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Gene Therapy

Gene Therapy for Sickle Cell Disease (Restore Trial)

Phase 1 & 2
Recruiting
Research Sponsored by Kamau Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
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)
Must not have
Prior hematopoietic stem cell transplant (HSCT) or gene therapy
Prior or current malignancy, myeloproliferative disorder, significant coagulation disorder, or immunodeficiency disorder
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights

Summary

This trial is testing a new drug for safety and effectiveness in people with severe sickle cell disease.

Who is the study for?
This trial is for people aged 12-40 with severe Sickle Cell Disease who've had multiple acute chest syndrome episodes or recurrent severe pain crises, despite treatment. They must be generally well-functioning (good performance status). Those with prior gene therapy, a perfect sibling donor match, active infections, pregnancy/breastfeeding, or certain genetic risks are excluded.
What is being tested?
The study tests nula-cel Drug Product in about 15 participants to see if it's safe and effective for treating severe Sickle Cell Disease. It's an early-phase trial where all enrolled patients receive the experimental treatment and are monitored for results.
What are the potential side effects?
Since this is a first-in-human study of nula-cel, specific side effects aren't listed but may include typical stem cell transplant-related risks like infection risk increase due to immune system effects and potential infusion reactions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have had 4 or more severe pain crises in the last 2 years despite treatment.
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I have had 2 or more acute chest syndrome episodes in the last 2 years.
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I can carry out most of my everyday activities without help.
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I am between 12 and 40 years old.
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I have severe sickle cell disease with frequent pain or lung problems.
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I am between 12 and 40 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have had a stem cell transplant or gene therapy.
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I have or had another cancer, blood disorder, clotting issue, or immune system disorder.
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I do not have any active and serious infections.
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I have a sibling who is a perfect match for a bone marrow donation.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Trial Design

1Treatment groups
Experimental Treatment
Group I: nula-cel Drug ProductExperimental Treatment1 Intervention
nula-cel Drug Product is a human autologous CRISPR-Cas9 edited and sickle mutation-corrected HSPC product.

Find a Location

Who is running the clinical trial?

Kamau TherapeuticsLead Sponsor
Graphite Bio, Inc.Lead Sponsor
Mathew Porteus, MD, PhDStudy DirectorKamau Therapeutics
~6 spots leftby Jul 2026
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