Clinical trials: Abnormalities, Multiple
Prader-Willi Syndrome, Hyperphagia
Oxytocin vs. Placebo for the Treatment Hyperphagia in Children and Adolescents With Prader-Willi Syndrome
The investigators propose a randomized double-blind 8 week treatment trial of intranasal oxytocin (IN-OXT) vs. placebo in 24 subjects aged 5 to 18 years with PWS in order to assess IN-OXT's affect on (1) Eating behaviors (2)…
- Trial name
- OXT-PWS (NCT02629991)
- Testing
- Intranasal Oxytocin (IN-OXT)
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- Montefiore Medical Center
Angelman Syndrome
Use of Eye Tracking to Study Social Perception Abnormalities in Children With Angelman Syndrome
Angelman syndrome (AS) is a rare neurogenetic disorder that affects approximately 1 in 15,000 children - approximately 500,000 people worldwide.
- Trial name
- EYEANGEL (NCT06737718)
- Testing
- Eye tracking, Data collection from patients' medical files, questionnaires
- Where
- France
- Volunteers
- 60 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Unverricht-Lundborg Disease
Brivaracetam as add-on Treatment of Unverricht-Lundborg Disease in Adolescents and Adults
The study will compare the efficacy and safety of brivaracetam with placebo in patients with Unverricht-Lundborg disease.
- Trial name
- NCT00357669
- Testing
- Brivaracetam 25 mg, Brivaracetam 50 mg
- Where
- 13 sites in Finland, France and 5 more
- Volunteers
- 50 people
- Sponsor
- UCB Pharma SA
Atopic Dermatitis
Evaluation of NM26-2198 in Healthy Subjects and in Patients With Moderate-to-severe Atopic Dermatitis (AD)
This is a randomized, double-blind, placebo-controlled, single- and multiple ascending dose study of subcutaneous (SC) administration of NM26-2198 in healthy volunteers and adult patients with moderate to-severe AD to evaluate…
- Trial name
- NCT05859724
- Testing
- NM26-2198
- Where
- 15 sites in United States, Canada and 2 more
- Volunteers
- 126 people, healthy volunteers welcome
- Sponsor
- Yellow Jersey Therapeutics AG
Fabry Disease, Chronic Kidney Disease, Stage IV (Severe)
Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme
This study was designed to determine appropriate treatment with Fabrazyme at a biweekly dose of either 1 mg/kg or 3 mg/kg in a population of patients with severe renal disease burden.
- Trial name
- NCT00837824
- Testing
- Fabrazyme (agalsidase beta)
- Where
- 9 sites in United States
- Volunteers
- 20 people
- Sponsor
- Genzyme, a Sanofi Company
Dyslipidemias, Familial Hypercholesterolemia
Study of ARO-ANG3 in Healthy Volunteers and in Dyslipidemic Patients
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetcs and pharmacodynamics of single- and multiple doses of ARO-ANG3 in healthy adult volunteers and in dyslipidemic patients including familial…
- Conditions
- Dyslipidemias, Familial Hypercholesterolemia, Hypertriglyceridemia
- Trial name
- NCT03747224
- Testing
- ARO-ANG3, sterile normal saline (0.9% NaCl)
- Where
- 6 sites in Australia, New Zealand
- Volunteers
- 93 people, healthy volunteers welcome
- Sponsor
- Arrowhead Pharmaceuticals
TSC, Tuberous Sclerosis Complex
Long-term, Prospective Study Evaluating Clinical and Molecular Biomarkers of Epileptogenesis in a Genetic Model of Epilepsy - Tuberous Sclerosis Complex
The primary objective of clinical part of EPISTOP project is to identify the clinical and molecular biomarkers of epileptogenesis in a prospective clinical study of patients with TSC.
- Conditions
- TSC, Tuberous Sclerosis Complex, Epilepsy
- Trial name
- EPISTOP (NCT02098759)
- Testing
- epilepsy early diagnosis protocol
- Where
- 9 sites in Austria, Belgium and 6 more
- Volunteers
- 100 people
- Sponsor
- Sergiusz Jozwiak
Polycystic Liver Disease, Hepatomegaly
Open-Label Extension of LOCKCYST Trial
Positive data originating from two polycystic liver patients treated with somatostatin analogues, showed a volume reduction of 38.3% and 14.9%.
- Conditions
- Polycystic Liver Disease, Hepatomegaly, Liver Diseases, Polycystic Kidney
- Trial name
- LOCKCYST-ext (NCT00771888)
- Testing
- lanreotide
- Where
- 2 sites in Belgium, Netherlands
- Volunteers
- 54 people
- Sponsor
- Radboud University Medical Center
Prader-Willi Syndrome
Study of Tesomet With Open-label Extension in Subjects With Prader-Willi Syndrome
This study will evaluate the safety and efficacy of Tesomet (tesofensine + metoprolol) in subjects with PWS.
- Trial name
- PWS (NCT05198362)
- Testing
- Tesomet
- Where
- United States
- Sponsor
- Saniona
Bardet-Biedl Syndrome
The Effect of GLP1 Agonists on Weight Loss in BBS Cohort in the UK
The goal of this observational study is to learn about the long-term effects of GLP1 agonists (weight loss jab, including Mounjaro) in patients with Bardet-Biedl Syndrome (BBS) who take GLP1 agonists to treat high body weight.
