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

Part of paid clinical trials in Los Angeles, California.

Sponsor
New York Medical College
Study ID
NCT02675959
Phase
PHASE2
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
ALL
Age
6 Months - 34 Years
Healthy Volunteers
Not accepted

Interventions

  • Defibrotide — DRUG
    defibrotide will be given prophylactically prior to AlloSCT to determine if it decreases the incidence of SOS in this high risk population, and determine that it is safe and feasible to give along with myeloimmunoablative therapy and allogeneic transplant.

Study Details

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.

Key Dates

Start date
Jul 1, 2017
Status verified
May 2025
Primary completion
Dec 31, 2026
Completion
Dec 31, 2027

Study Design

Enrollment
40 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Defibrotide prophylaxis
    defibrotide will be given prior to and during myeloablative immunotherapy conditioning (MAIC) followed by familial haploidentical (FHI) allogeneic stem cell transplantation (AlloSCT) with CD34 enrichment and t-cell addback in patients with high-risk sickle cell disease or beta thalassemia to reduced the risk and rate of the development of sinusoidal obstructive syndrome (SOS).

Primary Outcome Measure

All patients will be monitored for known and unknown side effects of defibrotide with daily physical exams while in the hospital and then as needed in addition to daily laboratory values including chemistries, hematology labs as needed [ Time Frame: 100 days ]

Central Contacts

Locations (4)

FacilityCityStateZIPSite coordinators
University of California Los AngelesLos AngelesCalifornia90095
Theodore B Moore, MD
[email protected]
(310) 825-6708
Andres Vargas
[email protected]
310-794-8929
Theodore B Moore, MD (PRINCIPAL_INVESTIGATOR)
University of FloridaGainesvilleFlorida32610-0278
Jordan Milner, MD
[email protected]
Giselle Moore-Higgs, PhD
[email protected]
New York Medical CollegeValhallaNew York10595
Sandra Fabricatore, RN, PNP
[email protected]
914-594-2152
Erin Morris, RN
[email protected]
714-964-5359
Mitchell S Cairo, MD (PRINCIPAL_INVESTIGATOR)
Medical College of WisconsinMilwaukeeWisconsin53226
Julie A Talano, MD
[email protected]
414-955-4185
Isabella Puls
[email protected]
Julie A Talano, MD (PRINCIPAL_INVESTIGATOR)

Find similar trials in Los Angeles, CA

By condition
Sickle cell disease
By research site
University of California Los Angeles· Los Angeles, CAUniversity of Florida· Gainesville, FLNew York Medical College· Valhalla, NYMedical College of Wisconsin· Milwaukee, WI

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