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Neural and structural adaptation of human skeletal muscle to overstretch and overload through mechanical stimuli delivered by dynamometry and exoskeletons

The primary objective of this study is to establish the relationships between structural and neural adaptation of human skeletal muscle when muscles are exposed to overstretch and overload through exercise.

Individualized trajectories of upper arm disease progression in Duchenne muscular dystrophy patients

Main objective1. To assess the longitudinal trajectories of upper arm flexor muscle MRI parameters, muscle force and upper arm function in a cohort of ambulant and non-ambulant DMD patients, to assess the value of this muscle as target for…

A nationwide natural history study in Becker muscular dystrophy: Modeling a slowly progressive neuromuscular disease to prepare for clinical trials.

The primary objective is to develop a disease model predicting clinical meaningful changes based on a combination of results of tests on clinical function, muscle MRI, echocardiography and circulating biomarkers applicable in various stages of…

A Phase 1 Study to Investigate the Absorption, Metabolism, and Excretion of PTC857 Following Oral Administration of a Single Dose of 14C-PTC857 at Steady-State Conditions in Healthy Male Volunteers.

In this study, we will investigate how quickly and to what extent the new compound PTC857 is absorbed, metabolized, and eliminated from the body. Both unlabeled and radioactively labeled PTC857 will be administered. Unlabeled PTC857 will be given…

An Open-Label Uncontrolled Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety and Activity of Nipocalimab in Children Aged 2 to less than 18 years with Generalized Myasthenia Gravis

This study has been transitioned to CTIS with ID 2022-502539-21-00 check the CTIS register for the current data. The purpose of this study is to determine the effect of nipocalimab on total serum immunoglobulin G (IgG) in pediatric participants 2 to…

Brain INvolvement in Dystrophinopathies: BIND: Deep functional phenotyping of Duchenne Muscular Dystrophy and Becker Muscular Dystrophy patients (WP5) Part 2

The primary objective of the second part of the study, which is the focus of this application, is to perform a comparative analysis of the cognitive profile and MRI outcomes of DMD/BMD patients.

Phase IIIb, open-label, single-arm, multi-center study to evaluate the safety, tolerability and efficacy of OAV101 administered intrathecally (1.2 x 1014 vector genomes) to participants 2 < 18 years of age with spinal muscular atrophy (SMA) who have discontinued treatment with nusinersen (Spinraza®) or risdiplam (Evrysdi®)

The purpose of this study is to characterize the safety and tolerability of OAV101 IT in participants who have discontinued treatment with nusinersen (Spinraza®) or risdiplam (Evrysdi®). The data from this study will expand on the data generated…

Phase 3, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients with Later-Onset Spinal Muscular Atrophy Receiving Background Nusinersen or Risdiplam Therapy

Primary-Assess the efficacy of apitegromab compared with placebo using the HFMSE in patients 2 through 12 years oldKey secondary-Assess the efficacy of apitegromab compared with placebo based on the number of patients with clinical improvement in…

Changes in movement profile related to use of a soft-robotic glove during high-demand tasks

The primary objective is to examine the effect of use of the assistive Carbonhand system during strenuous ADL tasks on the kinematic movement profile, compared to not using Carbonhand. Secondary objectives are to examine whether pain or discomfort…

A Multicenter, Open-label Extension (OLE) Study to Evaluate the Safety, Pharmacodynamics, and Clinical Effects of WVE-004 in Patients with C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD)

Primaire Objective: - To evaluate the safety and tolerability of long-term treatment with the study drug in patients with ALS or FTD with a documented mutation in the C9orf72 geneSecondary Objective: - To evaluate the clinical and pharmacodynamic (…

Withdrawal of Tiratricol Treatment in Males with Monocarboxylate Transporter 8 Deficiency (MCT8 Deficiency): A Double-blind, Randomized, Placebo-controlled Study

This study has been transitioned to CTIS with ID 2024-516124-34-00 check the CTIS register for the current data. Primary Objective:• To evaluate the effects of withdrawal of tiratricol treatment (placebo group) on serum total triiodothyronine (T3)…

A Randomized, Blinded, Placebo-Controlled, Phase 1 Single Ascending Dose Study in Healthy Adult Male Volunteers and an Open-Label Multiple Ascending Dose Study in Pediatric SMA Participants Previously Treated with Onasemnogene Abeparvovec (Zolgensma*) to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB115

This study has been transitioned to CTIS with ID 2023-505643-39-00 check the CTIS register for the current data. Part A: PrimaryTo evaluate the safety and tolerability of single ascending dose of BIIB115 administered via IT bolus injection to…

Improving symptomatic treatment with pyridostigmine and amifampridine: a randomized double-blinded, placebo controlled crossover trial in patients with myasthenia gravis (IMPACT-MG)

The aim of this study is to investigate the efficacy and tolerability of pyridostigmine monotherapy and amifampridine as add-on compared to placebo in patients with MG.

Development of a remote, patient-centric, clinical trial model using digital healthcare technology

Primary objective: To assess the feasibility and adherence of home-based monitoring of multiple aspects of disease progression in patients with ALS in order to develop a widely supported set of reliable digital parameters to capture data, collected…

A PHASE 3, OPEN-LABEL EXTENSION OF COURAGE-ALS (CY 5031)

To assess the long-term safety and tolerability of reldesemtiv in patients with ALS

A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study Evaluating Safety and Efficacy of CORT113176 (Dazucorilant) in Patients with Amyotrophic Lateral Sclerosis (DAZALS)

This study has been transitioned to CTIS with ID 2024-514082-19-00 check the CTIS register for the current data. The aim of this phase 2 study is to investigate the safety and efficacy of dazucorilant in the target ALS patient population.

A multicenter, open-label extension study to investigate the long-term safety of FAB122 in patients with Amyotrophic Lateral Sclerosis.

To evaluate the long-term safety of FAB122 in patients with ALS.

A Phase IIIb, Open Label Extension Study Evaluating The Safety And Tolerability of AMX0035 Up To 108 Weeks In Adult Participants with Amyotrophic Lateral Sclerosis (ALS) Previously Enrolled In Study A35-004 (PHOENIX)

To assess safety and tolerability of AMX0035 for treatment of ALS.

An Open-Label, Multicenter, Extension Trial to Evaluate the Long-Term Safety and Efficacy of Apitegromab in Patients with Type 2 and Type 3 Spinal Muscular Atrophy Who Completed Previous Investigational Trials of Apitegromab

This study has been transitioned to CTIS with ID 2024-511654-42-00 check the CTIS register for the current data. -Evaluate the long-term safety and tolerability of apitegromab in patients with Type 2 and Type 3 SMA-Evaluate the long-term efficacy of…

A Phase 1-3 Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION363 in Amyotrophic Lateral Sclerosis patients with Fused in Sarcoma mutations (FUS-ALS)

This study has been transitioned to CTIS with ID 2024-512163-31-00 check the CTIS register for the current data. Primary objective: To evaluate the clinical efficacy of ION363 in clinical functioning and survival in FUS-ALS patients. Secondary…