Clinical trials: Multiple Disability
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
Down Syndrome
Safety, Tolerability and Immunogenicity of ACI-24 Vaccine in Adults With Down Syndrome
The purpose of this study is to test in adults with Down Syndrome the safety, tolerability and immunogenicity of a vaccine, ACI-24.
- Trial name
- 3-Star (NCT02738450)
- Testing
- ACI-24 low dose, ACI-24 high dose
- Where
- 4 sites in United States
- Volunteers
- 20 people
- Sponsor
- AC Immune SA
Multiple Sclerosis, Multiple Sclerosis, Relapsing-Remitting
Impact of the Cionic Neural Sleeve on Mobility in Multiple Sclerosis
The purpose of this research is to support the clinical value of the Cionic Neural Sleeve for individuals diagnosed with multiple sclerosis (MS).
- Conditions
- Multiple Sclerosis, Multiple Sclerosis, Relapsing-Remitting, Multiple Sclerosis, Chronic Progressive
- Trial name
- NCT05964829
- Testing
- Cionic Neural Sleeve NS-100, Walking Program
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Cionic, Inc.
Down Syndrome, Obstructive Sleep Apnea
Effects of Hypoglossal Nerve Stimulation on Cognition and Language in Down Syndrome and Obstructive Sleep Apnea
This study is a prospective, single-arm study conducted under a common implant and follow-up protocol. The objective will be to follow fifty-seven (57) adolescents and young adults (10-21 years of age), with Down syndrome…
- Trial name
- NCT04801771
- Testing
- Inspire Upper Airway Stimulation (UAS) System
- Where
- 8 sites in United States
- Volunteers
- 57 people
- Sponsor
- Inspire Medical Systems, Inc.
Aneuploidy, Trisomy 21
Non-Invasive Chromosomal Evaluation of Trisomy Study
This study is being conducted to provide clinically annotated samples to support continued improvements in the Ariosa Test content, methodology, specimen processing and quality control.
- Conditions
- Aneuploidy, Trisomy 21, Trisomy 18, Trisomy 13
- Trial name
- NICHE (NCT02201862)
- Where
- 3 sites in United States
- Volunteers
- 2,000 people, healthy volunteers welcome
- Sponsor
- Cindy Cisneros
Down Syndrome (Trisomy 21)
Qualitative Effects of AEF0217 in Down Syndrome People
Following the AEF0217-102 trial (NCT05748405), which investigated the effects of AEF0217 versus placebo, participants with Down syndrome and their caregivers who took part in the trial are invited to share their perceptions of…
- Trial name
- NCT07413224
- Where
- Spain
- Volunteers
- 28 people
- Sponsor
- Aelis Farma
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.
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.
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.
Exercise
A Single-blinded, Controlled, Multi-centre Study of Effects of Exercise in Participants With Multiple Sclerosis
The primary objective of the investigation is to determine whether the addition of exercise (resistance training or modified physiotherapy) improves functional capacity in Multiple Sclerosis (MS) participants undergoing Disease…
- Trial name
- ACTIMS (NCT01065090)
- Where
- 14 sites in Australia, Denmark and 4 more
- Volunteers
- 38 people
- Sponsor
- Biogen
Prader-Willi Syndrome
Study to Assess the Efficacy and Safety of Eutropin in Prader-Willi Syndrome
Evaluate the efficacy and safety after treatment of Eutropin® inj. compared to Genotropin® in infants/toddlers with Prader-Willi syndrome
- Trial name
- NCT02204163
- Testing
- Eutropin, Genotropin
- Where
- 3 sites in South Korea
- Volunteers
- 34 people
- Sponsor
- LG Life Sciences
Multiple Sclerosis
The MS-LINK™ Outcomes Study
The Multiple Sclerosis (MS) Leadership and Innovation Network (MS-LINK™) is comprised of networks working cooperatively to advance Multiple Sclerosis (MS) science and improve MS participant outcomes.
- Trial name
- NCT04735406
- Where
- 9 sites in United States
- Volunteers
- 2,182 people
- Sponsor
- EMD Serono Research & Development Institute, 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
Prader-Willi Syndrome
A Study to Assess the Long-Term Safety of Pharmaceutical Grade Synthetic Cannabidiol Oral Solution in Participants With Prader-Willi Syndrome
The objective of this study is to assess the long-term safety and tolerability of Cannabidiol Oral Solution (CBD) in participants with Prader-Willi Syndrome.
- Trial name
- NCT03458416
- Testing
- Cannabidiol Oral Solution
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Radius Pharmaceuticals, Inc.
Fragile X Syndrome
Clinical Study Of caNNabidiol in childrEn and adolesCenTs With Fragile X (CONNECT-FX)
This trial will evaluate the efficacy and safety of ZYN002, a clear cannabidiol gel that can be applied to the skin (called transdermal application) twice a day for the treatment of behavioral symptoms of Fragile X Syndrome…
- Trial name
- CONNECT-FX (NCT03614663)
- Testing
- ZYN002 - Cannabidiol Transdermal Gel
- Where
- 21 sites in United States, Australia and 1 more
- Volunteers
- 212 people
- Sponsor
- Zynerba Pharmaceuticals, Inc.
Autism Spectrum Disorder, Autism
Dynamic Quantification of Social-Visual Engagement in Autism Spectrum Disorder (ASD)
The goal of this clinical study is to learn about the utility and performance of the EarliPoint System (™): Evaluation for Autism Spectrum Disorder to diagnose and assess autism spectrum disorder (ASD) in children ages 31-96…
- Trial name
- MEASURE-ASD2 (NCT05806216)
- Testing
- EarliPoint Diagnosis, Clinically Certain Expert Clinician Diagnosis (CC-ECD)
- Where
- 9 sites in United States
- Volunteers
- 929 people, healthy volunteers welcome
- Sponsor
- EarliTec Diagnostics, 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.
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.
Relapsing Remitting Multiple Sclerosis
A Study of Orally Administered BGC20-0134 (Structured Lipid) in Patients With Relapsing Remitting Multiple Sclerosis (RRMS)
To determine the efficacy and safety of an oral drug (BGC20-0134) in patients with relapsing remitting multiple sclerosis. Specifically, the cumulative number of new gadolinium enhancing lesions after 24 weeks of treatment with…
- Trial name
- NCT01037907
- Testing
- Pleneva TM BGC20-0134
- Where
- 35 sites in Belgium, France and 4 more
- Volunteers
- 173 people
- Sponsor
- Boston Scientific Corporation
Pregnant Individuals, Aneuploidy
The ADVANCE (Assay Development and Validation for Pre-Natal and Obstetric Conditions) Study is the Largest U.S.-Based Prospective Study Demonstrating a Circulating Fetal Cell (CFC) Based Approach to Non-invasive Fetal Risk Assessment
The goal of the ADVANCE (Assay Development and Validation for Pre-Natal and Obstetric Conditions) study is to compare the concordance of results of a novel non-invasive circulating fetal cell (CFC) assay to the results of…
- Conditions
- Pregnant Individuals, Aneuploidy, Down Syndrome (Trisomy 21), 22q11.2 Deletion Syndrome
- Trial name
- ADVANCE (NCT07643896)
- Where
- 9 sites in United States
- Volunteers
- 1,000 people
- Sponsor
- BillionToOne Inc.