PALLADIUM · Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq
Trial completed
BioMarin PharmaceuticalPhase 4 NCT06780332 Registry history of NCT06780332 on ClinicalTrials.gov (opens the registry)
Updated
3 trials changed in the last 30 days: 0 stopped, 0 with notable changes, 3 with minor changes only.
Trial completed
BioMarin PharmaceuticalPhase 4 NCT06780332 Registry history of NCT06780332 on ClinicalTrials.gov (opens the registry)
Updated
Enrollment closed, study ongoing
Also in this update: Trial sites reduced: 41 → 38 locations
BioMarin PharmaceuticalPhase 2 NCT06668805 Registry history of NCT06668805 on ClinicalTrials.gov (opens the registry)
Updated
Trial sites expanded: 25 → 28 locations
BioMarin PharmaceuticalPhase 2/3 NCT07441876 Registry history of NCT07441876 on ClinicalTrials.gov (opens the registry)
Updated
| Phase 1 | 1 | |
| Phase 1/2 | 3 | |
| Phase 2 | 7 | |
| Phase 2/3 | 1 | |
| Phase 3 | 5 | |
| Phase 4 | 1 |
| NCT ID | Title | Condition | Status | Updated |
|---|---|---|---|---|
| Phase 4 | ||||
| NCT07477691 | Immune Modulation During Palynziq® Treatment in Adults (IMPALA) | Phenylketonuria | Recruiting | 2026-06-17 |
| Phase 3 | ||||
| NCT07073014 | Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia | Hypochondroplasia | Enrolling by Invitation | 2026-09-09 |
| NCT05270837 | Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With… | Phenylketonuria (PKU) | Active, Not Recruiting | 2026-07-21 |
| NCT06224907 | Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe… | Hemophilia A | Active, Not Recruiting | 2026-03-19 |
| NCT03424018 | An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With… | Achondroplasia | Active, Not Recruiting | 2026-03-13 |
| NCT06455059 | Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia | Hypochondroplasia | Active, Not Recruiting | 2026-01-20 |
| Phase 2/3 | ||||
| NCT07441876 | Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With… | Achondroplasia | Recruiting | 2026-09-24 |
| Phase 2 | ||||
| NCT06382155 | A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature | Idiopathic Short Stature | Recruiting | 2026-05-22 |
| NCT07126262 | A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36… | Hypochondroplasia | Recruiting | 2026-03-31 |
| NCT06668805 | A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or… | Noonan Syndrome | Active, Not Recruiting | 2026-09-23 |
| NCT07573631 | An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in… | Duchenne Muscular Dystrophy (DMD) | Enrolling by Invitation | 2026-07-24 |
| NCT02724228 | A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children… | Achondroplasia | Active, Not Recruiting | 2026-03-13 |
| NCT03989947 | An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With… | Achondroplasia | Active, Not Recruiting | 2026-03-13 |
| NCT04554940 | A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia | Achondroplasia | Active, Not Recruiting | 2026-03-13 |
| Phase 1/2 | ||||
| NCT06280209 | A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and… | Duchenne Muscular Dystrophy | Active, Not Recruiting | 2026-06-25 |
| NCT04480567 | AAV Gene Therapy Study for Subjects with PKU | Phenylketonuria (PKU) | Active, Not Recruiting | 2024-12-12 |
| NCT05121376 | A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema | Hereditary Angioedema | Active, Not Recruiting | 2024-05-16 |
| Phase 1 | ||||
| NCT06738017 | Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants | Alpha 1-Antitrypsin Deficiency | Active, Not Recruiting | 2026-05-06 |
| Not specified | ||||
| NCT06168201 | VIrtual STudy in Achondroplasia for the US (VISTA) | Achondroplasia | Recruiting | 2026-06-30 |
| NCT06309979 | A Study to Assess Growth in Children With Idiopathic Short Stature | Idiopathic Short Stature | Recruiting | 2026-06-23 |
| NCT05813678 | A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace) | Phenylketonuria (PKU) | Recruiting | 2026-03-25 |
| NCT05579548 | A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to… | Phenylketonuria, Maternal | Recruiting | 2026-03-24 |
| NCT06305234 | A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq… | Phenylketonuria (PKU) | Recruiting | 2026-03-24 |
| NCT06212947 | A Multicenter Multinational Observational Study of Children With Hypochondroplasia | Hypochondroplasia | Recruiting | 2025-04-03 |
| NCT04476862 | Cerliponase Alfa Observational Study in the US | Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 | Active, Not Recruiting | 2026-06-30 |
Duchenne Muscular Dystrophy (2) · Hemophilia (2) · Heart Disease (1)