Phase 3 Completed N=30 Treatment

Stem Cell Transplantation for Sickle Cell Anemia

Sickle Cell Disease

Source: ClinicalTrials.gov NCT01877837 ↗

Enrolled (actual)

Serious AEs

52.0%

Results posted

Nov 2022

Primary outcomePrimary: Number of Participants With Graft Failure — 3 Participants

◆ Published Evidence

No publication linked

No peer-reviewed publication reporting this trial's results has been linked yet. This can indicate results are unpublished — a known publication-bias signal. We re-check periodically.

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.

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Participants With Graft Failure	3	—
SECONDARY Overall Survival	23	—

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

View full record on ClinicalTrials.gov →

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. Informational only — not medical advice.