Clinical trials: Disability Evaluation
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
Crohn's Disease, Rheumatoid Arthritis
An ObserVatIonal STudy of the Effectiveness of AdaLimumab on Health and Disability Outcomes in New Zealand Patients With Immune-Mediated InflammaTorY Diseases (VITALITY)
For public health purposes disability is becoming increasingly important as an outcome measure. Despite this, there are few data on the effectiveness of adalimumab on disability outcomes in patients with immune-mediated…
- Conditions
- Crohn's Disease, Rheumatoid Arthritis, Psoriasis
- Trial name
- VITALITY (NCT02451839)
- Volunteers
- 168 people
- Sponsor
- AbbVie
Hunter Syndrome
Safety and Efficacy of Hunterase
The objective of this study is to determine the safety and efficacy of once weekly dosing of idursulfase-beta 0.5mg/kg administered by intravenous(IV) infusion for Hunter syndrome patients < 6 years old.
- Trial name
- GC1111 (NCT01645189)
- Testing
- Hunterase
- Where
- South Korea
- Volunteers
- 6 people
- Sponsor
- Green Cross Corporation
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
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.
Mucopolysaccharidosis II
To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome Patients
The purpose of this study is to evaluate the safety and efficacy of GC1111 (recombinant human iduronate-w-sulfatase) in Hunter Syndrome (Mucopolysaccharidosis II) patients
- Trial name
- NCT01301898
- Testing
- GC1111_0.5mg/kg, GC1111_1.0mg/kg, Elaprase_0.5mg/kg
- Where
- South Korea
- Volunteers
- 31 people
- Sponsor
- Green Cross Corporation
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
Fragile X Syndrome (FXS), Fragile X Syndrome
Safety, Tolerability, and Preliminary Effectiveness of CTH120 in Fragile X Syndrome
The purpose of this Phase IIa study is to evaluate the safety, tolerability, and effectiveness of CTH120 in adult males with Fragile X syndrome.
- Trial name
- FXS-CTH120-01 (NCT07654114)
- Testing
- CTH120
- Where
- 2 sites in Spain
- Volunteers
- 30 people
- Sponsor
- Connecta Therapeutics, S.L.
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.
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
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.
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
Prader-Willi Syndrome
A Study of CSTI-500 in Patients With Prader-Willi Syndrome
This is a proof-of-concept, open-label, dose-escalation study to evaluate the safety, tolerability, pharmacokinetics, and efficacy of CSTI-500 in participants with genetically confirmed Prader-Willi Syndrome (PWS) who are 13 to…
- Trial name
- NCT07348601
- Testing
- CSTI-500
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- ConSynance Therapeutics
Multiple Sclerosis
Evaluate Safety and Efficacy of Apitox Add-on Therapy for Improving Disability and Quality of Life in MS Patients
Determine the effects of Apitox add-on therapy on the progression of disability in all forms of multiple sclerosis (MS) utilizing the Expanded Disability Status Scale (EDSS) and the MS Functional Composite (MSFC) measure. b.
- Trial name
- NCT03710655
- Testing
- Apitox - pure honeybee toxin
- Volunteers
- 468 people
- Sponsor
- Apimeds, Inc.
Mucopolysaccharidosis (MPS), Hunter Syndrome
Observational Study to Evaluate Neurodevelopmental Status in Pediatric Patients With Hunter Syndrome (MPS II)
Hunter syndrome (Mucopolysaccharidosis II, [MPS II]) is a rare, genetically linked lysosomal storage disease (LSD) caused by deficiency of the enzyme, iduronate-2-sulfatase (I2S).
- Trial name
- NCT01822184
- Where
- 7 sites in United States, Argentina and 3 more
- Volunteers
- 100 people
- Sponsor
- Shire
Down Syndrome, Edwards Syndrome
Non-Invasive Screening for Fetal Aneuploidy
The purpose of this study is to detect whole chromosome abnormalities in maternal blood.
- Trial name
- NCT02317965
- Testing
- Maternal Blood Draw
- Where
- 10 sites in United States
- Volunteers
- 340 people
- Sponsor
- Progenity, Inc.
Glut1 Deficiency Syndrome, De Vivo Disease
Early Diagnosis of the GLUT1 Deficiency Syndrome With a Blood Based Test
The study aims at validating the diagnostic performances of the METAglut1, a blood in vitro diagnostic test, for the simple and early diagnosis of the Glut1 deficiency syndrome (Glut1DS, or De Vivo disease).
- Conditions
- Glut1 Deficiency Syndrome, De Vivo Disease, Seizures, Movement Disorders
- Trial name
- METAglut1 (NCT03722212)
- Testing
- METAglut1
- Where
- 28 sites in France
- Volunteers
- 636 people
- Sponsor
- METAFORA biosystems
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
Fragile X Syndrome
Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome
This Phase IIb study is designed to assess whether 3 doses of AFQ056 are safe and effective in treating the behavioral symptoms of Fragile X Syndrome.
- Trial name
- NCT01357239
- Testing
- AFQ056
- Where
- 38 sites in United States, Australia and 14 more
- Volunteers
- 139 people
- Sponsor
- Novartis Pharmaceuticals
Diffuse Cutaneous Systemic Sclerosis
Efficacy and Safety of Belumosudil in Subjects With Diffuse Cutaneous Systemic Sclerosis
This was a phase 2, open-label, single-cohort, multicenter trial of belumosudil in participants with Diffuse Cutaneous Systemic Sclerosis (dcSSc).
- Trial name
- dcSSC (NCT04680975)
- Testing
- Belumosudil
- Where
- 6 sites in United States
- Volunteers
- 10 people
- Sponsor
- Kadmon, a Sanofi Company