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

Therapuetic Areas:Hematology
Age Range:2 - 20
Start Date:January 2012
End Date:December 2019
Contact:Mitchell S Cairo, MD

Use our guide to learn which trials are right for you!

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

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

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.

Inclusion Criteria:

- Homozygous Hemoglobin S Disease, or Hemoglobin S Beta0/+ thalassemia

- Patients must demonstrate one or more of the following Sickle Cell Disease

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

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

- 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
We found this trial at
40 Sunshine Cottage Road
Valhalla, New York 10595
(914) 594-4000
Principal Investigator: Mitchell S Cairo, MD
Phone: 914-594-2150
New York Medical College The College was founded in 1860 by a group of New...
Valhalla, NY
Click here to add this to my saved trials
747 52nd St
Oakland, California 94609
(510) 428-3000
Children's Hospital and Research Center Oakland For nearly 100 years, Children's Hospital & Research Center...
Oakland, CA
Click here to add this to my saved trials
Chicago, Illinois 60611
Phone: 312-227-4873
Chicago, IL
Click here to add this to my saved trials
Cincinnati, Ohio 45229
Cincinnati, OH
Click here to add this to my saved trials
Los Angeles, California 90095
Principal Investigator: Theodore Moore, MD
Phone: 310-825-6708
Los Angeles, CA
Click here to add this to my saved trials
Milwaukee, Wisconsin 53226
Principal Investigator: Julie Talano, MD
Phone: 414-955-4185
Milwaukee, WI
Click here to add this to my saved trials
Saint Louis, Missouri 63110
Principal Investigator: Shalini Shenoy, MD
Phone: 314-454-6018
Saint Louis, MO
Click here to add this to my saved trials