- Trial name
- NCT07602803
- Where
- United Kingdom
- Volunteers
- 300 people
- Sponsor
- The Queen Elizabeth Hospital
Smith Magenis Syndrome
Melatonin in Adults With SMS
Sleep-Wake and behavioral disorders in Smith Magenis Syndrome (SMS) are strongly linked to an inversion of the nychtemeral secretion of melatonin. This inversion have been described in children with SMS.
- Trial name
- SMS-adults (NCT03492970)
- Testing
- Hourly dosing of the nychtemeral secretion of melatonin
- Where
- France
- Volunteers
- 10 people
- Sponsor
- Hôpital le Vinatier
Multiple Osteochondroma, Osteogenesis Imperfecta
Resilience and Coping in a Rare Skeletal Disease Population to Face Coronavirus (COVID-19) Outbreak Distress: a Longitudinal Study
In the COVID-19 outbreak context, people living with rare diseases have been highly troubled with anxiety, loneliness, and depression.
- Conditions
- Multiple Osteochondroma, Osteogenesis Imperfecta, Ollier Disease, Maffucci Syndrome
- Trial name
- RELOAD (NCT04844697)
- Where
- Italy
- Volunteers
- 15 people
- Sponsor
- Istituto Ortopedico Rizzoli
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Diet as a Potential Treatment for Autosomal Dominant Polycystic Kidney Disease
The purpose of this study is to learn if dietary habits can affect vasopressin secretion in patients with autosomal dominant polycystic kidney disease.
- Trial name
- NCT02225860
- Testing
- Diet and water adjustment
- Where
- United States
- Volunteers
- 34 people
- Sponsor
- Tufts Medical Center
Myotonic Dystrophy Type 1 (DM1)
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1
The primary purpose of the study is to evaluate the safety and tolerability of multiple intravenous (IV) doses of DYNE-101 administered to participants with Myotonic Dystrophy Type 1 (DM1).
- Trial name
- ACHIEVE (NCT05481879)
- Testing
- DYNE-101
- Where
- 20 sites in United States, Australia and 6 more
- Volunteers
- 127 people
- Sponsor
- Dyne Therapeutics
Nystagmus, Congenital, Nystagmus, Acquired
Open-lable Extension Study on Safety and Efficacy of Neramexane to Treat Congenital and Acquired Nystagmus
The purpose of this study is to investigate the long-term safety, tolerability and efficacy of neramexane mesylate in the treatment of congenital idiopathic nystagmus (CIN).
- Conditions
- Nystagmus, Congenital, Nystagmus, Acquired, Multiple Sclerosis
- Trial name
- NCT00799942
- Testing
- Neramexane mesylate
- Where
- United Kingdom
- Volunteers
- 48 people
- Sponsor
- Merz Pharmaceuticals GmbH
Angelman Syndrome
Angelman Syndrome Natural History Study
The goal of this study is to conduct a prospective, longitudinal natural history study of children and adults with Angelman Syndrome using investigator-observed and parent-reported outcome measures to obtain data that will be…
- Trial name
- NCT04507997
- Where
- 11 sites in United States, Canada
- Volunteers
- 300 people
- Sponsor
- Boston Children's Hospital
Cystic Fibrosis
Effects of a Partially Supervised Conditioning Program in CF
Physical activity and exercise have become an accepted and valued component of Cystic Fibrosis care. Regular physical activity and exercise can slow the rate of decline of pulmonary function, improve physical fitness, and enhance…
- Trial name
- ACTIVATE-CF (NCT01744561)
- Testing
- Exercise Intervention
- Where
- 28 sites in United States, Austria and 6 more
- Volunteers
- 155 people
- Sponsor
- Wuerzburg University Hospital
Autosomal Dominant Polycystic Kidney
Renal Oxygen Consumption, Insulin Sensitivity, and Daily Caloric Restriction in ADPKD
The proposed research is a pilot study assessing kidney oxidative metabolism and insulin sensitivity after a 2-year weight loss intervention in those with autosomal dominant polycystic kidney disease who are overweight or obese.
- Trial name
- EXPLORE (NCT06496542)
- Testing
- Daily caloric restriction, Standard advice control
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- University of Colorado, Denver
Neonatal Disorder
Design of a Non-invasive Multi-modal Neonatal Monitoring System
This is a preliminary study whose objectives are to define the clinical use cases and the constraints of the implementation of a multi-sensor image-sound system.
- Trial name
- VARI-NEONAT (NCT03270410)
- Testing
- Data collection
- Where
- France
- Volunteers
- 35 people
- Sponsor
- Rennes University Hospital
Congenital Adrenal Hyperplasia
Androgen Reduction in Congenital Adrenal Hyperplasia, Phase 1
Children with congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency tend to have elevated circulating levels of androgens, which can accelerate skeletal maturation and adversely impact adult height.
- Trial name
- NCT02574910
- Testing
- Abiraterone acetate
- Where
- 4 sites in United States
- Volunteers
- 4 people
- Sponsor
- University of Texas Southwestern Medical Center
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