Clinical trials: Motor Neuron Disorder
- Condition Motor Neuron Disorder
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Spinal Muscular Atrophy (SMA)
Virtual Reality for the Mitigation of Anxiety During Intrathecal Administration in Participants With Spinal Muscular Atrophy
The primary objective of the study is to evaluate anxiety level during intrathecal administration (IT) under standard of care (SOC) and virtual reality (VR) conditions using a reliable self-rating scale.
- Trial name
- REALITY (NCT05354414)
- Testing
- Virtual reality, Standard of Care
- Where
- 12 sites in France
- Volunteers
- 63 people
- Sponsor
- Biogen
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
Muscular Atrophy, Spinal
Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy
Open-label, multi-center clinical study is to assess the safety, tolerability, pharmacokinetic (PK), pharmacodynamics (PD), and efficacy of Risdiplam (RO7034067) in infants with Type 1 spinal muscular atrophy (SMA).
- Trial name
- FIREFISH (NCT02913482)
- Testing
- Risdiplam
- Where
- 32 sites in United States, Belgium and 14 more
- Volunteers
- 62 people
- Sponsor
- Hoffmann-La Roche
Spinal Muscular Atrophy
A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA)
The primary objective is to examine the clinical efficacy of multiple doses of nusinersen (ISIS 396443) administered intrathecally to participants with Infantile-Onset Spinal Muscular Atrophy (SMA).
- Trial name
- NCT01839656
- Testing
- nusinersen
- Where
- 4 sites in United States, Canada
- Volunteers
- 21 people
- Sponsor
- Biogen
Amyotrophic Lateral Sclerosis
Individual Patient Expanded Access IND of Autologous HBadMSCs for the Treatment of Amyotrophic Lateral Sclerosis
The drug for this submission is Hope Biosciences' autologous, adipose-derived culture-expanded mesenchymal stem cells (HB-adMSCs) for the treatment of a single patient with Amyotrophic Lateral Sclerosis (ALS).
- Trial name
- NCT04514952
- Testing
- HB-adMSCs
- Where
- United States
- Sponsor
- Hope Biosciences Research Foundation
Spinal Muscular Atrophy
A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies
The primary objective is to evaluate long-term safety and tolerability of nusinersen (ISIS 396443) administered by intrathecal (IT) injection to participants with Spinal Muscular Atrophy (SMA) who previously participated in…
- Trial name
- SHINE (NCT02594124)
- Testing
- nusinersen
- Where
- 47 sites in United States, Australia and 12 more
- Volunteers
- 292 people
- Sponsor
- Biogen
Spinal Muscular Atrophy, SMA
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
This double-blind, Phase 2, multiple-dose study will be conducted to evaluate the PK/PD, efficacy, safety, and tolerability of apitegromab in subjects <2 years old with 5q autosomal recessive SMA who have delayed motor milestones…
- Conditions
- Spinal Muscular Atrophy, SMA, Spinal Muscular Atrophy Type 2, Spinal Muscular Atrophy Type 3
- Trial name
- OPAL (NCT07047144)
- Testing
- Apitegromab, Nusinersen, Risdiplam
- Where
- 23 sites in United States, Belgium and 5 more
- Volunteers
- 52 people
- Sponsor
- Scholar Rock, Inc.
Spinal Muscular Atrophy
A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy
This is a multi-center, exploratory, non-comparative, and open-label study to investigate the safety, tolerability, PK, and PK/PD relationship of risdiplam in adults, children and infants with Spinal Muscular Atrophy (SMA)…
- Trial name
- Jewelfish (NCT03032172)
- Testing
- Risdiplam
- Where
- 24 sites in United States, Belgium and 7 more
- Volunteers
- 174 people
- Sponsor
- Hoffmann-La Roche
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
Infantile-onset Spinal Muscular Atrophy
Expanded Access Program (EAP) for Nusinersen in Participants With Infantile-onset (Consistent With Type 1) Spinal Muscular Atrophy (SMA)
To provide access to nusinersen to eligible patients with Infantile-onset Spinal Muscular Atrophy (SMA) (consistent with Type 1) to address a high-unmet medical need.
