Clinical trials: Functional Deformities
Cystic Fibrosis
A Study Evaluating Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis and F/MF Genotypes
This study will evaluate the efficacy and safety of elexacaftor (ELX) / tezacaftor (TEZ) / ivacaftor (IVA) triple combination (TC) in subjects 6 through 11 years of age with cystic fibrosis (CF) who are heterozygous for F508del…
- Trial name
- NCT04353817
- Testing
- ELX/TEZ/IVA, IVA
- Where
- 34 sites in Australia, Canada and 8 more
- Volunteers
- 121 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Infantile Colics, Constipation
Randomized, Blinded, Placebo-Controlled Evaluation of BioAmicus Complete
This is a randomized, single-blind, placebo-controlled study to evaluate the efficacy of the dietary supplement BioAmicus Complete (oral drops; a mixture of 10 strains of lacto- and bifidobacteria) in managing functional…
- Conditions
- Infantile Colics, Constipation, Infantile Diarrhea, Atopic Dermatitis
- Trial name
- NCT07444008
- Testing
- BioAmicus Complete
- Where
- Russia
- Volunteers
- 140 people
- Sponsor
- NovoNatum Ltd
Retinitis Pigmentosa
Retina Implant Pilot Trial to Evaluate Safety & Efficacy in Blind Patients Having Degenerated Photo-receptors
The outcome of this trial will reveal the possibilities of the retinal implant to improve the situation of patients with hereditary retinal blindness caused by degenerations of the outer retina.
- Trial name
- NCT00515814
- Testing
- Retina implant is surgically placed into subretinal position
- Where
- 2 sites in Germany
- Volunteers
- 15 people
- Sponsor
- Retina Implant AG
Congenital Bleeding Disorder, Haemophilia A
Impact of Pain on Functional Impairment and Quality of Life in Adults With Hemophilia
This study is conducted in the United States of America (USA). The aim of the study is to assess the impact of pain on functional impairment and quality of life in adult persons with hemophilia (PWH) with and without inhibitors…
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia A With Inhibitors, Haemophilia B
- Trial name
- NCT01988532
- Testing
- No treatment given
- Where
- United States
- Volunteers
- 381 people
- Sponsor
- Novo Nordisk A/S
Familial Hypercholesterolemia
Effect of APL180 on Endothelial Function in Familial Hypercholesterolemia Patients
This study will evaluate the effect of APL180 on endothelial function measured by forearm venous occlusion plethysmography in patients with familial hypercholesterolemia.
- Trial name
- NCT00751608
- Testing
- APL180
- Sponsor
- Novartis Pharmaceuticals
Cardiomyopathy, Hypertrophic, Cardiomyopathy, Hypertrophic, Familial
Open-Label Study of Perhexiline in Patients With Hypertrophic Cardiomyopathy and Moderate to Severe Heart Failure
The purpose of this study is to evaluate the effect of perhexiline on exercise performance (efficacy) and safety in patients with hypertrophic cardiomyopathy and moderate-to-severe heart failure following dosing for 16 weeks.
- Trial name
- NCT02862600
- Testing
- Perhexiline, Use of bioanalytical assay to monitor plasma levels of perhexiline
- Where
- 11 sites in United States
- Volunteers
- 35 people
- Sponsor
- Heart Metabolics Limited
Hallux Valgus
Validation of Patient Reported Outcome Measures for Use in Hallux Valgus
The purpose of the study is to explore whether the mFFI is fit for purpose in adults with hallux valgus (HV).
- Trial name
- NCT03846687
- Where
- 3 sites in United States
- Volunteers
- 21 people
- Sponsor
- Ipsen
Joint Disease
Post Market Clinical Follow-Up Study Protocol for PROFEMUR® Gladiator Cemented Femoral Stems
MicroPort Orthopedics (MPO) is conducting this post market clinical follow-up (PMCF) study to evaluate the safety and efficacy of its total hip arthroplasty (THA) European Union (EU) .
- Trial name
- NCT02397746
- Testing
- total hip arthroplasty implant
- Volunteers
- 75 people
- Sponsor
- MicroPort Orthopedics Inc.
Knee Osteoarthritis, Traumatic Arthritis of Knee
Post Market Clinical Follow-Up Study for EVOLUTION® BIOFOAM® Tibia and EVOLUTION® CS/CR Porous Femur
MicroPort (MPO) is conducting this PMCF study to evaluate the safety and effectiveness of its EVOLUTION® BIOFOAM® Tibia and EVOLUTION® Cruciate Sacrificing/Cruciate Retaining (CS/CR) Porous Femur components, including EVOLUTION®…
- Conditions
- Knee Osteoarthritis, Traumatic Arthritis of Knee, Avascular Necrosis, Degenerative Joint Disease of Knee
- Trial name
- NCT04301622
- Testing
- Primary Knee implants
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- MicroPort Orthopedics Inc.
Cystic Fibrosis in Children
The Effectiveness of the Jamboxx Respiratory Therapy Device: Study 2
The Effectiveness of the Jamboxx Respiratory Therapy Device in Treatment of Patients with Decreased Respiratory Function is a proposal for investigation of the application of gaming to improving respiratory health.
