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A Double-Blind, Placebo-Controlled, Dose-Escalating, Phase 1 Study to Determine the Safety, Tolerability, Pharmacokinetics and Effect on Circulating Alpha-1 Antitrypsin Levels of ARC-AAT in Healthy Volunteer Subjects and in Patients with Alpha-1 Antitrypsin Deficiency (AATD)

The primary objective of the study is Study to determine the Safety, Tolerability, Pharmacokinetics and Effect on Circulating Alpha-1 Antitrypsin Levels of ARC-AAT in Patients with Alpha-1 Antitrypsin Deficiency (AATD)

A Phase 3, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation

primary objective: To evaluate the long-term safety and tolerability of lumacaftor in combination with ivacaftor in subjects with cystic fibrosis (CF), homozygous or heterozygous for the F508del-cystic fibrosis transmembrane conductance regulator (…

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Crossover Study to Evaluate the
Efficacy and Safety of Ivacaftor and VX-661 in Combination With Ivacaftor in Subjects Aged
12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation, and a
Second Allele With a CFTR Mutation Predicted to Have Residual Function

To evaluate the efficacy of VX-661 in combination with ivacaftor and ivacaftormonotherapy through 8 weeks of treatment in subjects with cystic fibrosis (CF)who are heterozygous for the F508del mutation on the CF transmembraneconductance regulator (…

Efficacy of RAD001/everolimus in Autism and NeuroPsychological deficits in children with TSC (RAPIT-trial)

The objective of this study is to determine whether influencing the protein pathway thought to underlie TSC by Everolimus treatment will improve cognitive abilities in these children.

A randomized, double-blind, placebo-controlled, parallel group study to evaluate the efficacy and safety of AFQ056 in adolescent patients with Fragile X Syndrome

The purpose of the study is to assess the efficacy and safety in adolescents of AFQ056 in treating behavioral symptoms characteristic of patients with FXS. Data from this study will be used to support the registration of AFQ056 in this population…

A Phase 3, Randomized, Double Blind, Placebo Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation

Primary ObjectiveTo evaluate the efficacy of lumacaftor in combination with ivacaftor throughat Week 24 in subjects with cystic fibrosis (CF) who are homozygous for the F508del CFTR mutation on the CF transmembrane conductance regulator (CFTR)…

A Multi-Centre, Randomised, Double Blind, Placebo Controlled, Parallel Group Study of TA 8995 in Patients with Mild Dyslipidaemia, Alone and In Combination with Statin Therapy

The primary objective of this study is to evaluate the efficacy of different doses of TA 8995, a cholesteryl ester transfer protein (CETP) inhibitor, on the elevation of high density lipoprotein cholesterol (HDL C) and reduction of low density…

A phase III, randomized, double blind, placebo-controlled clinical study to assess the efficacy and safety of GSK2402968 in subjects with Duchenne muscular dystrophy (DMD114044)

Primary: to assess efficacy of subcutaneous 6 mg/kg GSK2402968 versus placebo administered over 48 weeks in ambulant subjects with DMD. Secondary: Safety, tolerability, PK, quality of life.

Topical rapamycin to treat fibrofolliculomas in Birt-Hogg-Dubé syndrome

Primary Objective: To determine whether topical application of rapamycin can lead to reduction in size and/or number of fibrofolliculomas in BHD patients and may prevent the growth of new ones. Secondary Objectives: Safety, formula acceptability and…

The adenosine receptor; a new pharmacological tool in the treatment of sepsis.

Does antagonism of the adenosine receptor by caffeine lead to an increased LPS-induced inflammatory reaction and an increase in (subclinical) tissuedamage?Does the C34T-polymorphism of the enzyme AMP-deaminase lead to a decreased inflammatory…

Pharmacological and genetic imaging of executive function deficits

With this study we want to identify the common aetiology and neurobiology of EFD, by employing pharmacological and genetic imaging in three neuropsychiatric disorders. We want to identify an underlying functional and structural model for EFD,…

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate
the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation

To evaluate the efficacy of VX-661 in combination with ivacaftor through 24 weeks of treatment in subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene.

A Phase 2, Randomized, Double blind, Controlled Study to Evaluate the Safety and Efficacy of VX 440 Combination Therapy in Subjects Aged 12 Years and Older With Cystic Fibrosis

1. Primary Objectives• To evaluate the safety and tolerability of VX 440 monotherapy and VX 440 in dual and triple combination with VX-661 and IVA• To evaluate the efficacy of VX 440 monotherapy and VX 440 in dual and triple combination with VX 661…

A Phase IIa, randomized, double-blind, placebo-controlled study to evaluate multiple doses of GLPG2222 in subjects with Cystic Fibrosis who are homozygous for the F508del mutation

To evaluate the safety and tolerability of 4 different doses of GLPG2222 administered orally and q.d. for 29 days in adult subjects with CF whoare homozygous for the F508del CFTR mutation.Secondary objectives:To assess changes in biomarkers of CFTR…

A Phase 1/2 Study of VX-445 in Healthy Subjects and Subjects With Cystic Fibrosis

Parts D and E: Primary Objectives:• To evaluate the safety and tolerability of VX-445 in TC with TEZ and IVA in subjects with cystic fibrosis (CF)• To evaluate the efficacy of VX-445 in TC with TEZ and IVA in subjects with CFSecondary Objectives• To…

A randomized, double-blind, placebo-controlled, parallel-group study to evaluate the efficacy and safety of evinacumab in patients with homozygous familial hypercholesterolemia

The primary objective of the study is:* To demonstrate the reduction of low-density lipoprotein cholesterol (LDL-C) by evinacumab 15 mg/kg intravenously (IV) in comparison to placebo after 24 weeks in patients with homozygous familial…

A Phase 3, Randomized, Double-blind, Placebo-controlled Multicenter Study with an Open-Label Extension to Evaluate the Efficacy and Safety of Givosiran in Patients with Acute Hepatic Porphyrias (ENVISION)

Primary:Evaluate the effect of subcutaneous (SC) givosiran (ALN-AS1), compared to placebo, on the rate of porphyria attacks requiring hospitalization, urgent healthcare visit, or intravenous (IV) hemin administration at home in patients with acute…

A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del
Mutation and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)

Primary objective: - To evaluate the efficacy of VX-445/TEZ/IVA in CF subjects who are heterozygous for F508del and a gating or residual function mutation (F/G and F/RF genotypes).Secondary objectives: - To evaluate the safety of VX-445/TEZ/IVA- To…

A single-center, randomized, double-blind, placebo-controlled, parallel-group studie investigating the safety, tolerability and pharmacokinetics of single- and multiple-ascending subcutaneous doses of TA-46 in healthy volunteers

The study will be conducted in 94 healthy male and female volunteers. The study consists of 3 parts: Part A, Part B and Part C.Part A will be conducted in 40 healthy male and female volunteers divided into 5 groups with 8 volunteers each.Part B will…

A PHASE II, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL GROUP STUDY TO EVALUATE THE SAFETY, EFFICACY, AND PHARMACODYNAMICS OF 52 WEEKS OF TREATMENT WITH BASMISANIL IN PARTICIPANTS AGED 2 TO 14 YEARS OLD WITH DUP15Q SYNDROME FOLLOWED BY A 2-YEAR OPTIONAL OPEN-LABEL EXTENSION

Part 1:To evaluate the effects of 52 weeks of treatment with basmisanil on core symptom domains of Dup15q syndrome (language and social skills) and dailyfunctioning. Part 2:• To evaluate the tolerability and safety of up to 3 years of treatment with…