An internal pilot design for prospective cancer screening trials with unknown disease prevalence
Conclusion Conducting an internal pilot study without adjusting the critical value can cause Type I error rate inflation in small samples, but not in large samples. An internal pilot approach usually achieves goal power and, for most studies with sample size greater than 50, requires no Type I error correction. Further, we have provided a flexible and accurate approach to bound Type I error below a goal level for studies with small sample size. (Source: Trials)
Source: Trials - October 13, 2015 Category: Research Source Type: clinical trials

HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy
Conditions:   Fanconi Anemia;   Severe Marrow Failure;   Myelodysplastic Syndrome (MDS);   Acute Myelogenous Leukemia (AML)Interventions:   Drug: Busulfan;   Drug: Cyclophosphamide;   Drug: Fludarabine;   Drug: rabbit ATG;   Drug: G-CSF;   Biological: Peripheral blood stem cellSponsors:   Children's Hospital Medical Center, Cincinnati;   Memorial Sloan Kettering Cancer CenterRecruiting - verified April 2015 (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - April 28, 2014 Category: Research Source Type: clinical trials

Use Massive Parallel Sequencing and Exome Capture Technology to Sequence the Exome of Fanconi Anemia Children and Their Patents
Conditions:   Fanconi Anemia;   Autosomal or Sex Linked Recessive Genetic Disease;   Bone Marrow Hematopoiesis Failure, Multiple Congenital Abnormalities, and Susceptibility to Neoplastic Diseases.;   Hematopoiesis Maintainance.Interventions:   Genetic: human whole exome;   Genetic: whole genomicSponsors:   Chinese Academy of Medical Sciences;   Xiaofan ZhuAvailable - verified November 2013 (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - November 21, 2013 Category: Research Source Type: clinical trials