Achondroplasia Clinical Trials

66 clinical trials for Achondroplasia across international registries, including 18 currently recruiting participants. Compare status, phase, eligibility criteria and locations.

• A Clinical Trial to Investigate Long-term Safety, Tolerability, and Efficacy of Weekly Subcutaneous Doses With TransCon CNP in Children and Adolescents With Achondroplasia — RECRUITING · PHASE2
• A Study of TYRA-300 in Children With Achondroplasia: BEACH301 — RECRUITING · PHASE2
• Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia — RECRUITING · PHASE2
• Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH) — RECRUITING · PHASE2
• Determination of the appropriate tracheal tube size in children and adults with short stature due to skeletal dysplasia — RECRUITING
• VIrtual STudy in Achondroplasia for the US (VISTA) — RECRUITING
• A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide in Adolescents (12 - 18 Years of Age) With Achondroplasia. — RECRUITING · PHASE2
• A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia — RECRUITING · PHASE2
• Achondroplasia Natural History Multicenter Clinical Study — RECRUITING
• Prospective Longitudinal Monocentric Study to Measure Limb Movement in Patients With FGFR3-related Skeletal Dysplasia — RECRUITING
• A Study in Children With Achondroplasia — RECRUITING
• Extension Study of Infigratinib in Children With Achondroplasia (ACH) — RECRUITING · PHASE2
• PHASE 2B, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED CLINICAL TRIAL, PRECEDED BY A SINGLE ASCENDING DOSE PORTION AND A PHASE 2 OPEN-LABEL PORTION, TO EVALUATE THE SAFETY AND EFFICACY OF ORAL INFIGRATINIB IN INFANTS AND YOUNG CHILDREN WITH ACHONDROPLASIA — RECRUITING · PHASE2
• Evaluate the Efficacy and Safety of KK8398 in Patients With Achondroplasia（AOBA Study） — RECRUITING · PHASE3
• A MULTICENTER, PHASE 2, DOSE-ESCALATION/DOSE-EXPANSION STUDY OF TYRA-300 IN CHILDREN WITH ACHONDROPLASIA WITH OPEN GROWTH PLATES: BEACH301 — RECRUITING · PHASE2
• ATTACH: A PHASE 2, MULTICENTER, LONG-TERM, OPEN LABEL EXTENSION TRIAL EVALUATING SAFETY, TOLERABILITY, AND EFFICACY OF SUBCUTANEOUS DOSES OF TRANSCON CNP ADMINISTERED ONCE WEEKLY IN CHILDREN AND ADOLESCENTS WITH ACHONDROPLASIA — RECRUITING · PHASE2
• PHASE 2, OPEN-LABEL, LONG-TERM, EXTENSION (OLE) STUDY OF INFIGRATINIB, AN FGFR 1-3-SELECTIVE TYROSINE KINASE INHIBITOR, IN CHILDREN WITH ACHONDROPLASIA: PROPEL OLE — RECRUITING · PHASE2
• Registry for Patients With Achondroplasia / Hypochondroplasia (OMPR-Ach/Hy) — RECRUITING
• A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Multiple Subcutaneous Doses of TransCon CNP Administered Once Weekly in Children With Achondroplasia — COMPLETED · PHASE2
• A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children With Achondroplasia — COMPLETED · PHASE4
• A Phase 3, Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children with Achondroplasia — TERMINATED · PHASE3
• Phase 2, Open-Label, Long-Term, Extension (OLE) Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children with Achondroplasia: PROPEL OLE — TERMINATED · PHASE2
• A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Children With Achondroplasia — COMPLETED · PHASE2
• A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia — COMPLETED · PHASE2
• A PHASE 3, RANDOMIZED, DOUBLE-BLIND, ACTIVE CONTROLLED, MULTICENTER TRIAL INVESTIGATING THE EFFICACY AND SAFETY OF DUAL THERAPY WITH ONCE-WEEKLY NAVEPEGRITIDE AND ONCE-WEEKLY LONAPEGSOMATROPIN COMPARED TO NAVEPEGRITIDE MONOTHERAPY AND LONAPEGSOMATROPIN MONOTHERAPY IN CHILDREN AND ADOLESCENTS WITH ACHONDROPLASIA — NOT_YET_RECRUITING · PHASE3
