Clinical trials: Disability
Insulin Resistance, Obesity
Pilot Study of GLY-LOW Supplementation in Postmenopausal Women With Obesity
A combination of generally regarded as safe (GRAS) compounds named GLY-LOW, which included: alpha lipoic acid, pyridoxamine, nicotinamide, piperine and thiamine, were examined in pre-clinical experiments.
- Conditions
- Insulin Resistance, Obesity, Cardiovascular Diseases, Cognitive Impairment
- Trial name
- NCT06242535
- Testing
- GLY-LOW
- Where
- United States
- Volunteers
- 13 people, healthy volunteers welcome
- Sponsor
- Hoskinson Health and Wellness Clinic
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
Upper Extremity Impairments, Cerebral Palsy (CP)
Obi Medical Robot: Evaluating Effectiveness Related to Usability
This mixed-methods usability study evaluates the effectiveness of the Obi Gen 3 robotic feeding device in meeting user needs among providers, caregivers, and patients.
- Conditions
- Upper Extremity Impairments, Cerebral Palsy (CP), ALS - Amyotrophic Lateral Sclerosis, Rett Syndrome
- Trial name
- NCT07151950
- Testing
- One-week trial of Obi Medical device, which provides robotic assistance with feeding to persons with upper extremity impairments who are unable to self-feed in the traditional manner using utensils
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Desin LLC
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.
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
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
Cerebral Adrenoleukodystrophy (CALD)
A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)
This trial assessed the efficacy and safety of autologous cluster of differentiation 34 (CD34+) hematopoietic stem cells, transduced ex-vivo with Lenti-D lentiviral vector (also called elivaldogene autotemcel or eli-cel), for the…
- Trial name
- NCT01896102
- Testing
- Lenti-D Drug Product (eli-cel)
- Where
- 8 sites in United States, Argentina and 4 more
- Volunteers
- 32 people
- Sponsor
- Genetix Biotherapeutics Inc.
Sarcopenia, Gait Disorders in Old Age
A Double-blind, Placebo-controlled, Randomized INTerventional Clinical Trial (SARA-INT)
The main objective of the study is to evaluate the effect of two daily doses of BIO101 versus placebo on mobility function as measured by gait speed using the 400MW test.
- Conditions
- Sarcopenia, Gait Disorders in Old Age, Muscle Weakness
- Trial name
- SARA-INT (NCT03452488)
- Testing
- BIO101
- Where
- 22 sites in United States, Belgium
- Volunteers
- 233 people
- Sponsor
- Biophytis
Prader-Willi Syndrome
Study to Evaluate Efficacy, Safety, and Tolerability of RGH-706 in Prader-Willi Syndrome
RGH-706 is a novel, potent, and orally active MCHR1 antagonist drug candidate discovered and being developed by Gedeon Richter Plc. for weight management.
- Trial name
- NCT05322096
- Testing
- RGH-706
- Where
- 23 sites in United States, Czechia and 3 more
- Volunteers
- 39 people
- Sponsor
- Gedeon Richter Plc.
Severe Knee Pain, Severe Knee Disability
Persona Revision Knee System EMEA PMCF Study
The purpose of this post-market clinical follow-up study is to confirm the long-term safety, performance and clinical benefits of the Persona Revision Knee System Implants and Instrumentation in revision Total Knee Arthroplasty.
- Conditions
- Severe Knee Pain, Severe Knee Disability, Revision Arthroplasty
- Trial name
- NCT07843966
- Where
- 2 sites in Denmark, Spain
- Volunteers
- 238 people
- Sponsor
- Zimmer, GmbH
Fragile X Syndrome, Autism
Effects of CX516 on Functioning in Fragile X Syndrome and Autism
This study will investigate whether CX516 can improve attention, memory, language, or behavior in adults with Fragile X Syndrome and/or Autism. CX516 is an AMPAKINE® compound. AMPAKINE compounds enhance synaptic strength.
