p47Phase 1/2NCT06253507
pCCLCHIM-p47 (Lentiviral Vector Transduced CD34 Plus Cells) in Patients With p47 Autosomal Recessive Chronic Granulomatous Disease (AR-CGD)
A Phase 1/2 study testing Cryopreserved Autologous CD34+ cells transduced with pCCLCHIM-p47 in people with p47 and Autosomal Recessive, run by National Institute of Allergy and Infectious Diseases (NIAID) at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 3 to 65 years
- Conditions the study is for
- p47, Autosomal Recessive, Chronic Granulomatous Disease
- What is being tested?
- Cryopreserved Autologous CD34+ cells transduced with pCCLCHIM-p47
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- 25 Jun 2024to 31 Mar 2027
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- In order to be eligible to participate in this study, an individual must meet all of the following criteria:
- -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
- activity of the NADPH-oxidase.
- 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.
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- 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 or at their local facility. 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.
Who cannot join
- 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.
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- 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
- 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 21 days before the infusion of transduced, autologous CD34+ cells
- Acute infection diagnosed but not treated at time of enrollment. Patients will be delayed for enrollment until infection has resolved or if not resolving with adequate standard medical care, the patient will then be re-eligible for treatment as this would have them meeting the inclusion criteria of a refractory infection. (Patients that develop an infection during the conditioning [after enrollment and having started the busulfan] will proceed with the study.)
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
About this study
Official title: A Phase I/II, Non-Randomized, Open-Label Study of pCCLCHIM-p47 (Lentiviral Vector Transduced CD34+ Cells) in Patients With p47 Autosomal Recessive Chronic Granulomatous Disease (AR-CGD)
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.
Read more
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.
Read the detailed description
Study Description:
This is a phase I/II, non-randomized, open-label study of a single infusion of autologous CD34+ cells transduced ex vivo with pCCLCHIM-p47 in 5 patients with p47-AR CGD conditioned with high dose busulfan.
Objectives:
Primary Objectives:
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 at 6 months.
Secondary Objectives:
- Safety
- Long term engraftment (assessments at 1 year, 2 years, and 3 years)
- Event Free Survival
- Clinical Efficacy and Health Assessment
Exploratory Objectives:
- Assessment of biomarker utility
- Evaluation of the human microbiome during pCCLCHIM-p47 gene therapy.
- Patient reported assessment of efficacy (QOL)
Endpoints:
Primary Endpoint of this study will be engraftment of genetically modified cells as defined by presence of >10% oxidase positive cells at 6 months.
- Secondary Endpoints will consist of:
- Safety: assessed by recording of the incidence of adverse events.
- Record clinical adverse events and clinically significant laboratory abnormalities.
- Evaluate overall incidence of adverse events for the study as a whole.
- Monitor the incidence of serious adverse events.
- Measure overall survival at 3 years post-transplant
- Assessment of vector safety by vector insertion site analysis (VISA) and replication competent lentivector (RCL) testing.
- Long term multilineage engraftment, as measured by:
- Expression of p47 phox in neutrophils at 6, 12, 18, 24, and 36 months.
- Hematological reconstitution by 90 days post treatment (absolute neutrophil count >500 cells/microliter on three consecutive samples over at least ten days).
- NADPH Oxidase activity in monocytes by dihydrorhodamine (DHR) flow cytometry at 6, 12, 18, 24, and 36 months.
- Percentage of gene modified CD34+ hemopoietic cells in BM at 6, 12, 18, 24, 30, and 36 months as assessed by vector copy number (VCN) qPCR.
- VCN in cell lineages present in PB cells at months 1, 3, 6, 9, 12, 18, 24, and 36.
- Event free Survival
- Resolution of either underlying inflammation and/or infection related to P47 deficiency without recurrence for 3 years.
- Rate of severe (as defined by CTCAE v 5.0 - grade 3 to 5) CGD related infections after withdrawal of antimicrobial prophylaxis in time periods 6-12 and 6-36 months post treatment.
- Assessment of clinical efficacy and health by:
- The nutritional status (height, weight, serum albumin) at 36 months.
- Growth and development of pediatric subjects at 36 months.
