Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT00957931
• Date of registration: 12/08/2009
• Prospective Registration: No
• Primary sponsor: Stanford University
• Public title: Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs
• Scientific title: Pilot Study MUD HCT:Pts High Risk Sickle Cell,Other Non-Malignant RBC Disorders- Reduced Intensity Preparative Regimen, HAPLO-Identical Mesenchymal Stromal Cells
• Date of first enrolment: March 2009
• Target sample size: 6
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00957931
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Sandhya Kharbanda, M.D.
• Affiliation: Stanford University

Key inclusion & exclusion criteria

Inclusion Criteria:

- Patients with sickle cell disease (SCD) 1-25 years of age with an HLA-identical, but
unrelated, donor or 1 human leukocyte antigen (HLA) allele mismatched bone marrow or
up to 2 HLA antigen mismatched umbilical cord blood (UCB) donor with one or more of
the following:

- Stroke, central nervous system (CNS) hemorrhage or a neurologic event lasting
longer than 24 hours.

- Acute chest syndrome with a history of recurrent hospitalizations or exchange
transfusions.

- Recurrent vaso-occlusive pain, 3 or more episodes per year for 3 years or more
years; or recurrent priapism.

- Impaired neuropsychological function and/or abnormal cerebral MRI scan or
abnormal transcranial Doppler (TCD).

- Stage I or II sickle lung disease.

- Sickle nephropathy (moderate or severe proteinuria or a glomerular filtration
rate (GFR) 30-50% of the predicted normal value).

- Bilateral proliferative retinopathy and major visual impairment in at least one
eye.

- Osteonecrosis of multiple joints with documented destructive changes.

- Requirement for chronic transfusions but with RBC alloimmunization >2 antibodies
during long term transfusion therapy.

- Failure of hydroxyurea (HU) therapy.

- Patients aged 0-21 years with transfusion dependent alpha- or beta-thalassemia who
have an HLA-identical or 1 HLA allele mismatched bone marrow or up to 2 HLA mismatched
UCB donor.

- Patients aged 0-21 years with Diamond-Blackfan anemia who have an HLA-identical or 1
HLA allele mismatched bone marrow or up to 2 HLA mismatched UCB donor. Diamond-
Blackfan anemia patients will only be eligible if they have failed steroid therapy.

Exclusion Criteria:

- Patients with one or more of the following:

- Karnofsky or Lansky performance score <70 (See Appendices I and II).

- Stage III-IV lung disease (Appendix III).

- GFR<30% predicted normal values.

- Pregnant or lactating females.

- Active serious infection whereby patient has been on intravenous antibiotics for
one week prior to study entry.

- Any patient with AIDS or HIV seropositivity.

- Any patient with invasive aspergillus infection within one year of study entry.

- Psychologically incapable of undergoing bone marrow transplant (BMT) with
associated strict isolation or documented history of medical non-compliance.



Age minimum: 1 Year
Age maximum: 25 Years
Gender: All

Health Condition(s) or Problem(s) studied

Diamond-Blackfan Anemia

Thalassemia

Sickle Cell Disease

Intervention(s)

Biological: Mesenchymal Stromal Cells

Procedure: Bone marrow transplantation

Primary Outcome(s)

Count of Participants With Stable Engraftment Post Hematopoietic Cell Transplantation (HCT) [Time Frame: Up to 1 year]

Secondary Outcome(s)

Overall Survival 6 Months Following HCT [Time Frame: 6 months]

Count of Participants With Disease-free Survival 6 Months Following HCT [Time Frame: 6 months]

Overall Survival 1 Year Following HCT [Time Frame: 1 year]

Count of Participants With Disease-free Survival 1 Year Following HCT [Time Frame: 1 year]

Secondary ID(s)

MSC01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

University of Minnesota

University of Alabama at Birmingham

Results

• Results available: Yes
• Date Posted: 06/08/2018
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00957931