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Chemotherapy

Stem Cell Transplant for Sickle Cell Disease

Q: Are there specific requirements to qualify for this research project?

This study is seeking to enrol 98 patients that suffer from anemia and <a href="https://www.withpower.com/clinical-trials/sickle-cell">sickle cell</a> between the ages of 2 and 80. To be eligible, potential participants must have experienced at least one episode of acute chest syndrome resulting in admission to intensive care requiring non-mechanical ventilatory support (i.e. simple nasal cannula, face mask that requires oxygen content [venti mask, non-rebreather], continuous positive airway pressure [CPAP], bilevel positive airway pressure [BiPAP], high flow nasal cannula [HFNC] or invasive mechanical ventilatory support delivered by endotr

Q: Do elderly patients qualify for this trial?

As long as they are between the ages of 2 and 80, patients meet the age requirements for this trial.

Q: Are there any current openings for this clinical research?

This clinical trial is actively recruiting patients, as indicated on the listing on clinicaltrials.gov. The listing was first posted on April 6th, 2017 and was last edited on November 16th, 2022.

Q: How many volunteers are taking part in this experiment?

That is accurate, the clinical trial referenced is recruiting patients at this time. The listing was first posted on 4/6/2017 and updated as recently as 11/16/2022. They are enrolling 98 individuals total across 1 site.

Q: Are there any other clinical trials that have used haploidentical stem cells in their research?

Out of the 986 ongoing trials concerning haploidentical stem cell transplants, 178 have reached Phase 3. Most research surrounding this topic is based in San Antonio, <a href="https://www.withpower.com/clinical-trials/texas">Texas</a>; however, there are 29372 locations running studies related to this treatment.

Q: Can you please explain what haploidentical stem cell transplantation is typically used to treat?

Haploidentical stem cell transplants are not only used to treat organ transplantation, but also <a href="https://www.withpower.com/clinical-trials/multiple-sclerosis">multiple sclerosis</a>, <a href="https://www.withpower.com/clinical-trials/leukemia">leukemia</a>, myelocytic, acute, thrombocythemia, and essential.

Phase 1 & 2

Waitlist Available

Led By Courtney F Joseph, 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

Glomerular filtration rate >60 mL/min/1.73m^2 by cystatin C-based or iothalamate-based or other equivalent GFR testing

Ejection fraction greater than or equal to 35%

Timeline

Screening 3 weeks

Treatment Varies

Follow Up +30, +60, +100, 6months, 12 months, 18months, 24months annually

Awards & highlights

Study Summary

This trial is testing a new transplant regime to see if it is effective, safe and well tolerated in people with sickle cell disease.

Who is the study for?

Adults over 18 with sickle cell disease and severe complications like stroke, high blood flow in the lungs, liver issues, serious lung problems or silent strokes on MRI. They need a half-matched relative donor, good heart and kidney function, and can't be pregnant or breastfeeding. People with uncontrolled infections or major illnesses that could interfere with stem cell transplant recovery are excluded.Check my eligibility

What is being tested?

The trial is testing a new bone marrow transplant method using donors who are half tissue matches for adults with severe sickle cell disease. It includes taking stem cells from the donor's blood after drug stimulation, preparing the recipient's body with radiation and immune system drugs before infusing these cells through a central vein line.See study design

What are the potential side effects?

Possible side effects include reactions to medications like cyclophosphamide and sirolimus (such as nausea or low blood counts), risks from low-dose radiation exposure, complications from having a central line (like infection), and graft-versus-host disease where donated cells attack the recipient's body.

Eligibility Criteria

Inclusion Criteria

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

Select...

My kidney function test shows a filtration rate above 60 mL/min.

Select...

My heart pumps blood effectively.

Select...

I am 18 years old or older.

Select...

I have sickle cell disease and am at high risk for stroke or early death.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ +30, +60, +100, 6months, 12 months, 18months, 24months annually

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~+30, +60, +100, 6months, 12 months, 18months, 24months annually

This trial's timeline: 3 weeks for screening, Varies for treatment, and +30, +60, +100, 6months, 12 months, 18months, 24months annually for reporting.

Treatment Details

Study Objectives

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

Primary outcome measures

The percentage of patients at 100 days (+/- 1 week) post-transplant with sustained donor type hemoglobin on hemoglobin electrophoresis (HbS less than 50% when donors have sickle cell trait and <10% when donors have normal hemoglobin), who do ...

Secondary outcome measures

chimeric status of recipients

Trial Design

1Treatment groups

Experimental Treatment

Group I: recipientExperimental Treatment6 Interventions

recipient

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Hydroxyurea

2006

Completed Phase 4

~3620

cyclophosphamide

1994

Completed Phase 3

~8140

sirolimus

2006

Completed Phase 4

~1510

pentostatin

2005

Completed Phase 2

~350

0 / 4Eligibility criteria met

Find a Location

Who is running the clinical trial?

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

3,830 Previous Clinical Trials

47,302,699 Total Patients Enrolled

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

3 Previous Clinical Trials

304 Total Patients Enrolled

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

5 Previous Clinical Trials

326 Total Patients Enrolled

Media Library

Cyclophosphamide (Chemotherapy) Clinical Trial Eligibility Overview. Trial Name: NCT03077542 — Phase 1 & 2

Sickle Cell Disease Research Study Groups: recipient

Sickle Cell Disease Clinical Trial 2023: Cyclophosphamide Highlights & Side Effects. Trial Name: NCT03077542 — Phase 1 & 2

Cyclophosphamide (Chemotherapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03077542 — Phase 1 & 2

Frequently Asked Questions

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

Are there specific requirements to qualify for this research project?

"This study is seeking to enrol 98 patients that suffer from anemia and sickle cell between the ages of 2 and 80. To be eligible, potential participants must have experienced at least one episode of acute chest syndrome resulting in admission to intensive care requiring non-mechanical ventilatory support (i.e. simple nasal cannula, face mask that requires oxygen content [venti mask, non-rebreather], continuous positive airway pressure [CPAP], bilevel positive airway pressure [BiPAP], high flow nasal cannula [HFNC] or invasive mechanical ventilatory support delivered by endotr"

Answered by AI

Do elderly patients qualify for this trial?

"As long as they are between the ages of 2 and 80, patients meet the age requirements for this trial."

Answered by AI

Are there any current openings for this clinical research?

"This clinical trial is actively recruiting patients, as indicated on the listing on clinicaltrials.gov. The listing was first posted on April 6th, 2017 and was last edited on November 16th, 2022."

Answered by AI

How many volunteers are taking part in this experiment?

"That is accurate, the clinical trial referenced is recruiting patients at this time. The listing was first posted on 4/6/2017 and updated as recently as 11/16/2022. They are enrolling 98 individuals total across 1 site."

Answered by AI

Are there any other clinical trials that have used haploidentical stem cells in their research?

"Out of the 986 ongoing trials concerning haploidentical stem cell transplants, 178 have reached Phase 3. Most research surrounding this topic is based in San Antonio, Texas; however, there are 29372 locations running studies related to this treatment."

Answered by AI

Can you please explain what haploidentical stem cell transplantation is typically used to treat?

"Haploidentical stem cell transplants are not only used to treat organ transplantation, but also multiple sclerosis, leukemia, myelocytic, acute, thrombocythemia, and essential."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease

2017. "Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03077542.

All > Sickle Cell Disease > Sickle Cell Anemia