Health Trials

Congenital, Hereditary, and Neonatal Diseases and Abnormalities

2

Review clinical trials related to Congenital, Hereditary, and Neonatal Diseases and Abnormalities. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Not yet recruiting

Prenatal Transplantation for Fetuses With Fanconi Anemia

The investigators aim to evaluate the safety and efficacy of in utero hematopoietic stem cell transplantation (IUHSCT) for the treatment of fetuses diagnosed with Fanconi anemia (FA) during pregnancy.

Participants needed: 12
Trial details
Phase: Phase 1, Phase 2Biological sex: AllType: InterventionalSponsor: Agnieszka CzechowiczUpdated: Jun 16, 2026Locations: 2
Eligibility criteria

Male or female fetuses from 19^0/7 - 28^0/7 weeks gestational age at time of tra... [+3]

Fetal Participant Exclusion Criteria: Major anatomic or genetic anomalies that c... [+1]

Read more
Status: Recruiting

Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)

The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment rostudirsen (DYNE-251) administered intravenously (IV) every 4 weeks to ambulatory Duchenne muscular dystrophy (DMD) participants, 4 to 18 years of age, with dystrophin mutations amenable to exon 51 skipping.

Participants needed: 90
Trial details
Phase: Phase 3Age: 4-18Biological sex: MaleType: InterventionalSponsor: Dyne TherapeuticsUpdated: May 27, 2026Locations: 1
Eligibility criteria

Ambulatory male with confirmed diagnosis of DMD and with a mutation in the dystr... [+2]

Receipt of ongoing immunosuppressive therapy (other than glucocorticoids) within... [+6]

Read more