Clinical trials: Disabilities Multiple
- Condition Disabilities Multiple
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Fragile X Syndrome
Clinical Study to Assess the Pharmacokinetics, Safety and Tolerability of Single and Multiple Oral Doses of AFQ056 in Children With Fragile X Syndrome (FXS)
The aim of this study is to characterize the pharmacokinetics and safety/tolerability of AFQ056 in children with Fragile X Syndrome(FXS)
- Trial name
- NCT01482143
- Testing
- AFQ056
- Where
- 4 sites in United States, Spain
- Volunteers
- 21 people
- Sponsor
- Novartis Pharmaceuticals
Prader-Willi Syndrome
Study to Evaluate Efficacy, Safety, and Tolerability of RGH-706 in Prader-Willi Syndrome
RGH-706 is a novel, potent, and orally active MCHR1 antagonist drug candidate discovered and being developed by Gedeon Richter Plc. for weight management.
- Trial name
- NCT05322096
- Testing
- RGH-706
- Where
- 23 sites in United States, Czechia and 3 more
- Volunteers
- 39 people
- Sponsor
- Gedeon Richter Plc.
Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS)
The primary purpose of this study is to evaluate the safety and tolerability of ION440.
- Trial name
- NCT06430385
- Testing
- ION440
- Where
- 12 sites in United States, Austria and 3 more
- Volunteers
- 48 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Hunter Syndrome
Hunter Outcome Survey (HOS)
The purpose of this study is to collect data that will increase understanding of Hunter syndrome. The data from HOS may provide guidance to healthcare professionals about disease treatment options.
- Trial name
- HOS (NCT03292887)
- Where
- United States
- Volunteers
- 1,443 people
- Sponsor
- Shire
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.
Down Syndrome, Cognitive Dysfunction
Evaluating The Efficacy And Safety Of Donepezil Hydrochloride (Aricept) In The Treatment Of The Cognitive Dysfunction Exhibited By Children With Down Syndrome, Aged 6 To 10
The purpose of this study is to determine the efficacy and safety of donepezil hydrochloride (Aricept) in the treatment of cognitive dysfunction shown by children with Down syndrome, aged 6 to 10 years.
- Trial name
- NCT00754013
- Testing
- Aricept (Donepezil hydrochloride)
- Where
- 2 sites in United States
- Volunteers
- 9 people
- Sponsor
- Eisai Inc.
Prader-Willi Syndrome
A Study of Pitolisant in Participants With Prader-Willi Syndrome
The purpose of this study is to assess safety of pitolisant in eligible participants with Prader-Willi syndrome (PWS) who completed the End of Treatment (EOT) visit in a parent study (HBS-101-CL-002 [Open Label Extension (OLE)]…
- Trial name
- NCT07219485
- Testing
- Pitolisant
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- Harmony Biosciences Management, Inc.
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.
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
Cerebral Palsy, Neurological Conditions
Safety and Efficacy Study of Oral Glycopyrrolate Liquid for the Treatment of Pathologic (Chronic Moderate to Severe) Drooling in Pediatric Patients 3 to 18 Years of Age With Cerebral Palsy or Other Neurologic Conditions
This is an open-label clinical research study of an oral glycopyrrolate liquid for the treatment of chronic moderate to severe drooling in patients with cerebral palsy or other neurological conditions.
- Conditions
- Cerebral Palsy, Neurological Conditions, Mental Retardation, Sialorrhea
- Trial name
- NCT00491894
- Testing
- Oral Glycopyrrolate Liquid
- Where
- 7 sites in United States
- Volunteers
- 137 people
- Sponsor
- Shionogi
Autism Spectrum Disorder
An Exploratory Open Label Study of EPI-743 (Vincerinone TM) in Children With Autism Spectrum Disorder
The investigators hypothesize that EPI-743 may provide clinical benefit to children with Autism Spectrum Disorder.
- Trial name
- Autism (NCT02226458)
- Testing
- EPI-743
- Sponsor
- Edison 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.
