Phase 1b/2a Trial of Allogeneic HSCT From an HLA-partially Matched Related or Unrelated Donor Aft… (NCT06986382) | Clinical Trial Compass
Not Yet RecruitingPhase 1/2
Phase 1b/2a Trial of Allogeneic HSCT From an HLA-partially Matched Related or Unrelated Donor After TCRab+ T-cell/CD19+ B-cell Depletion for Patients With Monogenic and/or Early-onset Medically Refractory Crohn Disease
United States14 participantsStarted 2025-07
Plain-language summary
This research study is investigating whether alpha beta T-cell depleted hematopoietic stem cell transplant (HSCT) can be an immune system replacement for Crohn disease patients and whether this is safe and effective for patients with early onset, medically refractory Crohn disease.
Who can participate
Age range2 Years – 30 Years
SexALL
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Inclusion criteria
✓. history of corticosteroid-dependence despite trials of at least two biologic or small molecule therapies of different mechanisms,
✓. significant toxicity, or adverse effects related to such medical therapy;
✓. Disease not amenable to surgical therapy without risk of short bowel syndrome or permanent ileostomy;
✓. Requirement for long-term parenteral nutrition;
✓. Intolerable extraintestinal symptoms (e.g., arthritis, dermatitis); iii. Presence of any of the following features associated with high genetic contribution to disease:
Exclusion criteria
✕. Ulcerative colitis
✕. CD and associated extraintestinal manifestations responsive to medical therapy without corticosteroid-dependence or significant toxicity or adverse effects
✕. Known or suspected functionally deleterious mutation in a gene that meets either of the following expression criteria:
✕. Specifically expressed in epithelial or stromal cells, but not expressed in lymphohematopoietic cells (e.g., TTC7A)
✕. Expected to be more functionally deleterious in cell types other than lymphohematopoietic cells than in lymphohematopoietic cell types
✕. Active hemophagocytic lymphohistiocytosis (HLH). Patients with a history of hemophagocytic lymphohistiocytosis (HLH) are eligible, if there is no current clinical, histological, or biochemical evidence of HLH activity.
What they're measuring
1
Number of patients with myeloid engraftment
Timeframe: Day 42 post-HSCT
2
Number of patients with Grade III-IV acute graft vs host disease (GVHD)
Timeframe: Day 90 and 180 post-HSCT
3
Number of patients in remission of Crohn disease (Calprotectin)
Timeframe: 2 years post-HSCT
4
Number of patients in remission of Crohn disease (wPCDAI or CDAI)