Clinical trials: Disability, Evaluation
Secondary Progressive Multiple Sclerosis (SPMS)
A Study to Evaluate the Efficacy and Safety of Remibrutinib in Secondary Progressive Multiple Sclerosis
The purpose of this study is to provide efficacy and safety data for remibrutinib in patients with secondary progressive multiple sclerosis (SPMS)
- Trial name
- REMASTER (NCT07225504)
- Testing
- Remibrutinib (blinded), Remibrutinib (Open label)
- Where
- 296 sites in United States, Argentina and 34 more
- Volunteers
- 1,275 people
- Sponsor
- Novartis Pharmaceuticals
Fragile X Syndrome
Biomarker and DNA Collection in Subjects Participating in Protocol 22003
The subjects that meet the inclusion and exclusion criteria and consent to participate in protocol 22003 will be offered participation in 22003A which will evaluate secreted protein before and after treatment with STX209 and…
- Trial name
- NCT00892580
- Testing
- STX209
- Where
- 8 sites in United States
- Volunteers
- 32 people
- Sponsor
- Seaside Therapeutics, Inc.
Aneuploidy, Down Syndrome
Clinical Evaluation of the SEQureDx Trisomy Test in Low Risk Pregnancies
Pregnant women with low risk indicators for fetal chromosomal aneuploidy will be enrolled. Study blood will be collected in the first or second trimester at a scheduled prenatal screening visit, processed to plasma, and stored…
- Conditions
- Aneuploidy, Down Syndrome, Noninvasive Prenatal Screening
- Trial name
- NCT01597063
- Testing
- SEQureDx Trisomy Test
- Where
- 27 sites in United States
- Volunteers
- 3,333 people
- Sponsor
- Sequenom, Inc.
Fragile X Syndrome
Long-term, Safety and Tolerability Study of AFQ056 in Adolescent Patients With Fragile X Syndrome (Open-label)
The purpose of this study is to generate long-term safety, tolerability and efficacy data for AFQ056 in eligible adolescent patients with FXS who have participated in the CAFQ056B2214 study, the PK study CAFQ056B2131, or another…
- Trial name
- NCT01433354
- Testing
- AFQ056
- Where
- 28 sites in United States, Australia and 11 more
- Volunteers
- 119 people
- Sponsor
- Novartis Pharmaceuticals
Prader-Willi Syndrome
A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone Injection in Patients With Prader-Willi Syndrome
To evaluate the effectiveness of rhGH (Recombinant human growth hormone) injection for improving motor development in patients with PWS.
- Trial name
- NCT03554031
- Testing
- Recombinant Human Growth Hormone (rhGH) Injection
- Where
- 6 sites in China
- Volunteers
- 30 people
- Sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
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.
Fragile X Syndrome
An Open Label Extension Study in Subjects With Fragile X Syndrome
This study will enroll subjects who have completed Protocols 209FX301, 209FX302, or are currently participating in Protocol 2202 into a long-term study in which all subjects will receive active drug (arbaclofen).
- Trial name
- 209FX303 (NCT01555333)
- Testing
- arbaclofen
- Where
- 23 sites in United States
- Volunteers
- 357 people
- Sponsor
- Seaside Therapeutics, Inc.
Down Syndrome-Associated Alzheimer's Disease (DS-AD)
A Study to Evaluate Mivelsiran in Adult Participants With Early-Stage Down Syndrome-Associated Alzheimer's Disease (DS-AD)
The purpose of the study is to evaluate the effect of mivelsiran in adult participants with early-stage DS-AD and to characterize the safety, tolerability, and pharmacodynamics (PD) of mivelsiran.
- Trial name
- NCT07636811
- Testing
- Mivelsiran
- Where
- 10 sites in United States, Spain
- Volunteers
- 58 people
- Sponsor
- Alnylam Pharmaceuticals
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.
Healthy
A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of OP-101 After Intravenous Administration in Healthy Volunteers
A clinical study to measure the effect of OP-101 after being administered intravenously in healthy volunteers.
- Trial name
- NCT03500627
- Testing
- OP-101
- Where
- United States
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- Orpheris, Inc.
