Clinical trials: Disability, open to volunteers
Degenerative Lumbar Spinal Stenosis
Efficacy and Tolerance Study of B-DYN Versus Conventional Fusion in the Treatment of Lumbar Stenosis on the Degree of Post-operative Functional Incapacity (BDYNCLIN)
Degenerative Lumbar Spinal Stenosis (LSS) is an extremely common condition affecting more than 102 million people each year. It is a narrowing of the spinal canal in the lower back.
- Trial name
- BDYNCLIN (NCT04407338)
- Testing
- B-DYN, Conventional Bolted fusion (with or without cage)
- Where
- 2 sites in France
- Volunteers
- 216 people
- Sponsor
- Quanta Medical
RETT Syndrome With Proven MECP2 Mutation, Rett Syndrome
Efficacy and Safety of NTI164 in Children and Young Adults With Rett Syndrome
The FENRTT2 study will investigate the efficacy and safety of a medicinal cannabis plant extract with extremely low THC (delta-9-tetrahydrocannabinol), NTI164, on Rett syndrome (RTT) in a crossover design.
- Trial name
- TRANSCEND (NCT07257978)
- Testing
- NTI164
- Where
- Australia
- Volunteers
- 40 people
- Sponsor
- Fenix Innovation Group
Multiple Sclerosis
MS-Related Disability Worsening and Thalamic Volume Loss
PENTAGON is an observational, non-interventional, multicenter, prospective cohort study conducted at approximately 20 neurology centers and affiliated radiology centers across Germany.
- Trial name
- PENTAGON (NCT07753928)
- Testing
- Disease-Modifying Therapies (DMTs)
- Where
- 2 sites in Germany
- Volunteers
- 2,400 people
- Sponsor
- Jung Diagnostics GmbH
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
Dyslexia, Reading Disability
Brain Stimulation Paired With Structured Reading Instruction in Children With Dyslexia
This study investigates whether transcranial electrical stimulation (tES) delivered during structured reading instruction affects reading proficiency in children with developmental dyslexia and related reading disabilities.
- Trial name
- NCT07762365
- Testing
- Active transcranial electrical stimulation (tES)
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- General Neuro Inc.
Hearing Disability, Depression
Reduction of Depression Through Dual Treatment: Digital CBT & NBHWC Coaching
The purpose of this study is to see if a mobile-delivered mental health program called Toivoa-002, which combines digital cognitive behavioral therapy (CBT) with personal coaching, can effectively reduce depression in adults with…
- Conditions
- Hearing Disability, Depression, Mobility Disability
- Trial name
- NCT07195292
- Testing
- Toivoa-002 - A digital Cognitive Behavioral Therapy program combined with NBHWC-certified coaching.
- Where
- 4 sites in United States
- Volunteers
- 200 people
- Sponsor
- Toivoa Inc
Low Back Pain, Chronic Low Back Pain (CLBP)
Spinery Basivertebral Nerve Ablation for Vertebrogenic Chronic Low Back Pain
The goal of this clinical trial is to learn whether the Spinery® RF Generator and its sterile single-use accessories can safely lower disability and pain in adults with long-term low back pain that is thought to come from changes…
- Trial name
- BASILEA (NCT07696507)
- Testing
- Single Bipolar Ablation, Double Parallel Bipolar Ablation, Double Cross Bipolar Ablation
- Where
- 3 sites in Italy
- Volunteers
- 69 people
- Sponsor
- Axon srl
Danon Disease
A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
This is a single arm Phase 2 trial to evaluate the efficacy and safety of RP-A501, a recombinant adeno-associated virus serotype 9 (rAAV9) vector containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B)…
- Trial name
- NCT06092034
- Testing
- RP-A501
- Where
- 6 sites in United States, Germany and 2 more
- Volunteers
- 18 people
- Sponsor
- Rocket Pharmaceuticals 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
Mucopolysaccharidosis II
An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007
This is a multiregional open-label extension (OLE) to assess the safety, tolerability, and efficacy of long-term treatment with tividenofusp alfa (DNL310), an investigational central nervous system (CNS)-penetrant intravenous…
- Trial name
- NCT06075537
- Testing
- tividenofusp alfa
- Where
- 30 sites in United States, Argentina and 12 more
- Volunteers
- 99 people
- Sponsor
- Denali Therapeutics Inc.
