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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
~3780
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
~1140

Find a Location

Who is running the clinical trial?

M.D. Anderson Cancer CenterLead Sponsor
2,966 Previous Clinical Trials
1,804,781 Total Patients Enrolled
Kris M MahadeoPrincipal InvestigatorM.D. Anderson Cancer Center
2 Previous Clinical Trials
40 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Does this research allow for participation from geriatric individuals?

"This medical trial is enrolling individuals aged between 2 and 30 years old."

Answered by AI

Is it possible for me to take part in this experiment?

"Qualified applicants for this research must have thalassemia and be between 2 and 30 years of age. Our team is seeking around 40 participants in total."

Answered by AI

In what medical conditions is Hematopoietic Cell Transplantation commonly prescribed?

"Hematopoietic Cell Transplantation can be applied to combat various conditions like synovitis, ophthalmia, sympathetic disorders, and some types of lung cancer."

Answered by AI

How many individuals are actively taking part in this research protocol?

"Affirmative. Clinicaltrials.gov contains data that suggests this research study is currently enrolling 40 participants from one site; the trial was first listed on December 29th 2020 and last edited November 3rd 2022."

Answered by AI

Are there other research projects analyzing the effects of Hematopoietic Cell Transplantation?

"Currently, 1759 clinical trials exploring Hematopoietic Cell Transplantation are underway with 354 being Phase 3. Mishawaka Indiana is hosting several such studies while 51579 other sites around the world have ongoing research into this field."

Answered by AI

Are there any openings left for participants in this research initiative?

"Clinicaltrials.gov confirms that this research initiative is presently recruiting, with the initial posting occurring on December 29th 2020 and a recent update made to it in November 3rd 2022."

Answered by AI
Recent research and studies
clinicaltrials.govStudy
Pre-transplant Immunosuppression and Donor Stem Cell Transplant for the Treatment of Severe Hemoglobinopathies
2020. "Pre-transplant Immunosuppression and Donor Stem Cell Transplant for the Treatment of Severe Hemoglobinopathies". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04776850.
~0 spots leftby Apr 2025
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