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Clinical Trials About "Vertex Receives CHMP Positive Opinion ORKAMBI lumacaftor ivacaftor" RSS

10:36 EDT 19th September 2018 | BioPortfolio

We list hundreds of Clinical Trials about "Vertex Receives CHMP Positive Opinion ORKAMBI lumacaftor ivacaftor" on BioPortfolio. We draw our references from global clinical trials data listed on ClinicalTrials.gov and refresh our database daily.

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Showing "Vertex Receives CHMP Positive Opinion ORKAMBI lumacaftor ivacaftor" Clinical Trials 1–25 of 5,200+

Extremely Relevant

A Drug-Drug Interaction Study of N91115 +/- Rifampin in Healthy Adult Subjects

The present study is designed to support dose selection of N91115 in a planned Phase 2 study in CF patients who are on co-formulated lumacaftor/ivacaftor (Orkambi) treatment. Rifampin is being used in this drug-drug interaction (DDI) study to assess its effects on the PK of N91115, since rifampin's metabolic properties are believed to be similar to lumacaftor. The Orakambi NDA was approved July 1, 2015. It is important to understand the potential effects of Orkambi (lumacafto...


Real-life Follow-up of Cystic Fibrosis Patients Treated With Ivacaftor+Lumacaftor (Orkambi*)

The purpose of the study is to examine the real-life safety and effectiveness of the novel combination ivacaftor+lumacaftor in eligible patients with cystic fibrosis (CF). All patients with CF were eligible if they were 12 years and older, started ivacaftor+lumacaftor outside of a clinical trial between December 15th 2017 and December 15th 2018 in an accredited CF center in France. Patient followed-up is based on standardized recommendation of the French Cystic Fibrosis Society...

The Effect of Combination Lumacaftor and Ivacaftor on Markers of Hyperglycemia

The purpose of this research study is to find out if the combined therapy lumacaftor-ivacaftor affects glycemia in patient with cystic fibrosis.


Lum-Iva-biota: Exploring the Respiratory Mycobiota and Microbiota Profile in French CF Patients Taking Lumacaftor-Ivacaftor

In 2015, VERTEX company - producing already KALYDECO (IVACAFTOR, VX-770) potentiator molecule that is recommended for the treatment of CF patients aged ≥ 6 y, with CFTR mutation altering the channel regulation (class III mutations) as G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549Nou S549R) - was allowed by the Federal Drug Administration (FDA) and European Medicines Agency (EMEA) for producing and using the association LUMACAFTOR-IVACAFTOR in clinical trials to ma...

: TRANSITION: An Observational Study of Transition From Lumacaftor/Ivacaftor to Tezacaftor/Ivacaftor (Tez/Iva)

This study is a single center study of clinical and laboratory outcomes in patients ≥ 12 who transition from use of Orkambi to tez/iva. Clinical and laboratory measurements will be measured at baseline, 1 month, 3 months, and 6 months after initiation of tez/iva. The length of study participation will be approximately 6 months.

Orkambi Exercise Study

Shortness of breath (dyspnea) during exercise is a major source of distress and is a commonly reported symptom in patients with cystic fibrosis (CF). A recent treatment option known as Orkambi, which combines the drugs lumacaftor and ivacaftor, may be used in patients with CF to help improve lung health. However, the effects of this combination therapy on dyspnea and exercise performance, a known predictor of survival in CF, are not clear. The investigators aim to understand th...

ICM to Evaluate the Activation of p.Phe508del-CFTR by Lumacaftor in Combination With Ivacaftor

The academic investigator - initiated trial will evaluate in a postapproval setting whether, and if yes, to what extent and variability, the treatment with lumacaftor in combination with ivacaftor reverses the p.Phe508del CFTR - mediated basic defect in p.Phe508del homozygous subjects with cystic fibrosis under real life conditions.

Effect of Lumacaftor-ivacaftor on Glucose Handling and Tolerance in Cystic Fibrosis Phe508del

The purpose of this research study is to find out if the combined therapy lumacaftor-ivacaftor effects how people with cystic fibrosis respond to an oral glucose tolerance test, a test for diabetes.

Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor

Study 110 is a Phase 3, multicenter study in subjects aged 6 years and older with cystic fibrosis (CF) who are homozygous for the F508del-CF transmembrane conductance regulator (CFTR) mutation and who participated in Study 109 (NCT02514473) or Study 011B (NCT01897233). Study 110 is designed to evaluate the safety and efficacy of long term treatment of lumacaftor in combination with ivacaftor.

Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del

This is a Phase 3, 2-part (Part A and Part B), open-label, multicenter study evaluating the pharmacokinetics (PK), safety, tolerability, and pharmacodynamics (PD) of multiple doses of Lumacaftor/Ivacaftor (LUM/IVA) in subjects 2 through 5 years of age (inclusive) with Cystic Fibrosis (CF), homozygous for F508del. Subjects who participate in Part A may participate in Part B, if patient meet the eligibility criteria.

