Genome-Edited Cells for Sickle Cell Disease

Recruiting in Palo Alto (17 mi)

+8 other locations

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1

Waitlist Available

Sponsor: Novartis Pharmaceuticals

No Placebo Group

Trial Summary

What is the purpose of this trial?

This trial uses genetically modified blood stem cells from patients to treat Sickle Cell Disease. It works by reducing a gene's activity to increase beneficial fetal hemoglobin, aiming to lessen disease complications. The treatment involves a gene therapy that helps produce a type of hemoglobin that reduces the effects of the disease.

Research Team

Eligibility Criteria

This trial is for people aged 2-40 with sickle cell disease who've had severe symptoms like pain crises, acute chest syndrome, or stroke. It's open to those who can't tolerate or haven't benefited from hydroxyurea therapy and have a performance status over 70%. People with liver issues, iron overload, certain infections or cancers, previous transplants or gene therapy are not eligible.

Inclusion Criteria

I have stopped or refused hydroxyurea due to side effects or choice.

I am mostly active and can care for myself.

I have had severe symptoms like pain crises, chest issues, or needed regular blood transfusions.

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Exclusion Criteria

I do not have severe or worsening artery or brain vessel disease.

I have an active cancer, blood disorder, genetic abnormalities, or a weak immune system.

I have not had brain surgery or procedures for Moyamoya disease in the last year.

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Treatment Details

Interventions

HIX763 (Gene Therapy)
OTQ923 (Gene Therapy)

Trial OverviewThe study tests OTQ923 - a genome-edited stem cell treatment aimed at increasing fetal hemoglobin to alleviate sickle cell complications. Participants receive their own modified cells back to potentially reduce the severity of their condition.

Participant Groups

1Treatment groups

Experimental Treatment

Group I: OTQ923Experimental Treatment1 Intervention

Single intravenous infusion of OTQ923 Part A - Adults treated with OTQ923; Part B - Children age 2-17 treated with OTQ923 based on review of data from Part A by Health agency after a formal interim analysis.