Clinical trials: Neuron Disease, Motor
Amyotrophic Lateral Sclerosis
Effects of ODM-109 on Respiratory Function in Patients With Amyotrophic Lateral Sclerosis
In the double-blind, cross-over part of the study, ODM-109 capsules and placebo capsules for ODM-109 will be administered for 2 weeks separated by a 19-23 days wash-out period.
- Trial name
- ALS (NCT02487407)
- Testing
- ODM-109
- Where
- 11 sites in Germany, Ireland and 2 more
- Volunteers
- 66 people
- Sponsor
- Orion Corporation, Orion Pharma
Spinal Muscular Atrophy
An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA)
This objectives of this study are to evaluate the safety, tolerability, and pharmacokinetics of a single dose of nusinersen (ISIS 396443) administered intrathecally to participants with Spinal Muscular Atrophy (SMA).
- Trial name
- SMNRx (NCT01494701)
- Testing
- nusinersen
- Where
- 4 sites in United States
- Volunteers
- 28 people
- Sponsor
- Biogen
Amyotrophic Lateral Sclerosis
A Study to Investigate the Safety and Pharmacodynamics of a Single Intrathecal Injection (IT) of INS1202 in Participants With Amyotrophic Lateral Sclerosis (ALS)
The primary objective of this dose-finding study is to evaluate the safety, tolerability and pharmacodynamics of single dose of INS1202 via IT administration in participants ≥ 18 to <80 years of age with ALS who carry superoxide…
- Trial name
- ARMOR (NCT07290062)
- Testing
- INS1202
- Where
- 7 sites in United States
- Volunteers
- 23 people
- Sponsor
- Insmed Gene Therapy LLC
Muscular Atrophy, Spinal
A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When Given Before Symptoms Appear in Babies With Genetically Diagnosed Spinal Muscular Atrophy (SMA)
In this study, researchers will learn more about the effects and safety of BIIB115, also known as salanersen. Specifically, researchers will learn more about how salanersen works in babies who have been diagnosed with SMA through…
- Trial name
- STELLAR-1 (NCT07221669)
- Testing
- Salanersen
- Where
- 14 sites in United States, Australia and 5 more
- Volunteers
- 30 people
- Sponsor
- Biogen
Amyotrophic Lateral Sclerosis
A Study of VRG50635 in Participants With Amyotrophic Lateral Sclerosis (ALS)
The primary purpose of this study is to evaluate the safety and tolerability of VRG50635 in participants with ALS.
- Trial name
- NCT06215755
- Testing
- VRG50635
- Where
- 7 sites in Belgium, Canada and 2 more
- Volunteers
- 54 people
- Sponsor
- Verge Genomics
Amyotrophic Lateral Sclerosis
Registry Study of Assistive Devices, Medicines and Healthcare Measures in ALS, SMA and Other Neurological Diseases.
This registry study aims to collect data on the provision of assistive devices, medicines, and other healthcare measures, such as ventilation therapy and nutrition support, in patients with Amyotrophic lateral sclerosis (ALS)…
- Trial name
- NCT05852418
- Where
- 16 sites in Germany
- Volunteers
- 25,000 people
- Sponsor
- Ambulanzpartner Soziotechnologie APST GmbH
Muscular Atrophy, Spinal
A Study to Collect Blood Samples From Patients With Spinal Muscular Atrophy for Biomarker Analysis
In this single center study blood samples for biomarker analysis will be collected from patients with spinal muscular atrophy. Up to 21 mL blood will be drawn from eligible patients at a single visit.
- Trial name
- NCT01910168
- Where
- 2 sites in United States
- Volunteers
- 36 people
- Sponsor
- Hoffmann-La Roche
Neurogenic Dysphonia, Neurogenic Dysphagia
Neurogenic Dysphonia/Dysphagia Registry
The Neurogenic Dysphonia/Dysphagia Registry is designed to be purely observational (i.e. non-interventional, exploratory). Patient data collected from the registry is expected to be consistent with any information which can be…
- Trial name
- StrongVoice (NCT02493491)
- Where
- 12 sites in United States
- Volunteers
- 146 people
- Sponsor
- Merz North America, Inc.
SMA - Spinal Muscular Atrophy
Safety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injectionin the Treatment of Patients With Type 2 Spinal Muscular Atrophy (SMA) - Phase III
This trial employs a multicenter, randomized, open-label, standard-of-care-controlled design and plans to enroll 50 patients with Type 2 SMA aged 2 to 12 years who have previously received nusinersen.
- Trial name
- NCT06971094
- Testing
- GC101 adeno-associated virus injection
- Where
- 7 sites in China
- Volunteers
- 50 people
- Sponsor
- GeneCradle Inc
Amyotrophic Lateral Sclerosis (ALS)
A Phase II Multi-centre, Extension Study to Investigate the Long Term Safety of ONO-2506PO in Patients Diagnosed With Amyotrophic Lateral Sclerosis (ALS)
The objective of this study is to investigate the long term safety of ALS patients taking ONO-2506PO.
- Trial name
- NCT00694941
- Testing
- ONO-2506PO
- Where
- 25 sites in Belgium, France and 5 more
- Volunteers
- 15 people
- Sponsor
- Ono Pharmaceutical Co., Ltd.
