NCT02165007CompletedPhase 1
Haploidentical Hematopoietic Stem Cell Transplantation
HAPLOIDENTICAL HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR CHILDREN WITH SICKLE CELL DISEASE AND THALASSEMIA USING CD34+ POSITIVE SELECTED GRAFTS
Source: ClinicalTrials.gov · Last updated 19 Dec 2024
Brief summary (registry, in English)
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.
- Status (registry)
- Completed
- Phase (registry)
- Phase 1
- Study type
- Interventional
- Enrollment
- 7 (actual)
- First submitted
- 13 Jun 2014
- Start date
- Jan 2015
- Primary completion date
- Jan 2024
- Lead sponsor
- Catherine Bollard
- Diseases
- Sickle cell disease, Thalassaemia
- NCT number
- NCT02165007
What you commit to
- Study type: Interventional · Phase 1
- Participants enrolled: 7
- From Jan 2015 to the primary completion date, Jan 2024
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 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.
- 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
Other illness
- 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)
Other
- Life expectancy less than 6 months
- Pregnant or breastfeeding patients
- Patients with suitable 8/8 HLA matched related and unrelated donors
Who the study is looking for
- 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.
Age in the registry: - to 22 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Save your city in your profile to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Childrens National Medical Center Coordinator of this site? Confirm its status | Washington D.C. | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.