BridgeMD
A phase I/II, non randomized, monocentric open-label study of autologous CD34+ cells transduced with the G1XCGD lentiviral vector in patients with X-Linked Chronic Granulomatous Disease
EU CTIS ID: 2024-512790-27-00
What this study is testing
Evaluation of safety and efficacy by biochemical and functional reconstitution in progeny of engrafted cells and stability at 12 months.
- Phase I and Phase II (Integrated)- First administration to humans
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- a. Male X-CGD patients >23 months of age. Youngest patients (>1 month and ≤ 23 months) may be enrolled at physician’s appreciation; in this case mobilization of peripheral HSC may be replaced by two bone marrow harvests.
- b. Molecular diagnosis confirmed by DNA sequencing and supported by laboratory evidence for absent or reduction > 70% of the biochemical activity of the NAHPD-oxidase.
- c. At least one ongoing or resistant or at high risk of relapse severe infection and/or inflammatory complications requiring hospitalisation despite conventional therapy.
- d. No HLA-matched donor available after 3 months search, unless the risk of waiting for a potential match or for performing an allogeneic transplant is considered unacceptable.
- e. No co-infection with Human Immunodeficiency Virus (HIV) or hepatitis B virus (HBs Ag positive) or hepatitis C virus (anti-HCV Ab positive).
- f. written informed consent for adult patient.
You likely can't join if
- a. 10/10 HLA identical (A, B, C, DR, DQ) family or unrelated.
- b. Contraindication for leukapheresis (anaemia Hb <8g/dl, cardiovascular instability, severe coagulopathy).
- c. Contraindication for administration of conditioning medication and any component of the Investigational Medicinal Product (IMP) preparation.
- d. Administration of gamma interferon within 30 days before the infusion of transduced autologous CD34+ cells.
- e. Tested positive (definitive) for the presence of multiple types (2 or more) of anti-platelet antibodies
- f. Tested positive (definitive) for the presence of anti-HLA (Class I & II) antibodies.
See the full eligibility criteria
- a. Male X-CGD patients >23 months of age. Youngest patients (>1 month and ≤ 23 months) may be enrolled at physician’s appreciation; in this case mobilization of peripheral HSC may be replaced by two bone marrow harvests.
- b. Molecular diagnosis confirmed by DNA sequencing and supported by laboratory evidence for absent or reduction > 70% of the biochemical activity of the NAHPD-oxidase.
- c. At least one ongoing or resistant or at high risk of relapse severe infection and/or inflammatory complications requiring hospitalisation despite conventional therapy.
- d. No HLA-matched donor available after 3 months search, unless the risk of waiting for a potential match or for performing an allogeneic transplant is considered unacceptable.
- e. No co-infection with Human Immunodeficiency Virus (HIV) or hepatitis B virus (HBs Ag positive) or hepatitis C virus (anti-HCV Ab positive).
- f. written informed consent for adult patient.
- g. Parental/guardian and where appropriate child’s signed consent/assent.
- a. 10/10 HLA identical (A, B, C, DR, DQ) family or unrelated.
- b. Contraindication for leukapheresis (anaemia Hb <8g/dl, cardiovascular instability, severe coagulopathy).
- c. Contraindication for administration of conditioning medication and any component of the Investigational Medicinal Product (IMP) preparation.
- d. Administration of gamma interferon within 30 days before the infusion of transduced autologous CD34+ cells.
- e. Tested positive (definitive) for the presence of multiple types (2 or more) of anti-platelet antibodies
- f. Tested positive (definitive) for the presence of anti-HLA (Class I & II) antibodies.
- g. Participation in another experimental therapeutic protocol within 6 months prior to baseline and during the study period.
- h. 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 completion of the study.
- i. Patient/Parent/Guardian unable or unwilling to comply with the protocol requirements
The study team makes the final eligibility decision.
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 male, 0-17 years, 18-64 years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.