Depleted Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia After Being Conditioned With a Regimen Containing Briquilimab

Part of paid clinical trials in Stanford, California.

Sponsor
Porteus, Matthew, MD
Study ID
NCT04784052
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

  • Fanconi Anemia

Eligibility Criteria

Sex
ALL
Age
2 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • JSP191 — DRUG
    Participants will receive a single IV dose at start of conditioning
  • CliniMACS Prodigy System — DEVICE
    The device used to remove the αβ+T cells from donor stem cell transplant before being given to the recipient
  • Depleted Stem Cell Transplant — BIOLOGICAL
    TCRαβ+ T-cell/CD19+ B-cell depleted hematopoietic cells will be administered by IV after completion of conditioning regimen.
  • Rabbit Anti-Thymoglobulin (rATG) — BIOLOGICAL
    3 consecutive daily doses of rATG will be given by IV during conditioning
  • Cyclophosphamide — DRUG
    4 consecutive daily doses of cyclophosphamid will be given by IV during conditioning
  • Fludarabine — DRUG
    4 consecutive daily doses of fludarabine will be given by IV during conditioning
  • Rituximab — DRUG
    1 dose of rituximab will be given at the end of conditioning

Study Details

The objective of this clinical trial is to develop a cell therapy for Fanconi Anemia which enables enhanced donor hematopoietic and immune reconstitution with decreased toxicity by transplanting depleted stem cells from a donor with and without using an experimental antibody treatment called JSP-191 as a part of conditioning. This experimental treatment will hopefully cause fewer side effects than chemotherapy (the current standard of care method). Participants will be administered the conditioning regimen, are assessed until they receive the depleted stem cell infusion, and will be followed for up to 2 years after the cell infusion.

Key Dates

Start date
Dec 7, 2021
Status verified
Jan 2026
Primary completion
Dec 31, 2027
Completion
Dec 31, 2028

Study Design

Enrollment
18 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Depleted Stem Cell Transplant with JSP-191 Conditioning
    Participants will receive an infusion of donor stem cells which have been depleted of αβ+T cells using the CliniMACS System device. Before the stem cell transplant, they will receive a reduced-intensity preparative regimen containing JSP191 in combination with rATG, cyclophosphamide, fludarabine and rituximab.
  • Experimental: Depleted Stem Cell Transplant without JSP-191 Conditioning
    Participants will receive an infusion of donor stem cells which have been depleted of αβ+T cells using the CliniMACS System device. Before the stem cell transplant, they will receive a reduced-intensity preparative regimen containing rATG, cyclophosphamide, fludarabine and rituximab.

Primary Outcome Measure

Number of participants without grade 3 and 4 treatment-emergent adverse events (TEAEs) (infusion related reactions). [ Time Frame: From start of conditioning regimen administration until cell infusion (up to 30 days) ]

Locations (1)

FacilityCityStateZIPSite coordinators
Stanford UniversityStanfordCalifornia94305
Rajni Agarwal, MD
[email protected]
650-725-9250

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By research site
Stanford University· Stanford, CA

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