Minimizing Toxicity in HLA-identical Sibling Donor Transplantation for Children With Sickle Cell Disease

Part of paid clinical trials in Washington D.C., District of Columbia.

Sponsor
Robert Nickel
Study ID
NCT03587272
Phase
PHASE2
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
ALL
Age
2 Years - 25 Years
Healthy Volunteers
Not accepted

Interventions

  • Alemtuzumab, low dose total body irradiation, Sirolimus — DRUG
    The conditioning regimen (SUN regimen) will consist of alemtuzumab daily for 5 days (total dose 1 mg/kg) and low dose total body irradiation (TBI) 300 cGY on Day -2 with gonadal shielding if possible. The HSCT graft will be G-CSF mobilized PBSCs with minimum CD34+ of 5 x106/kg recipient weight, goal of 10 x 106/kg (no upper limit). A peripheral blood stem cell (PBSC) graft was selected as it engrafts faster than bone marrow and would lead to shorter period of neutropenia and infection and less thrombocytopenia and need for platelet transfusion. GVHD prophylaxis will be sirolimus with a loading dose 3 mg/m2 on day -1. Sirolimus will be continued at 1 mg/m2/day starting on day 0 and dose adjusted to maintain a target trough level 5-15 ng/mL for the first 3 months and 5-10 ng/mL for the remainder of the first year.

Study Details

This multisite prospective study seeks to determine if HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus (Sickle transplant Using a Nonmyeloablative approach, "SUN") can decrease the toxicity of transplant while achieving a high cure rate for children with sickle cell disease (SCD).

Key Dates

Start date
Apr 17, 2018
Status verified
Mar 2026
Primary completion
Nov 30, 2030
Completion
Nov 30, 2030

Study Design

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

Arms

  • Experimental: SUN regimen
    Alemtuzumab, low dose total body irradiation, Sirolimus HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus (Sickle transplant Using a Nonmyeloablative approach, "SUN") can decrease the toxicity of transplant while achieving a high cure rate for children with sickle cell disease (SCD).

Primary Outcome Measure

Acute GVHD [ Time Frame: 100 days post transplant ]

Central Contacts

Locations (5)

FacilityCityStateZIPSite coordinators
Children's National Health SystemWashington D.C.District of Columbia20010
Maryanne Odinakachukwu
[email protected]
202-476-2957
Fahmida Hoq, MBBS, MS
[email protected]
202-476-3634
Robert Nickel, MD (PRINCIPAL_INVESTIGATOR)
Ann & Robert H. Lurie Children's Hospital of ChicagoChicagoIllinois60611
Olga Jonas
[email protected]
312.227.4871
Ella Ramsey
[email protected]
Sonali Chaudhury, MD (PRINCIPAL_INVESTIGATOR)
Columbia UniversityNew YorkNew York10032
Jaclyn Dosik
[email protected]
212-305-0866
Monica Bhatia (PRINCIPAL_INVESTIGATOR)
Levine Children's HospitalCharlotteNorth Carolina28203
Janice Hollifield
[email protected]
980-442-2342
Michael Kent, MD (PRINCIPAL_INVESTIGATOR)
Nationwide Children's HospitalColumbusOhio43205
Hemalatha Rangarajan
[email protected]
614-355-1689
Hemalatha Rangarajan, MD (PRINCIPAL_INVESTIGATOR)

Find similar trials in Washington D.C., DC

By condition
Sickle cell disease
By research site
Children's National Health System· Washington D.C., DCAnn & Robert H. Lurie Children's Hospital of Chicago· Chicago, ILColumbia University· New York, NYLevine Children's Hospital· Charlotte, NCNationwide Children's Hospital· Columbus, OH

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