Gene Therapy for Sickle Cell Disease
(Restore Trial)
What You Need to Know Before You Apply
What is the purpose of 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.
Do I have to stop taking my current medications for the trial?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
What makes the GPH101 drug unique for treating sickle cell disease?
What data supports the effectiveness of the treatment Nula-cel for sickle cell disease?
Who Is on the Research Team?
Mathew Porteus, MD, PhD
Principal Investigator
Kamau Therapeutics
Are You a Good Fit for This Trial?
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.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Conditioning
Participants undergo myeloablative conditioning prior to receiving nula-cel infusion
Treatment
Participants receive nula-cel via IV infusion
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- GPH101 Drug Product
Find a Clinic Near You
Who Is Running the Clinical Trial?
Kamau Therapeutics
Lead Sponsor
Graphite Bio, Inc.
Lead Sponsor