9 results
To improve overall survival of patients with a early-stage high grade uterine leiomyosarcoma.
Comparison of steady-state cysteamine-trough WBC cystine levels between Cystagon® and RP103 over 3 months for each treatment period.
The objective of this pivotal study is to assess the PK and PD as well as safety and tolerability of RP103 compared to Cystagon® in patients with nephropathic cystinosis. Results of this Phase 3 study will be used to support the registration…
The objective of this pivotal study is to assess the long-term safety, tolerability, pharmacokinetics and pharmacodynamics of RP103 in pediatric and adult patients with nephropathic cystinosis. Results of this Phase 3 study will be used to support…
The aim of the trial is to individualize treatment for each patient by adapting it to early response and to treat only those patients with addiotional radiotherapy who show an inadequate treatment response. The degree of treatment response is…
The aim of the trial is to individualize treatment for each patient by adapting it to early response and thus to continue intensive treatment only with those patients wo show an inadequate treatment response.For patients who show a good initial…
The aim of this trial is to individualize and thus to optimize treatment for each patient by adapting it to the individual response. The treatment response is determined by means of FDG-PET after 2 cycles of escalated BEACOPP + 2 cycles of ABVD.The…
To determine the efficacy of treatment with mitapivat compared with placebo, as assessed by the reduction in transfusion burden in pediatric subjects with pyruvate kinase deficiency (PK deficiency) who are regularly transfused.
To determine the efficacy of treatment with mitapivat compared with placebo, as assessed by the increase in Hb concentrations in pediatric subjects with PK deficiency who are not regularly transfused