Clinical trials: Disabilities Multiple
Respiratory Syncytial Virus Infection
Synagis® Liquid 50mg, 100mg for Intramuscular Injection Special Investigation in Immunocompromised Children With Synagis®
This post marketing observational study (PMOS) was conducted in Japan during the 2013-2014 and 2014-2015 Respiratory Syncytial Virus (RSV) seasons to assess the safety and effectiveness of palivizumab for the prevention of…
- Trial name
- NCT02016690
- Volunteers
- 312 people
- Sponsor
- AbbVie
Down Syndrome
Non-drug Study to Assess the Suitability of Assessment Scales in Japanese Individuals With Down Syndrome Aged 6-30
To evaluate the suitability (i.e. number of tests completed and number of participants completing the tests, variance estimate of baseline and of the change from baseline values) of neurocognitive tests and functioning scales in…
- Trial name
- NCT02431117
- Where
- 7 sites in Japan
- Volunteers
- 43 people
- Sponsor
- Chugai Pharmaceutical
Prader-Willi Syndrome, Obesity
A Study of Setmelanotide in Patients With Prader-Willi Syndrome
This is a Phase 2, open-label study designed to assess the safety and efficacy of setmelanotide in patients with obesity due to Prader-Willi Syndrome (PWS) who are 6 to 65 years of age.
- Conditions
- Prader-Willi Syndrome, Obesity, Hyperphagia
- Trial name
- NCT06772597
- Testing
- Setmelanotide
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Prader-Willi Syndrome
A Study of Pitolisant in Patients With Prader-Willi Syndrome
This is a Phase 3, randomized, double-blind, placebo-controlled, multicenter, global clinical study to assess the efficacy and safety of pitolisant in patients living with Prader-Willi syndrome.
- Trial name
- NCT06366464
- Testing
- Pitolisant tablet
- Where
- 57 sites in United States, Australia and 11 more
- Volunteers
- 134 people
- Sponsor
- Harmony Biosciences Management, Inc.
Trisomy 13, Trisomy 18
High Risk Multiple Gestation Study
The objectives of the clinical study are to demonstrate the accuracy of our proprietary algorithm method to determine the genetic health of the developing fetuses in a multiple gestation pregnancy from a maternal blood sample.
- Conditions
- Trisomy 13, Trisomy 18, Trisomy 21, Sex Chromosome Abnormalities
- Trial name
- NCT02278874
- Where
- 4 sites in United States
- Volunteers
- 99 people, healthy volunteers welcome
- Sponsor
- Natera, Inc.
Prader-Willi Syndrome
A Phase 3 Extension Study of RAD011 (Cannabidiol Oral Solution) in Patients With Prader-Willi Syndrome
This is a Phase 3 open-label extension (OLE) study in patients diagnosed with Prader-Willi Syndrome (PWS) who completed the Maintenance Period of the randomized, placebo-controlled Phase 2/3 study SCOUT-015.
- Trial name
- NCT05387798
- Testing
- RAD011
- Sponsor
- Radius Pharmaceuticals, Inc.
Obesity, Over-weight
An Efficacy, Safety, and Pharmacokinetics Study of Beloranib in Obese Subjects With Prader-Willi Syndrome
The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics for certain doses of beloranib in obese subjects with Prader-Willi Syndrome.
- Conditions
- Obesity, Over-weight, Prader-Willi Syndrome
- Trial name
- NCT01818921
- Testing
- ZGN-440 sterile diluent, 1.2 mg ZGN-440 for injectable suspension, 1.8 mg ZGN-440 for injectable suspension
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- Zafgen, Inc.
Mucopolysaccharidosis Type II, Metabolic Diseases
A Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers
It is a phase I open-label single-dose, dose-escalation cohort study to evaluate of the tolerance, safety, and pharmacokinetics of GNR-055 in healthy volunteers
- Trial name
- NCT06475404
- Testing
- verenafusp alfa 6 mg/kg, verenafusp alfa 9 mg/kg, verenafusp alfa 12 mg/kg
- Where
- Russia
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- AO GENERIUM
Down Syndrome, Edwards Syndrome
Whole Blood Specimen Collection From Pregnant Subjects
To obtain whole blood specimens from pregnant subjects to be used for research and development and clinical validation studies of prenatal assays.
- Conditions
- Down Syndrome, Edwards Syndrome, Patau Syndrome, Klinefelter Syndrome
- Trial name
- NCT02430584
- Testing
- Observational - no intervention
- Where
- United States
- Volunteers
- 5,000 people, healthy volunteers welcome
- Sponsor
- Progenity, Inc.
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.
Trisomy 21 and Other Fetal Aneuploidy
Automated Screen for Fetal Aneuploidy
The purpose of this study is to develop and evaluate a blood test and automated microfluidic test platform for the prenatal screening of fetal aneuploidy.
