Phase 3
Completed N=7
Allogeneic Transplantation For Severe Osteopetrosis
Severe Osteopetrosis
Source: ClinicalTrials.gov NCT00775931 ↗
Enrolled (actual)
7
Serious AEs
42.9%
Results posted
Jul 2019
Primary outcomePrimary: Number of Patients Who Achieved Donor Cell Engraftment — 5; 0 Participants
◆ Published Evidence
No publication linked
No peer-reviewed publication reporting this trial's results has been linked yet. This can indicate results are unpublished — a known publication-bias signal. We re-check periodically.
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.
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 | — |
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
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. Informational only — not medical advice.