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Immunosuppressive Agent

Nonmyeloablative Stem Cell Transplant for Sickle Cell Anemia and Thalassemia

Phase 2

Recruiting

Led By Matthew M Hsieh, 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

-Age greater than or equal to 4 years

--E. Sickle hepatopathy defined as EITHER ferritin >1000mcg/L OR direct bilirubin >0.4 mg/dL at baseline

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 10 years

Awards & highlights

Study Summary

This trial is testing a new variation of stem cell transplant that uses low dose radiation and a combination of immunosuppressive drugs to see if it helps the body better accept donor stem cells.

Who is the study for?

This trial is for people aged 4 and older with severe sickle cell disease or beta-thalassemia, who are at high risk of complications not improved by other treatments. They must have a matched family donor willing to donate stem cells. Exclusions include serious infections within the last month, pregnancy, lactation, or any major illness that could interfere with transplant survival.Check my eligibility

What is being tested?

The study tests a new transplant method using low-dose radiation and immunosuppressive drugs (cyclophosphamide, pentostatin, sirolimus) to see if they help patients better accept donated stem cells. Participants will undergo various procedures including blood tests and bone marrow sampling before receiving the treatment in hospital.See study design

What are the potential side effects?

Potential side effects may include reactions to medication like cyclophosphamide or alemtuzumab such as nausea and hair loss; organ inflammation from sirolimus; skin irritation from radiotherapy; immune system weakening leading to increased infection risk.

Eligibility Criteria

Inclusion Criteria

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

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I am at least 4 years old.

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I have sickle cell-related liver issues with high ferritin or bilirubin levels.

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I have kidney problems due to sickle cell disease, including high creatinine, low filtration rate, or need for dialysis.

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I have a family donor who is a complete match for my transplant.

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I have beta-thalassemia with moderate to severe iron overload.

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I have severe sickle cell disease with major organ damage or complications not improved by current treatments.

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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 severe sickle cell disease not improved by standard treatments.

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I have beta-thalassemia with significant iron overload.

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I have had acute chest syndrome twice or once while on hydroxyurea.

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

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I've been hospitalized at least 3 times last year for pain crises, even while on treatment.

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I need regular blood transfusions and my hemoglobin doesn't improve much on hydroxurea.

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I have had at least 2 long-lasting erections.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 10 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~10 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and 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

Determine regimen failure rate, defined as graft rejection, severe GVHD (acute GVHD grade 3 or higher or extensive chronic GVHD), or prolonged donor red cell aplasia (>2 years post-HSCT)

Secondary outcome measures

Examine the level of chimerism required to maintain both graft survival as well as hematologic normalcy using a regimen containing pentostatin, cyclophosphamide, alemtuzumab, and low total body irradiation.

Trial Design

2Treatment groups

Experimental Treatment

Group I: patients with preexisting antibodiesExperimental Treatment5 Interventions

patients with preexisting antibodies (major ABO mismatch or otheranti-donor red cell antibody). The primary endpoint forthis group will be different than the first cohort. It is very likely that red cell aplasia (6 months to 2 years posttransplant) and prolonged duration of red cell transfusion are expected in this second group, thus a later time point to determine treatment success is justified.

Group II: male donor - female recipientExperimental Treatment5 Interventions

The first cohort of patients will be male donor - femalerecipients to see if this new regimen will yield higher rate of durable donor leukocyte chimerism. We will also measure anti-A, anti-B, and/or other red cell antibody titers from this initial cohort to determine the feasibility of transplanting patients with pre-existing antibodies (major ABO mismatch or other anti-donor red cell antibody)

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Cyclophosphamide

1995

Completed Phase 3

~3770

Radiotherapy

2017

Completed Phase 3

~2610

Alemtuzumab

2004

Completed Phase 4

~1890

Pentostatin

2000

Completed Phase 3

~1300

Sirolimus

2013

Completed Phase 4

~2750

0 / 14Eligibility criteria met

Find a Location

Who is running the clinical trial?

National Institutes of Health Clinical Center (CC)NIH

383 Previous Clinical Trials

880,840 Total Patients Enrolled

National Cancer Institute (NCI)NIH

13,665 Previous Clinical Trials

40,925,692 Total Patients Enrolled

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

3,837 Previous Clinical Trials

47,851,432 Total Patients Enrolled

Media Library

Cyclophosphamide (Immunosuppressive Agent) Clinical Trial Eligibility Overview. Trial Name: NCT02105766 — Phase 2

Stem Cell Transplantation Research Study Groups: male donor - female recipient, patients with preexisting antibodies

Stem Cell Transplantation Clinical Trial 2023: Cyclophosphamide Highlights & Side Effects. Trial Name: NCT02105766 — Phase 2

Cyclophosphamide (Immunosuppressive Agent) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02105766 — Phase 2

Frequently Asked Questions

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

How many individuals have committed to participating in this research?

"Affirmative. Clinicaltrials.gov hosts evidence that this clinical trial is currently searching for candidates, with the first posting on April 21st 2014 and a recent update from November 11th 2022. The medical study requires 162 participants enrolled at 2 sites."

Answered by AI

Are there any other investigations into the efficacy of Alemtuzumab?

"As of the present, there are 926 ongoing trials concerning Alemtuzumab with 170 in Phase 3. Although Philadelphia, Pennsylvania boasts a plethora of studies for this medication, 28982 other sites across the world are running clinical investigations related to it."

Answered by AI

Are there any open slots to take part in this clinical research?

"According to the details on clinicaltrials.gov, this study is actively recruiting participants. The trial was announced April 21st 2014 and last updated November 11th 2022."

Answered by AI

What hazards have been identified in regards to Alemtuzumab treatment?

"Due to the Phase 2 nature of this clinical trial, there is some evidence indicating alemtuzumab's safety but none attesting to its efficacy; thus our team has assessed it with a score of 2."

Answered by AI

What conditions is Alemtuzumab typically prescribed for?

"Alemtuzumab can be leveraged to treat a broad range of medical conditions, including organ transplantation, multiple sclerosis and acute myelocytic leukemia."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure

2014. "Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02105766.

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