Clinical trials: Disability, Evaluation
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
Mucopolysaccharidosis II
Long-term Follow-Up for RGX-121
RGX-121-5101 is an observational long-term follow-up study to the RGX-121-101 or RGX-121-1102 studies where participants received RGX-121, a gene therapy intended to deliver a functional copy of the iduronate-2-sulfatase gene…
- Trial name
- NCT04597385
- Testing
- Long-term Follow-Up
- Where
- 4 sites in United States, Brazil
- Volunteers
- 27 people
- Sponsor
- REGENXBIO Inc.
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.
Fragile X Syndrome
Safety and Efficacy of AFQ056 in Adult 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
- NCT01253629
- Testing
- AFQ056
- Where
- 31 sites in United States, Australia and 8 more
- Volunteers
- 175 people
- Sponsor
- Novartis Pharmaceuticals
Fragile X Syndrome
Long-term, Safety, Tolerability and Efficacy Study of AFQ056 in Adult Patients With Fragile X Syndrome
The purpose of this study is to generate long-term safety, tolerability and efficacy data for AFQ056 in eligible adult patients with FXS who have participated in the CAFQ056A2212 (NCT01253629).study and patients who have…
- Trial name
- NCT01348087
- Testing
- AFQ056
- Where
- 28 sites in United States, Australia and 8 more
- Volunteers
- 148 people
- Sponsor
- Novartis Pharmaceuticals
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.
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.
Fragile X Syndrome
Biomarker Testing and DNA Collection in Subjects Participating in Protocol 22001
The subjects that meet the inclusion and exclusion criteria and consent to participate in protocol 22001 will be offered participation in 22001A which will evaluate secreted protein before and after treatment with STX209 and…
- Trial name
- NCT00823368
- Testing
- STX209
- Where
- 11 sites in United States
- Sponsor
- Seaside Therapeutics, Inc.
Dyslexia
Evaluation of the Performance and Safety of the Atoldys/ Lexilens Glasses Developed to Improve the Reading of Dyslexic Subjects
The aim of this study is to evaluate the efficacy and safety of a new medical device (Atoldys/ Lexilens glasses) on the improvement of reading skills of young dyslexic subjects.
- Trial name
- NCT04586621
- Where
- 3 sites in France
- Volunteers
- 41 people, healthy volunteers welcome
- Sponsor
- ABEYE
Neurodevelopmental Disorders, Autism Spectrum Disorder
A Game-based Neurodevelopmental Assessment for Young Children
The aim of this study is to evaluate a novel tablet game-based neurodevelopmental assessment tool for young children aged 3 to 8 years old.
- Conditions
- Neurodevelopmental Disorders, Autism Spectrum Disorder, Attention Deficit Hyperactivity Disorder, Dyslexia
- Trial name
- BRIGHTEN (NCT05780853)
- Testing
- Tablet Game-based Assessment - YARN assessment service, Adaptive Behavior Assessment System, Third Edition (ABAS-3), Demographic questionnaire
- Where
- United Kingdom
- Volunteers
- 590 people, healthy volunteers welcome
- Sponsor
- Brightlobe Limited
Developmental Disabilities, Feeding Disorder
Tube Feeding Tolerance After Switch to Peptide Based Formula in Children With Developmental Delay
A retrospective chart review to assess feeding tolerance in children who had been switched from an intact protein formula to a Peptide based formula due to feeding intolerance in a pediatric facility for the developmentally…
- Trial name
- NCT02912065
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Société des Produits Nestlé (SPN)
Mucopolysaccharidosis II
Mucopolysaccharidosis Type II Natural History
Mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, is caused by a deficiency of iduronate-2-sulfatase (IDS) leading to an accumulation of glycosaminoglycans (GAGs) in tissues of MPS II patients, resulting in…
- Trial name
- NCT03529786
- Where
- 3 sites in United States, Brazil and 1 more
- Volunteers
- 36 people
- Sponsor
- REGENXBIO Inc.
