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Gene Therapy for X-Linked Chronic Granulomatous Disease

This study is testing a gene therapy called pCCLChimGp91lentiviral vector in people with X-Linked Chronic Granulomatous Disease (CGD). CGD is a condition where the immune system doesn't work properly due to a gene mutation. Researchers want to see if this gene therapy can help make the immune system more normal and reduce the risk of infections. The treatment involves taking your own stem cells, adding a normal gene to them, and then giving them back to you. The study will also use other medications like Busulfan (a conditioning drug), Tocilizumab (a monoclonal antibody), Eltrombopag (to help blood cell production), and Sirolimus (to prevent rejection after transplant). The main goals are to check the safety of the treatment and how well it works after 6 months and 1 year. You can join if you are between 3 and 60 years old and have a confirmed diagnosis of X-linked CGD.

Study design
This is a Phase I/II, non-randomized, single-site study that is open-label, meaning everyone knows what treatment is being given. It plans to enroll 10 participants.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
The study will measure how well the treatment works at 6 months and 1 year after the infusion.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT07113743

Part B- G1X-CGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease

Enrolling by Invitation
PHASE1Ages 3–60InterventionalTreatment
National Institute of Allergy and Infectious Diseases (NIAID)
~10 participants
Updated 2026-09-08 on ClinicalTrials.gov
What's tested:BusulfanTocilizumabEltrombopagSirolimuspCCLChimGp91lentiviral vector containing the human gp91 phox (CYBB) gene

At a glance

Recruiting sites
0 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Safety
Measured over Throughout the study
+1 more outcome measured

Conditions

Chronic Granulomatous Disease (CGD)

Explore related trials

X-linked Chronic Granulomatous DiseaseNADPH Oxidase Complex

NCT07113743

Where you'd take part

This study runs at 1 site. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • National Institutes of Health Clinical Center

    Bethesda, Marylandno site contact published

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Study leadership

  • Elizabeth M Kang, M.D. · PRINCIPAL_INVESTIGATOR · National Institute of Allergy and Infectious Diseases (NIAID)

Who to contact

This trial hasn't published a contact. View it on ClinicalTrials.gov

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Eligibility criteria

Inclusion

Must have confirmed molecular diagnosis of X-linked CGD confirmed by deoxyribonucleic acid (DNA) sequencing and supported by laboratory evidence for absent or reduction \>90% of the biochemical activity of the NADPH-oxidase.
At least 1 prior ongoing or refractory severe infection and/or inflammatory complications requiring hospitalization despite conventional therapy.
No 10/10 HLA-matched donor available after initial search of National Marrow Donor Program (NMDP) registries within the last year.
Must weigh at least 15 kg.
Male or female, and must be at least 3 years of age but no older than 60.
Parent/guardian must be willing to sign and date informed consent form for child and where appropriate, child may sign assent.
Stated willingness to comply with all study procedures and availability for the duration of the study.
Ability to take oral medication and be willing to adhere to the prophylactic regimen.
Apheresis of patients for the hematopoietic stem cells collected as a part of this protocol will be performed according to the Standard of Care apheresis practices established in the NIH CC Department of Transfusion Medicine or at their local facility. If performed at
For apheresis, pediatric patients:
Must weigh at least 15 kg body weight;
Preserved renal function (creatinine \<=2.5 mg/dL; \<=3+ proteinuria); preserved hepatic function (bilirubin \<=2.0 mg/dl);
Must be negative for co-infection with human immunodeficiency virus (HIV) or hepatitis B virus (HBsAg positive) or hepatitis C virus (HCV ribonucleic acid (RNA) positive), adenovirus, parvovirus B 19 or toxoplasmosis or mycobacterial infection (prior or current).
For females of reproductive potential, must agree to use of 2 highly effective contraception throughout study participation and for at least 3 months after the study.
For females:
Condoms, male or female, with or without a spermicide;
Diaphragm or cervical cap with spermicide;
Intrauterine device;
Contraceptive pills or patch, Norplant, Depo-Provera, or other FDA- approved contraceptive method;
For males of reproductive potential: use of condoms or other methods to ensure effective contraception with partner.
Agreement to adhere to Lifestyle Considerations throughout study duration.
Ability of subject (Patient/Legal Guardian) to understand and the willingness to sign a written informed consent document.
For the Natural History Protocol (05-I-0213): All patients must be willing to allow storage of blood samples for future studies.
Must provide a durable power of attorney (DPA) for health care decisions to an appropriate adult relative or guardian in accordance to NIH-200 "NIH Advance Directive for Health Care and Medical Research Participation."

Exclusion

Patient/Parent/Guardian unable or unwilling to comply with the protocol requirements.
Contraindication for leukapheresis (anemia Hb \<8 g/dl, cardiovascular instability, severe coagulopathy).
Patients who are unable to lie prone during the bone marrow harvesting procedure (in the case of bone marrow harvest, contraindication to general anesthesia).
Have a 10/10 HLA identical (A,B,C,DR,DQ) family or unrelated adult donor unless there is deemed to be an unacceptable risk associated with an allogeneic procedure.
Tested positive (definitive) for the presence of multiple types (2 or more) of anti-platelet antibodies.
Altered organ function as outlined below observed within 8 weeks of entering this trial.
Chemistry Lab abnormalities: Serum sodium \>= 156 mmol/L or \<= 129 mmol/L, potassium \>= 6.1 mmol/L or \<= 2.9 mmol/L, calcium \>= 3.2 mmol/L or \< 1.74 mmol/L , magnesium \>= 1.24 mmol/L or \< 0.39 mmol/L, phosphate \>= 5.1 mmol/L or \< 1.9 mmol/L.
Serum transaminases \> 5X the upper limit of normal (ULN).
General
Expected survival \< 6 months.
Major congenital anomaly.
Known allergic reactions to components of busulfan or dimethyl sulfoxide (DMSO) or contraindication for administration of conditioning medication.
Evidence of active malignant disease.
Treatment with another investigational drug or other intervention within 6 months.
Unable to undergo apheresis as per the NIH CC Department of Transfusion Medicine Standard of Care apheresis procedures.
Administration of gamma-interferon within 21 days before the infusion of transduced, autologous CD34+ cells.
Any other condition that, in the opinion of the Investigator, may compromise the safety or compliance of the patient or would preclude the patient from successful study completion.

What this trial measures

  • SafetyThroughout the study

    1\) The primary safety objective of this procedure will be assessed by recording the incidence of adverse events. a) Record clinical adverse events and clinically significant laboratory abnormalities. b) Evaluate overall incidence of adverse events for the study as a whole. c) Monitor the incidence of serious adverse events.

  • Efficacy6 months and 1 year

    The primary efficacy objective of this study will be determined by measuring the percentage of subjects who have \>= 10% oxidase positive granulocytes by DHR flow cytometry at month 6 and 12 after transplant.