Browse Studies — Arctuva

Phase 2

A Phase 2 Double-Blinded, Randomized, Placebo-Controlled Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of Intravenous Administration of ARGX-119 in Pediatric Participants Aged 5 to Less Than 18 Years With Spinal Muscular Atrophy

This study aims to find the correct dose of ARGX-119 for children with SMA. The study will also look at how safe the study drug is, how well it works, how it m…

Spinal Muscular Atrophy (SMA)

Estimated

~$10 – $7,500

argenxNCT07287982

Observational

Feasibility and Preliminary Effectiveness of HABIT-ILE + Functional Strength Training in Children With Spinal Muscular Atrophy (SMA): a Prospective Single-group Intervention Trial.

This single-arm pilot study will assess the preliminary effectiveness of an intensive motor skill intervention (HABIT-ILE) combined with functional strength tr…

Spinal Muscular Atrophy (SMA)

Estimated

~$10 – $6,250

Teachers College, Columbia UniversityNCT07488338

Understanding the Diagnostic Pathway and Treatment Experience of Patients With Spinal Muscular Atrophy (SMA)

Evaluate the diagnostic journey, patient experience, and disparities in the treatment of Spinal Muscular Atrophy (SMA) in the MedStar Health System.

Spinal Muscular Atrophy (SMA)

Estimated

~$10 – $1,125

Medstar Health Research InstituteNCT07223489

A Prospective, Long-Term 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 lead…

Spinal Muscular Atrophy (SMA)

Estimated

~$10 – $1,050

Novartis PharmaceuticalsNCT04174157