Phase I/II Study of Allogeneic Stem Cell Transplantation to Treat Clinically Aggressive Sickle Cell Disease (SCD)

About the study

The investigators propose to determine the engraftment and transplant related morbidity and mortality after a non-myeloablative allogeneic hematopoietic stem cell transplant protocol using immune- suppressive agents and low-dose total body irradiation (TBI) without standard chemotherapy in patients with aggressive sickle cell disease who are not candidates for or experienced complications from hydroxyurea therapy.

Fully HLA matched siblings will be used as donors for hematopoietic stem cells to reduce the risk of morbidity and mortality in this cohort of patients.

Study point of contact

Lani Krauz, RN
312-413-0242
[email protected]
Damiano Rondelli, MD
312-996-6179
[email protected]

Locations

1 United States site

Age

16 to 60 Years

Phase

Phase 1/Phase 2

Study type

Interventional

Age

16 Years - 60 Years

Gender

All

Interventions

Procedure

Drug

Compensation

Unknown

participation requirements

Patients with sickle cell disease, subtype Hgb SS, SC, or SB disease who are on chronic transfusion therapy for a prior stroke or those patients who were intolerant of hydroxyurea therapy or were being treated with hydroxyurea therapy and were complicated by at least one of the following:

Stroke or central nervous system event lasting longer than 24 hours
Frequent vaso-occlusive pain episodes, defined as ≥ 3 per year severe enough to interfere with the patient’s normal daily function or require medical attention in the clinic, emergency room, acute care center, or hospital
Recurrent episodes of priapism, defined as ≥ 2 per year requiring emergency room visits
Acute chest syndrome with recurrent hospitalizations, defined as ≥ 2 lifetime events
Red-cell alloimmunization (≥ 2 antibodies) during longterm transfusion therapy
Bilateral proliferative retinopathy with major visual impairment in at least one eye
Osteonecrosis of 2 or more joints
Sickle cell nephropathy
Stage I or II sickle lung disease
Symptoms of pulmonary hypertension and mean pulmonary artery pressure > 25mmHg
Age 16-60 years
Karnofsky performance status of 70 or higher
Adequate cardiac function, defined as left ventricular ejection fraction ≥ 40%
Adequate pulmonary function, defined as diffusion lung capacity of carbon monoxide ≥ 50%
Estimated GFR ≥ 30mL/min as calculated by the modified MDRD equation
ALT ≤ 3x upper limit of normal
No evidence of chronic active hepatitis or cirrhosis
HIV-negative
Patient is not pregnant
History of compliance with medications and medical care
Patient is able and willing to sign informed consent
Patient has an HLA-identical matched related donor

Locations

  • Chicago, Illinois, United States, University of Illinois at Chicago, 60612 [Recruiting]
Last updated 2022-02-16