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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
History of three or more severe pain crises per year in the 2-year period preceding enrollment despite the institution of supportive care measures (i.e. a pain management plan and/or treatment with hydroxyurea);
History of two or more episodes of acute chest syndrome (ACS) in the 2-year period preceding enrollment despite the institution of supportive care measures (i.e. asthma therapy and/or hydroxyurea);
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 Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
You have had three or more severe pain episodes every year for the past two years, despite trying different ways to manage the pain.
Select...
You have experienced two or more episodes of acute chest syndrome in the past two years, even after receiving asthma treatment and/or hydroxyurea.
Select...
If you have sickle cell disease (SCD), you cannot participate if you have one or more of the following conditions: i. Severe pain crisis ii. Chest syndrome iii. Stroke iv. Acute chest syndrome v. Multi-organ failure
Select...
You have had a serious brain problem or any lasting neurological issues for more than a day.

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%
Epstein-Barr virus (EBV)
50%
Bacteriemia
33%
Central Nervous System Toxicity
17%
Cytomegalovirus infection (CMV)
17%
BK virus (BKV)
17%
Thrombotic microangiopathy (TMA)
17%
Non-engraftment
17%
Deep vein thrombosis (DVT)
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
Total Body Irradiation
2006
Completed Phase 3
~820
Bone Marrow Transplant (BMT)
2019
Completed Phase 2
~10
Cyclophosphamide
1995
Completed Phase 3
~3780
Fludarabine monophosphate
2008
Completed Phase 3
~480
Hydroxyurea
2006
Completed Phase 4
~3620
Thiotepa
2008
Completed Phase 3
~2150

Find a Location

Who is running the clinical trial?

Emory UniversityLead Sponsor
1,626 Previous Clinical Trials
2,560,697 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
~2 spots leftby Mar 2025