This study is in progress, not accepting new patients

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

a study on Sickle Cell Anemia Anemia

Summary

Eligibility
for people ages 2-20 (full criteria)
Location
at UCLA UCSF
Dates
study started
completion around
Principal Investigator
by Theodore Moore (ucla)

Description

Summary

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

Official Title

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

Details

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.

Keywords

Sickle Cell Disease, stem cell transplantation, haploidentical, Sickle Cell Anemia, CD34 selected T-cell depleted allogeneic SCT, Haplo Stem Cell Transplantation

Eligibility

You can join if…

Open to people ages 2-20

  • Homozygous Hemoglobin S Disease, or Hemoglobin S Beta0/+ thalassemia
  • Patients must demonstrate one or more of the following Sickle Cell Disease Complications
    1. Clinically significant neurologic event (stroke) or any neurologic deficit lasting >24 hours that is accompanied by an infarct on cerebral MRI
    2. Minimum of two episodes of acute chest syndrome.
    3. Recurrent painful events (at least 3 in the 2 years prior to enrollment).
    4. Abnormal TCD study requiring starting on chronic transfusion therapy.
    5. 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.

You CAN'T join if...

  • 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

Locations

  • University of California Los Angeles (UCLA)
    Los Angeles California 90095 United States
  • Children's Hospital and Research Center Oakland
    Oakland California 94609 United States

Lead Scientist at University of California Health

  • Theodore Moore (ucla)
    Professor of Clinical, Pediatrics, Medicine. Authored (or co-authored) 87 research publications

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
New York Medical College
Links
Consortium website for Parent-to-Child (haplo) Bone Marrow Transplant for Sickle Cell Disease (SCD)
ID
NCT01461837
Phase
Phase 2 research study
Study Type
Interventional
Participants
About 21 people participating
Last Updated