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Monoclonal Antibodies

Stem Cell Transplant for Sickle Cell Disease

Phase 2

Waitlist Available

Led By Monica Bhatia, MD

Research Sponsored by Columbia 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 day +30, 60, 100, 180, 365, 730, and 1005

Awards & highlights

Study Summary

This trial is testing whether lower doses of chemo, followed by a stem cell transplant, will be successful in treating a patient's disease with fewer side effects.

Eligible Conditions

Sickle Cell Disease
Beta Thalassemia

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ day 30, day 60, day 100, day 180, 1 year, 2 years, 3 years, 5 years, 10 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~day 30, day 60, day 100, day 180, 1 year, 2 years, 3 years, 5 years, 10 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and day 30, day 60, day 100, day 180, 1 year, 2 years, 3 years, 5 years, 10 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Prevalence of toxicity associated with moderately ablative therapy (busulfan/fludarabine/alemtuzumab) and allogeneic stem cell transplantation in selected patients with Sickle Cell Disease (SCD) and Beta Thalassemia (BT)

Secondary outcome measures

Incidence of acute and chronic graft versus host disease (GVHD) following moderately ablative therapy and allogeneic stem cell transplantation in selected patients with SCD and BT

Incidence of primary and secondary graft failure

Percent of mixed donor chimerism

+3 more

Trial Design

2Treatment groups

Experimental Treatment

Group I: SCD groupExperimental Treatment4 Interventions

Sickle Cell Disease patients receiving chemotherapy (Busulfan, Fludarabine and Alemtuzumab) will undergo allogeneic stem cell transplant.

Group II: BT groupExperimental Treatment4 Interventions

Beta Thalassemia patients receiving chemotherapy (Busulfan, Fludarabine and Alemtuzumab) will undergo allogeneic stem cell transplant.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Busulfan

FDA approved

Fludarabine

FDA approved

Alemtuzumab

FDA approved

Allogeneic stem cell transplant

2005

Completed Phase 2

~260

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

Columbia UniversityLead Sponsor

1,431 Previous Clinical Trials

2,460,780 Total Patients Enrolled

Monica Bhatia, MDPrincipal InvestigatorColumbia University

3 Previous Clinical Trials

73 Total Patients Enrolled

Media Library

Alemtuzumab (Monoclonal Antibodies) Clinical Trial Eligibility Overview. Trial Name: NCT00408447 — Phase 2

Sickle Cell Disease Research Study Groups: BT group, SCD group

Sickle Cell Disease Clinical Trial 2023: Alemtuzumab Highlights & Side Effects. Trial Name: NCT00408447 — Phase 2

Alemtuzumab (Monoclonal Antibodies) 2023 Treatment Timeline for Medical Study. Trial Name: NCT00408447 — Phase 2

Frequently Asked Questions

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

How does the level of risk associated with this treatment compare to other treatments?

"This is a Phase 2 trial, so while there is some evidence that the treatment is safe, its efficacy has not yet been proven. Our team at Power estimates that it scores a 2 on our safety scale."

Answered by AI

Are there any current vacancies in this trial for people who wish to participate?

"Currently, this particular clinical trial is not admitting any more patients. It was originally posted on September 1st, 2004 but the listing was edited for the last time on August 18th, 2022. There are other 442 trials seeking beta-thalassemia patients and 242 trials recruiting for this treatment actively admitting patients right now."

Answered by AI

For which kind of patients is this clinical trial designed?

"Researchers conducting this study are looking to enroll 53 individuals that have beta-thalassemia. The age requirement for participants is between 1 month and 30 years old. There are a few other requirements, the most notable being: Age ≤30."

Answered by AI

What is the primary reason this therapy is used?

"This immunotherapy can help patients with autoimmune hemolytic anemia as well as multiple sclerosis, kidney transplant rejection, and B-cell lymphoma."

Answered by AI

Can you share any other instances in which this treatment has been studied?

"Currently, 442 clinical trials are underway to research this treatment. Of those, 34 are in the final stage of testing before approval. The majority of these trials are based in Philadelphia, but there are studies taking place at 4706 other locations around the world."

Answered by AI

Are participants in this study required to be under 20 years of age?

"From the eligibility requirements, it is apparent that patients as young as one month old to those who are thirty years old may enroll in this trial."

Answered by AI

How many study participants are being treated with this medication?

"This study is not admitting any more patients at the moment. The clinical trial was first posted on September 1st, 2004 and updated for the last time on August 18th, 20212. However, there are 442 other trials actively recruiting patients and 242 studies currently running that might admit beta-thalassemia patients."

Answered by AI

Recent research and studies

Bone Marrow TransplantationJournal

A Pilot Study of Reduced Intensity Conditioning and Allogeneic Stem Cell Transplantation from Unrelated Cord Blood and Matched Family Donors in Children and Adolescent Recipients

Del Toro, G, P Satwani, L Harrison, Y-K Cheung, M Brigid Bradley, D George, D J Yamashiro, et al.. 2004. “A Pilot Study of Reduced Intensity Conditioning and Allogeneic Stem Cell Transplantation from Unrelated Cord Blood and Matched Family Donors in Children and Adolescent Recipients”. Bone Marrow Transplantation. Springer Science and Business Media LLC. doi:10.1038/sj.bmt.1704399.

Biology of Blood and Marrow TransplantationJournal

Reduced-intensity Allogeneic Stem Cell Transplantation in Adults and Children with Malignant and Nonmalignant Diseases: End of the Beginning and Future Challenges

Satwani, Prakash, Lauren Harrison, Erin Morris, Gustavo Del Toro, and Mitchell S. Cairo. 2005. “Reduced-intensity Allogeneic Stem Cell Transplantation in Adults and Children with Malignant and Nonmalignant Diseases: End of the Beginning and Future Challenges”. Biology of Blood and Marrow Transplantation. Elsevier BV. doi:10.1016/j.bbmt.2005.04.002.

Bone Marrow TransplantationJournal

Reduced Toxicity, Myeloablative Conditioning with BU, Fludarabine, Alemtuzumab and SCT from Sibling Donors in Children with Sickle Cell Disease

Bhatia, M, Z Jin, C Baker, M B Geyer, K Radhakrishnan, E Morris, P Satwani, et al.. 2014. “Reduced Toxicity, Myeloablative Conditioning with BU, Fludarabine, Alemtuzumab and SCT from Sibling Donors in Children with Sickle Cell Disease”. Bone Marrow Transplantation. Springer Science and Business Media LLC. doi:10.1038/bmt.2014.84.

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