- Frequency of severe infections (days requiring IV antibiotics and/or hospitalization for treatment) at 36 months.
- Presence of inflammatory complications (days requiring hospitalizations) at 36 months.
Tertiary Endpoints:
- Utility of Biomarker assessment
- Stool collection for microbiome analysis (optional)
- Patient Quality of life questionnaire collection
- Conditions
- p47, Autosomal Recessive, Chronic Granulomatous Disease
- Being tested
- Cryopreserved Autologous CD34+ cells transduced with pCCLCHIM-p47
- Keywords
- p47 Phox, Autosomal Recessive, Chronic Granulomatous Disease, Gene Therapy, Lenti Vector
- Medical subject headings
- Granulomatous Disease, Chronic, Granulomatous Disease, Chronic, Autosomal Recessive, Cytochrome B-Positive, Type I
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cryopreserved Autologous CD34+ cells transduced with pCCLCHIM-p47Biological
Cryopreserved autologous CD34+ cells transduced ex vivo with the pCCLCHIM-p47 vector containing the human p47phox (NCF1) gene in final formulation and container closure system, ready for intended medical use.
The minimum cell dose for infusion is 3 x 10^6 CD34+/kg.
| Study group | Role | What they receive |
|---|---|---|
| Single arm | Experimental | Not described Receives: Biological: Cryopreserved Autologous CD34+ cells transduced with pCCLCHIM-p47 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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: 6 months to 1 year.
Secondary outcomes (4)
Safety
assessed by recording of the incidence of adverse events for the study as a whole
Measured: Length of study
Long term Engraftment
Measured: 6, 12, 18, 24, and 36 months
Event Free Survival
Measured: 3 years
Clinical Efficacy and Health Assessment
Measured: 24 and 36 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Feb 2024
- Study start25 Jun 2024
- Primary completion31 Mar 2027 (estimated)
- Study completion31 Mar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for p47Studies of the same condition, open and under way.
- More trials run by National Institute of Allergy and Infectious Diseases (NIAID)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Elizabeth M Kang, M.D., Principal investigator, National Institute of Allergy and Infectious Diseases (NIAID)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06253507
- Sponsor's protocol ID
- 10001562
- Other IDs
- 001562-I
- Status last verified
- 20 Oct 2026
- First submitted
- 9 Feb 2024
- Last updated
- 3 Nov 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II, Non-Randomized, Open-Label Study of pCCLCHIM-p47 (Lentiviral Vector Transduced CD34+ Cells) in Patients With p47 Autosomal Recessive Chronic Granulomatous Disease (AR-CGD)
- ClinicalTrials.gov ID
- NCT06253507
- Acronym / sponsor ID
- 10001562
- Conditions
- p47, Autosomal Recessive, Chronic Granulomatous Disease
- Keywords
- p47 Phox, Autosomal Recessive, Chronic Granulomatous Disease, Gene Therapy, Lenti Vector
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Enrolling by invitation
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)US National Institutes of Health
- Study start
- 25 Jun 2024 (actual)
- Primary completion
- 31 Mar 2027 (estimated)
- Study completion
- 31 Mar 2027 (estimated)
- First posted
- 12 Feb 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Study Description:
This is a phase I/II, non-randomized, open-label study of a single infusion of autologous CD34+ cells transduced ex vivo with pCCLCHIM-p47 in 5 patients with p47-AR CGD conditioned with high dose busulfan.
Objectives:
Primary Objectives:
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 at 6 months.
Secondary Objectives:
- Safety
- Long term engraftment (assessments at 1 year, 2 years, and 3 years)
- Event Free Survival
- Clinical Efficacy and Health Assessment
Exploratory Objectives:
- Assessment of biomarker utility
- Evaluation of the human microbiome during pCCLCHIM-p47 gene therapy.
- Patient reported assessment of efficacy (QOL)
Endpoints:
Primary Endpoint of this study will be engraftment of genetically modified cells as defined by presence of >10% oxidase positive cells at 6 months.
- Secondary Endpoints will consist of:
- Safety: assessed by recording of the incidence of adverse events.
- Record clinical adverse events and clinically significant laboratory abnormalities.
