EDIT-301 for Sickle Cell Disease

Recruiting in Palo Alto (17 mi)

+23 other locations

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: Editas Medicine, Inc.

No Placebo Group

Approved in 2 Jurisdictions

Trial Summary

What is the purpose of this trial?

This trial is testing a new treatment called EDIT-301 for people aged 12 to 50 with severe sickle cell disease. The treatment uses the patient's own stem cells, which are changed in a lab and then put back into their body to help fight the disease.

Research Team

Eligibility Criteria

This trial is for adults with severe sickle cell disease who have had at least two major pain episodes per year despite treatment. They must be generally well-functioning (Karnofsky Performance Status ≥ 80). People can't join if they've had a stem cell transplant, have certain allergies or infections, advanced liver disease, cancer, immune disorders, or an HLA-matched donor.

Inclusion Criteria

I have a severe form of sickle cell disease.

I can carry out normal activities with minimal symptoms.

I've had 2 or more severe pain crises a year despite treatment.

Exclusion Criteria

My organs are not functioning properly.

I am not allergic to plerixafor, busulfan, or any related medications.

I have or had another cancer or a condition that weakens my immune system.

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Treatment Details

Interventions

EDIT-301 (Gene Therapy)

Trial OverviewThe study tests EDIT-301's effectiveness and safety in treating severe sickle cell disease. Participants will receive this experimental therapy to see how well it works and what side effects it may cause compared to standard treatments.

Participant Groups

1Treatment groups

Experimental Treatment

Group I: EDIT-301Experimental Treatment1 Intervention

EDIT-301 (autologous gene edited (CD)34+ hematopoietic stem cells) will be administered as a one-time intravenous infusion.

EDIT-301 is already approved in Canada for the following indications: