Granulomatous disease, chronic, autosomal recessive, cytochrome b-positive, type 1
MONDO:0009309Any chronic granulomatous disease in which the cause of the disease is a mutation in the NCF1 gene.
Also known as: NCF1 chronic granulomatous disease, chronic granulomatous disease 1, autosomal recessive, chronic granulomatous disease caused by mutation in NCF1, granulomatous disease, chronic, autosomal recessive, cytochrome b-positive, type 1, CDG1, CGD, autosomal recessive cytochrome B-positive, type 1, Ncf1, deficiency of, Soc2, deficiency of
45 clinical trials for this condition and its sub-types, 3 tagged with Granulomatous disease, chronic, autosomal recessive, cytochrome b-positive, type 1 itself.
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New gene therapy aims to fix immune system in rare disease
Disease control By invitation onlyThis study tests a new gene therapy for people with chronic granulomatous disease (CGD), a genetic disorder that weakens the immune system and makes them prone to serious infections. The treatment takes the patient's own blood stem cells, adds a missing gene in the lab, and puts …
Phase 1/2 • Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) • Aim: Disease control
Last updated Sep 17, 2026 00:00 UTC
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New shot could help Immune-Compromised patients fight infections
Disease control OngoingThis phase 3 trial is testing a new medicine called Newnorm, a concentrated antibody solution given as a shot under the skin, in 50 people with primary immunodeficiency (a condition where the immune system doesn't work properly). The goal is to see if Newnorm can prevent serious …
Phase 3 • Sponsor: Octapharma • Aim: Disease control
Last updated Aug 08, 2026 00:03 UTC
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New antibody therapy aims to shield kids with rare immune disease from deadly infections
Disease control OngoingThis study tests a medicine called Kedrion IVIG 10% in 30 children with primary immunodeficiency, a condition where the immune system doesn't work properly. The treatment is made from donated antibodies and is given through a vein to help prevent serious bacterial infections. Res…
Phase 3 • Sponsor: Kedrion S.p.A. • Aim: Disease control
Last updated Jun 27, 2026 13:00 UTC
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Quick-Push immune therapy studied for safety and ease
Disease control OngoingThis study looks at whether giving Cutaquig® (a medicine that helps fight infections) by a faster manual push method is safe and easy for adults with primary or secondary immunodeficiency. About 36 participants will use the rapid push method at home. The goal is to see if this ap…
Sponsor: University of Alberta • Aim: Disease control
Last updated Jun 27, 2026 12:31 UTC
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New antibody therapy tested for long-term safety in immune-deficient patients
Disease control OngoingThis study tests the long-term safety of TAK-881, a subcutaneous immunoglobulin treatment, in 53 adults and children with primary immunodeficiency diseases (PIDD) who completed a prior study. Participants receive regular infusions every 2-4 weeks and are monitored for side effect…
Phase 3 • Sponsor: Takeda • Aim: Disease control
Last updated Jun 27, 2026 12:05 UTC
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Groundbreaking gene therapy aims to fix immune system in rare disease
Disease control By invitation onlyThis early-stage trial tests a new gene therapy (PM359) for people with a rare immune disorder called chronic granulomatous disease (CGD). The treatment takes a patient's own blood stem cells, fixes a specific genetic error using a precise editing tool called prime editing, and r…
Phase 1/2 • Sponsor: Prime Medicine, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:04 UTC
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Scientists collect skin cells to pave way for future immune disease cures
Knowledge-focused By invitation onlyThis study collects small skin samples from people with primary immunodeficiency or immune regulation disorders, as well as from healthy volunteers. Researchers will use these cells to create cell lines for laboratory research. The goal is to better understand immune diseases and…
Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) • Aim: Knowledge-focused
Last updated Sep 02, 2026 00:00 UTC