• A Multicenter, Randomized, Operationally Seamless Phase 2/3 Study to Evaluate the Efficacy and Safety of BMN 333 versus Vosoritide in Children with Achondroplasia — NOT_YET_RECRUITING · PHASE3
• Prospective Clinical Assessment Study in Children With Achondroplasia (ACH) — COMPLETED
• A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia — COMPLETED · PHASE3
• A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia — ACTIVE_NOT_RECRUITING · PHASE2
• A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH) — ACTIVE_NOT_RECRUITING · PHASE2
• An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia — ACTIVE_NOT_RECRUITING · PHASE2
• An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia — ACTIVE_NOT_RECRUITING · PHASE3
• 3D Gait analysis with people with achondroplasia — OTHER
• A Phase 2 Clinical Trial to Evaluate Efficacy, Safety, and Tolerability of Navepegritide in Combination With Lonapegsomatropin in Children With Achondroplasia — ACTIVE_NOT_RECRUITING · PHASE2
• A MULTICENTER, RANDOMIZED, OPERATIONALLY SEAMLESS PHASE 2/3 STUDY TO EVALUATE THE EFFICACY AND SAFETY OF BMN 333 VERSUS VOSORITIDE IN CHILDREN WITH ACHONDROPLASIA — NOT_YET_RECRUITING · PHASE2
• Phase 2b, Randomized, Double-Blind, Placebo-Controlled Clinical Trial, Preceded by a Single Ascending Dose Portion and a Phase 2 Open-Label Portion, to Evaluate the Safety and Efficacy of Oral Infigratinib in Infants and Young Children with Achondroplasia — TERMINATED · PHASE2
• A Study of ABSK061 to Assess Safety, Tolerability, Pharmacokinetics, and Efficacy in Children With Achondroplasia — NOT_YET_RECRUITING · PHASE1
• A Phase 3, Multicenter, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Infigratinib in Children 3 to <18 Years of Age with Achondroplasia: PROPEL 3 — OTHER · PHASE3
• A PHASE 2, MULTICENTER, DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED TRIAL, EVALUATING SAFETY, TOLERABILITY, AND EFFICACY OF SUBCUTANEOUS DOSES OF TRANSCON CNP ADMINISTERED ONCE WEEKLY FOR 52 WEEKS IN INFANTS (0 TO <2 YEARS OF AGE) WITH ACHONDROPLASIA FOLLOWED BY AN OPEN LABEL EXTENSION (OLE) PERIOD — NOT_YET_RECRUITING · PHASE2
• Study of Infigratinib in Children With Achondroplasia — COMPLETED · PHASE2
• A PHASE 2B, MULTICENTER, DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED TRIAL EVALUATING EFFICACY AND SAFETY OF SUBCUTANEOUS DOSES OF NAVEPEGRITIDE ADMINISTERED ONCE WEEKLY FOR 52 WEEKS IN ADOLESCENTS (12-<18 YEARS OF AGE) WITH ACHONDROPLASIA — NOT_YET_RECRUITING · PHASE2
• Observational Study Investigating Clinical & Anthropometric Characteristics of Children With Achondroplasia. — TERMINATED
• A Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia — OTHER · PHASE2
• A Study to Learn About Recifercept in Patients With Achondroplasia — COMPLETED
• A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia — COMPLETED · PHASE2
• A Multi-center, Longitudinal, Observational Study of Children With Achondroplasia — COMPLETED
• A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia — TERMINATED · PHASE2
• Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia — TERMINATED · PHASE2
• A PHASE 3, MULTICENTER, DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF INFIGRATINIB IN CHILDREN 3 TO <18 YEARS OF AGE WITH ACHONDROPLASIA: PROPEL 3 — COMPLETED · PHASE3
• Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants — TERMINATED · PHASE1

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