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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)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 12 and 24 months after hct

Awards & highlights

Study 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.Check my eligibility

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.See study design

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.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 12 and 24 months after hct

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~12 and 24 months after hct

This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 and 24 months after hct for reporting.

Treatment Details

Study Objectives

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

Primary outcome measures

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 outcome measures

Change From Baseline in Pain Scores using Numerical Rating Scale or Faces Pain Rating Scale at 100 days, 6 months and 12 months post-transplant

Disease free survival rate

Event-free survival rate

+9 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%

Renal failure

13%

Pleuracentesis

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%

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

0 / 10Eligibility criteria met

Find a Location

Who is running the clinical trial?

City of Hope Medical CenterLead Sponsor

565 Previous Clinical Trials

1,921,445 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

Frequently Asked Questions

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

What criteria must an individual meet in order to enroll in this research study?

"This clinical trial is recruiting 15 individuals ages 1 to 30 who are suffering from anemia and sickle cell, respecting the following criteria: periodic priapism requiring medical attention, osteonecrosis of at least two joints despite supportive care treatments, prior RBC transfusion therapy (8+ units per year for over 12 months), one or more severe vaso-occlusive pain crises in 2 years preceding enrollment even with a solid treatment plan/hydroxyurea administration, as well as history of acute chest syndrome episodes in that same time span."

Answered by AI

How many participants are enrolled in this clinical trial at the maximum capacity?

"Affirmative. According to the clinicaltrials.gov website, this investigation is actively recruiting volunteers and has been since 2/2/2018. Last edited on 6/14/2022, it requires 15 participants from a single site."

Answered by AI

Does the approved age range for this clinical experiment include individuals above 40 years of age?

"To be considered for this clinical trial, prospective participants must fall between 1 year and 30 years of age. Additionally, 131 trials are available to individuals that younger than 18 years old whereas 85 studies target patients aged 65 or above."

Answered by AI

Has the FDA given approval for Hematopoietic stem cell transplantation?

"Our team at Power gives hematopoietic stem cell transplantation a safety rating of 1 due to its Phase 1 status, meaning that the amount of data available on efficacy and safety is limited."

Answered by AI

Are there any vacancies for this trial at the present time?

"Affirmative. Per clinicaltrials.gov, this medical research is actively seeking volunteers; the trial was first published on February 2nd 2018 and recently updated on June 14th 2022. Fifteen individuals must be sourced from a single site for participation in the study."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Haploidentical Transplantation With Pre-Transplant Immunosuppressive Therapy for Patients With Sickle Cell Disease

2018. "Haploidentical Transplantation With Pre-Transplant Immunosuppressive Therapy for Patients With Sickle Cell Disease". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03279094.

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