Ischemic Stroke, Chronic Stroke
Investigation of Neural Stem Cells in Ischemic Stroke
A study of stereotactic, intracerebral injection of CTX0E03 neural stem cells into patients with moderate to moderately severe disability as a result of an ischemic stroke.
- Conditions
- Ischemic Stroke, Chronic Stroke, Hemiparesis, Arm Paralysis
- Trial name
- PISCES III (NCT03629275)
- Testing
- CTX0E03 Drug Product and delivery device
- Where
- 23 sites in United States
- Volunteers
- 15 people
- Sponsor
- ReNeuron Limited
Obesity & Overweight, Mobility Disability
Proof-of-Concept Study Evaluating Total Body Weight, Physical Function & Safety of Enobosarm in Patients Treated With GLP-1 Receptor Agonist, for Weight Loss
The primary objective of this study is to assess the effect of enobosarm on total body weight
- Conditions
- Obesity & Overweight, Mobility Disability, HOMA-IR
- Trial name
- PLATEAU (NCT07446998)
- Testing
- Enobosarm, Semaglutide (Wegovy) weekly injection
- Where
- 13 sites in United States
- Volunteers
- 239 people
- Sponsor
- Veru 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
Acute Ischemic Stroke
A Study to Evaluate the Effects of YM872 on Brain Function and Disability When Administered in Combination With Alteplase (Tissue Plasminogen Activator)
The purpose of this study is to determine if YM872 in combination with t-PA can reduce disability and brain damage from stroke. YM872 or placebo will be given as a continuous intravenous (iv) infusion for 24 hours.
- Trial name
- NCT00044057
- Testing
- YM872 (zonampanel), t-PA (alteplase)
- Where
- 118 sites in United States, Austria and 3 more
- Sponsor
- Astellas Pharma Inc
Rett Syndrome
A Phase 1/2/3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study)
The primary objectives of this study are to evaluate the safety of a single intrathecal (IT) dose of TSHA-102 in females with typical Rett syndrome, to select the TSHA-102 dose with the best benefit/risk profile based on the…
- Trial name
- NCT05606614
- Testing
- TSHA-102
- Where
- 9 sites in United States, Canada
- Volunteers
- 17 people
- Sponsor
- Taysha Gene Therapies, Inc.
Rett Syndrome
Evaluation of the Efficacy, Safety, and Tolerability of Sarizotan in Rett Syndrome With Respiratory Symptoms
This study evaluates the safety, tolerability and efficacy of Sarizotan in reducing respiratory abnormalities in Rett Syndrome in an initial double blind 24 week period followed by an open label treatment phase of up to 168 weeks…
- Trial name
- STARS (NCT02790034)
- Testing
- Sarizotan low dose, Sarizotan high dose
- Where
- 14 sites in United States, Australia and 3 more
- Volunteers
- 129 people
- Sponsor
- Newron Pharmaceuticals SPA
Dyslexia
Evaluation of Speech Rhythm Training in Dyslexic Readers Aged 7 to 9 Years
Studies of dyslexia have shown altered oscillatory activity in the low gamma band (~25-35 Hz) in the left auditory cortex. Neural oscillations around 30 Hz constitute the basic sampling rate of speech, from which the ability to…
- Trial name
- RnDys (NCT05996497)
- Testing
- behavioral measures, Electrophysiological measures, Training 1
- Where
- France
- Volunteers
- 160 people
- Sponsor
- Institut Pasteur
Mucopolysaccharidosis Type II, Metabolic Diseases
A Multi-cohort 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
- NCT04539340
- Testing
- GNR-055
- Where
- Russia
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- AO GENERIUM
X-Linked Adrenoleukodystrophy (X-ALD), Cerebral Adrenoleukodystrophy (CALD)
Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD)
Study ALD-103 will be a multi-site, global, prospective and retrospective data collection study that is designed to evaluate outcomes of allo-HSCT in male subjects with CALD ≤17 years of age.
- Conditions
- X-Linked Adrenoleukodystrophy (X-ALD), Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD)
- Trial name
- NCT02204904
- Testing
- Allo-HSCT
- Where
- 13 sites in United States, Argentina and 5 more
- Volunteers
- 59 people
- Sponsor
- Genetix Biotherapeutics Inc.
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.