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Ephedrine as add-on therapy for patients with myasthenia gravis

The main objective of this pilot study of n-of-one trials is to determine the effect of add-on treatment with ephedrine for all participants enrolled in this small series of n-of-one trials. Secondary objectives include determining the effect in…

Probiotics in atherosclerosis: a pilot study

The objective of the study is to see whether oral administration of probiotics can modulate certain immunological markers which are associated with accelarated atherosclerosis in patients with ANCA associated vasculitis.

A Randomized, controlled, cross-over Study to assess Hypoallergenicity of an extensively hydrolyzed whey protein Infant formula in children with cow's milk allergy using amino-based infant formula as a reference.

The primary study objective is to assess the hypoallergenicity of an extensively hydroloyzed whey protein infant formula in children with cow's milk allergy.The second study objective is to assess the long-term effects on growth and tolerance…

oral PHA-022121 for the acute treatment and prophylaxis Of angioedema attacks in Patients with Acquired C1-Inhibitor Deficiency

Primary objectives: to evaluate the efficacy of three different single doses of PHA-022121 versus placebo in achieving angioedema symptom relief during acute attacks and the efficacy of prophylactic treatment with PHA-022121 versus placebo in…

A randomized, double-blind, placebo-controlled, phase II, cross-over clinical trial evaluating the
efficacy and safety of KVD900, an oral plasma kallikrein inhibitor, in the on-demand treatment of
angioedema attacks in adult subjects with hereditary angioedema type I or II

Primary Objective:• To investigate the efficacy of KVD900 compared to placebo in halting the progression of a peripheral or abdominal attack of hereditary angioedema (HAE).Secondary Objectives:• To investigate the safety and tolerability of KVD900…

Leflunomide and Hydroxychloroquine combination therapy
for primary Sjögren*s Syndrome

This study has been transitioned to CTIS with ID 2024-510904-36-00 check the CTIS register for the current data. To assess clinical efficacy and safety of Leflunomide/Hydroxychloroquine in pSS patients in a phase IIb placebo-controlled randomized…

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Crossover Study to Evaluate the Efficacy of Lumacaftor/Ivacaftor Combination Therapy in Subjects With Cystic Fibrosis Who Have an A455E-CFTR Mutation

Primary ObjectiveTo evaluate the efficacy of lumacaftor/ivacaftor combination therapy (LUM/IVA) in subjects with cystic fibrosis (CF) 12 years of age and older who have at least one A455E mutation.Other Objectives* To explore the association between…