Haplo T-Cell Depleted Transplantation in High-Risk Sickle Cell Disease (HaploSCD)

New York Medical College

Status and phase

Active, not recruiting

Phase 2

Conditions

Sickle Cell Disease

Treatments

Drug: CD34 selected T-cell depleted allogeneic SCT

Study type

Interventional

Funder types

Other

Industry

Identifiers

NCT01461837

NYMC526-4090

FD-R-0004090 (Other Grant/Funding Number)

Details and patient eligibility

About

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

Full description

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.

Enrollment

21 patients

Sex

All

Ages

2 to 20 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

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.

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