Pediatrics

A Phase II, Open-Label, Two-Arm Study of Therapeutic Iobenguane (131I) as single agent or in combination with Vorinostat for Recurrent or Progressive High-Risk Neuroblastoma Subjects (OPTIMUM Trial)

Cancer Pediatrics Brain and Spinal Cord Cancer Pediatric Subjects Female Subjects Male Subjects

The purpose of this study is to evaluate the efficacy and safety of 131I-MIBG in combination with Vorinostat in patients with Recurrent or Progressive neuroblastoma

Tuberous Sclerosis Complex (TSC) Biosample Repository and Natural History Database

Pediatrics Pediatric Subjects Adult Subjects Female Subjects Male Subjects

Long-Term Open-Label Study to Assess the Safety and Efficacy of Vatiquinone in Patients With Friedreich Ataxia

Pediatrics Pediatric Subjects Adult Subjects

The primary objective of this study is to assess the long-term safety of vatiquinone in participants with Friedreich ataxia (FA) previously exposed to vatiquinone.

Assessing Pediatric Endpoints in DM1 (ASPIRE-DM1)

Pediatrics Pediatric Subjects

The overall goal of the study is to establish valid clinical endpoint assessments for children with congenital myotonic dystrophy type 1 and childhood myotonic dystrophy type 1, and develop biomarkers for the condition.

A 2-part Phase 2 Study of Safety, Pharmacokinetics and Biomarkers in Children with Duchenne Muscular Dystrophy including a Randomized, Double-Blind, Placebo-Controlled Part A, Followed by an Open-Label Part B

Pediatrics Pediatric Subjects

The LYNX study is a 2-part, multicenter, Phase 2 study of safety, pharmacokinetics and biomarkers in children with Duchenne muscular dystrophy including a randomized, double-blind, placebo-controlled part A, followed by an open-label part B.

A two-part multicenter study: a randomized, double-blind, placebocontrolleddose- escalation safety phase (Part 1) followed by double-blind, placebocontrolled, adaptive phase (Part 2) study to evaluate the safety and efficacy of AB-1003 in adult subjec…

Pediatrics Adult Subjects

The purpose of this study is to evaluate the safety and tolerability of a single intravenous infusion of AB-1003 in adults diagnosed with limb girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). Participants will be treated in sequential, dose-level cohorts. (Part 1)

A Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Ambulatory and Non-Ambulatory Participants with Spinal Muscular Atrophy with Open-Label Extension (RESILIENT)

Pediatrics Pediatric Subjects Adult Subjects

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 or risdiplam or have a history of onasemnogene abeparvovec-xioi, compared to placebo.

University of Iowa Genetic and Rare Diseases Biorepository.

Pediatrics Pediatric Subjects Adult Subjects Female Subjects Male Subjects

Multiomic characterization of induced pluripotent stem cells(iPSC) models of Hereditary Spastic Paraplegias and ataxias.

Pediatrics Pediatric Subjects Adult Subjects Female Subjects Male Subjects

ARST2032: A Prospective Phase 3 Study of Patients with Newly Diagnosed Very Low-Risk and Low-Risk Fusion Negative Rhabdomyosarcoma

Cancer Pediatrics Sarcoma Pediatric Subjects Adult Subjects

Rhabdomyosarcoma is a type of cancer that occurs in the soft tissues in the body. This phase III trial aims to maintain excellent outcomes in patients with very low risk rhabdomyosarcoma (VLR-RMS) while decreasing the burden of therapy using treatment with 24 weeks of vincristine and dactinomycin (VA) and examines the use of centralized molecular risk stratification in the treatment of rhabdomyosarcoma. Another aim of the study it to find out how well patients with low risk rhabdomyosarcoma (LR-RMS) respond to standard chemotherapy when patients with VLR-RMS and patients who have rhabdomyosarcoma with DNA mutations get separate treatment. Finally, this study examines the effect of therapy intensification in patients who have RMS cancer with DNA mutations to see if their outcomes can be improved.

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