Phase 3
N=30
Stem Cell Transplantation for Sickle Cell Anemia
Sickle Cell Disease
Bottom Line
View on ClinicalTrials.gov: NCT01877837 ↗Enrolled (actual)
30
Serious AEs
52.0%
Results posted
Nov 2022
Primary outcome: Primary: Number of Participants With Graft Failure — 3 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Alemtuzumab (Drug); Fludarabine (Drug); Melphalan (Drug); Stem Cells (Procedure)
- Age
- Pediatric, Adult · 2+ yrs
- Sex
- All
- Sponsor
- Hackensack Meridian Health
- Primary completion
- Sep 2021
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Graft Failure |
3 | — |
| SECONDARY Overall Survival |
23 | — |
Summary
This protocol will be investigating the use of stem cell transplantation, in related donors, to cure sickle cell disease. Sickle cell disease is a recessive disorder caused by a point mutation that results in the substitution of valine for glutamic acid at the sixth position in the B-chain of hemoglobin. This leads to sickling of the red blood cells under many conditions, such as hypoxia, dehydration, and hyperthermia. The sickling leads to vaso-occlusion, which causes irreversible damage in almost all systems in the body, including the central nervous system (CNS), lungs, heart, bones, eyes, liver, and kidneys.
Eligibility Criteria
Inclusion Criteria
- Patient Eligibility
- Matched sibling donors (9-10/10 marrow/PBSC or 5-6/6 UCB (single or double) with a total TNC dose of greater than 5 x 107/kg recipient weight)
- Age 2-30
- Hb SS, S-thal0, S-thal+, SC
- Evidence of ongoing hemolysis: Hb 5%, LDH > 500, TB>2
- Karnofsky/Lansky score >50
- LVSF>26% or LVEF>40%
- DLCO >40% or O2 sat >85% for those patients that can't perform PFTs
- GFR >70 and serum creatinine 1000, liver biopsy should be done and show no evidence of bridging fibrosis or cirrhosis
- Exclusion criteria
- Evidence of uncontrolled bacterial, viral, or fungal infection within one month prior to initiation of the conditioning regimen
- Pregnant or breastfeeding
- HIV positive
- Written informed consent not obtained
Data sourced from ClinicalTrials.gov (NCT01877837). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.