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
Prader-Willi Syndrome
A 2-Part Study to Assess Efficacy, Safety and Tolerability of BMB-101 for the Treatment of Patients With Prader-Willi Syndrome.
The goal of this clinical trial is to evaluate the safety and effects of a new drug called BMB-101 in people with Prader-Willi Syndrome (PWS).
- Trial name
- NCT07266324
- Testing
- BMB-101
- Where
- 2 sites in Australia
- Volunteers
- 16 people
- Sponsor
- Bright Minds Biosciences Pty Ltd
Fragile X Syndrome (FXS), Autism Spectrum Disorder (ASD)
A Study to Investigate the Effects and Safety of Codabakalner for the Treatment of FXS in Male Participants and Adult Participants With ADS
This is a Study to Investigate the Effects and Safety of Codabakalner for the Treatment of Fragile X Syndrome in Male Participants and Adult Participants with Autism Spectrum Disorders
- Trial name
- NCT07439510
- Testing
- Codabakalner
- Volunteers
- 120 people
- Sponsor
- Spinogenix
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
Prader-Willi Syndrome
A Study of Pitolisant in Patients With Prader-Willi Syndrome
This is a Phase 3, randomized, double-blind, placebo-controlled, multicenter, global clinical study to assess the efficacy and safety of pitolisant in patients living with Prader-Willi syndrome.
- Trial name
- NCT06366464
- Testing
- Pitolisant tablet
- Where
- 57 sites in United States, Australia and 11 more
- Volunteers
- 134 people
- Sponsor
- Harmony Biosciences Management, Inc.
Prader-Willi Syndrome
A Study of Pitolisant in Participants With Prader-Willi Syndrome
The purpose of this study is to assess safety of pitolisant in eligible participants with Prader-Willi syndrome (PWS) who completed the End of Treatment (EOT) visit in a parent study (HBS-101-CL-002 [Open Label Extension (OLE)]…
- Trial name
- NCT07219485
- Testing
- Pitolisant
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- Harmony Biosciences Management, Inc.
Aging, Mortality
Longevity Metrics AI/ML Development Study
This study builds AI models that score diagnostic screening tests, and that predict screening results, clinical judgment, and life expectancy.
- Conditions
- Aging, Mortality, Cardiovascular Disease Prevention, Metabolic Syndrome
- Trial name
- NCT07808619
- Testing
- AIML scored health screening battery
- Where
- United States
- Volunteers
- 1,000,000 people, healthy volunteers welcome
- Sponsor
- Longevity Metrics, Inc.
Primary Progressive Multiple Sclerosis, Primary Progressive MS
A Phase 2a Study of SUN-627 in Participants With Non-Active Secondary Progressive and Primary Progressive Multiple Sclerosis (SPMS/PPMS)
The goal of this Phase 2 clinical trial is to investigate the safety and tolerability of SUN-627 in men and women aged 18-70 years old with a diagnosis of non-active SPMS/PPMS.
- Conditions
- Primary Progressive Multiple Sclerosis, Primary Progressive MS, Primary Progressive Multiple Sclerosis (PPMS), Secondary Progressive MS (SPMS)
- Trial name
- NCT07840326
- Testing
- SUN-627
- Where
- 7 sites in United States
- Volunteers
- 20 people
- Sponsor
- Sundance Biosciences, Inc.
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.
Aging, Mortality
Human Observatory Study
The Human Observatory Study is a prospective observational and ecological surveillance study building a continuously-updating world model for human health, disease, and death at the individual and population level.
- Conditions
- Aging, Mortality, All-cause Mortality, Life Expectancy
- Trial name
- HOS (NCT07646782)
- Testing
- Multi-Domain Ecological and Clinical Data Linkage
- Where
- United States
- Volunteers
- 1,000,000 people, healthy volunteers welcome
- Sponsor
- Longevity Metrics, Inc.