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TransCon CNP administered once-weekly in children and adolescents with achondroplasia who have completed a prior TransCon CNP clinical trial. Participants who complete a prior TransCon CNP trial and meet all eligibility criteria will be invited to continue into the long-term open label extension trial to receive 100 µg CNP/kg/week of TransCon CNP. Trial treatment will be completed when the participant reaches 16 years of age for females and 18 years of age for males and have femur and tibial epiphyseal closure. TransCon CNP treatment will continue if femur and tibial epiphyseal closure is not confirmed at the age of 16 years for females, and 18 years for males. Treatment with TransCon CNP will be completed once femur and tibial epiphyseal closure is confirmed by radiographic imaging. The trial duration is individual for each trial participant. Visits will occur every 12-14 weeks throughout the trial.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| TransCon CNP 100 mcg | Experimental | TransCon CNP 100 mcg delivered once weekly by subcutaneous injection |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| TransCon CNP | Drug | TransCon CNP drug product is a lyophilized powder in a single-use vial. Prior to use, the lyophilized powder is reconstituted with sterile water for injection and administered by subcutaneous injection via syringe and needle. |
| Measure | Description | Time Frame |
|---|---|---|
| Safety and Tolerability | Incidence of Treatment-Emergent Adverse Events | Through trial completion, an average of 10 years |
| Height Z-scores | Number of standard deviations | Through trial completion, an average of 10 years |
| Measure | Description | Time Frame |
|---|---|---|
| Annualized Growth Velocity | cm per year | Through trial completion, an average of 10 years |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Claus Strange | Ascendis Pharma A/S | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Ascendis Pharma Investigational Site | Little Rock | Arkansas | 72202 | United States | ||
| Ascendis Investigational Site |
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Patients will roll over from previous TransCON CNP clinical trials
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| Aurora |
| Colorado |
| 80045 |
| United States |
| Ascendis Investigational Site | Wilmington | Delaware | 19803 | United States |
| Ascendis Investigational Site | Saint Paul | Minnesota | 55102 | United States |
| Ascendis Pharma Investigational Site | Columbia Falls | Montana | 65212 | United States |
| Ascendis Pharma Investigational Site | Buffalo | New York | 14203 | United States |
| Ascendis Investigational Site | Houston | Texas | 77030 | United States |
| Ascendis Pharma Investigational Site | Madison | Wisconsin | 53705 | United States |
| Ascendis Pharma Investigational Site | Parkville | Victoria | 3052 | Australia |
| Ascendis Investigational Site | Linz | 4020 | Austria |
| Ascendis Investigational Site | Montreal | H3T 1C5 | Canada |
| Ascendis Pharma Investigational Site | Copenhagen | 2100 | Denmark |
| Ascendis Investigational Site | Berlin | 13353 | Germany |
| Ascendis Pharma Investigational Site | Dublin | D01 YC76 | Ireland |
| Ascendis Investigational Site | Auckland | 1023 | New Zealand |
| Ascendis Investigational Site | Coimbra | 3000-602 | Portugal |
| Ascendis Investigational Site | Vitoria-Gasteiz | 1008 | Spain |
| ID | Term |
|---|---|
| D000130 | Achondroplasia |
| D004392 | Dwarfism |
| ID | Term |
|---|---|
| D001848 | Bone Diseases, Developmental |
| D001847 | Bone Diseases |
| D009140 | Musculoskeletal Diseases |
| D010009 | Osteochondrodysplasias |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D004700 | Endocrine System Diseases |
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