- Trial name
- NCT03522480
- Testing
- Jamboxx Respiratory Therapy Device, Autogenic Draining Training
- Where
- United States
- Sponsor
- My Music Machines Inc.
Healthy Volunteers, Polycystic Kidney, Autosomal Dominant
Effect of Venglustat in Patients With Renal Impairment
Primary Objective: To study the effect of mild, moderate and severe renal impairment on the pharmacokinetics (PK) of Venglustat following a single dose.
- Trial name
- NCT03687554
- Testing
- Venglustat GZ/SAR402671
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Genzyme, a Sanofi Company
Cystic Fibrosis
A Phase 3 Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Heterozygous for F508del and a Minimal Function Mutation (F/MF)
The purpose of this study evaluates the efficacy and safety of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants who were heterozygous for F508del and a minimal function mutation (F/MF participants).
- Trial name
- NCT05033080
- Testing
- VX-121/TEZ/D-IVA, ELX/TEZ/IVA, IVA
- Where
- 139 sites in United States, Australia and 10 more
- Volunteers
- 435 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
Pilot Study Testing the Effect of Ivacaftor on Lung Function in Subjects With Cystic Fibrosis and Residual CFTR Function
This study is a multiple within participant crossover study to evaluate the effect of ivacaftor on lung function in participants aged 12 years and older with cystic fibrosis (CF) who have phenotypic or molecular evidence of…
- Trial name
- NCT01685801
- Testing
- Ivacaftor
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Gastrointestinal Disorder, Functional, Regurgitation, Gastric
Transition to Infant Formula Feeding on Gastrointestinal Regurgitation (TIGER) Study
To compare the frequency of regurgitation and other digestive outcomes between infants who receive an infant formula based on whole goat milk and an infant formula based on cow milk proteins during the transition period from…
- Conditions
- Gastrointestinal Disorder, Functional, Regurgitation, Gastric, Colic, Infantile, Constipation - Functional
- Trial name
- TIGER (NCT05363553)
- Testing
- Infant formula based on whole goat milk, infant formula based on cow milk proteins
- Where
- Thailand
- Volunteers
- 146 people, healthy volunteers welcome
- Sponsor
- Dairy Goat Co-operative (N.Z.) Limited
Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM), Amyloidosis
Cardiac Magnetic Resonance and Echocardiography Study With Acoramidis
The study aims to evaluate the effect of acoramidis on cardiac functional and structural improvement in participants with ATTR-CM.
- Conditions
- Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM), Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis
- Trial name
- ASCEND-ATTR (NCT07695701)
- Testing
- Acoramidis (AG10) oral tablet
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- Eidos Therapeutics, a BridgeBio company
Leber Congenital Amaurosis (LCA), Retinitis Pigmentosa (RP)
Natural History Study in Inherited Retinal Disease Subjects Caused by Mutations in RPE65 or LRAT
To evaluate the natural history of visual function in subjects with IRD phenotypically diagnosed as Leber congenital amaurosis (LCA) or retinitis pigmentosa (RP) caused by RPE65 or LRAT gene mutations.
- Trial name
- NCT02575430
- Testing
- No treatment: retrospective chart review
- Where
- 9 sites in United States, Canada and 5 more
- Volunteers
- 59 people
- Sponsor
- QLT Inc.
Cystic Fibrosis
SD Cystic Fibrosis Study
Subject with Cystic Fibrosis have increased clearance of many drugs. Based on pre-clinical data SB656933 was found to have low clearance and high bio-availability.
- Trial name
- NCT00605761
- Testing
- SB656933
- Where
- 3 sites in United States
- Volunteers
- 9 people
- Sponsor
- GlaxoSmithKline
Rheumatoid Arthritis, Osteoarthritis
G7 BiSpherical Acetabular Shell PMCF Study
The primary objective of this study is to obtain implant survivorship and clinical outcomes data for the commercially available G7 BiSpherical Acetabular Shell.
- Conditions
- Rheumatoid Arthritis, Osteoarthritis, Noninflammatory Degenerative Joint Disease, Avascular Necrosis
- Trial name
- NCT03266874
- Where
- 5 sites in France, Germany and 2 more
- Volunteers
- 160 people
- Sponsor
- Zimmer Biomet
Hemophilia B
A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B
Participants in this study have a genetic mutation, specifically in the coagulation (blood clotting) Factor 9 gene that causes severe or moderately severe hemophilia B.
- Trial name
- BEYOND-9 (NCT06379789)
- Testing
- REGV131, LNP1265
- Where
- 52 sites in United States, Australia and 7 more
- Volunteers
- 130 people
- Sponsor
- Regeneron Pharmaceuticals
Infantile Colic, Infantile Functional Gastrointestinal Disorders
A Phase I Study to Evaluate the Oral Absorption of Nepadutant in Infants
The purpose of the study is to evaluate the gastrointestinal absorption of nepadutant after single dose as oral solution (and the effect of age on its oral absorption) in infants.
- Trial name
- NCT00655083
- Testing
- Nepadutant
- Where
- 3 sites in United States
- Volunteers
- 21 people
- Sponsor
- Menarini Group