MedTrace Logo

Reduced Intensity AlloTransplant For Osteopetrosis

NCT00638820 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 3

Last updated 2017-12-28

Study results available
· View outcomes & findings →

Summary

We believe that hematopoietic stem cell transplantation (HSCT) will help subjects with Osteopetrosis generate functioning osteoclasts, and by so doing assist in the resolution of the abnormal bone architecture, and the anemia and bone marrow failure that is also characteristic of this disease. However, we have found in past studies that approximately 30% of Osteopetrosis patients do not engraft. Therefore, in this study, we plan to use a different combination of pre-transplant drugs to try to make transplants safer for this disease, as well as to provide a second infusion of stem cells in patients with matched related or unrelated donors. The purpose of this research is to find a safer and more effective means of performing stem cell transplantation in patients with Osteopetrosis, using chemotherapy and radiation designed to bring about engraftment and lessen transplant mortality.

Conditions

  • Osteopetrosis

Interventions

PROCEDURE

Stem Cell or Umbilical Cord Blood Transplantation

Stem Cell (unrelated or matched related donor grafts (both peripheral blood and marrow) infusion on Day 0 and 42; Umbilical Cord Blood on Day 0 and 42

DRUG

Campath, Busulfan, Clofarabine

* 12 Campath-1H 0.3 mg/kg intravenously (IV) over 2 hours * 11 Campath-1H 0.3 mg/kg intravenously over 2 hours * 10 Campath-1H 0.3 mg/kg intravenously over 2 hours * 9 Busulfan \<12 kg: 2.2 mg/kg/dose IV every 12 hours \>12 kg: 1.6 mg/kg/dose IV every 12 hours * 8 Busulfan \<12 kg: 2.2 mg/kg/dose IV every 12 hours \>12 kg: 1.6 mg/kg/dose IV every 12 hours * 7 "Rest" * 6 Clofarabine 40 mg/m2 intravenously over 2 hours * 5 Clofarabine 40 mg/m2 intravenously over 2 hours * 4 Clofarabine 40 mg/m2 intravenously over 2 hours * 3 Clofarabine 40 mg/m2 intravenously over 2 hours * 2 Clofarabine 40 mg/m2 intravenously over 2 hours

PROCEDURE

Total Lymphoid Irradiation

Dose 500 cGy via anteroposterior (AP) and posteroanterior (PA) fields (250 cGy AP and 250 cGy PA).

Sponsors & Collaborators

  • Masonic Cancer Center, University of Minnesota

    lead OTHER

Principal Investigators

  • Paul Orchard, MD · Masonic Cancer Center, University of Minnesota

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
45 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2007-09-30
Primary Completion
2008-05-31
Completion
2008-05-31

Countries

  • United States

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00638820 on ClinicalTrials.gov