite appropriate supportive care measures:
* recurrent severe VOC (≥ 4 episodes in the preceding 2 years)
* ACS (≥ 2 episodes in the prior 2 years with at l…
Contacts