NCT06253507
pCCLCHIM-p47 (Lentiviral Vector Transduced CD34 Plus Cells) in Patients With p47 Autosomal Recessive Chronic Granulomatous Disease (AR-CGD)
Enrolling by Invitation
PHASE1Ages 3–65InterventionalTreatmentNational Institute of Allergy and Infectious Diseases (NIAID)Government-funded
~10 participants
Updated 2026-02-23 on ClinicalTrials.gov
What's tested:Cryopreserved Autologous CD34+ cells transduced with pCCLCHIM-p47
At a glance
Recruiting sites
0 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
To evaluate the efficacy of pCCLCHIM-p47 transduced autologous CD34+ cells treatment in p47 AR-CGD patients as measured by engraftment of genetically modified cells.
Measured over 6 months to 1 year.
Conditions
Where it's being run
1 sites across 1 statesMaryland1
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
Do you actually qualify for this trial?
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Eligibility criteria
Inclusion
Must have confirmed genetic diagnosis of p47 AR-CGD by identification of a mutation in the responsible genes or protein analysis demonstrating lack of P47 expression and supported by laboratory evidence for absent or reduction \> 95% of the biochemical
Must weigh at least 15 kg.
Adult Patient must be willing to sign and date informed consent form.
Parent/guardian must be willing to sign and date informed consent form for child and where appropriate, child may sign assent.
State willingness to comply with all study procedures and availability for the duration of the study.
Male or female must be at least 3 years of age.
Do not have an appropriately HLA matched donor (related or unrelated)
Must have had at least a history of a prior CGD related infection and/or an ongoing/refractory severe infection requiring hospitalization and/or inflammatory complications requiring hospitalization despite conventional therapy.
Patients will be collected as per standard of care mobilization and apheresis procedures as performed at the Clinical Center. Patients will have consented onto the gene therapy study prior to collection; however products collected prior to the study for other protocols may be used as part of the back up.
Patient must weigh at least 15 kilograms body weight.
Normal female donors of childbearing potential may be entered if using effective contraception and having a negative serum pregnancy test within one week of beginning G-CSF administration.
Ability to take oral medication and be willing to adhere to the prophylactic regimen.
For females of reproductive potential, must agree to use 2 forms of highly effective contraception throughout study participation (for a minimum of 3 months after having received the genetically modified cells but preferably for the first 2 years.)
For females (will be counseled on appropriate combinations for best efficacy):
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 FDAapproved contraceptive method.
Having a male partner who has previously undergone a vasectomy.
For males of reproductive potential: use of condoms or other methods to ensure effective contraception prevention with partner.
Agreement to adhere to Lifestyle Considerations throughout study duration.
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".
All patients must be willing to allow storage of blood samples for gene therapy studies.
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.
Exclusion
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%
Uncorrected congenital cardiac malformation with clinical symptomatology
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
Expected survival \< 6 months.
Major congenital anomaly.
Contraindication for administration of conditioning medication.
Current or active malignancy or prior hematologic malignancy.
Uncontrolled hypertension (systolic greater than 1 SD of the mean expected for age despite adequate medication).
Uncontrolled tachycardia with resting heart rate greater than 1 standard deviation for age group despite medications.
Neurologic deficits determined to interfere with ability to comply with study interventions.
Known serious or anaphylactic allergic reactions to components of the Busulfan or to DMSO.
Treatment with another investigational drug or other intervention within 6 months.
Unable to undergo apheresis:
Patients who are hemodynamically unstable (systolic or diastolic blood pressure fall of 20 mm Hg from the stable patient s baseline measurement) or requiring mechanical respiratory assistance are excluded.
History of vasculitis or any illness that precludes apheresis as defined by the Cellular Engineering Department.
Administration of gamma-interferon within 30 days before the infusion of transduced, autologous CD34+ cells.
What this trial measures
- To evaluate the efficacy of pCCLCHIM-p47 transduced autologous CD34+ cells treatment in p47 AR-CGD patients as measured by engraftment of genetically modified cells.6 months to 1 year.