Gene Correction Treatment Tested for Severe Sickle Cell Disease
Tests a gene-corrected stem cell treatment in people with severe sickle cell disease.
Key facts
- Who runs it
- Kamau Therapeutics
- Where
- Columbus, Los Angeles, New York, Palo Alto, and St Louis, United StatesSee the 6 sites
- Trial code
- Registry title
- Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease
- Status
- Recruiting
- Phase
- Phase 1/2
- People taking part
- 15 people, planned
- Started
- 15 Nov 2021
- Expected to end
- 31 Dec 2028
- Type of study
- Interventional
- Treatments tested
- nula-cel Drug Product
- Record updated
- 2 July 2026
Who can take part
AI summary
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for people aged 12 to 40 with severe sickle cell disease. It tests a gene-corrected form of their own blood stem cells.
- Age
- 12 to 40 years (the registry header lists 12 to 50 years)
- Sex
- All sexes can join.
- Disease stage
- Severe sickle cell disease: at least 4 severe pain crises in the past 2 years, or at least 2 episodes of acute chest syndrome in the past 2 years with at least one in the past year. Performance status of 80 or higher.
- Earlier treatment
- Must have had these events despite appropriate supportive care. Cannot have had a prior stem cell transplant or gene therapy.
Main reasons for not taking part
- A 10/10 HLA-matched sibling donor is available
- Prior stem cell transplant or gene therapy
- Current or past cancer, bone marrow overgrowth disorder, or a significant clotting or immune disorder
- Active bacterial, viral, fungal or parasitic infection
- Pregnancy, or breastfeeding after giving birth
The registry criteria, word for word
Show all 11 criteriaShow less
Who can take part
- ≥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 least one episode in the past year)
- Lansky/Karnofsky performance status of ≥ 80
Who cannot take part
- Prior HSCT or gene therapy
- Prior or current malignancy or myeloproliferative or a significant coagulation or immunodeficiency disorder
- Clinically significant and active bacterial, viral, fungal or parasitic infection
- Available 10/10 HLA-matched sibling donor
- Pregnancy or breastfeeding in a postpartum female
- Presence of a chromosomal abnormality/mutation that may put the participant at an increased risk for MDS or AML per investigator's judgment
Check if you might fit
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 12 and 50 years old?
Where in United States
- Nationwide Children's HospitalColumbus, United StatesContact: Dalena SandersonTaking people
- Children's Hospital Los AngelesLos Angeles, United StatesContact: Bridget BaileyTaking people
- Columbia University Irving Medical CenterNew York, United StatesContact: Camila BarrosTaking people
- Memorial Sloan KetteringNew York, United StatesContact: Maria Paes PenaTaking people
- Lucile Packard Children's HospitalPalo Alto, United StatesContact: Stanford Intake TeamTaking people
- Washington UniversitySt Louis, United StatesContact: Maggie NashTaking people
About the trial
What the trial wants to find out
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.
The group in the trial
- nula-cel Drug ProductTreatment being tested
nula-cel Drug Product is a human autologous CRISPR-Cas9 edited and sickle mutation-corrected HSPC product.
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorData and sources
- Registry record
- Listed on ClinicalTrials.gov.
- Official title
- A Phase I/II Study of Nula-cel in Autologous CD34+ Hematopoietic Stem Cells to Convert HbS to HbA for Treating Severe Sickle Cell Disease
- Plain-language title
- Gene Correction Treatment Tested for Severe Sickle Cell DiseaseGenerated from the registry record. The registry title is the official one.
- AI summary
- From the trial's eligibility criteria. Read by machine on 12 November 2026.
- Fit questions
- Built from NCT04819841, who can take part
- Record updated
- 2 July 2026