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Monoclonal Antibodies

Bone Marrow Transplant for Sickle Cell Disease

Phase 2
Recruiting
Led By Ann Haight, MD
Research Sponsored by Emory University
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
- Asymptomatic cerebrovascular disease, as evidenced by one the following: Silent cerebral infarction with at least one lesion measuring at least 3 mm in one dimension that is visible on two planes on the most recent brain MRI, or, cerebral arteriopathy, as evidenced by conditional TCD (TAMMV>170cm/sec but <200cm/sec) on two separate scans >2 weeks apart). If patient has a conditional TCD, then a brain MRI/MRA to evaluate for vasculopathy is required
- Previous clinical stroke, defined as a neurological deficit lasting longer than 24 hours plus new finding on head CT or brain MRI/MRA
Timeline
Screening 3 weeks
Treatment Varies
Follow Up year 2
Awards & highlights

Study Summary

This trial is testing a bone marrow transplant as a possible treatment for sickle cell disease in children. Eligible participants will undergo a pre-transplant evaluation, conditioning, and then the transplant itself, followed by routine care and checkups for at least 5 years.

Who is the study for?
This trial is for pre-adolescent children under 13 with sickle cell disease who've had multiple episodes of acute chest syndrome or vaso-occlusive pain, and have a healthy sibling donor also under 13. Candidates must not have severe neurological impairments, organ dysfunction, HIV infection, or uncontrolled infections.Check my eligibility
What is being tested?
The study tests the effectiveness of a bone marrow transplant from an HLA-matched sibling in treating sickle cell disease. Participants will undergo chemotherapy (Fludarabine, Alemtuzumab, Melphalan) to prepare their body for the transplant.See study design
What are the potential side effects?
Potential side effects include weakened immune system leading to increased risk of infections; nausea; hair loss; mouth sores due to chemotherapy; and graft-versus-host disease where the new cells may attack the recipient's body.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have a brain condition without symptoms, confirmed by specific brain scans.
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I have had a stroke with symptoms lasting over 24 hours and confirmed by brain scans.
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I've had at least 3 painful episodes a year that needed strong painkillers or hospital visits.
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I have symptoms of sickle cell disease.
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I have had 2 or more episodes of acute chest syndrome in my life.
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I've had 3 or more episodes of chest pain and blocked blood flow yearly for 3 years.
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I have had at least 3 acute chest syndrome events requiring blood transfusions.
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I am between 2 and 12 years old with a sickle cell disease.
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My sibling under 13 can donate to me and doesn't have sickle cell disease.
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My condition is considered severe.
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I've had 2 or more painful episodes needing strong painkillers at a hospital or clinic.
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I do not have symptoms of HbSS or HbSβ°thalassemia.
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My condition is not considered severe.
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I have had at least 3 episodes of acute chest syndrome or pain crises in my life.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~year 2
This trial's timeline: 3 weeks for screening, Varies for treatment, and year 2 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Immune Suppression-free, Rejection-free Survival
Secondary outcome measures
CMV Invasive Disease
Chronic GVHD
Cytomegalovirus (CMV) Viremia
+12 more

Trial Design

1Treatment groups
Experimental Treatment
Group I: Reduced Intensity Conditioning with FAMExperimental Treatment3 Interventions
Children with SCD will received reduced intensity conditioning with fludarabine, alemtuzumab and melphalan (FAM) during HSCT with a HLA matched sibling donor
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Fludarabine
FDA approved
Alemtuzumab
FDA approved
Melphalan
FDA approved

Find a Location

Who is running the clinical trial?

Emory UniversityLead Sponsor
1,632 Previous Clinical Trials
2,556,419 Total Patients Enrolled
Ann Haight, MDPrincipal InvestigatorEmory University
2 Previous Clinical Trials
40 Total Patients Enrolled

Media Library

Alemtuzumab (Monoclonal Antibodies) Clinical Trial Eligibility Overview. Trial Name: NCT04018937 — Phase 2
Sickle Cell Disease Research Study Groups: Reduced Intensity Conditioning with FAM
Sickle Cell Disease Clinical Trial 2023: Alemtuzumab Highlights & Side Effects. Trial Name: NCT04018937 — Phase 2
Alemtuzumab (Monoclonal Antibodies) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04018937 — Phase 2

Frequently Asked Questions

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

Is enrollment for this investigation still a possibility?

"Per information hosted on clinicaltrials.gov, this medical trial is actively recruiting participants as of August 3rd 2022; the study was initially posted in March 22nd 2019."

Answered by AI

How many venues are currently hosting this experiment?

"Currently, there are 11 study sites that are taking part in this trial. These locations span from Kent to Charleston and Jackson as well as other nearby towns. To reduce travel needs, it may be beneficial to seek out the closest centre for enrollment purposes."

Answered by AI

What potential harms could Fludarabine pose to patients?

"Fludarabine's safety was assessed and ranked as a 2, since the Phase 2 clinical trial has only produced some evidence of its protective properties but no data on efficacy."

Answered by AI

Does the eligibility criterion for this research project include adults aged 18 or older?

"The intended patient population for this trial are minors between the ages of two and thirteen."

Answered by AI

How many individuals are currently engaged in this medical research?

"Affirmative. According to information found on clinicaltrials.gov, this medical research project is in its recruitment phase. It was initially posted on March 22nd 2019 and updated most recently on August 3rd 2022. The study needs 58 volunteers across 11 healthcare sites."

Answered by AI

Who meets the requirements to participate in this experiment?

"Aspiring participants of this research should meet the criteria of anemia, sickle cell, and between 2 to 13 years old. The trial is seeking 58 volunteers in total."

Answered by AI

What conditions has Fludarabine been administered to treat?

"Fludarabine is a viable treatment for multiple sclerosis, as well as organ transplant rejection, kidney issues, B-lymphocyte overproduction, and amyloidosis."

Answered by AI

Has there been prior research into the efficacy of Fludarabine?

"Fludarabine was first clinically investigated in 1997 at the City of Hope Comprehensive Cancer Center. Since then, 654 trials have been completed and 463 are presently recruiting participants. Notably, a significant portion of these studies originate from Kent, Ohio."

Answered by AI
~21 spots leftby Jan 2027