Clinical trials: Multiple Disability
Prader-Willi Syndrome
A Study of Oral ARD-101 in Patients With Prader-Willi Syndrome
A Phase 2, Single-Arm, Open-Label Study to Evaluate the Safety and Efficacy of ARD-101 in Patients with Prader-Willi Syndrome
- Trial name
- NCT05153434
- Testing
- ARD-101
- Where
- 2 sites in United States
- Volunteers
- 19 people
- Sponsor
- Aardvark Therapeutics, Inc.
Multiple Sclerosis, Secondary Progressive
BG00012 and Delay of Disability Progression in Secondary Progressive Multiple Sclerosis
The primary objective of the study is to investigate whether treatment with BG00012 (dimethyl fumarate) compared with placebo slows the accumulation of disability not related to relapses in participants with secondary progressive…
- Trial name
- INSPIRE (NCT02430532)
- Testing
- dimethyl fumarate
- Where
- 18 sites in United States, Belgium and 4 more
- Volunteers
- 58 people
- Sponsor
- Biogen
Down Syndrome
Blood Flow Regulation in Individuals With Down Syndrome - Training Study
Work capacity is an important predictor of declining health or physical function, and of mortality, and is commonly measured as peak oxygen consumption.
- Trial name
- NCT04854122
- Testing
- Combined exercise intervention
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- University of Nevada, Las Vegas
Hypothyroidism, Trisomy 21
Use of Liquid Stable Levothyroxine in Trisomy 21 Pediatric Patients
Children with levothyroxine (T21) have developmental delay and other functional gastrointestinal (GI) issues that may negatively affect L-T4 tolerability and absorption.
- Trial name
- NCT04747275
- Testing
- Liquid stable levothyroxine (L-T4) Tirosint-SOL, Oral tablet levothyroxine (L-T4)
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Children's Mercy Hospital Kansas City
Multiple Sclerosis, Pathologic Processes
Upper Extremity Function in Multiple Sclerosis Patients With Advanced Disability Treated With Ocrevus
The investigators are measuring the effectiveness of Ocrevus™ in helping patients with hand or arm weakness, especially if posed by a more advanced MS patient than those included in the clinical trials.
- Conditions
- Multiple Sclerosis, Pathologic Processes, Demyelinating Diseases, Nervous System Diseases
- Trial name
- NCT03562975
- Testing
- Ocrelizumab
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- University of South Florida
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.
Down Syndrome
Promoting Health Through Parent Empowerment and the Activation of Routines (PrO-PEAR)
This study will examine the feasibility of the Promoting Health through Parent Empowerment and the Activation of Routines (Pro-PEAR) intervention using a open case-series for intervention optimization followed by a pilot RCT in…
- Trial name
- Pro-PEAR (NCT05020366)
- Testing
- Promoting Health through Parent Empowerment and the Activation of Routines, Enhanced Usual Care
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- University of Pittsburgh
Down Syndrome, Alzheimer Disease
The Promotion of Physical Activity for the Prevention of Alzheimer's Disease in Adults With Down Syndrome
The objectives of this study are to determine the feasibility and potential efficacy of remotely delivered group exercise sessions to increase daily moderate to vigorous physical activity in adults with Down syndrome, relative to…
- Trial name
- NCT04048759
- Testing
- Remote Low, Remote High, Personal Coach
- Where
- United States
- Volunteers
- 81 people
- Sponsor
- University of Kansas Medical Center
Aneuploidy
National Prevalence and Impact of Noninvasive Prenatal Testing
Information on pregnant women undergoing non-invasive prenatal testing (NIPT) at one of the Obstetrix Medical Groups Outpatient Centers between January 2012 and June 2014 will be retrospectively gathered and an analysis of the…
- Trial name
- NCT02284399
- Testing
- non-invasive prenatal testing
- Where
- 7 sites in United States
- Volunteers
- 4,488 people, healthy volunteers welcome
- Sponsor
- Obstetrix Medical Group
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.
