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Chemotherapy

Bone Marrow Transplant (BMT) for Sickle Cell Disease (TRANSFORM Trial)

Phase 2
Waitlist Available
Led By Lakshmanan Krishnamurti, MD
Research Sponsored by Emory University
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 one year
Awards & highlights

TRANSFORM Trial Summary

This trial is testing if bone marrow transplants from half-matched related donors can be used to treat people with severe sickle cell disease. Before transplant, patients receive chemotherapy and radiation.

Eligible Conditions
  • Sickle Cell Disease

TRANSFORM Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to one year
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to one year 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) Rate
Late Graft Rejection Rate
Primary Graft Rejection Rate
+1 more
Secondary outcome measures
Chimerism Rate Following Hematopoietic Cell Transplantation for Sickle Cell Disease
Cumulative Incidence of Neutrophil Engraftment and Platelet Engraftment.
Frequency of Idiopathic Pneumonia Syndrome (IPS)
+5 more

Side effects data

From 2021 Phase 2 trial • 10 Patients • NCT02757885
67%
Fever
67%
Graft-versus-host disease (GVHD)
50%
Bacteriemia
50%
Epstein-Barr virus (EBV)
33%
Central Nervous System Toxicity
17%
Deep vein thrombosis (DVT)
17%
BK virus (BKV)
17%
Non-engraftment
17%
Cytomegalovirus infection (CMV)
17%
Thrombotic microangiopathy (TMA)
100%
80%
60%
40%
20%
0%
Study treatment Arm
Bone Marrow Recipient

TRANSFORM Trial Design

1Treatment groups
Experimental Treatment
Group I: Bone Marrow RecipientExperimental Treatment7 Interventions
Single arm open label study: Participants with sickle cell disease (SCD) will receive bone marrow from a human leukocyte antigen (HLA) mismatched donor.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Bone Marrow Transplant (BMT)
2019
Completed Phase 2
~10
Fludarabine monophosphate
2008
Completed Phase 3
~480
Cyclophosphamide
1995
Completed Phase 3
~3770
Total Body Irradiation
2006
Completed Phase 3
~820
Hydroxyurea
2006
Completed Phase 4
~3620
Thiotepa
2008
Completed Phase 3
~2210

Find a Location

Who is running the clinical trial?

Emory UniversityLead Sponsor
1,642 Previous Clinical Trials
2,563,996 Total Patients Enrolled
Lakshmanan Krishnamurti, MDPrincipal Investigator - Emory University
Children's Healthcare of Atlanta at Egleston
ArMedical Forces Medical College (Medical School)
University Of Mn Medical Sch (Residency)
9 Previous Clinical Trials
871 Total Patients Enrolled

Frequently Asked Questions

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

How many people are signing up for this experiment?

"Yes, an update on September 13th indicates that this study is actively recruiting the 15 participants it needs from a single site."

Answered by AI

What is the minimum age requirement for participants in this test?

"The age limit for inclusion in this study is 40 years old, with a minimum age of 15."

Answered by AI

What are some of the risks associated with BMT?

"Since there is only some data supporting safety, and none yet to suggest it is effective, BMT received a score of 2."

Answered by AI

Can you give me some more information on other BMT clinical trials?

"Out of the 960 ongoing trials studying Bone Marrow Transplants (BMT), 170 are currently in Phase 3. Most of these studies taking place in Philadelphia, however there are 29043 locations running clinical trials for this treatment globally."

Answered by AI

To whom does this study appeal?

"This study is looking for 15 participants between the ages of 15 and 40 who have anemia or sickle cell. Participants must also meet the following criteria: -Clinically significant neurologic event (stroke) or any neurological deficit lasting > 24 hours;-Karnofsky/Lansky performance score ≥ 60; -Disease severity: Participants with SCD who have 1 or more of the following (i-v); -Age: Patients must be 15 - 40 years of age inclusive OR if younger than 15 years must be pubertal; -History of three or"

Answered by AI

Are there any patients currently needed for this research project?

"According to the clinicaltrials.gov website, this study is still open for enrollment and actively recruiting patients. The trial was originally posted on April 1st, 2016 with a most recent update on September 13th, 2020."

Answered by AI

What types of cancer respond best to treatment with BMT?

"BMT is a potential treatment for malignant melanoma of the skin as well as other conditions such as leukemia, neoplasm metastasis, and sickle cell anemia."

Answered by AI

Why was this research project undertaken?

"The primary objective of this study is to evaluate the rate of late graft rejection. Secondary objectives include assessing the frequency of idiopathic pneumonia syndrome (IPS) and stroke, as well as the veno-occlusive disease (VOD) rate."

Answered by AI
Recent research and studies
clinicaltrials.govStudy
Transplantation for Patients With Sickle Cell Disease From Mismatched Family Donors of Bone Marrow
Suhag Parikh 2019. "Transplantation for Patients With Sickle Cell Disease From Mismatched Family Donors of Bone Marrow". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02757885.
~2 spots leftby May 2025
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