Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases

NCT03367546 | Terminated Phase 2

• 2 other identifiers: 2017LS101MT2017-30
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

Brief Summary

This is a Phase II study for the use of T-cell replete reduced intensity conditioning (RIC) haploidentical donor allogeneic hematopoietic cell transplantation (HaploHCT) for individuals with high-risk non-malignant diseases who lack a suitable HLA-matched sibling donor.

Conditions

Sickle Cell Disease; Thalassemia; High Risk Hematologic Disorders; Cerebral Adrenoleukodystrophy; Inherited Metabolic Disorders

Keywords

SCD; cALD

Outcome Measures

Primary Outcomes (1)

• Neutrophil Recovery

  Incidence of neutrophil recovery by day +42

  Day 42

Secondary Outcomes (2)

• Overall Survival (OS)

  1 year

• Primary Graft Failure (neutropenic and non-neutropenic)

  Day 42

Study Arms (1)

rATG, FLU/CY/TBI, & Thiotepa

EXPERIMENTAL

Anti-Thymocyte Globulin - Rabbit (rATG), Fludarabine (Fludara), Cyclophosphamide (Cytoxan, Neosar), Total Body Irradiation (TBI), \& Thiotepa

Procedure: Blood and Marrow Transplant

Interventions

Blood and Marrow Transplant PROCEDURE

Reduced intensity conditioning (RIC) with rabbit ATG, fludarabine, cyclophosphamide, thiotepa and low dose (2 Gy) total body irradiation followed by T-cell replete, unmanipulated, haploidentical related donor stem cell transplant (HaploHCT) and post-transplant cyclophosphamide (PTCy)

rATG, FLU/CY/TBI, & Thiotepa

Eligibility Criteria

• Age: 0 Years - 25 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Sickle Cell Disease (SCD)
• \* If diagnosis of SCD must meet one or more of the following disease characteristics:
• Stroke, CNS hemorrhage or a neurologic event lasting longer than 24 hours, or abnormal cerebral MRI or cerebral arteriogram or MRI angiographic study and impaired neuropsychological testing
• 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 abnormal cerebral MRI scan
• 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
• RBC alloimmunization
• Transfusion Dependent Alpha- or Beta-Thalassemia
• Other Non-Malignant Hematologic Disorders:
• Transfusion dependent or involve other potential life-threatening cytopenias, including but not limited to Paroxysmal Nocturnal Hemoglobinuria, Glanzmann's Thrombasthenia, Severe Congenital Neutropenia and Shwachman-Diamond Syndrome

You may not qualify if:

• Availability of a suitable HLA-matched related donor
• Uncontrolled infection
• Pregnant or breastfeeding
• HIV positive

Sponsors & Collaborators

• Masonic Cancer Center, University of Minnesota: lead

Study Sites (1)

Masonic Caner Center at University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

MeSH Terms

Conditions

Anemia, Sickle Cell; Thalassemia

Interventions

Blood Specimen Collection

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Specimen Handling; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Punctures; Surgical Procedures, Operative; Investigative Techniques

Study Officials

• Christen L Ebens, MD, MPH

  University of Minnesota - Pediatrics Blood and Marrow Transplant

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 5, 2017
• First Posted: December 8, 2017
• Study Start: July 2, 2018
• Primary Completion: December 19, 2022
• Study Completion: December 19, 2022
• Last Updated: July 12, 2024

Record last verified: 2024-07

Locations

US (1)