Clinical trials: Progressive Muscular Atrophy
SMA - Spinal Muscular Atrophy
An OLIP Study (Open Label Individual Patient) of Nusinersen in a Participant With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersen
This study is an open label individual patient study of the drug Nusinsersen.
- Trial name
- NCT07838779
- Testing
- Nusinersen Injectable Product
- Where
- Canada
- Volunteers
- 1 people
- Sponsor
- London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's
Ageing
Study to Assess the Effect of Leucine Along With Resistance Exercise on Muscle Strength and Quality of Life in Elders
The purpose of this study is to determine whether a nutritional supplement with an amino acid called leucine along with resistance exercise are effective in the improvement of muscle strength and quality of life in elderly people.
- Trial name
- NCT00929929
- Testing
- L-leucine., Maltodextrin., Exercise program.
- Where
- Spain
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Hospital Clinic of Barcelona
Spinal Muscular Atrophy
Mechanisms and Treatment of Exercise Intolerance and Persistent Fatigue in Spinal Muscular Atrophy
This study will focus on the pathophysiological underpinnings of reduced exercise capacity and fatigue in ambulatory patients with spinal muscular atrophy (SMA).
- Trial name
- NCT05518773
- Testing
- Observational
- Where
- United States
- Volunteers
- 34 people
- Sponsor
- Columbia University
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3
Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam
This Phase 3 trial (Study SRK-015-003) was conducted in patients ≥2 years old at Screening, who were previously diagnosed with later-onset spinal muscular atrophy (SMA) (i.e., Type 2 and Type 3 SMA) and were receiving an approved…
- Conditions
- Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA
- Trial name
- SAPPHIRE (NCT05156320)
- Testing
- Apitegromab
- Where
- 50 sites in United States, Belgium and 7 more
- Volunteers
- 188 people
- Sponsor
- Scholar Rock, Inc.
Duchenne Muscular Dystrophy, Spinal Muscular Atrophy
Optimum Insufflation Capacity in NMD
Patients with underlying neuromuscular disorder (NMD) often suffer from weakness in the inspiratory and expiratory muscles. Consequently they do not have the strength to generate the minimum flow of 160 to 300 liters/minute for…
- Conditions
- Duchenne Muscular Dystrophy, Spinal Muscular Atrophy, Chronic Respiratory Insufficiency
- Trial name
- NCT01981915
- Testing
- IPPB, LIAM
- Where
- Germany
- Volunteers
- 40 people
- Sponsor
- Universität Duisburg-Essen
Cancer Cachexia, Omega 3
Effects of Dietary Supplementation With omega3-fatty Acids (Fish Oil) and Progressive Resistance Training on Skeletal Muscle Status in Gynaecological, Gastrointestinal and Urological Cancer Patients
Cancer cachexia is a common and prognostically relevant complication of advanced malignancies, characterized by systemic inflammation, increased catabolism, and reduced nutritional intake, leading to a progressive loss of…
- Conditions
- Cancer Cachexia, Omega 3, Resistance Training, Cancer
- Trial name
- SUPPORT (NCT07643558)
- Testing
- Omega 3 FA (Fish Oil), Training
- Where
- Germany
- Volunteers
- 288 people
- Sponsor
- University of Erlangen-Nürnberg Medical School
Metastatic Castration Resistant Prostate Cancer
A Study of AC176 for the Treatment of Metastatic Castration Resistant Prostate Cancer
This clinical trial is evaluating a drug called AC176 in participants with metastatic castration resistant prostate cancer (mCRPC) who have progressed on at least two prior systemic therapies.
- Trial name
- NCT05241613
- Testing
- AC176
- Where
- 5 sites in United States
- Volunteers
- 28 people
- Sponsor
- Accutar Biotechnology Inc
Anesthesia, Spinal, Spinal Puncture Complications
Ultrasound-assisted or Landmark-based Intrathecal Administration of Nusinersen in Adult Patients With Spinal Muscular Atrophy (The EchoSpin Study)
Nusinersen (Spinraza, Biogen Inc, Boston, MA), the first treatment approved by FDA and EMA for all Spinal Muscular Atrophy (SMA) subtypes, is an antisense oligonucleotide that is administered intrathecally through a lumbar…
- Conditions
- Anesthesia, Spinal, Spinal Puncture Complications, Spinal Muscular Atrophy, Ultrasonography
- Trial name
- EchoSpin (NCT05644899)
- Testing
- Interlaminar ultrasound-assisted intrathecal administration of nusinersen, Landmark-based intrathecal administration of nusinersen
- Where
- Italy
- Volunteers
- 51 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Inclusion Body Myositis, Myotonic Dystrophy
Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders
Currently, there are no standardised fall risk scores or guidelines on when to use appropriate assistive gait devices (AGDs) for people with neuromuscular disorders (NMDs).