- Evaluate overall incidence of adverse events for the study as a whole.
- Monitor the incidence of serious adverse events.
- Measure overall survival at 3 years post-transplant
- Assessment of vector safety by vector insertion site analysis (VISA) and replication competent lentivector (RCL) testing.
- Long term multilineage engraftment, as measured by:
- Expression of p47 phox in neutrophils at 6, 12, 18, 24, and 36 months.
- Hematological reconstitution by 90 days post treatment (absolute neutrophil count >500 cells/microliter on three consecutive samples over at least ten days).
- NADPH Oxidase activity in monocytes by dihydrorhodamine (DHR) flow cytometry at 6, 12, 18, 24, and 36 months.
- Percentage of gene modified CD34+ hemopoietic cells in BM at 6, 12, 18, 24, 30, and 36 months as assessed by vector copy number (VCN) qPCR.
- VCN in cell lineages present in PB cells at months 1, 3, 6, 9, 12, 18, 24, and 36.
- Event free Survival
- Resolution of either underlying inflammation and/or infection related to P47 deficiency without recurrence for 3 years.
- Rate of severe (as defined by CTCAE v 5.0 - grade 3 to 5) CGD related infections after withdrawal of antimicrobial prophylaxis in time periods 6-12 and 6-36 months post treatment.
- Assessment of clinical efficacy and health by:
- The nutritional status (height, weight, serum albumin) at 36 months.
- Growth and development of pediatric subjects at 36 months.
- Frequency of severe infections (days requiring IV antibiotics and/or hospitalization for treatment) at 36 months.
- Presence of inflammatory complications (days requiring hospitalizations) at 36 months.
Tertiary Endpoints:
- Utility of Biomarker assessment
- Stool collection for microbiome analysis (optional)
- Patient Quality of life questionnaire collection
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single arm | Experimental | Biological: Cryopreserved Autologous CD34+ cells transduced with pCCLCHIM-p47 |
Who may qualify
- Ages
- 3 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (25 listed)
- In order to be eligible to participate in this study, an individual must meet all of the following criteria:
- -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
- activity of the NADPH-oxidase.
- Must weigh at least 15 kg.
- Adult Patient must be willing to sign and date informed consent form.
Key exclusion criteria (40 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Cryopreserved Autologous CD34+ cells transduced with pCCLCHIM-p47Cryopreserved autologous CD34+ cells transduced ex vivo with the pCCLCHIM-p47 vector containing the human p47phox (NCF1) gene in final formulation and container closure system, ready for intended medical use. The minimum cell dose for infusion is 3 x 10^6 CD34+/kg. | Biological | Single arm |
Primary outcome
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.
Time frame: 6 months to 1 year.
4 secondary outcomes
Safety
Time frame: Length of study
Long term Engraftment
Time frame: 6, 12, 18, 24, and 36 months
Event Free Survival
Time frame: 3 years
Clinical Efficacy and Health Assessment
Time frame: 24 and 36 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Nov 2026.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
- Individual participant dataSharing planned: No
Related trials for p47
RecruitingPhase 1/2
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A Study of Selpercatinib (LOXO-292) in Participants With Advanced Solid Tumors, RET Fusion-Positive Solid Tumors, and Medullary Thyroid Cancer (LIBRETTO-001)
Non-Small Cell Lung Cancer, Medullary Thyroid Cancer
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A Study of Oral LOXO-292 (Selpercatinib) in Pediatric Participants With Advanced Solid or Primary Central Nervous System (CNS) Tumors
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RecruitingPhase 1/2
Lentiviral Gene Therapy for p47 AR-CGD
P47-Phox, Deficiency of
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Elizabeth M Kang, M.D. principal investigator
National Institute of Allergy and Infectious Diseases (NIAID)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Cryopreserved Autologous CD34+ cells transduced with pCCLCHIM-p47 in people with p47 and Autosomal Recessive, run by National Institute of Allergy and Infectious Diseases (NIAID) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 25 inclusion and 40 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: enrolling by invitation, as of the sponsor's last update on 3 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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. (time frame: 6 months to 1 year.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Nov 2026.