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MUNC13.4 - A phase I/II Open Label non randomized study, monocentric, single arm, evaluating Safety and Efficacy of Gene Therapy of FHL 3 caused by mutations in the human UNC13D gene by transplantation of a single dose of autologous CD34+ cells transduced ex vivo with the UNC13D LV vector expressing the UNC13D cDNA
EU CTIS ID: 2023-507334-24-00
What this study is testing
assessing the initial safety of treatment with MUNC-CD34 and MUNC-T3, including the mobilisation procedure, conditioning regimen and transplantation with LV-EF1a-UNC13D lentiviral vector gene modified autologous hematopoietic stem cells combined with transduced autologous T-cell in Munc 13.4 deficient patients.
- Human Pharmacology (Phase I)- 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
- 1. Patient aged from 3 months up to 17 years old
- 2. Patient with a FHL caused by mutation of the UNC13D gene.
- 3. Complete remission is defined by the normalization of clinical and laboratory parameters:
- 4. Patient eligible for an allogeneic HSCT in absence of an HLA geno-identical donor (at diagnostic or after failure of a previous HSCT (rejection or loss of the graft))
- 5. Parental, guardian’s patient signed informed consent.
- 6. For patients of childbearing age : willing to use an effective method of contraception during the trial and for at least 12 months post-infusion
You likely can't join if
- 1) Active CNS encephalitis related to HLH
- 2) Existence of a matched –sibling donor
- 3) Unwillingness to return for follow-up during the 2 years study and lifelong for off study review.
- 4) HIV-1 or 2 or HTLV1 infections.
- 5) Patient on AME (state medical aid) (unless exemption from affiliation)
- 6) Pregnancy or breast feeding in a post-partum female
See the full eligibility criteria
- 1. Patient aged from 3 months up to 17 years old
- 2. Patient with a FHL caused by mutation of the UNC13D gene.
- 3. Complete remission is defined by the normalization of clinical and laboratory parameters:
- 4. Patient eligible for an allogeneic HSCT in absence of an HLA geno-identical donor (at diagnostic or after failure of a previous HSCT (rejection or loss of the graft))
- 5. Parental, guardian’s patient signed informed consent.
- 6. For patients of childbearing age : willing to use an effective method of contraception during the trial and for at least 12 months post-infusion
- 7. Affiliation to Social Security
- 1) Active CNS encephalitis related to HLH
- 2) Existence of a matched –sibling donor
- 3) Unwillingness to return for follow-up during the 2 years study and lifelong for off study review.
- 4) HIV-1 or 2 or HTLV1 infections.
- 5) Patient on AME (state medical aid) (unless exemption from affiliation)
- 6) Pregnancy or breast feeding in a post-partum female
- 7) Diagnosis of significant psychiatric disorder of the subject that could seriously impeded the ability to participate in the study
- 8) Known allergies, hypersensitivity, or intolerance to any of busulfan, fludarabine, rituximab, G-CSF, plerixafor or excipients, or similar compounds
- 9) Participation in another clinical study with an investigational drug within 30 days of inclusion.
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 all sexes, 0-17 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.