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Cell Therapy

Stem Cell Transplant for Sickle Cell Anemia

Q: What is the typical reason that people in Cohort 2 take this medication?

<p>Organ transplantation, <a href="https://www.withpower.com/clinical-trials/multiple-sclerosis">multiple sclerosis</a>, and <a href="https://www.withpower.com/clinical-trials/leukemia">leukemia</a>, myelocytic, acute can <a href="https://www.withpower.com/clinical-trials/all">all</a> be treated using Cohort 2.</p>

Phase 1 & 2

Waitlist Available

Led By Courtney D Fitzhugh, M.D.

Research Sponsored by National Heart, Lung, and Blood Institute (NHLBI)

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Patients with sickle cell disease (HB SS, SC, or SBeta(0)-thal) at high risk for disease-related morbidity or mortality, defined by having severe end-organ damage (A, B, C, or D) or potentially modifiable complication(s) not ameliorated by hydroxyurea (E):

5.3.4 No history of congestive heart failure or unstable angina, and no history of stroke)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up year 5

Awards & highlights

Study Summary

This trialstudies a new kind of bone marrow transplant to treat people with anemias like sickle cell and thalassemia. Donors give stem cells, recipients get low-dose radiation and immunosuppressant drugs.

Who is the study for?

Adults diagnosed with severe congenital anemias like sickle cell disease or Beta-thalassemia, who have a half-matched family donor. Candidates should be at high risk due to organ damage or complications not improved by other treatments. They must understand and consent to the trial, not be pregnant, and meet specific health criteria.Check my eligibility

What is being tested?

The trial is testing a bone marrow transplant (BMT) using stem cells from half-matched donors combined with low-intensity radiation and immunosuppressant drugs (Alemtuzumab, Sirolimus, Cyclophosphamide). It aims to cure anemia without full chemotherapy's side effects.See study design

What are the potential side effects?

Possible side effects include reactions to immunosuppressants like increased infection risk, liver issues from cyclophosphamide, infusion-related reactions from Alemtuzumab and Sirolimus, as well as potential damage from low-dose irradiation.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I have sickle cell disease with severe complications not improved by hydroxyurea.

Select...

I have never had congestive heart failure, unstable angina, or a stroke.

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I haven't followed my iron removal treatment as prescribed since my first transfusion.

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I have sickle cell-related liver issues with high ferritin or bilirubin and low platelets.

Select...

I've been hospitalized more than once a year despite taking the highest dose of hydroxyurea I can tolerate.

Select...

I have a family member who is a partial genetic match for a donation.

Select...

I have thalassemia with significant iron overload due to issues like liver problems, poor treatment history, or enlarged liver.

Select...

I have had a stroke confirmed by an MRI or need regular transfusions due to artery issues in my brain.

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I have kidney problems due to sickle cell disease.

Select...

I have had episodes of severe pain due to blocked blood flow.

Select...

I am eligible for a stem cell transplant and have been treated with hydroxyurea.

Select...

I am 18 years old or older.

Select...

I understand and am willing to sign the informed consent; if under age, I have parental consent.

Select...

My hemoglobin S level is 50% or higher, or I have beta thalassemia intermedia.

Select...

I have thalassemia with significant iron overload.

Select...

My heart's tricuspid valve has a high blood flow speed.

Select...

My liver extends more than 2cm below my rib cage.

Select...

I have experienced acute chest syndrome.

Select...

I have been taking the highest dose of hydroxyurea I can tolerate for 6 months.

Select...

I've been hospitalized at least 3 times in the past year.

Select...

My donor is a half-matched family member.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ year 5

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~year 5

This trial's timeline: 3 weeks for screening, Varies for treatment, and year 5 for reporting.

Treatment Details

Study Objectives

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

Primary outcome measures

Patients With Donor Type Hemoglobin

Secondary outcome measures

Chimeric Value That is Required to Maintain Graft Survival and Hematologic Normalcy.

Number of Participants Overall Survival

Number of Participants That Experienced a Transplant-related Mortality

+7 more

Trial Design

3Treatment groups

Experimental Treatment

Group I: Cohort 3Experimental Treatment5 Interventions

PBSC transplant with 100 mg/kg post-transplant cyclophosphamide (PT-Cy)

Group II: Cohort 2Experimental Treatment5 Interventions

PBSC transplant with 50 mg/kg post-transplant cyclophosphamide (PT-Cy)

Group III: Cohort 1Experimental Treatment4 Interventions

PBSC transplant with no post-transplant cyclophosphamide (PT-Cy)

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Alemtuzumab

2004

Completed Phase 4

~1890

PBSC Transplant

2012

Completed Phase 2

~40

Sirolimus

2013

Completed Phase 4

~2750

Cyclophosphamide

1995

Completed Phase 3

~3780

0 / 21Eligibility criteria met

Find a Location

Who is running the clinical trial?

National Heart, Lung, and Blood Institute (NHLBI)Lead Sponsor

3,833 Previous Clinical Trials

47,306,473 Total Patients Enrolled

25 Trials studying Sickle Cell Anemia

2,435 Patients Enrolled for Sickle Cell Anemia

Courtney D Fitzhugh, M.D.Principal InvestigatorNational Heart, Lung, and Blood Institute (NHLBI)

5 Previous Clinical Trials

358 Total Patients Enrolled

Media Library

PBSC Transplant (Cell Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT00977691 — Phase 1 & 2

Sickle Cell Anemia Research Study Groups: Cohort 3, Cohort 1, Cohort 2

Sickle Cell Anemia Clinical Trial 2023: PBSC Transplant Highlights & Side Effects. Trial Name: NCT00977691 — Phase 1 & 2

PBSC Transplant (Cell Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT00977691 — Phase 1 & 2

Frequently Asked Questions

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

What is the typical reason that people in Cohort 2 take this medication?

"Organ transplantation, multiple sclerosis, and leukemia, myelocytic, acute can all be treated using Cohort 2."

Answered by AI

Does this research include test subjects that are older than 45 years of age?

"According to the information provided, the minimum age requirement for this clinical trial is 2 years old and the maximum age limit is 80 years old."

Answered by AI

How many people are being given the chance to participate in this clinical trial?

"As of right now, this specific study is not recruiting any more participants. Although, it's worth noting that the clinical trial was first posted on December 14th 2009 and edited most recently on October 13th 2022. There are a total of 415 other trials for anemia, sickle cell and 961 studies exploring different aspects of Cohort 2 that are currently admitting patients."

Answered by AI

Are we able to sign people up for the trial at this time?

"This study is not currently looking for patients, as the last update on clinicaltrials.gov was from October 13th, 2022. The study was originally posted on December 14th, 2009. Although this particular study has completed recruitment, there are 1,376 other studies that are actively recruiting patients."

Answered by AI