Clinical trials: Effect Neonatal
Prader-Willi Syndrome
Phase 2 Trial to Evaluate Safety and Efficacy of Setmelanotide (RM-493) in Obese Participants With Prader-Willi Syndrome
The purpose of this study was to evaluate the effects of a once daily subcutaneous injectable formulation of setmelanotide in obese participants with Prader-Willi syndrome on tolerability, weight loss, and hyperphagia-related…
- Trial name
- NCT02311673
- Testing
- Setmelanotide
- Where
- 5 sites in United States
- Volunteers
- 40 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Gout, Food-drug Interaction
Study to Investigate the Effects of Food on Relative Bioavailability of ABP-671 Tablets in Healthy Subjects
This is a single center, open-label, single-dose, 2-way randomized crossover design in which 12 healthy subjects will be randomized to 1 of 2 treatment sequences (AB or BA).
- Trial name
- NCT04303039
- Testing
- ABP-671
- Where
- United States
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Atom Therapeutics Co., Ltd
Osteogenesis Imperfecta
Multicenter,Single-arm Study to Evaluate Efficacy, Safety, & Pharmacokinetics of Denosumab in Children w/ OI
This is a prospective, multicenter, single-arm study in children 2 to 17 years of age with OI to evaluate efficacy and safety of denosumab.
- Trial name
- OI (NCT02352753)
- Testing
- Denosumab
- Where
- 49 sites in United States, Australia and 11 more
- Volunteers
- 153 people
- Sponsor
- Amgen
Sickle Cell Disease
Study to Evaluate the Effect of Ticagrelor Versus Placebo in Reducing Vaso-Occlusive Crises Rate in Pediatric Patients With Sickle Cell Disease.
The purpose of this study is to compare the effect of ticagrelor vs placebo for the reduction of Vaso-Occlusive crises in paediatric patients with Sickle Cell Disease
- Trial name
- HESTIA5 (NCT04293172)
- Testing
- Brilinta
- Sponsor
- AstraZeneca
Friedreich Ataxia
A Study to Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich's Ataxia
In this study, researchers will learn more about omaveloxolone, also known as BIIB141 or SKYCLARYS®. Omaveloxolone is already approved for people with Friedreich's Ataxia (FA) who are 16 years of age or older.
- Trial name
- BRAVE (NCT06953583)
- Testing
- Omaveloxolone
- Where
- 34 sites in United States, Australia and 14 more
- Volunteers
- 255 people
- Sponsor
- Biogen
Dermatitis, Atopic
A Study to Evaluate the Safety, Tolerability, Drug Levels, and Drug Effects of BMS-986326 in Participants With Atopic Dermatitis
The purpose of this study is to assess the safety, tolerability, drug levels, drug effects, and impact on disease severity of BMS-986326 in participants with moderate-to-severe atopic dermatitis (AD).
- Trial name
- NCT06248814
- Testing
- BMS-986326
- Where
- 13 sites in Czechia, France and 3 more
- Volunteers
- 64 people
- Sponsor
- Bristol-Myers Squibb
Huntington's Disease
A Study to Evaluate the Effect of SAGE-718 on Cognitive Function in Participants With Huntington's Disease (HD)
The primary purpose of this study is to evaluate the effect of SAGE-718 on cognitive performance and functioning in participants with HD.
- Trial name
- NCT05107128
- Testing
- SAGE-718
- Where
- 49 sites in United States, Australia and 2 more
- Volunteers
- 189 people
- Sponsor
- Supernus Pharmaceuticals, Inc.
Dermatitis Atopic
An Open-label, DDI Study to Investigate the Effects of Amlitelimab on the PK of Selected Cytochrome P450 Substrates
This is an open-label, single group, 2-period, Phase 1, single-sequence study. The study duration will be up to 342 days. The treatment period will be up to Week 29, where Week 29 is defined as End of Treatment (last amlitelimab…
- Trial name
- NCT06686628
- Testing
- Amlitelimab, Midazolam, Caffeine
- Where
- Germany
- Volunteers
- 23 people
- Sponsor
- Sanofi
Duchenne Muscular Dystrophy
A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy.
The purpose of this study is to understand the safety and effects of an experimental gene therapy called fordadistrogene movaparvovec. We are seeking participants from previous Pfizer interventional studies.
- Trial name
- NCT05689164
- Testing
- fordadistrogene movaparvovec
- Where
- 12 sites in United States
- Volunteers
- 7 people
- Sponsor
- Pfizer
Hypercholesterolemia, Hyperlipidemia, Familial Combined
Study to Compare Welchol and TriCor to TriCor Alone in Patients With High Cholesterol
The primary objective of this study is to compare the effect of Welchol in combination with TriCor compared to TriCor alone on low-density lipoprotein cholesterol (LDL-C) in patients with high cholesterol.
- Trial name
- NCT00754039
- Testing
- colesevelam HCl tablets and fenofibrate tablets
- Where
- 10 sites in United States
- Volunteers
- 129 people
- Sponsor
- Daiichi Sankyo
Atopic Dermatitis
Vaccine Responses in Tralokinumab-Treated Atopic Dermatitis - ECZTRA 5 (ECZema TRAlokinumab Trial No. 5)
The purpose of this trial is to test if treatment with the trial drug, tralokinumab, can affect the body's immune response to vaccines.
