Hematopoietic Stem Cell Transplant For Patients With Dyskeratosis Congenita and Severe Aplastic Anemia
This is an open label, single arm, phase II clinical trial designed to evaluate the safety
and efficacy of the treatment regimen. Efficacy will be measured by long-term engraftment
of the transplanted cells.The primary endpoint of neutrophil engraftment is defined as an
absolute neutrophil count (ANC) >5 x 108/L (first of three consecutive laboratory
measurements on different days) with at least 10% donor cells by day 100. We will evaluate
the proportion of success (P) and its 95% confidence interval (CI) for the entire group.
The null hypothesis of 90% engraftment will be rejected if 4 or more patients fail to
engraft out of 15 evaluable patients. The secondary endpoints of regimen related mortality,
acute and chronic graft-versus-host disease (GVHD) and secondary malignancies will be
estimated by cumulative incidence treating non-event deaths as a competing risk. Survival
will be estimated by Kaplan-Meier methods. Immune reconstitution will be summarized with
SAA and DC arms will be analyzed separately.
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Defined as an absolute neutrophil count (ANC) >5 x 10^8/L (first of three consecutive laboratory measurements on different days) with at least 10% donor cells by day 100. Demonstrate sustained engraftment after a fludarabine based preparative regimen in patients with dyskeratosis congenita followed by HCT.
Jakub Tolar, M.D., Ph.D.
Masonic Cancer Center, University of Minnesota
United States: Institutional Review Board
|Masonic Cancer Center, University of Minnesota||Minneapolis, Minnesota 55455|