Multiple Sclerosis
A Retrospective Study to Understand the Clinical Management of Subjects With Multiple Sclerosis at a Site in Taiwan Over the Last 10 Years
This is an observational, non comparative, non-randomised, open-label, retrospective, single centre study planned to collect the data of subjects diagnosed with multiple sclerosis (MS) as per Poser or McDonald criteria between…
- Trial name
- NCT01084798
- Where
- Taiwan
- Volunteers
- 123 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Prader-Willi Syndrome
Cannabidiol Oral Solution for the Treatment of Patients With Prader-Willi Syndrome
The primary objective of this study is to assess the efficacy of cannabidiol oral solution on hyperphagia-related behavior in patients with Prader-Willi Syndrome (PWS).
- Trial name
- NCT02844933
- Testing
- Cannabidiol
- Where
- 7 sites in United States
- Volunteers
- 7 people
- Sponsor
- Radius Pharmaceuticals, Inc.
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.
Microdeletion Syndromes, Trisomy 21
Development of Non-invasive Prenatal Test for Microdeletion and Other Genetic Syndromes Based on Cell Free DNA
The purpose of this study is to collect blood from families with a child who has been diagnosed with a chromosomal disorder including microdeletions in order to further develop a non-invasive prenatal screening test based on…
- Conditions
- Microdeletion Syndromes, Trisomy 21, Trisomy 18, Trisomy 13
- Trial name
- Microdel Triad (NCT02109770)
- Where
- 2 sites in United States
- Volunteers
- 216 people
- Sponsor
- Natera, Inc.
Down Syndrome
Evaluating The Efficacy And Safety Of Donepezil Hydrochloride (HCl) (Aricept) In Treating Cognitive Dysfunction Exhibited By Children With Down Syndrome
The purpose of this study is to determine whether donepezil HCl is effective and safe in improving cognitive dysfunction exhibited by children and adolescents with Down syndrome (DS).
- Trial name
- NCT00570128
- Testing
- Donepezil HCl
- Where
- 31 sites in United States
- Volunteers
- 129 people
- Sponsor
- Eisai Inc.
Autism Spectrum Disorder, Autism
Treatment Monitoring in Autism Spectrum Disorder (ASD) in Children
The goal of this clinical study is to learn about the utility and performance of the EarliPoint(™) System: Evaluation for Autism Spectrum Disorder to monitor changes in a child's verbal ability, non-verbal learning, and social…
- Conditions
- Autism Spectrum Disorder, Autism, Developmental Delay, Autism, Early Infantile
- Trial name
- MEASURE-ASD1 (NCT05675371)
- Testing
- Assessment by Expert Clinician During Behavioral Intervention (ABA or similar), Assessment by the EarliPoint Assessment For Autism Spectrum Disorder
- Where
- 9 sites in United States
- Volunteers
- 325 people, healthy volunteers welcome
- Sponsor
- EarliTec Diagnostics, Inc
Down Syndrome, Cognitive Dysfunction
Evaluating The Efficacy And Safety Of Donepezil Hydrochloride (Aricept) In The Treatment Of The Cognitive Dysfunction Exhibited By Children With Down Syndrome, Aged 11 To 17
The purpose of this study is to determine the efficacy and safety of donepezil hydrochloride (Aricept) in the treatment of the cognitive dysfunction shown by children with Down syndrome, aged 11 to 17.
- Trial name
- NCT00754052
- Testing
- Aricept (donepezil hydrochloride)
- Where
- 2 sites in United States
- Volunteers
- 8 people
- Sponsor
- Eisai Inc.
Down Syndrome
A Study of RG1662 in Adults and Adolescents With Down Syndrome (CLEMATIS)
This multi-center, randomized, double-blind, 3-arm, parallel-group, placebo-controlled study will evaluate the efficacy and safety of RG1662 in adults and adolescents with Down syndrome.
- Trial name
- NCT02024789
- Testing
- RG1662
- Where
- 37 sites in United States, Argentina and 8 more
- Volunteers
- 173 people
- Sponsor
- Hoffmann-La Roche