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Chemotherapy

Haploidentical Bone Marrow Transplant for Sickle Cell Disease

Q: Could you please share how many other times this Haploidentical Bone Marrow Transplantation clinical trial has been conducted?

Haploidentical Bone Marrow Transplantation was first studied in 1997. As of now, there have been a total of 1442 completed studies with 981 trials actively recruiting patients. Many of these studies are being conducted in <a href="https://www.withpower.com/clinical-trials/washington">Washington</a> D.C.

Q: Could I be included as a subject in this experiment?

This trial is looking for 80 patients with anemia, <a href="https://www.withpower.com/clinical-trials/sickle-cell">sickle cell</a> between the ages of 5 and 45. Most notable, applicants should meet the following criteria: Umbilical cord blood or peripheral blood stem cell donors will not be accepted., A Karnofsky/Lansky performance score of ≥ 60., An HLA haploidentical first degree relative donor (parents, siblings or half siblings, or children) with 2, 3, or 4 (out of 8) HLA-mismatches who is willing and able to donate bone marrow. A unidirectional mismatch in either the graft versus host or host versus graft direction

Q: What are some of the risks associated with Haploidentical Bone Marrow Transplantation?

While Phase 2 trials are not as reliable in terms of efficacy, the existing data on Haploidentical Bone Marrow Transplantation suggests that it is a safe medical procedure.

Q: Are we still enrolling patients for this clinical trial?

No, this study is not currently recruiting patients. It was last updated on October 11th, 2022 and had originally been posted on October 3rd, 2017. There are 1183 other trials with open recruitment at the moment.

Q: What types of leukemia does Haploidentical Bone Marrow Transplantation typically target?

Haploidentical Bone Marrow Transplantation is a treatment for <a href="https://www.withpower.com/clinical-trials/malignant-melanoma">malignant melanoma</a> of skin that can also be used to fight <a href="https://www.withpower.com/clinical-trials/leukemia">leukemia</a>, neoplasm metastasis, and <a href="https://www.withpower.com/clinical-trials/sickle-cell-anemia">sickle cell anemia</a>.

Q: Does this research include elderly individuals in its tests?

According to the guidelines for who can participate in this research, individuals must be between 5 and 45 years old.

Q: How many participants are being included in this clinical trial?

This particular study is no longer enrolling patients. The listing originally went up on October 3rd, 2017 but the last update was on October 11th, 2022. However, there are currently 981 trials for Haploidentical Bone Marrow Transplantation and 202 clinical trials actively searching for patients with anemia or <a href="https://www.withpower.com/clinical-trials/sickle-cell">sickle cell</a> disease.

Q: Are there other hospitals in this state that are also running this trial?

Currently, this study is enrolling patients at 27 sites. The locations are located in <a href="https://www.withpower.com/clinical-trials/washington">Washington</a>, Buffalo and Cleveland along with other locations 27 other locations. To minimize travel demands, it is important to select the site nearest you if you enroll.

Phase 2

Waitlist Available

Research Sponsored by Medical College of Wisconsin

Eligibility Criteria Checklist

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

Must have

Serum conjugated (direct) bilirubin < 2x upper limit of normal for age as per local laboratory. Participants with hyperbilirubinemia as the result of hyperhemolysis, or a severe drop in hemoglobin post blood transfusion, are not excluded.

Liver MRI using a validated methodology per institutional preference (T2* or R2* or by ferriscan [R2 MRI]) for estimation of hepatic iron content is required for participants who are currently receiving ≥8 packed red blood cell transfusions per year for ≥1 year or have received ≥20 packed red blood cell transfusions (lifetime cumulative). Participants who have hepatic iron content ≥ 10 mg Fe/g liver dry weight by liver MRI must have a Gastroenterology/hepatology consultation with liver biopsy and histological examination including documentation of the absence of cirrhosis, bridging fibrosis, and active hepatitis.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1 and 2 years

Awards & highlights

Study Summary

This trial is testing whether haploidentical BMT (using a donor who is a half-match to the patient) is effective and safe in patients with sickle cell disease.

Who is the study for?

This trial is for children (5-14.99 years) and adults (15-45.99 years) with severe sickle cell disease who have a family member willing to donate bone marrow that partially matches their HLA type. Participants must be in good physical condition, not pregnant or breastfeeding, and agree to use contraception if of childbearing potential.Check my eligibility

What is being tested?

The study tests the effectiveness and safety of haploidentical bone marrow transplantation in treating severe sickle cell disease. It includes medications like Hydroxyurea, Rabbit-ATG, Thiotepa, Fludarabine, Cyclophosphamide plus Total Body Irradiation and Mesna as part of the treatment process.See study design

What are the potential side effects?

Possible side effects include immune reactions from the transplant leading to graft-versus-host disease, infections due to weakened immunity post-transplantation, organ damage from chemotherapy drugs or radiation therapy used before transplantation.

Eligibility Criteria

Inclusion Criteria

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

Select...

My direct bilirubin levels are within twice the normal limit, not due to blood transfusion issues.

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I need a liver MRI because I've had many blood transfusions.

Select...

I have a relative who can donate bone marrow and matches me partially in HLA.

Select...

