NADPH Oxidase Correction in mRNA-transfected Granulocyte-enriched Cells in Chronic Granulomatous Disease (CGD)

Recruiting Phase 1 Interventional Study
Chronic Granulomatous Disease Infection
No Placebo Group Every participant receives an active treatment — no one gets a placebo.
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At a Glance
Age
18 – 75
Sex
Male
Trial phase
Phase 1
Study type
Interventional
Purpose
Treatment
Participants needed
25 (estimated)
Sponsor
National Institute of Allergy and Infectious Diseases (NIAID) · NIH
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About This Trial
Background: CGD is caused by a gene mutation. For people with CGD, their cells cannot kill germs well, so they can get frequent or life-threatening infections. Researchers want to see if a new procedure can help a person s cells kill germs for a short time. It uses messenger RNA (mRNA) to deliver correct instructions for the gene mutation to the cells. Objective: To test a procedure in which mR…
Trial Locations
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Eligibility Criteria
* INCLUSION CRITERIA: Individuals must meet all of the following criteria to be eligible for study participation: * Males aged 18 to 75 years * CGD confirmed by DHR and deficiency subtype confirmed by protein analysis and/or genetic sequencing * Has a physician at home for follow-up care * Able to…
Contacts

Joanna L Peterson

(301) 346-9780

joanna.peterson@nih.gov

CONTACT

Suk S De Ravin, M.D.

(301) 496-6772

sderavin@mail.nih.gov

CONTACT