Phase 3 N=7 Treatment

Allogeneic Transplantation For Severe Osteopetrosis

Severe Osteopetrosis

Bottom Line

View on ClinicalTrials.gov: NCT00775931 ↗

Enrolled (actual)

Serious AEs

42.9%

Results posted

Jul 2019

Primary outcome: Primary: Number of Patients Who Achieved Donor Cell Engraftment — 5; 0 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: umbilical cord blood transplantation (Procedure); Campath-1H (Drug); Total Lymphoid Irradiation (Radiation); Cyclophosphamide (Drug); Busulfan (Drug); Fludarabine monophosphate (Drug); marrow graft transplantation (Procedure)
Age: Pediatric, Adult · 0+ yrs
Sex: All
Sponsor: Masonic Cancer Center, University of Minnesota
Primary completion: Jun 2013

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Patients Who Achieved Donor Cell Engraftment	5; 0	—
SECONDARY Transplant Related Mortality at 100 Days	0; 0	—
SECONDARY Transplant Related Toxicity	3; 1	—
SECONDARY Incidence of Grade II - IV Acute Graft-versus-host Disease	2; 0	—

Summary

The purpose of this research is to explore what we believe may be 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. Prior multi-institutional data in past studies found that approximately 30% of Osteopetrosis patients do not engraft. Therefore, in this study, we utilize a reduced intensity design 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.

Eligibility Criteria

Inclusion Criteria

Patients eligible for transplantation under this protocol will be 45 years of age
Evidence of hepatic failure
Pulmonary dysfunction sufficient to significantly increase the risk of transplant.
Renal dysfunction with glomerular filtration rate (GFR) <30% of predicted.
Cardiac compromise sufficient to substantially increase the risk of transplantation
Severe, stable neurologic impairment.
Human immunodeficiency virus (HIV) positivity.
Pregnant or lactating females

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00775931). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.