This phase II trial studies how well naive T-cell depletion works in preventing chronic graft-versus-host disease in children and young adults with blood cancers undergoing donor stem cell transplant. ...
Conduct a cross-sectional analysis of the nutritional status of individuals with PKU treated with pegvaliase for one or more years or who have followed an unrestricted diet for 6 or more months while ...
This is a prospective, multicenter, observational study designed to collect gastrointestinal related data in patients with Cystic Fibrosis (CF).
This is a Phase 2 open-label, single-arm design study with dose escalation by subject cohort . The study is designed to monitor the PTG-300 safety profile, to obtain preliminary evidence of efficacy ...
ZYN002 is a pharmaceutically manufactured Cannabidiol that is developed as a clear gel that can be applied to the skin (called transdermal delivery). The gel will be applied to clean, dry, intact ...
Patients with severe immune thrombocytopenia (ITP) present with similarly low platelet counts but varying bleeding symptoms, making it difficult to predict the disease course and to decide on an ...
This First-in-Human (FIH) Phase 1/2 study will evaluate mRNA-3704 in patients with methylmalonic acidemia/aciduria (MMA) due to methylmalonyl-coenzyme A mutase (MUT) deficiency between 1 to 18 years ...
This is a multi-center cohort study of approximately 250 previously untreated patients (PUPs) with congenital moderate to severe hemophilia A or B in a network of up to 50 US Hemophilia Treatment ...
The purpose of this study is to test whether an external erectile prosthesis (The Elator™) is a feasible alternative to internal erectile prostheses for transgender men who have undergone a ...
In this research study the investigators want to learn more about how a medication called tranexamic acid (TXA) could help reduce bleeding during Periacetabular Osteotomy (PAO) surgery. TXA is ...