- Trial name
- NCT00054730
- Testing
- CX516 (Ampalex®)
- Where
- 2 sites in United States
- Sponsor
- RespireRx
Hunter Syndrome
Intensive Pharmacovigilance Program for Elaprase (SHP ELA-701)
The purpose of this non-interventional, observational study which is conducted in Mexico is to evaluate the safety profile of elaprase (idulsurfase) in participants with hunter syndrome (mucopolysaccharydosis II) being treated…
- Trial name
- NCT03582449
- Where
- 6 sites in Mexico
- Volunteers
- 5 people
- Sponsor
- Shire
Developmental Delay, Skeletal Abnormalities
Biomarker for Sly Disease (MPS VII) (BioSly)
Development of a new MS-based biomarker for the early and sensitive diagnosis of Sly disease from blood (plasma)
- Conditions
- Developmental Delay, Skeletal Abnormalities, Hepatomegaly, Splenomegaly
- Trial name
- BioSly (NCT02298699)
- Where
- 5 sites in Egypt, Germany and 2 more
- Sponsor
- CENTOGENE GmbH Rostock
Mucopolysaccharidosis II
Mucopolysaccharidosis Type II Observational
This is an observational study planned to document prospectively disease manifestation and neurocognitive course in pediatric patients with a clinical presentation consistent with neuronopathic ("severe") MPS II undergoing…
- Trial name
- NCT04591834
- Testing
- Observational
- Where
- 3 sites in United States, Canada
- Sponsor
- REGENXBIO Inc.
Amyloid Plaque, Beta-Amyloid
A Study to Assess the Effects of ACI-24.060 in Alzheimer's Disease and in Down Syndrome (ABATE Study)
The purpose of this study is to assess the safety, tolerability, immunogenicity and pharmacodynamic effects of ACI-24.060 in subjects with prodromal Alzheimer's disease and in non-demented adults with Down syndrome.
- Conditions
- Amyloid Plaque, Beta-Amyloid, DSAD, Prodromal Alzheimer's Disease
- Trial name
- NCT05462106
- Testing
- ACI-24.060 at Dose A in Study Part 1a, ACI-24.060 at Dose B in Study Part 1a, ACI-24.060 at Dose C in Study Part 1a
- Where
- 26 sites in United States, Spain and 1 more
- Volunteers
- 304 people
- Sponsor
- AC Immune SA
Anemia, Kidney Diseases
A Study to Assess Disability in Anemic Elderly Patients With Kidney Disease Receiving PROCRIT (Epoetin Alfa)
The purpose of this study is to assess disability in anemic patients over the age of 65 who have kidney disease and are receiving weekly PROCRIT® (Epoetin Alfa, a glycoprotein that stimulates red blood cell production).
- Trial name
- NCT00338468
- Testing
- epoetin alfa
- Volunteers
- 13 people
- Sponsor
- Ortho Biotech Products, L.P.
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.
Hunter Syndrome
Hunter Outcome Survey (HOS)
The purpose of this study is to collect data that will increase understanding of Hunter syndrome. The data from HOS may provide guidance to healthcare professionals about disease treatment options.
- Trial name
- HOS (NCT03292887)
- Where
- United States
- Volunteers
- 1,443 people
- Sponsor
- Shire
Intellectual Disability, Developmental Delay
Establishing the Clinical Utility of First StepDx PLUS and NextStepDx PLUS Study
The study uses a randomized controlled study design of pediatric neurologists and developmental pediatricians and front-line (primary care) pediatricians to determine if use of FirstStepDx PLUS and Next StepDx PLUS are associated…
- Trial name
- NCT02414438
- Testing
- FirstStepDX PLUS and NextStepDX PLUS Webinar, FirstStepDX PLUS and NextStepDX PLUS Test Results
- Where
- United States
- Volunteers
- 217 people, healthy volunteers welcome
- Sponsor
- Qure Healthcare, LLC
Fragile X Syndrome, Fragile X Syndrome (FXS)
Clinical Study of Cannabidiol in Children, Adolescents, and Young Adults With Fragile X Syndrome
This is a randomized, double-blind, placebo-controlled, multiple-center study, to assess the efficacy and safety of cannabidiol administered as ZYN002 for the treatment of children, adolescent, and young adult patients with…
- Trial name
- RECONNECT (NCT04977986)
- Testing
- ZYN002
- Where
- 27 sites in United States, Australia and 3 more
- Volunteers
- 257 people
- Sponsor
- Harmony Biosciences Management, Inc.