I am not using umbilical cord or peripheral blood stem cells for my treatment.

Select...

My liver tests are within the required range and I may need a liver MRI due to my transfusion history.

Select...

I have a family member who matches my HLA type and is willing to donate bone marrow.

Select...

My kidney function is within the normal range for my age.

Select...

I can care for myself but may need occasional help.

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My heart's pumping ability is within the required range.

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My oxygen levels are good and my lungs work well enough to breathe properly.

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I am between 5 and 14 years old.

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I can do most activities, my heart and lungs work well enough for treatment.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 1 and 2 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~1 and 2 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 and 2 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

Two-Year Post-Transplant Event Free Survival (EFS)

Secondary outcome measures

Chimerism

Disease Recurrence

Graft Rejection

+3 more

Trial Design

1Treatment groups

Experimental Treatment

Group I: Haploidentical TransplantationExperimental Treatment8 Interventions

A conditioning regimen with Hydroxyurea, rabbit-ATG, Thiotepa, Fludarabine, Cyclophosphamide, Total Body Irradiation, and Mesna will be administered prior to Haploidentical Bone Marrow Transplantation.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Haploidentical Bone Marrow Transplantation

2008

Completed Phase 2

~60

Hydroxyurea

2006

Completed Phase 4

~3620

Thiotepa

2008

Completed Phase 3

~2150

Fludarabine

2012

Completed Phase 3

~1100

Cyclophosphamide

1995

Completed Phase 3

~3780

Total Body Irradiation

2006

Completed Phase 3

~820

Mesna

2003

Completed Phase 2

~1380

0 / 12Eligibility criteria met

Find a Location

Who is running the clinical trial?

Medical College of WisconsinLead Sponsor

609 Previous Clinical Trials

1,162,521 Total Patients Enrolled

National Heart, Lung, and Blood Institute (NHLBI)NIH

3,833 Previous Clinical Trials

47,306,416 Total Patients Enrolled

National Cancer Institute (NCI)NIH

13,654 Previous Clinical Trials

40,933,073 Total Patients Enrolled

Media Library

Cyclophosphamide (Chemotherapy) Clinical Trial Eligibility Overview. Trial Name: NCT03263559 — Phase 2

Sickle Cell Disease Research Study Groups: Haploidentical Transplantation

Sickle Cell Disease Clinical Trial 2023: Cyclophosphamide Highlights & Side Effects. Trial Name: NCT03263559 — Phase 2

Cyclophosphamide (Chemotherapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03263559 — Phase 2

Frequently Asked Questions

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

Could you please share how many other times this Haploidentical Bone Marrow Transplantation clinical trial has been conducted?

"Haploidentical Bone Marrow Transplantation was first studied in 1997. As of now, there have been a total of 1442 completed studies with 981 trials actively recruiting patients. Many of these studies are being conducted in Washington D.C."

Answered by AI

Could I be included as a subject in this experiment?

"This trial is looking for 80 patients with anemia, sickle cell between the ages of 5 and 45. Most notable, applicants should meet the following criteria: Umbilical cord blood or peripheral blood stem cell donors will not be accepted., A Karnofsky/Lansky performance score of ≥ 60., An HLA haploidentical first degree relative donor (parents, siblings or half siblings, or children) with 2, 3, or 4 (out of 8) HLA-mismatches who is willing and able to donate bone marrow. A unidirectional mismatch in either the graft versus host or host versus graft direction"

Answered by AI

What are some of the risks associated with Haploidentical Bone Marrow Transplantation?

"While Phase 2 trials are not as reliable in terms of efficacy, the existing data on Haploidentical Bone Marrow Transplantation suggests that it is a safe medical procedure."

Answered by AI

Are we still enrolling patients for this clinical trial?

"No, this study is not currently recruiting patients. It was last updated on October 11th, 2022 and had originally been posted on October 3rd, 2017. There are 1183 other trials with open recruitment at the moment."

Answered by AI

What types of leukemia does Haploidentical Bone Marrow Transplantation typically target?

"Haploidentical Bone Marrow Transplantation is a treatment for malignant melanoma of skin that can also be used to fight leukemia, neoplasm metastasis, and sickle cell anemia."

Answered by AI

Does this research include elderly individuals in its tests?

"According to the guidelines for who can participate in this research, individuals must be between 5 and 45 years old."

Answered by AI

How many participants are being included in this clinical trial?

"This particular study is no longer enrolling patients. The listing originally went up on October 3rd, 2017 but the last update was on October 11th, 2022. However, there are currently 981 trials for Haploidentical Bone Marrow Transplantation and 202 clinical trials actively searching for patients with anemia or sickle cell disease."

Answered by AI

Are there other hospitals in this state that are also running this trial?

"Currently, this study is enrolling patients at 27 sites. The locations are located in Washington, Buffalo and Cleveland along with other locations 27 other locations. To minimize travel demands, it is important to select the site nearest you if you enroll."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Haploidentical Bone Marrow Transplantation in Sickle Cell Patients (BMTCTN1507)

2017. "Haploidentical Bone Marrow Transplantation in Sickle Cell Patients (BMTCTN1507)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03263559.

All > Sickle Cell > Sickle Cell Anemia