Gene Therapy for Sickle Cell Disease (GRASP Trial)

Palo Alto (17 mi)

Overseen byDavid Williams

Age: < 65

Sex: Any

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: David Williams

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?This trial uses gene therapy to treat patients with severe Sickle Cell Disease by modifying their own blood stem cells. The treatment aims to increase healthy hemoglobin levels by changing a specific gene. This approach could reduce painful episodes and improve overall health without needing a donor.

Eligibility Criteria

This trial is for people aged 13-40 with severe sickle cell disease (HbSS or HbS/β0 thalassemia) who've had at least 4 pain crises in the last 2 years. They need good organ function, no matching bone marrow donor, and can't be on chronic blood transfusions or have a history of stroke, certain infections like HIV/Hepatitis, liver issues from iron overload, or other conditions that could interfere with treatment.

Inclusion Criteria

I am between 13 and 40 years old.

I have had 4 or more severe pain episodes in the last 2 years.

I don't have a family member who is a perfect match for a bone marrow donation.

I have sickle cell disease with either HbSS or HbS/β0 thalassemia genotype.

Exclusion Criteria

I have had a stem cell transplant from a donor.

I have an abnormal TCD history and am now on hydroxyurea.

I have severe blood vessel problems in my brain.

I have recurring priapism that hasn't improved with treatment.

I am on a regular transfusion plan to prevent strokes.

I have a known bone marrow disorder or abnormal bone marrow cells.

I cannot take busulfan due to health reasons.

I have had a stroke or a neurological event that lasted more than a day.

I have a genetic condition that causes blood clots or I've had a blood clot before.

Treatment Details

The study tests gene therapy to increase fetal hemoglobin which doesn't sickle. Patients' own stem cells are modified using a virus vector targeting BCL11A gene to reduce sickling hemoglobin levels. This could potentially cure or improve their condition without needing a donor and may use less chemotherapy.

1Treatment groups

Experimental Treatment

Group I: Treatment ArmExperimental Treatment1 Intervention

Open-label, non-randomized, single arm study of a single infusion of autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a.