Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease

Phase 2 Recruiting 40 enrolled New York Medical College Baylor College of Medicine · collab Children's Hospital Los Angeles · collab Dana-Farber Cancer Institute · collab Johns Hopkins University · collab Medical College of Wisconsin · collab Tufts Medical Center · collab University of California, Los Angeles · collab

NaSingle-groupOpen-labelTreatment

Summary

This is a follow-up trial to NYMC 526 (NCT01461837) to assess the safety, efficacy and toxicity of administering Defibrotide prophylaxis for high-risk sickle cell or beta thalassemia patients undergoing a familial haploidentical allogeneic stem cell transplantation with CD34 enrichment and T-cell addback. This patient population historically has a risk of developing sinusoidal obstructive syndrome (SOS) and Defibrotide has demonstrated efficacy in treatment of SOS. The Funding Source is FDA OOPD.

Timeline

Start: 2017-07-01
Primary completion: 2026-12
Completion: 2027-12

Publications

Chu Y, Talano JA, Baxter-Lowe LA, Verbsky JW, Morris E, Mahanti H, Ayello J, Keever-Taylor C, Johnson B, Weinberg RS, Shi Q, Moore TB, Fabricatore S, Grossman B, van de Ven C, Shenoy S, Cairo MS. Donor chimerism and immune reconstitution following haploidentical transplantation in sickle cell disease. Front Immunol. 2022 Dec 9;13:1055497. doi: 10.3389/fimmu.2022.1055497. eCollection 2022.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Defibrotide	Oligonucleotide (other)	—	—

Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease

Summary

Timeline

Publications

Drugs

Indications