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Stem Cell Transplantation

Stem Cell Transplant for Sickle Cell Disease

Phase 1

Waitlist Available

Research Sponsored by Catherine Bollard

Eligibility Criteria Checklist

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

Must have

First allogeneic transplant

Pulmonary function: DLCO ≥40% (adjusted for hemoglobin) and FEV1≥50% in patients 7 years and older with normal cognitive function and able to perform the test adequately. If not able to complete the testing a CT chest will be required., oxygen saturation>91%

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 2 years

Awards & highlights

Study Summary

This trial will test the safety of a new stem cell transplant method for sickle cell disease and thalassemia.

Who is the study for?

This trial is for young people up to age 22 with severe sickle cell disease or thalassemia requiring regular blood transfusions and iron overload. They need a related donor partially matching their HLA type, good heart and kidney function, adequate lung capacity, normal liver enzymes, and no active infections or hepatitis. Pregnant individuals or those with HIV are excluded.Check my eligibility

What is being tested?

The study tests a less intense procedure of stem cell transplant from half-matched donors in patients with sickle cell disease or thalassemia. It aims to evaluate the safety of this approach using CD34+ selected peripheral blood stem cells.See study design

What are the potential side effects?

Potential side effects may include reactions to the stem cell infusion, increased risk of infection due to immune suppression from conditioning treatment prior to transplantation, organ inflammation, and possible graft-versus-host disease.

Eligibility Criteria

Inclusion Criteria

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

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I am undergoing my first transplant from a donor.

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My lung function is good and I can breathe well.

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My liver tests are within the required range.

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I am 22 years old or younger.

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I have severe sickle cell disease with significant complications.

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I have thalassemia and need regular blood transfusions, leading to too much iron in my body.

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My donor matches me on at least 4 but not all 8 HLA markers.

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My heart is functioning well, with good pumping ability.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 2 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~2 years

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

Transplantation

Secondary outcome measures

Overall survival

Other outcome measures

Chronic GVHD

Grades II-IV and III-IV acute GVHD

Graft failure

+3 more

Trial Design

1Treatment groups

Experimental Treatment

Group I: peripheral blood stem cell graft that are CD34+ selectedExperimental Treatment1 Intervention

peripheral blood stem cell graft that are CD34+ selected. All patients will undergo reduced intensity conditioning regimen which followed by infusion of a peripheral blood stem cell graft collected from haploidentical family donors that are CD34+ positively selected using the CliniMACS device and Sirolimus will be used for GVHD prophylaxis and given for 9 months post-transplant and then tapered off by one year (see intervention).

0 / 8Eligibility criteria met

Find a Location

Who is running the clinical trial?

Catherine BollardLead Sponsor

13 Previous Clinical Trials

304 Total Patients Enrolled

Children's National Research InstituteOTHER

210 Previous Clinical Trials

207,730 Total Patients Enrolled

Media Library

Haploidentical Hematopoietic Stem Cell Transplantation (Stem Cell Transplantation) Clinical Trial Eligibility Overview. Trial Name: NCT02165007 — Phase 1

Frequently Asked Questions

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

Have any other experiments been conducted utilizing CD34+ peripheral blood stem cell grafts?

"Currently, 125 trials concerning peripheral blood stem cell graft that are CD34+ selected are operational. Of those studies, 13 have progressed to the third and final phase of clinical testing. Although many experiments for this treatment take place in Cincinnati, Ohio; there are 1,074 sites worldwide running tests on this remedy."

Answered by AI

What is the aggregate size of the cohort involved in this research?

"Unfortunately, this medical trial has already concluded its recruitment and is no longer looking for participants. The initial posting date was January 1st 2015 with the latest update made on August 30th 2022. For those seeking alternative studies to join, there are currently 59 trials enrolling patients with thalassemia and 125 clinical trials recruiting peripheral blood stem cell grafts that have been CD34+ selected still actively searching for volunteers."

Answered by AI

What hazards are posed by the infusion of CD34+-selected peripheral blood stem cells?

"The safety of a CD34+ selected peripheral blood stem cell graft has only been minimally studied, therefore our team at Power gave it an initial score of 1."

Answered by AI

Are there any remaining opportunities to participate in this clinical research?

"Information on clinicaltrials.gov implies that this medical trial is no longer recruiting patients, as its initial posting was in 2015 and the most recent edit occurred in August of 2022. Nonetheless, there are currently 184 other trials actively searching for participants at present."

Answered by AI

What medical purposes are CD34+ selected peripheral blood stem cell grafts typically utilized for?

"Rejection of a transplanted liver can be mitigated by the use of CD34+ selected peripheral blood stem cells. Moreover, this form of treatment is also beneficial for treating different diseases such as kidney disease and renal angiomyolipomas."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Haploidentical Hematopoietic Stem Cell Transplantation

Catherine Bollard 2015. "Haploidentical Hematopoietic Stem Cell Transplantation". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02165007.

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