Cataract
Evaluation of the EpiGlare Tester for the Measurement of Glare-Induced Changes in BSCVA in Subjects With and Without Cataracts
The EpiGlare Tester will identify visual disability resulting from the glare experienced by subjects with cataracts in the glare-induced change in best spectacle corrected visual acuity (BSCVA).
- Trial name
- NCT04739397
- Testing
- Glare Testing
- Where
- United States
- Volunteers
- 89 people, healthy volunteers welcome
- Sponsor
- Epico, LLC
Fragile X Syndrome (FXS), Neurotypical Adults
Neural Biomarkers in Fragile X Syndrome
The purpose of this non-interventional test /re-test study is to assess neural biomarkers in adult subjects with Fragile X syndrome compared to those measured in a population of typically developing adults
- Trial name
- EXP-CTH120 (NCT06957054)
- Testing
- Any
- Where
- 2 sites in Spain
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Connecta Therapeutics, S.L.
Mucopolysaccharidosis Type II (MPS II)
RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)
RGX-121 is a gene therapy which is designed to deliver a functional copy of the iduronate-2-sulfatase (IDS) gene to the central nervous system.
- Trial name
- NCT04571970
- Testing
- RGX-121
- Where
- 2 sites in United States, Canada
- Volunteers
- 6 people
- Sponsor
- REGENXBIO Inc.
Adrenoleukodystrophy
A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients
This is a Phase II/III, randomized, double-blind, placebo-controlled, multicenter, two parallel-group study in male patients with the adrenomyeloneuropathy (AMN) phenotype of X-linked adrenoleukodystrophy (X-ALD) to assess the…
- Trial name
- Advance (NCT03231878)
- Testing
- Leriglitazone
- Where
- 10 sites in United States, France and 6 more
- Volunteers
- 116 people
- Sponsor
- Minoryx Therapeutics, S.L.
Fragile X Syndrome
Investigation of Sulindac (HLX-0201) and Gaboxadol (HLX-0206) in Male Fragile X Syndrome Patients Aged 13-40
This study is to investigate the safety, tolerability and efficacy of Sulindac (HLX-0201) and Gaboxadol (HLX-0206) in males with Fragile X Syndrome (FXS) with confirmed full FMR1 mutation treated over a 10 week period in an…
- Trial name
- IMPACT-FXS (NCT04823052)
- Testing
- Sulindac (HLX-0201), dose strength 1, Sulindac (HLX-0201), dose strength 2, Gaboxadol (HLX-0206)
- Where
- 10 sites in United States, Australia
- Sponsor
- Healx Limited
Healthy Volunteers, Down Syndrome
Leucettinib-21 First-in-Human Phase 1 in Healthy Volunteers and Subjects With Down Syndrome and Alzheimer's Disease
Leucettinib-21 First-in-Human Phase 1 Study in 6 Parts: Single (Part 1 and 5) and Multiple (Part 3 and 6) Ascending Doses, and Food-Effect (Part 2) in Healthy Subjects, and Single Dose (Part 4) in People with Down Syndrome (DS)…
- Conditions
- Healthy Volunteers, Down Syndrome, Alzheimer's Disease
- Trial name
- LEUCETTA (NCT06206824)
- Testing
- Leucettinib-21
- Where
- France
- Volunteers
- 152 people, healthy volunteers welcome
- Sponsor
- Perha Pharmaceuticals
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
Rett Syndrome
Rett REVOLUTION Trial: An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Rett Syndrome
The RETT REVOLUTION trial is a placebo-controlled, single-blinded, exploratory study with patients serving as their own control ("N of 1" trial design) where the safety and efficacy of vorinostat in the treatment of Rett syndrome…
- Trial name
- NCT07150013
- Testing
- Vorinostat (SAHA)
- Where
- Colombia
- Volunteers
- 15 people
- Sponsor
- Unravel Biosciences, Inc.