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Hematopoietic Cell Transplantation for Sickle Cell Disease

Phase < 1
Waitlist Available
Led By Kris M Mahadeo
Research Sponsored by M.D. Anderson Cancer Center
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 2 years post-hct
Awards & highlights

Study Summary

This trial is studying pre-transplant immunosuppression and donor stem cell transplant to see if it can treat patients with severe blood diseases.

Eligible Conditions
  • Sickle Cell Disease
  • Beta Thalassemia
  • Sickle Cell-Beta Thalassemia
  • Thalassemia
  • Sickle Cell Thalassemia

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 2 years post-hct
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 2 years post-hct for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Event-free survival (EFS)
Secondary outcome measures
Event-free survival
Incidence of acute graft-versus-host disease
Incidence of central nervous system toxicities including posterior reversible encephalopathy syndrome
+8 more

Side effects data

From 2010 Phase 2 trial • 21 Patients • NCT00060424
48%
Neutropenia
10%
Death following disease progression post transplant
10%
Thrombocytopenia
10%
Hyperbilirubinemia
10%
Hypoxia
5%
Typhlitis & Bowel Perforation
5%
Death following progression of GVHD
5%
Cardiac Arrhythmia and Seizure
5%
Death: Sepsis/Renal failure/ with history of GVHD
5%
Severe abnormal pain due to gut GVH
5%
Acute Pulmonary Embolism
5%
Perforated sigmoid diverticulitis
5%
Minimal hydronephosis
5%
Cholecystectomy
5%
Hypotension
5%
Increased creatinine
5%
Neurotoxicity
5%
Renal failure
5%
Tumor lysis syndrome
100%
80%
60%
40%
20%
0%
Study treatment Arm
Treatment (Enzyme Inhibitor, Transplant, GVHD Prophylaxis)

Trial Design

1Treatment groups
Experimental Treatment
Group I: Treatment (PTIS, HCT)Experimental Treatment11 Interventions
See Detailed Description.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Dexamethasone
2007
Completed Phase 4
~2590
Hematopoietic Cell Transplantation
2006
Completed Phase 2
~360
Plasmapheresis
2016
Completed Phase 3
~390
Rituximab
1999
Completed Phase 4
~1880
Cyclophosphamide
1995
Completed Phase 3
~3770
Busulfan
2008
Completed Phase 3
~1120
Tacrolimus
2011
Completed Phase 4
~4740
Fludarabine Phosphate
1997
Completed Phase 3
~2390
Mycophenolate Mofetil
1997
Completed Phase 4
~2380
Bortezomib
2005
Completed Phase 2
~1060

Find a Location

Who is running the clinical trial?

M.D. Anderson Cancer CenterLead Sponsor
2,987 Previous Clinical Trials
1,798,235 Total Patients Enrolled
Kris M MahadeoPrincipal InvestigatorM.D. Anderson Cancer Center
2 Previous Clinical Trials
40 Total Patients Enrolled
~0 spots leftby Jun 2025
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