Hyperphagia, Prader-Willi Syndrome
Pilot Study of Startle-response Test to Assess Transcranial Direct Current Stimulation-induced Modulation of Hyperphagia in Prader-Willi Syndrome
The purpose of this study is to determine the effects of transcranial direct current stimulation (tDCS) as it modifies hyperphagia in obese subjects, non-obese subjects, and subjects with Prader-Willi syndrome (PWS).
- Trial name
- NCT01863017
- Testing
- tDCS
- Where
- 3 sites in United States
- Volunteers
- 31 people, healthy volunteers welcome
- Sponsor
- University of Kansas Medical Center
Hypofibrinogenemia, Afibrinogenemia
Human Fibrinogen Concentrate in Pediatric Cardiac Surgery
The goal of the study is to determine whether the use of Human Fibrinogen Concentrate (RiaSTAP) will decrease blood loss and the need for component blood therapy in neonates and infants undergoing cardiopulmonary bypass.
- Conditions
- Hypofibrinogenemia, Afibrinogenemia, Bleeding Disorders
- Trial name
- RiaSTAP (NCT02822599)
- Testing
- RiaStAP, Saline
- Where
- 2 sites in United States
- Volunteers
- 30 people
- Sponsor
- Nicklaus Children's Hospital f/k/a Miami Children's Hospital
Down Syndrome, Mild Cognitive Impairment
Nicotine Treatment of Cognitive Decline in Down Syndrome
This study will ascertain whether nicotine is safe and tolerable in DS patients, help with dose-ranging of nicotine in DS, look for evidence of enhancements in cognitive functioning, and establish evidence for biological and…
- Trial name
- NCT01778946
- Testing
- Low Dose Nicotine (7mg), Moderate Dose Nicotine (14mg)
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Vanderbilt University Medical Center
Prader-Willi Syndrome, Obesity
Understanding the Role of Gut Microbiota in Hyperphagia in Prader-Willi Syndrome
This study aims to use a high-fiber supplementation, an intervention known to create shifts in the gut microbiota towards a healthier structure, to explore the relationship between gut microbiota, appetite control and feeding…
- Trial name
- PWSGUT (NCT05541003)
- Testing
- NBT-NM108
- Where
- United States
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Rutgers, The State University of New Jersey
Relapse Remitting Multiple Sclerosis
Study to Assess the Effect of Ofatumumab in Treatment Naïve, Very Early RRMS Patients Benchmarked Against Healthy Controls.
This study evaluates the impact of ofatumumab in Relapsing Remitting Multiple Sclerosis (RRMS) participants that are very early in the course of their disease using clinical and magnetic resonance imaging (MRI) outcomes.
- Trial name
- AGNOS (NCT05084638)
- Testing
- Ofatumumab
- Where
- 36 sites in United States, Puerto Rico
- Volunteers
- 180 people, healthy volunteers welcome
- Sponsor
- Novartis Pharmaceuticals
Down Syndrome, Physical Inactivity
GameSquad for Adults With Down Syndrome
The investigators are assessing acceptability, appropriateness, and feasibility of the adapted GameSquad-DS intervention using validated scales alongside qualitative data among young adults with Down Syndrome.
- Trial name
- NCT05473247
- Testing
- Ring-Fit Adventure Group
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- University of Kansas Medical Center
Multiple Sclerosis
Study of Teriflunomide in Reducing the Frequency of Relapses and Accumulation of Disability in Patients With Multiple Sclerosis
The primary objective was to determine the effect of teriflunomide on the frequency of relapses in patients with relapsing multiple sclerosis (MS).
- Trial name
- TEMSO (NCT00134563)
- Testing
- Teriflunomide
- Where
- 21 sites in United States, Austria and 19 more
- Volunteers
- 1,088 people
- Sponsor
- Sanofi
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.
Down Syndrome, Trisomy 21
Down Syndrome Metabolic Health Study
The purpose of this research study is to determine which measures best capture cardiovascular disease (CVD) risk and type 2 diabetes (T2DM) risk in children and adolescents with Down syndrome (DS).
- Trial name
- NCT01821300
- Where
- 2 sites in United States
- Volunteers
- 257 people, healthy volunteers welcome
- Sponsor
- Children's Hospital of Philadelphia
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