Rare Diseases Clinical Trials Update: Week 14, 2026

Published April 3, 2026 — 5 trials covered

By Victor Lafforgue, Founder of TrialsAlert

Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age

This Phase 3 trial is testing a new triple combination drug therapy for children aged 1 to 11 with cystic fibrosis who have specific genetic mutations. If this treatment works, it could improve lung function and quality of life by targeting the root cause of cystic fibrosis in young patients. The study is actively recruiting children worldwide, offering hope for earlier and more effective treatment options in this age group.

Pediatric Safety Follow-up Study of Prior Treatment With Romosozumab for Osteogenesis Imperfecta

This Phase 3 study focuses on the safety of romosozumab, a bone-strengthening treatment, in children and adolescents with osteogenesis imperfecta, a rare condition that causes fragile bones. It is recruiting participants who previously received romosozumab to better understand long-term safety in this young population. The results could help families and doctors make informed decisions about managing this challenging condition.

A Study of the Safety of Mibavademab in Pediatric and Adult Participants Switching From Metreleptin to Mibavademab for the Treatment of Generalized Lipodystrophy (GLD)

This Phase 3 study evaluates the safety of switching to mibavademab for children and adults with generalized lipodystrophy, a rare disorder affecting fat storage, who were previously treated with metreleptin. The study is no longer recruiting but remains active, with results expected after completion. This research could lead to a safer or better-tolerated treatment option for people living with this rare disease.

A Study to Assess How Effective and Safe NVD003 is for Treating Patients With Congenital Pseudarthrosis of the Tibia

This Phase 3 trial is comparing a new treatment called NVD003 with the current standard, iliac crest bone graft, in children with congenital pseudarthrosis of the tibia, a rare bone condition causing poor healing. The study is recruiting in several countries and plans to evaluate recovery and bone healing one year after surgery. Positive results could provide a new option to improve bone healing for affected children.

Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age

This Phase 4 study examines the long-term impact of Elaprase treatment on growth in children with Hunter syndrome who began therapy before age six. The results, available from multiple international sites, help to understand how this treatment influences height and weight over time. This information is important for families and healthcare providers to better manage growth and development in children with this rare condition.

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