Rare Diseases Clinical Trials Update: Week 26, 2026

Published June 26, 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.

Acetylsalicylic Acid Versus Placebo as an add-on Treatment to Local Non-steroidal Anti-inflammatory Drug for the Management of Thrombotic Episodes in Superficial Venous Malformations in Children Aged 6 to 17 Years

This study is exploring the use of aspirin as an additional treatment to help manage painful blood clots in children with superficial venous malformations, a rare condition affecting veins. If this treatment works, it could reduce pain and improve daily life for children aged 6 to 17 who experience these episodes. The trial is currently recruiting in France and aims to enroll 34 participants.

Genome Sequencing Strategies for Genetics Diagnosis of Patients With Intellectual Disability

This study looked at using whole genome sequencing to improve the diagnosis of intellectual disability in children. By analyzing the entire genome of children and their unaffected parents, researchers hope to find clearer genetic answers that current tests may miss. The trial has completed with over 3800 participants in France, offering valuable information for families seeking a diagnosis.

Gene-Modified Stem Cell Therapy for Children With Autosomal Recessive Osteopetrosis (ARO)

This early phase trial is testing a gene-modified stem cell therapy for young children with autosomal recessive osteopetrosis, a rare bone disease. The treatment aims to address the root genetic cause, which could reduce the need for lifelong supportive care. The study is recruiting in Italy and will follow patients for up to two years after treatment to monitor outcomes.

Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of SAR442501 in Pediatric Participants With Achondroplasia

This phase 2 trial is evaluating a new injectable medicine called SAR442501 in children with achondroplasia, a rare condition affecting bone growth. The goal is to see if the treatment is safe and helps improve bone development, potentially reducing complications. The study included children from birth to 12 years old and took place across several countries.

Immersive Virtual Reality Program in Down Syndrome

This study is testing an immersive virtual reality program designed to support physical and cognitive skills in people with Down syndrome. The program could offer a more engaging way to help individuals improve development and quality of life. The trial is currently enrolling 20 participants in Spain.

TrialsAlert subscribers received the full impact-classified briefing and doctor-ready report this week. Get weekly briefings at trialsalert.com.