- Trial name
- FAST1 (NCT03635359)
- Testing
- blood test
- Where
- 6 sites in Australia, Belgium and 4 more
- Volunteers
- 2,000 people, healthy volunteers welcome
- Sponsor
- BioCeryx
Fragile X Syndrome, Fragile X Syndrome (FXS)
Clinical Study of Cannabidiol in Children, Adolescents, and Young Adults With Fragile X Syndrome
This is a randomized, double-blind, placebo-controlled, multiple-center study, to assess the efficacy and safety of cannabidiol administered as ZYN002 for the treatment of children, adolescent, and young adult patients with…
- Trial name
- RECONNECT (NCT04977986)
- Testing
- ZYN002
- Where
- 27 sites in United States, Australia and 3 more
- Volunteers
- 257 people
- Sponsor
- Harmony Biosciences Management, Inc.
Secondary Progressive Multiple Sclerosis With Inflammatory Disease Activity
Swiss Study of the Impact of Mayzent on SPMS Patients in a Long-term Non-interventional Study
This study is a national, prospective, multicenter, non-interventional (observational) study with the aim to describe the impact of Siponimod treatment in a real-world SPMS population in Switzerland who are treated with Siponimod…
- Trial name
- SWISSMASIA (NCT04895202)
- Testing
- Siponimod
- Where
- 6 sites in Switzerland
- Volunteers
- 8 people
- Sponsor
- Novartis Pharmaceuticals
Multiple Sclerosis, Relapsing-Remitting
An Observational Study Comparing Multiple Sclerosis International Quality of Life Questionnaire (MusiQoL) and Multiple Sclerosis Quality of Life-54 Instrument (MSQOL-54) in Relapsing Multiple Sclerosis (RMS) Patients on Long-term Rebif® Therapy
Significant data from placebo-controlled clinical trials have demonstrated the efficacy of Rebif in relapsing remitting multiple sclerosis (RRMS) with reduction in relapse rate, delay in disability progression, and reduction in…
- Trial name
- COMPARE (NCT01141751)
- Volunteers
- 334 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Multiple Sclerosis, Relapsing Remitting
Sexual Dysfunction, Disability and Quality of Life in Patients With Multiple Sclerosis (MS)
This is an observational, prospective, non-interventional, non-controlled study planned to be conducted in subjects with Relapsing-Remitting Multiple Sclerosis (RRMS).
- Trial name
- NCT01080053
- Where
- Argentina
- Volunteers
- 306 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Sleep Disorders
Efficacy and Safety of Circadin® in the Treatment of Sleep Disturbances in Children With Neurodevelopment Disabilities
The purpose of this study is to establish the efficacy and safety of Circadin in children with neurodevelopmental disorders and to determine the dose, this randomized, placebo-controlled study is planned to evaluate the efficacy…
- Trial name
- NCT01906866
- Testing
- Circadin 2/5/10 mg
- Where
- 28 sites in United States, Finland and 3 more
- Volunteers
- 125 people
- Sponsor
- Neurim Pharmaceuticals Ltd.
Mucopolysaccharidosis Type II, Metabolic Diseases
A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II
This is phase 2/3 study to evaluate the safety, pharmacokinetics, pharmacodynamics, and efficacy of the investigational product GNR-055 in MPS II (Hunter syndrome) patients of different age groups.
- Trial name
- NCT05208281
- Testing
- GNR-055 1.0-2.0-3.0 mg/kg, GNR-055 2.0 mg/kg, GNR-055 3.0 mg/kg
- Where
- 5 sites in Russia
- Volunteers
- 32 people
- Sponsor
- AO GENERIUM
Down Syndrome, Amyloid Beta-protein
Phase II Study of Florbetaben (BAY94-9172) PET Imaging for Detection/Exclusion of Cerebral β-amyloid.
To determine the sensitivity of the visual assessment of BAY94-9172 PET images in detecting cerebral β-amyloid in individuals with Down Syndrome (DS) and specificity in individuals without DS.
- Trial name
- NCT00928304
- Testing
- Florbetaben (BAY94-9172)
- Where
- 2 sites in United States
- Volunteers
- 109 people, healthy volunteers welcome
- Sponsor
- Lantheus Biosciences Ltd.
Prader-Willi Syndrome
Open-Label Extension Study of DCCR in Patients With Prader-Willi Syndrome
The purpose of this is study is to evaluate the long-term safety of DCCR (diazoxide choline) extended-release tablets) in patients with Prader-Willi syndrome.
- Trial name
- NCT05701774
- Testing
- DCCR
- Where
- 22 sites in United States, United Kingdom
- Volunteers
- 77 people
- Sponsor
- Soleno Therapeutics, Inc.
Down Syndrome
Evaluating The Safety Of Donepezil Hydrochloride (Aricept) For Up To 1 Year In The Treatment Of The Cognitive Dysfunction Exhibited By Children With Down Syndrome - Follow-Up To A 10-Week, Double-Blind, Placebo-Controlled Trial
The purpose of this study is to determine the safety of donepezil hydrochloride (Aricept) in children with Down syndrome who have finished the preceding 10-week, double-blind study of donepezil hydrochloride.
- Trial name
- NCT00675025
- Testing
- Donepezil hydrochloride (Aricept)
- Where
- 31 sites in United States
- Volunteers
- 117 people
- Sponsor
- Eisai Inc.