HAPLOIDENTICAL HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR CHILDREN WITH SICKLE CELL DISEASE AND THALASSEMIA USING CD34+ POSITIVE SELECTED GRAFTS

Study point of contact

Fahmida Hoq, MBBS, MS
2024763634
[email protected]
Allistair Abraham, MD
2024765772
[email protected]

Locations

1 United States site

Age

< 22 Years

Phase

Phase 1

Study type

Interventional

Gender

All

Interventions

Drug

Compensation

Unknown

About the study

The study is designed as a Pilot/Phase 1 trial of reduced intensity Haploidentical HSCT in
patients with sickle cell disease and thalassemia. The purpose of the study is to assess the
safety and toxicity of reduced intensity conditioning haploidentical hematopoietic stem cell
transplantation.

participation requirements

– First allogeneic transplant

– Age up to 22 years

– Patients with severe sickle cell disease (stroke, elevated TCD velocities, >2 acute
chest syndrome, ongoing chronic red cell transfusion > 6 months)

– Patients with transfusion dependent thalassemia and evidence of iron overload

– Patients must have a related donor that is HLA-matched at >/=4 of 8 but <8/8 HLA-A, -B, -C and -DRB1 - Cardiac function: Shortening fraction >25%; ejection fraction >40%

– Estimated creatinine clearance greater than 50 mL/minute

– Pulmonary function: DLCO ≥40% (adjusted for hemoglobin) and FEV1≥50% in patients 7
years and older with normal cognitive function and able to perform the test
adequately. If not able to complete the testing a CT chest will be required., oxygen
saturation>91%

– Liver function: direct (conjugated) bilirubin < 2x the upper limit of normal and ALT/AST < 2.5x the upper normal limit. - Signed informed consent.

participation restrictions

– Life expectancy less than 6 months

– Patients with uncontrolled bacterial, viral or fungal infections (undergoing
appropriate treatment and with progression of clinical symptoms) within 1 month prior
to conditioning. Patients with febrile illness or suspected minor infection should
await clinical resolution prior to starting conditioning.

– Pregnant or breastfeeding patients

– Patients seropositive for the human immunodeficiency virus (HIV)

– Patient with active Hepatitis B or C determined by serology and/or NAAT

– Active hepatitis, bridging fibrosis or cirrhosis on liver biopsy (biopsy required for
patients on chronic transfusion therapy for > 1 year and evidence of iron overload
with ferritin >1000 ng/mL)

– Patients with suitable 8/8 HLA matched related and unrelated donors

– Patients who have an intolerance to or have received alemtuzumab in the prior 6 months
will be excluded from enrollment unless alemtuzumab is replaced with rabbit ATG in the
conditioning regimen

Locations

  • Washington, District of Columbia, United States, Childrens National Medical Center, 20010 [Recruiting]