Rare Diseases Clinical Trials Update: Week 24, 2026

Published June 12, 2026 — 5 trials covered

By Victor Lafforgue, Founder of TrialsAlert. Blog posts are AI-drafted from ClinicalTrials.gov source data and reviewed by the editorial team. See our editorial policy for details.

An Open-label Extension Trial to Evaluate the Long-term Safety of KVD900 (Sebetralstat) for On-Demand Treatment of Angioedema Attacks in Adolescent and Adult Patients With Hereditary Angioedema (HAE)

This Phase 3 trial tested KVD900, an oral medication designed to treat sudden attacks of hereditary angioedema (HAE) in adolescents and adults. The study involved 145 patients across 71 locations worldwide, focusing on the long-term safety of this new treatment. If proven safe and effective, KVD900 could offer a simpler, pill-based option instead of current treatments that require injections, potentially making it easier for patients to manage their condition and improving their daily lives.

Gene Transfer Study in Patients With Late Onset Pompe Disease

This early phase trial is exploring a gene transfer therapy called AT845 for adults with Late Onset Pompe Disease, a rare muscle disorder. It included 11 participants from the US and UK and aims to provide a treatment that addresses the root cause of the disease rather than just relieving symptoms. Success in this study could open the door to a new way of managing Pompe Disease that may slow or stop its progression.

Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PN

This Phase 1 trial focused on a new granule form of the drug selumetinib for young children with neurofibromatosis type 1 (NF1) who have difficult-to-treat tumors called plexiform neurofibromas. The study explored how the medication is processed in the body and its safety to find the best dosing for children aged 1 to under 7. Results from multiple countries are already available and may lead to a more manageable treatment option for very young patients facing this rare condition.

A Remote Study Using Technology to Assess Outcomes in DMD

This study tested a wearable device to monitor motor function in boys under four years old with Duchenne Muscular Dystrophy (DMD). Conducted in the United Kingdom, it allowed toddlers to wear watch-like devices at home for six months, reducing the need for hospital visits. This approach could make it easier to track motor skills and evaluate new treatments earlier in the disease course.

DMCRN-02-001: Assessing Pediatric Endpoints in DM1

This study is working to improve how doctors measure and understand congenital myotonic dystrophy type 1 (CDM) in children. Recruiting 50 children in the United States and Italy, it aims to develop better clinical assessments to track disease progression. Improved measurements can help guide future treatments to be more effective in managing this rare condition.

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