Observational Study of Glucose Tolerance Abnormalities in Patient With Cystic Fibrosis Homozygous for Phe 508 Del CFTR Treated by Lumacaftor-Ivacaftor

Cystic Fibrosis related diabetes (CFRD), a major factor of morbid-mortality in CF, is characterized by a preclinical phase of glucose intolerance particularly long reaching up to 10 years. At the physiopathology level, insulin secretion is determinant in the glucose tolerance abnormalities in CF. Indeed insulin secretion is dependent of the CFTR activity at the beta cell surface and inhibition of CFTR leads to a decrease in insulin secretion. Recently, the combination o...

Study of N91115 in Patients With CF Homozygous for the F508del-CFTR Mutation

This will be a double-blind, randomized, placebo-controlled, parallel group study. The purpose of this study is to investigate the efficacy and safety of N91115 in adult patients with CF who are homozygous for the F508del-CFTR mutation and being treated with lumacaftor/ivacaftor (Orkambi™).

Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Subjects 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del

This study will evaluate the safety and pharmacokinetics (PK) of lumacaftor (LUM) and ivacaftor (IVA) in subjects 1 to less than 2 years of age with cystic fibrosis (CF), homozygous for F508del (F/F).

A Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects With CF, Homozygous for the F508del-CFTR Mutation

To evaluate the efficacy and safety of lumacaftor in combination with ivacaftor in subjects aged 6 Through 11 years with cystic fibrosis (CF), homozygous for the F508del CF transmembrane conductance regulator (CFTR) mutation

A Study to Explore the Impact of Lumacaftor/Ivacaftor on Disease Progression in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del

This study will explore the impact of lumacaftor/ivacaftor (LUM/IVA) on disease progression in subjects aged 2 through 5 years with cystic fibrosis (CF), homozygous for F508del (F/F)

Tezacaftor/Ivacaftor Combination Therapy Expanded Access Program for Patients 12 Years of Age and Older With Cystic Fibrosis

To provide TEZ/IVA combination therapy to CF patients who are 12 years of age and older who completed Vertex TEZ/IVA combination therapy clinical studies (NCT02565914 or NCT03150719). To provide TEZ/IVA combination therapy to CF patients in critical need who are 12 years of age and older, homozygous for F508del.

iPS Cell Response to CFTR Modulators

Orkambi is currently approved for patients with CF carrying two copies of the common F508del variant, but not for those with the P67L mutation. This is a clinical study of 22 subjects carrying P67L, each given Orkambi for approximately four weeks. The researchers will monitor clinical endpoints that include FEV1, sweat chloride, and nasal potential difference. Cutaneous punch biopsy material will be obtained from each subject so that iPS cells can be differentiated into airway ...

Relevant

A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor

This is a Phase 3, randomized, double blind, placebo controlled, parallel group, multicenter study in people with CF who are homozygous for the F508del CFTR mutation.

A Study to Evaluate the Effect of VX-661 in Combination With Ivacaftor on Chest Imaging Endpoints in Subjects With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation

The primary purpose of study is to evaluate the treatment effect of VX-661 in combination with ivacaftor (VX-661/ivacaftor) on chest imaging endpoints using low-dose computed tomography (LDCT) at Week 72, and to evaluate the safety of VX-661/ivacaftor through Week 72

Impact of the Introduction of ORKAMBI on Anxiety, Depression, Quality of Life and Adherence of Adolescents and Young Adults

The aim of this project is to evaluate the psychological reshuffle induced by ORKAMBI. The particular focus of this study is the consequence of its introduction on anxiety, depression, quality of life and adherence to all cystic fibrosis (CF) treatment. To answer this question investigators will monitor the psychological function of CF adolescents and young adults treated with ORKAMBI and compare them to CF adolescents and young adults not treated with ORKAMBI.

Novel Therapeutic Approaches for Treatment of CF Patients With W1282X Premature Termination Codon Mutations

Based on previous clinical findings, we hypothesize that ivacaftor will have synergistic effects with drugs that facilitate truncated but partially active W1282X CFTR protein processing (tezacaftor) in patients with W1282X CFTR. In the current study, we propose to directly test the efficacy of tezacaftor/ivacaftor (TEZ/IVA) for W1282X CFTR therapy in the clinic in comparison to ivacaftor alone.

A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have a CFTR Gating Mutation

The purpose of this study is to evaluate the safety of ivacaftor treatment, and PK of ivacaftor and metabolites in subjects with cystic fibrosis (CF) who are

Evaluation of Ivacaftor in Patients Using Ataluren for Nonsense Mutations

The purpose of this study is to explore the combination of ataluren and ivacaftor as a treatment for patients with a specific cystic fibrosis mutation

A Phase 3 Study of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Who Have One F508del-CFTR Mutation and a Second Mutation That Has Been Demonstrated to be Clinically Responsive to Ivacaftor

This is a Phase 3, randomized, double-blind, ivacaftor-controlled, parallel-group, multicenter study in subjects aged 12 years and older with CF who are heterozygous for the F508del-CFTR mutation and a second CFTR allele with a gating defect that is clinically demonstrated to be ivacaftor responsive.

A Study to Evaluate the Safety of Long-term Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have a CFTR Gating Mutation

This is a Phase 3, 2-arm, multicenter study with an open-label ivacaftor arm and an observational arm to evaluate the safety and efficacy of long-term ivacaftor treatment in subjects with cystic fibrosis (CF) who are


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