pCCLCHIM-p47 (Lentiviral Vector Transduced CD34 Plus Cells) in Patients With p47 Autosomal Recessive Chronic Granulomatous Disease (AR-CGD)

Enrolling by invitation · Phase 1/Phase 2

Conditions studied: p47, Autosomal Recessive, Chronic Granulomatous Disease

In brief

Background: Chronic granulomatous disease (CGD) is a genetic disorder. People with CGD are missing a gene that affects their white blood cells. White cells are part of the immune system, and people with GCD are vulnerable to many infections. Researchers want to test a new treatment to replace the missing gene that may be safer than the current treatment for CGD. Objective: To test a new type of gene therapy in people with CGD. Eligibility: People aged 3 years or older with CGD. Design: Participants will undergo apheresis: Blood will be collected through a tube attached to a needle inserted in a vein; the blood will run through a machine that separates certain cells (stem cells); the remaining blood will be returned to the body through a second needle. The participant s stem cells will be modified in a laboratory to add the gene they are missing. Participants will stay in the hospital for about 40 days. For the first 10 days, they will undergo many exams, including imaging scans and tests of their heart and lung function. They will receive drugs to prepare their bodies for the gene therapy. They will receive a "central line": A hollow tube will be inserted into a vein in the chest, with a port opening above the skin. This port will be used to draw blood and administer drugs without the need for new needle sticks. For the gene therapy, each participant s own modified stem cells will be put into their body through the port. Participants will have 8 follow-up visits over 3 years.

Key facts

Study ID

NCT06253507

Run by

National Institute of Allergy and Infectious Diseases (NIAID)

People needed

10

Starts

2024-06-25

Expected to finish

2027-03-31

Last updated by the study team

2026-02-23

Who can join

Age: 3 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

You may not qualify if…

• An individual who meets any of the following criteria will be excluded from participation in this study:
• Patient/Parent/Guardian unable or unwilling to comply with the protocol requirements.
• Contraindication for leukapheresis (anemia Hb <8g/dL, cardiovascular instability, severe coagulopathy, uncontrolled seizure disorder).
• Pregnancy or lactation.
• Tested positive (definitive) for the presence of multiple types (2 or more) of anti-platelet antibodies.
• Patient will be excluded if they have any of the following within 8 weeks of entering the trial.
• Hematologic
• Anemia (hemoglobin < 8 g/dL).
• Neutropenia (absolute granulocyte count <1,000/mm3)
• Thrombocytopenia (platelet count < 100,000/mm3).
• Prothrombin Time (PT) or Partial thromboplastin time (PTT) > 2 X the upper limits of normal (ULN) (Patients with a correctable deficiency controlled on medication will not be excluded).
• Cytogenetic abnormalities known to be associated with hematopoietic defect on peripheral blood or bone marrow.
• Infectious
• --Evidence of infection with HIV-1 and -2, or active Hepatitis B, Hepatitis C, Adenovirus, Parvovirus, Toxoplasmosis, or any other uncontrolled viral infection.
• Pulmonary
• --Resting O2 saturation by pulse oximetry < 90% on room air.
• Cardiac
• Ejection fraction by Echocardiogram of less than 40%
• OR
• --Uncorrected congenital cardiac malformation with clinical symptomatology
• OR
• Abnormal EKG which with additional work up (including a Cardiology consult) indicates cardiac pathology incompatible with the use of high dose busulfan.
• -Hepatic
• Transaminases >5X upper limit of normal.
• General

Where it is running

• National Institutes of Health Clinical Center — Bethesda, Maryland, United States

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.