- Trial name
- ECZTRA 5 (NCT03562377)
- Testing
- Tralokinumab, Tdap vaccine, Meningococcal vaccine
- Where
- 51 sites in United States, Canada
- Volunteers
- 215 people
- Sponsor
- LEO Pharma
Classical Homocystinuria
The Effect of Dietary Management and Cysteine Supplementation on Growth Parameters and Biochemical Control for Pediatric Qatari Patients Affected with Classical B6 Non-responsive Homocystinuria.
Classical homocystinuria (HCU) is an autosomal recessive disorder caused by the deficiency of an enzyme cystathionine β-synthase (CβS) that affects the catabolic pathway of the amino acid methionine (Met) which leads to an…
- Trial name
- NCT06622577
- Testing
- Cysteine
- Where
- Qatar
- Volunteers
- 50 people
- Sponsor
- Hamad Medical Corporation
Atopic Dermatitis
A Study to Learn If the Study Medicine Called PF-08049820 Changes How the Body Processes the Other Study Medicines Called Oral Contraceptives, Midazolam, and Dabigatran in Healthy Adult Female Participants
The purpose of this study is to see how a medicine called PF-08049820 affects how other medicines move through the body. This information will help plan future studies.
- Trial name
- NCT07190430
- Testing
- PF-08049820, Midazolam, Dabigatran Etexilate
- Where
- United States
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Pfizer
Lipid Metabolism, Inborn Errors, Hyperlipidemias
Measure Liver Fat Content After ISIS 301012 (Mipomersen) Administration
This study will assess what, if any, effect that ISIS 301012 (mipomersen) has on liver triglyceride content in multiple groups of subjects with varying degrees of risk for hepatic steatosis.
- Conditions
- Lipid Metabolism, Inborn Errors, Hyperlipidemias, Metabolic Diseases, Hypolipoproteinemia
- Trial name
- NCT00362180
- Testing
- mipomersen
- Where
- Netherlands
- Volunteers
- 38 people, healthy volunteers welcome
- Sponsor
- Kastle Therapeutics, LLC
Skin Depressions, Skin Elasticity
A Study to Evaluate the Effect of Juvederm Volift With Lidocaine on Aging Hands in Participants Age 35 and Older
This study will evaluate the effect of Juvederm Volift with Lidocaine, an injectable gel implant, to treat aging hands of participants 35 and older.
- Conditions
- Skin Depressions, Skin Elasticity, Skin Laxity
- Trial name
- NCT04390581
- Testing
- Juvéderm® VOLIFT with Lidocaine
- Where
- France
- Volunteers
- 75 people
- Sponsor
- Allergan
Smith-Magenis Syndrome, Circadian
Evaluating the Effects of Tasimelteon vs Placebo on Sleep Disturbances in SMS
The aim of this study is to investigate tasimelteon vs. placebo on sleep disturbances of individuals with Smith-Magenis Syndrome.
- Trial name
- NCT02231008
- Testing
- tasimelteon
- Where
- 4 sites in United States
- Volunteers
- 49 people
- Sponsor
- Vanda Pharmaceuticals
Gout
Tolerization Reduces Intolerance to Pegloticase and Prolongs the Urate Lowering Effect
The purpose of this study is to evaluate the effect of a high zone tolerizing regimen of pegloticase on clinical outcome, as defined by an serum uric acid level <6 mg/dL, in patients with chronic, refractory gout.
- Trial name
- TRIPLE (NCT02598596)
- Testing
- Pegloticase, Azathioprine
- Where
- 11 sites in United States
- Volunteers
- 132 people
- Sponsor
- Ampel BioSolutions, LLC
Hemophilia A
Russian Kogenate Pediatric Study
A prospective study to evaluate the effect of rFVIII-FS in different prophylactic regimens on bleeding events frequency and development of arthropathy in Previously Treated and Minimally treated Hemophilia A pediatric population.
- Trial name
- NCT00632814
- Testing
- rFVIII-FS (Kogenate FS, BAY14-2222) 70 IU/kg, dosing once per week, rFVIII-FS (Kogenate FS, BAY14-2222), 70 IU/kg twice per week (30 IU/kg + 40 IU/kg), rFVIII-FS (Kogenate FS, BAY14-2222) 75 IU/kg, dosing three times per week (3 x 25 IU/kg)
- Where
- 4 sites in Russia
- Volunteers
- 32 people
- Sponsor
- Bayer
Huntington Disease
Within Subject Crossover Study of Cognitive Effects of Neflamapimod in Early-Stage Huntington Disease
This is a double-blind, placebo-controlled 2-period 10-week treatment within-subject crossover study of neflamapimod in early-stage Huntington disease (HD).
- Trial name
- NCT03980938
- Testing
- neflamapimod
- Where
- United Kingdom
- Volunteers
- 15 people
- Sponsor
- EIP Pharma Inc
Infantile Colic, Constipation
Randomized Placebo-controlled Study of L. Reuteri NCIMB 30351 in GI Functional Disorders and Food Allergy in Newborns
This study is a randomized, placebo-controlled, single-masked (blinded), post-marketing clinical study of a drug Lactobacillus Reuteri NCIMB 30351 drops in functional disorders of gastrointestinal tract and skin symptoms of food…
- Conditions
- Infantile Colic, Constipation, Infantile Diarrhea, Gastro Esophageal Reflux
- Trial name
- NCT04262648
- Testing
- Lactobacillus Reuteri
- Where
- 2 sites in Russia
- Volunteers
- 90 people
- Sponsor
- NovoNatum Ltd