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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%

Must not have

Patient with active Hepatitis B or C determined by serology and/or NAAT

Patients with suitable 8/8 HLA matched related and unrelated donors

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 2 years

Awards & highlights

All Individual Drugs Already Approved

Approved for 5 Other Conditions

No Placebo-Only Group

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.

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.

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.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have active Hepatitis B or C.

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I have a donor who is a perfect match for my transplant.

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

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Transplantation

Secondary study objectives

Overall survival

Other study objectives

Chronic GVHD

Grades II-IV and III-IV acute GVHD

Graft failure

+3 more

Awards & Highlights

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

Approved for 5 Other Conditions

This treatment demonstrated efficacy for 5 other conditions.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

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).

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Sirolimus

FDA approved

0 / 10Eligibility criteria met