- Conditions
- Inclusion Body Myositis, Myotonic Dystrophy, Limb-girdle and Facioscapulohumeral Muscular Dystrophies, Pompe Disease
- Trial name
- FBIndex (NCT06605612)
- Testing
- FBIndex
- Where
- Germany
- Volunteers
- 108 people
- Sponsor
- LMU Klinikum
ALS, PLS
ALS/MND Natural History Study Data Repository
This is a data repository for multi-site multi-protocol clinic-based Natural History Study of ALS and Other Motor Neuron Disorders (MND).
- Conditions
- ALS, PLS, MND (Motor Neurone DIsease), Kennedy Disease
- Trial name
- NCT05966038
- Where
- 18 sites in United States, Israel and 1 more
- Volunteers
- 5,000 people
- Sponsor
- Massachusetts General Hospital
Spinal Muscular Atrophy
A Registry of Cases of Spinal Muscular Atrophy
Because no long-term record of prenatal Spinal Muscular Atrophy (SMA) treatment exists, this study will address an important gap in the understanding of the safety and benefit of treatment while in the womb.
- Trial name
- NCT07805278
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- St. Jude Children's Research Hospital
Spinal Muscular Atrophy
Short and Long Term Treatment With 4-AP in Ambulatory SMA Patients
The purpose of this study is to assess whether 4-AP (Dalfampridine-ER, Ampyra) improves walking ability and endurance in adult patients with Spinal muscular atrophy (SMA) Type 3 compared to placebo and whether the duration of…
- Trial name
- NCT01645787
- Testing
- 4-aminopyridine
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Columbia University
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, Neuromuscular Disorders
Study of Feasibility to Reliably Measure Functional Abilities' Changes in Nonambulant Neuromuscular Patients Without Trial Site Visiting
Clinical trials organization in several neuromuscular disorders (NMD) has some specific issues. Nonambulant status and difficulties with transportation are among them.
- Trial name
- NCT02235090
- Testing
- Direct current stimulation of cervical spinal cord
- Where
- Ukraine
- Volunteers
- healthy volunteers welcome
- Sponsor
- Charitable Foundation Children with Spinal Muscular Atrophy
Spinal Muscular Atrophy
Antisense Oligonucleotide for Spinal Muscular Atrophy
This is a longitudinal, multiple-center, observational study of patients genetically confirmed chromosome 5q SMA to monitor the efficacy, safety, tolerability of SPINRAZA® (nusinersen) for up to 24 months.
- Trial name
- NCT05187260
- Testing
- Nusinersen
- Where
- 3 sites in China
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- First Affiliated Hospital of Fujian Medical University
Spinal Muscular Atrophy
A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA).
The primary objective of Part 1 of this study is to assess the safety and tolerability of Nusinersen in participants with SMA who are not eligible to participate in the clinical studies ISIS 396443-CS3B (NCT02193074) or ISIS…
- Trial name
- EMBRACE (NCT02462759)
- Testing
- Nusinersen
- Where
- 7 sites in United States, Germany
- Volunteers
- 21 people
- Sponsor
- Biogen
Motor Disorders
Patients With ALS and Other Motor Disorders Will be Treated With Umbilical or Placental Mesenchymal Cell Exosome Solution
Patients with ALS and other Motor Discorders will be treated with mesenchymal cell exosome solution.
- Trial name
- NCT07105371
- Testing
- Exosome Secretome
- Where
- 2 sites in United States, Greece
- Volunteers
- 70 people
- Sponsor
- The Foundation for Orthopaedics and Regenerative Medicine
Spinal Muscular Atrophy
Newborn Screening for Spinal Muscular Atrophy (SMA) - a Proof of Principle Study Using Anonymised Blood Spots.
Spinal muscular atrophy (SMA) is a motor neuron disorder caused by the absence of a functional survival of motor neuron 1, telomeric (SMN1) gene. Type I SMA, a lethal disease of infancy, accounts for the majority of cases.
- Trial name
- NCT05237544
- Where
- United Kingdom
- Volunteers
- 5,065 people, healthy volunteers welcome
- Sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust
Spinal Muscular Atrophy Type 3
Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3
A long term safety study of amifampridine phosphate in patients with spinal muscular atrophy (SMA) Type 3.
- Trial name
- SMA3 (NCT03819660)
- Testing
- Amifampridine Phosphate 10 MG Oral Tablet
- Where
- Italy
- Volunteers
- 13 people
- Sponsor
- Catalyst Pharmaceuticals, Inc.