Tundra Space

Tundra Space

Clinical Research Directory

Browse clinical research sites, groups, and studies.

Back to Studies
ACTIVE NOT RECRUITING
NCT02171104
PHASE2

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

Sponsor: Masonic Cancer Center, University of Minnesota

View on ClinicalTrials.gov

Summary

This single-institution, phase II study is designed to test the ability to achieve donor hematopoietic engraftment while maintaining low rates of transplant-related mortality (TRM) using busulfan- and fludarabine-based conditioning regimens with busulfan therapeutic drug monitoring (TDM) for patients with various inherited metabolic disorders (IMD) and severe osteopetrosis (OP).

Official title: MT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATG

Key Details

Gender

All

Age Range

Any - 55 Years

Study Type

INTERVENTIONAL

Enrollment

149

Start Date

2014-07-10

Completion Date

2029-07-14

Last Updated

2026-01-07

Healthy Volunteers

No

Interventions

BIOLOGICAL

Stem Cell Transplantation

Infusion given on Day 0

DRUG

IMD Preparative Regimen

* Anti-thymocyte Globulin (ATG) * Fludarabine * Busulfan

DRUG

Osteopetrosis Only Preparative Regimen

* Anti-thymocyte Globulin (ATG) * Fludarabine * Busulfan * Thiotepa

DRUG

Osteopetrosis Haploidentical Only Preparative Regimen

* Rituximab * Alemtuzumab * Busulfan * Fludarabine

DRUG

cALD SR-A (Standard-Risk, Regimen A)

N-acetylcysteine start day +1 through day +28

DRUG

cALD SR-B (Standard-Risk, Regimen B)

N-acetylcysteine start day +1through day +56

DRUG

cALD HR-D (High-Risk, Regimen C)

N-acetylcysteine and celecoxib start day of admission (prior to conditioning regimen) and continue through day +100

DRUG

cALD HR-D (High-Risk, Regimen D)

N-acetylcysteine, celecoxib, vitamin E and alpha lipoic acid start day of admission (prior to conditioning regimen) and continue through day +100

Locations (1)

Masonic Cancer Center, University of Minnesota

Minneapolis, Minnesota, United States