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Hematopoietic Stem Cell Transplantation

Stem Cell Transplant for Sickle Cell Disease

Phase 1
Recruiting
Led By Anna B. Pawlowska, MD
Research Sponsored by City of Hope Medical Center
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Diagnosis: Patients with sickle cell anemia (Hgb SS or SB° Thalassemia) with baseline Hgb S more than 60%
Significant neurologic event (stroke) or any neurological deficit lasting > 24 hours; or increased transcranial Doppler velocity (>200 m/s)
Must not have
Patients with any active malignancy are ineligible for this study, other than non-melanoma skin cancers
Prior autologous or allogeneic transplant
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 24 months
Awards & highlights

Summary

This trial is studying a new way to treat sickle cell disease using haploidentical donors (a donor with partial matching of the patient's HLA) and a new myeloablative preparative regimen.

Who is the study for?
This trial is for patients aged 1-30 with sickle cell anemia who have had severe symptoms like stroke, acute chest syndrome, or frequent pain crises despite treatment. They must not be pregnant, agree to use contraception, and have a half-matched (haploidentical) family donor because no fully matched donor is available.
What is being tested?
The study tests a new approach using two cycles of drugs to suppress the immune system before receiving stem cells from a half-matched family donor. The goal is to safely expand the pool of donors and achieve consistent results without causing harm.
What are the potential side effects?
Potential side effects include reactions related to immune suppression such as increased infection risk, possible damage to bone marrow function (myeloablation), and other complications that can arise from transplant procedures.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have sickle cell anemia with more than 60% Hgb S.
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I have had a stroke or a lasting neurological issue, or my brain blood flow speed is high.
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I've had one or more acute chest syndrome episodes in the last 2 years despite treatment.
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I have bone damage in two or more joints despite receiving care.
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I've had 8 or more blood transfusions yearly for over a year to prevent complications.
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I have a family donor who matches half of my genetic markers for a transplant.
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I've had severe pain crises yearly despite treatment.
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I have had repeated priapism treated with medication.
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I don't have a fully matched donor for a transplant.
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I am between 1 and 30 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I do not have any active cancer except for non-melanoma skin cancers.
Select...
I have had a stem cell transplant from myself or a donor.
Select...
I do not have any uncontrolled illnesses or infections.
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I am not currently on any experimental treatments or other cancer therapies.
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I have a donor who is a complete HLA match for me.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~24 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 24 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Rate of unacceptable adverse events that are defined as any of the following events that occur from start of pre-transplant immunosuppressive therapy to the first 100 days post HCT
Secondary study objectives
Disease free survival rate
Event-free survival rate
Immune reconstitution at day 100, 180 and 365
+4 more

Side effects data

From 2020 Phase 2 trial • 14 Patients • NCT00258427
75%
Infection
38%
Pneumonia
25%
Engraftment syndrome
25%
Acute kidney injury
25%
Hypertension
25%
Intubation
25%
Veno occlusive disease
13%
Pleuracentesis
13%
Renal failure
13%
GI bleeding
13%
Death
13%
diffuse alveolar hemorrhage
13%
Acute hypoxic respiratory failure
13%
Disseminated intravascular coagulation
13%
Drug Rash with Eosinophilia
13%
Eosinophilic gut
13%
Pneumoperitoneum
13%
Dialysis
13%
pulmonary hemorrhage
13%
Chest Tube Placement
13%
Chest Xray
13%
pericardial effusion
13%
Respiratory distress syndrome
100%
80%
60%
40%
20%
0%
Study treatment Arm
USB Arm
Sibling withoutCliniMACS
Marrow Isolex
Marrow Clinimacs

Trial Design

1Treatment groups
Experimental Treatment
Group I: Haploidentical stem cell transplantationExperimental Treatment1 Intervention
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Hematopoietic stem cell transplantation
2008
Completed Phase 2
~430

Find a Location

Who is running the clinical trial?

City of Hope Medical CenterLead Sponsor
596 Previous Clinical Trials
1,923,522 Total Patients Enrolled
Anna B. Pawlowska, MDPrincipal InvestigatorCity of Hope Medical Center

Media Library

Hematopoietic stem cell transplantation (Hematopoietic Stem Cell Transplantation) Clinical Trial Eligibility Overview. Trial Name: NCT03279094 — Phase 1
Sickle Cell Disease Research Study Groups: Haploidentical stem cell transplantation
Sickle Cell Disease Clinical Trial 2023: Hematopoietic stem cell transplantation Highlights & Side Effects. Trial Name: NCT03279094 — Phase 1
Hematopoietic stem cell transplantation (Hematopoietic Stem Cell Transplantation) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03279094 — Phase 1
~1 spots leftby Mar 2025
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