- Trial name
- NCT02865109
- Testing
- Nusinersen
- Where
- 7 sites in Colombia, New Zealand and 1 more
- Sponsor
- Biogen
Spinal Muscular Atrophy
A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam (ASCEND)
In this study, researchers will learn more about the use of a higher dose of nusinersen (BIIB058) in participants with spinal muscular atrophy (SMA).
- Trial name
- ASCEND (NCT05067790)
- Testing
- Nusinersen
- Where
- 22 sites in United States, Germany and 3 more
- Volunteers
- 45 people
- Sponsor
- Biogen
Spinal Muscular Atrophy 1
Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1
The purpose of this trial is to evaluate safety and efficacy of intravenous delivery of AVXS-101 as a treatment of spinal muscular atrophy Type 1 (SMN1).
- Trial name
- NCT02122952
- Testing
- AVXS-101
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Novartis Gene Therapies
Spinal Muscular Atrophy
A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety in Participants Aged 15 to 60 Years With Spinal Muscular Atrophy (SMA) Who Are Either New to SMA Treatment or Were Previously Treated With Risdiplam
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 individuals with SMA who are between the ages…
- Trial name
- SOLAR (NCT07444476)
- Testing
- Salanersen
- Where
- 3 sites in United States, China
- Volunteers
- 90 people
- Sponsor
- Biogen
Muscular Atrophy, Spinal
A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy (PIERRE-PK)
In this PIERRE-PK study, researchers will learn how the body processes nusinersen when it is given through the ThecaFlex DRx™ System, compared to when nusinersen is given by lumbar puncture (LP).
- Trial name
- PIERRE-PK (NCT06555419)
- Testing
- Nusinersen, ThecaFlex DRx System
- Where
- 21 sites in United States, France and 5 more
- Volunteers
- 78 people
- Sponsor
- Biogen
Spinal Muscular Atrophy
A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA)
The primary objective of this study is to examine the clinical efficacy of nusinersen (ISIS 396443) administered intrathecally to participants with later-onset Spinal Muscular Atrophy (SMA).
- Trial name
- CHERISH (NCT02292537)
- Testing
- Nusinersen
- Where
- 24 sites in United States, Canada and 8 more
- Volunteers
- 126 people
- Sponsor
- Biogen
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
Spinal Muscular Atrophy, Neuromuscular Diseases
A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy
This trial will study the efficacy and safety of taldefgrobep alfa as an adjunctive therapy for participants who are already taking a stable dose of nusinersen and/or risdiplam and/or have a history of onasemnogene abeparvovec…
- Conditions
- Spinal Muscular Atrophy, Neuromuscular Diseases, SMA
- Trial name
- RESILIENT (NCT05337553)
- Testing
- taldefgrobep alfa
- Where
- 53 sites in United States, Belgium and 7 more
- Volunteers
- 269 people
- Sponsor
- Biohaven Pharmaceuticals, Inc.
Spinal Muscular Atrophy
An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal Muscular Atrophy
This is an interventional study to evaluate safety and efficacy of AAV-hSMN1 in spinal muscular atrophy patients.
- Trial name
- NCT06288230
- Testing
- vesemnogene lantuparvovec
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Lantu Biopharma
Healthy Volunteer, Muscular Atrophy, Spinal
A Study to Learn About the Safety of BIIB115 Injections and How BIIB115 is Processed in the Bodies of Healthy Adult Male Volunteers and of Pediatric Participants With Spinal Muscular Atrophy Who Previously Took Onasemnogene Abeparvovec
In this study, researchers will learn about a study drug called BIIB115 in healthy adult male volunteers and in participants with spinal muscular atrophy (SMA). This study will focus on children with SMA.
- Trial name
- NCT05575011
- Testing
- BIIB115
- Where
- 16 sites in Belgium, Canada and 7 more
- Volunteers
- 62 people, healthy volunteers welcome
- Sponsor
- Biogen
Amyotrophic Lateral Sclerosis
Early Access Program for Ulefnersen in Children, Adolescents and Adults With FUS-associated Amyotrophic Lateral Sclerosis (FUS-ALS)
This expanded access program provides access to ulefnersen for eligible patients with FUS-associated amyotrophic lateral sclerosis (FUS-ALS) who meet protocol-defined eligibility criteria.
- Trial name
- NCT07862959
- Testing
- Ulefnersen
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.