By InvitationPhase 1/2

A Study of the Safety and Efficacy of Prime Editing (PM359) in Participants With p47phox Autosomal Recessive Chronic Granulomatous Disease (CGD )

United States, Canada, United Kingdom12 participantsStarted 2024-10-17

Plain-language summary

This is an open-label, single-arm, multicenter Phase 1/2 study evaluating the safety and efficacy of gene therapy by transplantation of Prime Edited autologous CD34+ stem cells modified ex vivo (PM359) in participants with autosomal recessive Chronic Granulomatous Disease (CGD) caused by mutations in the NCF1 (Neutrophil Cytosolic Factor 1) gene.

Who can participate

Age range6 Years

SexALL

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Exclusion criteria

✕. Contraindication to mobilization and apheresis, including severe allergic reaction to receipt of any medication or other drug substance required for mobilization and apheresis (e.g., G-CSF, plerixafor) or PM359 manufacture (e.g., DMSO).
✕. Contraindication to receipt of the conditioning agent, busulfan.

What they're measuring

Safety of administration of PM359, as quantified by frequency of adverse events (AEs) after drug product infusion

Timeframe: PM359 infusion through Month 12 after PM359 infusion

Percentage of participants with sustained reconstitution of NADPH oxidase activity in neutrophils

Timeframe: At Month 6 and Month 12 after PM359 infusion, as compared to baseline

Trial details

NCT IDNCT06559176

SponsorPrime Medicine, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2030-01

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