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Active, Not Recruiting

NCT Number: NCT01461837

Haplo T-Cell Depleted Transplantation in High-Risk Sickle Cell Disease

This study is being done to determine the safety and outcome (long-term control) of a high-dose chemotherapy regimen followed by an infusion of CD34 selected (immune cells) stem cells from a partially matched adult family member donor, called haploidentical stem cell transplantation, in high-risk sickle cell disease patients.

Funding Source - FDA OOPD

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Sickle Cell Disease Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies

Age range

2 year–20 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

University of California Los Angeles (UCLA), Los Angeles, California, United States

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About this study

The purpose of this study is to investigate host myeloimmunosuppressive conditioning followed by familial haploidentical T cell depleted allogeneic stem cell transplantation in patients with high risk Sickle Cell Disease (SCD). It is hypothesized that it will be safe and well tolerated, and result in sustained donor chimerism, acceptable engraftment and immune reconstitution. Also, that it will limit SCD related organ damage resulting in improved and/or stable neurological, neurocognitive, pulmonary and pulmonary vascular function and health related quality of life (QOL).

Patients 2-20.99 years of age with a diagnosis of high-risk SCD and with an unaffected HLA partially matched family donor and meeting eligibility criteria (inclusion and exclusion criteria) are eligible.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Homozygous Hemoglobin S Disease, or Hemoglobin S Beta0/+ thalassemia
  • Patients must demonstrate one or more of the following Sickle Cell Disease Complications
  • Clinically significant neurologic event (stroke) or any neurologic deficit lasting >24 hours that is accompanied by an infarct on cerebral MRI
  • Minimum of two episodes of acute chest syndrome.
  • Recurrent painful events (at least 3 in the 2 years prior to enrollment).
  • Abnormal TCD study requiring starting on chronic transfusion therapy.
  • At least one silent infarct lesion on a MRI scan of the head.
  • A familial haploidentical donor without homozygous sickle cell disease
  • Adequate organ function (renal, liver, cardiac and pulmonary function)
  • Karnofsky or Lansky (age appropriate) Performance Score ≥50%
  • Liver biopsy is optional to assess for iron overload in chronically transfused patients.

Exclusion criteria

  • Females who are pregnant or breast-feeding
  • SCD Patients with documented uncontrolled infection
  • SCD patients who have an unaffected HLA matched family donor willing to proceed to donation
  • Karnofsky/Lansky (age appropriate) Performance Score <50% (hemiplegia alone secondary to a previous stroke is not an exclusion)
  • Demonstrated lack of compliance with medical care.
  • Clinically significant fibrosis or cirrhosis of the liver
  • Previously received a HSCT

Treatment and study plan

CD34 selected T-cell depleted allogeneic SCT

Drug

Hydroxyurea (60 mg/kg/day) and azathioprine (3 mg/kg/day) day -59 to day -11; fludarabine (30 mg/m2) Days -17, -16, -15, -14, -13; busulfan (3.2 mg/kg/day) Days -12, -11, -10, -9; thiotepa (10 mg/kg IV) day -8; cyclophosphamide (50 mg/kg) Days -7, -6, -5, -4; TLI on day -3; rabbit ATG (2.0 mg/kg/day) day -5,-4,-3, and -2; Stem Cell infusion day 0

Other names: Familial haploidentical, T-cell depleted, allogeneic stem cell transplantation, high risk Sickle Cell Disease

Primary outcomes

  1. Treatment related events

    Time frame: 1 year

    Death, primary or late graft rejection, or recurrence of disease and acceptable rate of hematopoietic engraftment, acute and chronic graft-versus-host disease

Secondary outcomes

  1. neurological/neurocognitive status

    Time frame: 2 years

    Change from baseline in neurological/neurocognitive status

  2. Pulmonary/pulmonary vascular status

    Time frame: 2 years

    Change from baseline of Pulmonary/pulmonary vascular status

  3. Health-related quality of life

    Time frame: 4 years

    Change from baseline of Health-related quality of life (CHRIs-HSCT/CHRIs-General)

Sponsors and collaborators

Lead sponsor

New York Medical College

Other

Collaborators

  • Ann & Robert H Lurie Children's Hospital of Chicago
  • Medical College of Wisconsin
  • Miltenyi Biomedicine GmbH
  • Tufts Medical Center
  • UCSF Benioff Children's Hospital Oakland
  • University of California, Los Angeles
  • University of California, San Francisco
  • Washington University School of Medicine

Registry information

Official study title

Familial Haploidentical T-Cell Depleted Transplantation in High-Risk Sickle Cell Disease (IND 14359)

Acronym: HaploSCD

Important dates

Study start
2012
Primary completion
2025
Study completion
2026
First posted
Oct 28, 2011
Registry last updated
Aug 8, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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