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3
An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy
The TOPAZ study will assess the safety and efficacy of SRK-015 in later-onset Spinal Muscular Atrophy (SMA Type 2 and Type 3) in pediatric and adult patients.
- Conditions
- Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA
- Trial name
- TOPAZ (NCT03921528)
- Testing
- SRK-015
- Where
- 16 sites in United States, Italy and 2 more
- Volunteers
- 58 people
- Sponsor
- Scholar Rock, Inc.
Muscular Atrophy, Spinal
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy
This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene…
- Trial name
- HINALEA 2 (NCT05861999)
- Testing
- Risdiplam
- Where
- 17 sites in United States, Germany and 4 more
- Volunteers
- 19 people
- Sponsor
- Hoffmann-La Roche
Muscular Atrophy, Spinal
A Study of Dosing Patterns and Costs in Patients With Spinal Muscular Atrophy Receiving Disease Modifying Therapies
The aim of this study was to assess real-world dosing patterns, long-term healthcare costs, and characteristics of SMA patients who received treatment with DMTs. DMTs included onasemnogene abeparvovec, nusinersen, and risdiplam.
- Trial name
- NCT07378943
- Where
- United States
- Volunteers
- 4,114 people
- Sponsor
- Novartis Pharmaceuticals
Spinal Muscular Atrophy (SMA)
Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
Spinal muscular atrophy (SMA) is a neurogenetic disorder caused by a loss or mutation in the survival motor neuron 1 gene (SMN1) on chromosome 5q13, which leads to reduced SMN protein levels and a selective dysfunction of motor…
- Trial name
- NCT04174157
- Testing
- Prospective observational registry, Zolgensma
- Where
- 99 sites in United States, Greece and 9 more
- Volunteers
- 700 people
- Sponsor
- Novartis Pharmaceuticals
Neurological Disorder, ALS (Amyotrophic Lateral Sclerosis)
INdependence Through Endovascular Neuroprosthetic Technology (INTENT): an Early Feasibility Study
INdependence Through Endovascular Neuroprosthetic Technology (INTENT): an Early Feasibility Study
- Conditions
- Neurological Disorder, ALS (Amyotrophic Lateral Sclerosis), Motor Neuron Disease, ALS - Amyotrophic Lateral Sclerosis
- Trial name
- NCT07543367
- Testing
- Stentrode
- Where
- 5 sites in United States
- Volunteers
- 10 people
- Sponsor
- Synchron, Inc.
Amyotrophic Lateral Sclerosis, Sporadic, Amyotrophic Lateral Sclerosis
ALS Diagnosis From a Saliva Sample: a Non-coding RNA Analysis Approach
RNALS is a multicentre, transversal, diagnostic and non-interventional study carried out in ALS reference centers; in order to identify a diagnostic signature for Amyotrophic Lateral Sclerosis by analyzing of coding and…
- Trial name
- RNALS (NCT05928416)
- Testing
- Saliva sample
- Where
- 19 sites in France
- Volunteers
- 653 people, healthy volunteers welcome
- Sponsor
- ZIWIG
Muscular Atrophy, Spinal
A Real-World Study of Clinical Use of Available Treatments for Pediatric Patients With Spinal Muscular Atrophy
The aim of this study was to evaluate real-world treatment patterns and clinical outcomes in pediatric SMA patients in the Czech and Slovak Republics, using data from the Registry of muscular Dystrophy (REaDY).
- Trial name
- NCT07474311
- Where
- Czechia
- Volunteers
- 213 people
- Sponsor
- Novartis Pharmaceuticals
Metastatic Hormone-Sensitive Prostate Cancer (mHSPC)
TulmiSTAR-02: A Phase I/II Open-label Study of Tulmimetostat in Combination With Darolutamide vs. Darolutamide, and Tulmimetostat With Abiraterone in Patients With Metastatic Hormone-sensitive Prostate Cancer (mHSPC)
The purpose of this study is to evaluate the safety, tolerability, and efficacy of two different treatment combinations of tulmimetostat in participants with de novo or recurrent metastatic hormone-sensitive prostate cancer…
- Trial name
- TulmiSTAR-02 (NCT07190300)
- Testing
- Tulmimetostat, Darolutamide, Abiraterone
- Where
- 31 sites in United States, Australia and 12 more
- Volunteers
- 181 people
- Sponsor
- Novartis Pharmaceuticals
Amyotrophic Lateral Sclerosis, ALS
Open-label Study to Evaluate Safety, Tolerability and PK of BHV-0223 in ALS
Phase 1, open-label study of BHV-0223 in ALS.
- Conditions
- Amyotrophic Lateral Sclerosis, ALS, Lou Gehrig Disease, Lou Gehrig's Disease
- Trial name
- NCT03520517
- Testing
- BHV-0223
- Where
- 5 sites in United States
- Volunteers
- 22 people
- Sponsor
- Biohaven Pharmaceuticals, Inc.
SMA - Spinal Muscular Atrophy, Gene Therapy
Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
Phase 3 pivotal US trial studying open-label intravenous administration of onasemnogene abeparvovec-xioi in spinal muscular atrophy (SMA) Type 1 participants.
- Trial name
- STR1VE (NCT03306277)
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- 16 sites in United States
- Volunteers
- 22 people
- Sponsor
- Novartis Gene Therapies