Achondroplasia (DBCOND0028226)

Identifiers

Synonyms
Achondroplastic dwarf / Achondroplasia (disorder) / Hypochondroplasia / Hypochondroplasia (disorder) / Osteochondrodysplasia / Skeletal dysplasia / Congenital skeletal dysplasia (disorder)

Associated Data

Indicated Drugs and Targets
DrugDescriptionTargets
Vosoritide
An analogue of C-type naturietic peptide used to promote bone growth in pediatric patients with achondroplasia.
Clinical Trials
IdentifierTitleDrug(s)PurposePhaseStatus
NCT05598320
A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Children With Achondroplasiatreatment2 / 3active_not_recruiting
NCT06079398
A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared with Placebo in Infants (0 to <2 Years of Age) with AchondroplasiaNo drug interventionstreatment2recruiting
NCT05659719
A Study to Learn About Recifercept in Patients With AchondroplasiaNo drug interventionsNot AvailableNot Availablecompleted
NCT06164951
A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents with Achondroplasiatreatment3enrolling_by_invitation
NCT04265651
Study of Infigratinib in Children With Achondroplasiatreatment2active_not_recruiting
NCT03780153
The Norwegian Adult Achondroplasia StudyNo drug interventionsNot AvailableNot Availablecompleted
NCT04638153
A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasiaprevention2terminated
NCT03989947
An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasiatreatment2active_not_recruiting
NCT03583697
A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasiatreatment2completed
NCT04035811
Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)No drug interventionsNot AvailableNot Availablerecruiting
NCT05929807
A Clinical Trial to Investigate Long-term Safety, Tolerability, and Efficacy of Weekly Subcutaneous Doses With TransCon CNP in Children and Adolescents With Achondroplasiatreatment2 / 3enrolling_by_invitation
NCT03794609
Observational Study Investigating Clinical & Anthropometric Characteristics of Children With Achondroplasia.No drug interventionsNot AvailableNot Availableterminated
NCT01541306
C-Type Natriuretic Peptide and AchondroplasiaNo drug interventionsNot AvailableNot Availablecompleted
NCT00001536
Issues Surrounding Prenatal Genetic Testing for AchondroplasiaNo drug interventionsNot AvailableNot Availablecompleted
NCT03424018
An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasiatreatment3active_not_recruiting
NCT03875534
A Multi-center, Longitudinal, Observational Study of Children With AchondroplasiaNo drug interventionsNot AvailableNot Availablecompleted
NCT03197766
A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasiatreatment3completed
NCT01516229
Special Survey for Long Term ApplicationNot AvailableNot Availablecompleted
NCT01435629
A Survey Collecting Data on Adult Height in Patients With Achondroplasia Treated With SomatropinNot AvailableNot Availablecompleted
NCT04554940
A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasiatreatment2active_not_recruiting
NCT03449368
Lifetime Impact of Achondroplasia Study in Europe-LIAISENo drug interventionsNot AvailableNot Availablecompleted
NCT01590446
A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteerstreatment1completed
NCT05116046
Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasiatreatment2terminated
NCT05145010
Extension Study of Infigratinib in Children With Achondroplasia (ACH)treatment2enrolling_by_invitation
NCT02597881
Achondroplasia Natural History Multicenter Clinical StudyNo drug interventionsNot AvailableNot Availableenrolling_by_invitation
NCT05246033
A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Multiple Subcutaneous Doses of TransCon CNP Administered Once Weekly in Children With Achondroplasiatreatment2active_not_recruiting
NCT02724228
A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)treatment2active_not_recruiting
NCT05603936
Adaption and Testing of the Quality of Life in Short Stature Youth (QoLISSY) Questionnaire for Parents With Children From 0-4No drug interventionsNot AvailableNot Availablerecruiting
NCT04184817
Radiological Analysis on Patients With Achondroplasia DisorderNo drug interventionsNot AvailableNot Availableunknown_status
NCT02055157
A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasiatreatment2completed
NCT06433557
A Phase 2 Clinical Trial to Evaluate Efficacy, Safety, and Tolerability of Navepegritide in Combination With Lonapegsomatropin in Children With Achondroplasiatreatment2recruiting
NCT03872531
Lifetime Impact Study for AchondroplasiaNo drug interventionsNot AvailableNot Availablecompleted
NCT05353192
A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children With Achondroplasiatreatment4recruiting
NCT06168201
A Participant-mediated Observational Virtual Registry of Children With Achondroplasia in the United StatesNo drug interventionsNot AvailableNot Availablerecruiting
NCT01603095
A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With AchondroplasiaNo drug interventionsNot AvailableNot Availablecompleted
NCT05813314
Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participantstreatment1terminated
NCT05328050
Registry for Patients With Achondroplasia / Hypochondroplasia (OMPR-Ach/Hy)No drug interventionsNot AvailableNot Availablerecruiting
NCT04085523
A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasiatreatment2active_not_recruiting