BridgeMD
New treatment option for P47-Phox, Deficiency of
Official title Lentiviral Gene Therapy for p47 AR-CGD
ClinicalTrials.gov ID: NCT05207657
What this study is testing
What is Lentiviral vector transduced CD34+ cells?
Lentiviral vector transduced CD34+ cells is an investigational medicine, being studied as a potential treatment for p47-phox, deficiency of.
Also referred to as p47LV transduced autologous CD34+ cells.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- Chronic Granulomatous Disease (CGD) is a rare inherited disorder in which patients suffer from severe infection and inflammation. The first indications of disease usually appear in early childhood.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 23 and older
You may be able to join if
- p47 AR-CGD patients \> 23 months of age
- Molecular diagnosis confirmed by Deoxyribonucleic acid (DNA) sequencing and supported by laboratory evidence for absent or reduction \> 95% of the...
- At least one prior, ongoing or refractory severe infection and/or inflammatory complications requiring hospitalisation despite conventional therapy
- No 10/10 human leukocyte antigen (HLA)-matched donor available after initial search of National Marrow Donor Program (NMDP) registries performed...
- No co-infection with Human Immunodeficiency Virus (HIV) or hepatitis B virus (HBsAg positive) or hepatitis C virus (HCV ribonucleic acid (RNA)...
You likely can't join if
- Age ≤ 23 months or \> 35 kg body weight
- 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...
- Contraindication for leukapheresis (Haemoglobin \<8g/dl, cardiovascular instability, severe coagulopathy)
- Appropriate organ function as outlined below must be observed within 8 weeks of entering this trial. a) Haematologic i) Anaemia (hemoglobin \ 2 X the...
- General
- Expected survival \< 6 months.
See the full eligibility criteria
- p47 AR-CGD patients \> 23 months of age
- Molecular diagnosis confirmed by Deoxyribonucleic acid (DNA) sequencing and supported by laboratory evidence for absent or reduction \> 95% of the biochemical activity of the NAHPD-oxidase
- At least one prior, ongoing or refractory severe infection and/or inflammatory complications requiring hospitalisation despite conventional therapy
- No 10/10 human leukocyte antigen (HLA)-matched donor available after initial search of National Marrow Donor Program (NMDP) registries performed within the last year
- No co-infection with Human Immunodeficiency Virus (HIV) or hepatitis B virus (HBsAg positive) or hepatitis C virus (HCV ribonucleic acid (RNA) positive), Cytomegalovirus (CMV), adenovirus, parvovirus B 19 or...
- Written informed consent for adult patient
- Parental/guardian and, where appropriate, child's signed consent/assent
- Age ≤ 23 months or \> 35 kg body weight
- 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
- Contraindication for leukapheresis (Haemoglobin \<8g/dl, cardiovascular instability, severe coagulopathy)
- Appropriate organ function as outlined below must be observed within 8 weeks of entering this trial. a) Haematologic i) Anaemia (hemoglobin \ 2 X the upper limits of normal (ULN) (patients with a correctable deficiency...
- General
- Expected survival \< 6 months.
- Major congenital anomaly.
- Ineligible for autologous Haematopoietic Stem Cell Transplant (HSCT) by the criteria at the clinical site.
- Contraindication for administration of conditioning medication
- Known sensitivity to Busulfan
- Administration of gamma-interferon within 30 days before the infusion of transduced, autologous CD34+ cells
- Participation in another experimental therapeutic protocol within 6 months prior to baseline and during the study period
- 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
- Patient/Parent/Guardian unable or unwilling to comply with the protocol requirements.
The study team makes the final eligibility decision.
Where it's taking place
- London, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 23 months and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include London, United Kingdom. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.