
A Study Evaluating the Long-Term Safety of Elexacaftor Combination Therapy
Cystic FibrosisThis study evaluate the long-term safety and tolerability of elexacaftor (ELX)/tezacaftor (TEZ)/ ivacaftor (IVA) triple combination (TC) in participants with cystic fibrosis (CF) who are homozygous for F508del.

Phase 2 Combination Study With Escalating Doses of MS1819-SD on Top of a Stable Dose of PPEs
Cystic FibrosisCystic Fibrosis Gastrointestinal Disease1 moreThis is a Phase 2 study sponsored by AzurRx SAS and involves testing of a new medication for the compensation of exocrine pancreatic insufficiency (EPI) caused by cystic fibrosis (CF). The new medication is called MS1819 spray dried (MS1819-SD) which is a lipase produced by the Lip2 gene of Yarrowia lipolytica using recombinant DNA technology. The primary purpose of this study is to investigate the efficacy and safety of escalating doses of study drug on top of a stable dose of PPEs in CF patients who are not fully compensated by PPEs only. This enzyme has demonstrated an appropriate profile to compensate the pancreatic lipase (enzyme) deficiency that is common in CP (chronic pancreatitis) and CF patients. The design of the study is open-label, meaning that all eligible patients will receive the study drug MS1819-SD. The study drug dose will increase throughout the study during dose escalation visits in each treatment period; study includes a total of three treatment periods. The total duration of the MS1819-SD treatment phase is of 39-51 days. The total duration of patient participation in the study is of 69-81 days. Approximately 24 patients will be enrolled in this study.

Efficacy, Safety and Tolerability of NP-120 on Idiopathic Pulmonary Fibrosis and Its Associated...
Idiopathic Pulmonary FibrosisNP-120 (Ifenprodil) has been shown to mediate anti-inflammatory responses and reduce pulmonary fibrosis in a murine model of Idiopathic Pulmonary Fibrosis (IPF). In addition, NP-120 significantly reduced both cough frequency and onset in a guinea pig tussive model. The purpose of this proof-of-concept trial is to determine the efficacy of NP-120 in the treatment of IPF and its associated cough.

Light Emitting Diode-Red Light (LED-RL) Phototherapy for Skin Scarring Prevention
FibrosisSkin Scarring4 moreSkin scarring (fibrosis) is a common complication in the wound healing process and remains a therapeutic challenge. Scar formation often occurs following injury to the skin such as surgery, trauma, and burns. The goal of this study is to evaluate the safety and efficacy of visible red light as a modality to reduce skin scarring after mini-facelift surgery. Based on laboratory data, light emitting diode-red light (LED-RL) phototherapy may lessen post-surgical skin fibrosis clinically.

MN4000 for Treatment of CF and MND Patients in the Home Setting
Cystic FibrosisMotor Neuron Disease1 moreThe study was a non-randomized open label pilot study. It was an observational design conducted at one (1) site in the US. All enrolled subjects received treatment with the MN4000. This pilot study evaluated subject satisfaction with the therapy and adherence to the therapy during the 90-day treatment period, and also collected clinical outcome data. Outcomes were assessed before, during and after the MN4000 treatment period.

A Phase 3 Study of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for...
Cystic FibrosisThis study will evaluate the efficacy of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function mutation (F/MF subjects).

Study Evaluating the Efficacy and Safety of Obeticholic Acid in Subjects With Compensated Cirrhosis...
Compensated CirrhosisNonalcoholic SteatohepatitisThe primary objective of this study is to evaluate whether obeticholic acid (OCA; INT-747) can lead to histological improvement in fibrosis with no worsening of NASH in adults with compensated cirrhosis due to NASH.

Comparative Effect of Nigella Sativa and Conventional Management for OSMF
Oral Submucous FibrosisOral submucous fibrosis (OSMF) is a potentially malignant condition mainly characterized by inflammation and fibrosis of the submucosal tissues leading to marked rigidity and reduced opening of the jaws. OSMF has a multifactorial etiology but areca nut consumption is considered to be the most consistent factor in its pathogenesis. There is no definitive treatment available for treating OSMF. Conventional management involves physiotherapy exercises and treating the disease with certain medications such as steroids and homeopathic medications.

The Effects of Telerehabilitation on Muscle Function, Physical Activity and Sleep in Cystic Fibrosis...
Cystic FibrosisCovid191 moreThe aim of this study is to evaluate the peripheral muscle function, sleep disorders and physical activity level in children with cystic fibrosis who are physically inactive at home due to social isolation and to examine the effect of the 6-week online exercise protocol.

Streamlined Treatment of Pulmonary Exacerbations in Pediatrics
Cystic FibrosisSTOP PEDS is a pilot study of children with CF ages 6-18 across 10 sites in North America. The primary goal is to assess the acceptability and feasibility of a multicenter randomized trial comparing immediate antibiotics versus tailored therapy for pulmonary exacerbation (PEx) treatment in this population.