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The trial is a multicenter, double-blind, randomized, placebo-controlled, dose escalation trial of weekly TransCon CNP administered subcutaneously in prepubertal children 2 to 10 years old, inclusive, with Achondroplasia.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| TransCon CNP 6 mcg | Experimental | TransCon CNP 6 mcg CNP/kg delivered once weekly by subcutaneous injection. |
|
| TransCon CNP 20 mcg | Experimental | TransCon CNP 20 mcg CNP/kg delivered once weekly by subcutaneous injection. |
|
| TransCon CNP 50 mcg | Experimental | TransCon CNP 50 mcg CNP/kg delivered once weekly by subcutaneous injection. |
|
| TransCon CNP 100 mcg | Experimental | TransCon CNP 100 mcg CNP/kg delivered once weekly by subcutaneous injection. |
|
| Placebo | Placebo Comparator | Placebo mimicking 6, 20, 50, or 100 mcg CNP/kg delivered once weekly by subcutaneous injection. |
|
| Open-Label Extension Period: TransCon CNP | Experimental |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| TransCon CNP | Drug | TransCon CNP drug product is a lyophilized powder in a single-use vial containing either TransCon CNP 3.9 mg CNP-38/vial or TransCon CNP 0.80 mg CNP-38/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 |
|---|---|---|
| Annualized Height Velocity (cm/Year) After 52 Weeks of Double-blind Treatment | The primary efficacy analysis compared the difference in the primary efficacy endpoint between the TransCon CNP treatment group and the pooled placebo group using an ANCOVA model with the annualized height velocity (AHV) at Week 52 as the response variable, treatment (dose groups and placebo) and sex as factors, baseline age and baseline height SDS as the covariates, and based on the Full Analysis Set. | 52 weeks |
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Inclusion Criteria:
Exclusion Criteria:
Clinically significant findings at Screening that:
Have received treatment (>3 months) of human growth hormone (hGH) or other medications known to affect stature or body proportionality at any time
Have received any dose of medications intended to affect stature or body proportionality within the previous 6 months of Screening Visit
Have received any study drug or device intended to affect stature or body proportionality at any time
History or presence of injury or disease of the growth plate(s), other than Achondroplasia, that affects growth potential of long bones
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| Name | Affiliation | Role |
|---|---|---|
| Study Director, MD | Ascendis Pharma, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Ascendis Pharma Investigational Site | Little Rock | Arkansas | 72211 | United States | ||
| Ascendis Pharma Investigational Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37823031 | Result | Savarirayan R, Hoernschemeyer DG, Ljungberg M, Zarate YA, Bacino CA, Bober MB, Legare JM, Hogler W, Quattrin T, Abuzzahab MJ, Hofman PL, White KK, Ma NS, Schnabel D, Sousa SB, Mao M, Smith A, Chakraborty M, Giwa A, Winding B, Volck B, Shu AD, McDonnell C. Once-weekly TransCon CNP (navepegritide) in children with achondroplasia (ACcomplisH): a phase 2, multicentre, randomised, double-blind, placebo-controlled, dose-escalation trial. EClinicalMedicine. 2023 Oct 2;65:102258. doi: 10.1016/j.eclinm.2023.102258. eCollection 2023 Nov. | |
| 42319555 |
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A total of 60 participants were screened and 57 participants met eligibility criteria and were enrolled into the trial and randomized in the planned ratio of approximately 3:1 (TransCon CNP: Placebo) within sequential dose escalation cohorts. All 57 participants completed the 52-week double-blind period & entered the 104-week open-label extension (OLE) period, where they received TransCon CNP. All participants received up to a maximum of 100-mcg dose of TransCon CNP during the OLE period.
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| ID | Title | Description |
|---|---|---|
| FG000 | TransCon CNP 6 mcg | TransCon CNP 6 mcg CNP/kg delivered once weekly by subcutaneous injection. TransCon CNP: TransCon CNP drug product is a lyophilized powder in a single-use vial containing either TransCon CNP 3.9 mg CNP-38/vial or TransCon CNP 0.80 mg CNP-38/vial. Prior to use, the lyophilized powder is reconstituted with sterile water for injection and administered by subcutaneous injection via syringe and needle. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Double-Blind Period (Weeks 0 to 52) |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Dec 28, 2022 | Dec 27, 2023 |
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There are 5 cohorts enrolling approximately 60 subjects who will be randomized to receive either TransCon CNP or Placebo in a 3:1 ratio
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Participants who completed the 52-week blinded treatment period continued into the 104-week open-label extension period and received treatment with TransCon CNP (navepegritide) doses escalated up to a maximum of 100 mcg/kg delivered once weekly by subcutaneous injection.
|
|
| Placebo for TransCon CNP | Drug | Weekly subcutaneously injection of placebo. |
|
| Aurora |
| Colorado |
| 80045 |
| United States |
| Ascendis Pharma Investigational Site | Saint Paul | Minnesota | 55102 | United States |
| Ascendis Pharma Investigational Site | Columbia | Missouri | 65212 | United States |
| Ascendis Pharma Investigational Site | Buffalo | New York | 14203 | United States |
| Ascendis Pharma Investigational Site | Houston | Texas | 77030 | United States |
| Ascendis Pharma Investigational Site | Seattle | Washington | 98105 | United States |
| Ascendis Pharma Investigational Site | Madison | Wisconsin | 53705 | United States |
| Ascendis Pharma Investigational Site | Parkville | Victoria | 3052 | Australia |
| Ascendis Pharma Investigational Site | Linz | 4020 | Austria |
| Ascendis Pharma Investigational Site | Copenhagen | 2100 | Denmark |
| Ascendis Pharma Investigational Site | Berlin | 13353 | Germany |
| Ascendis Pharma Investigational Site | Dublin | D01 YC76 | Ireland |
| Ascendis Pharma Investigational Site | Auckland | 1023 | New Zealand |
| Ascendis Pharma Investigational Site | Coimbra | 3000-602 | Portugal |
| Derived |
| Brod M, McLeod L, Smith A. Assessing signs and impacts of achondroplasia: psychometric evaluation of the Achondroplasia Child Experience Measures. J Patient Rep Outcomes. 2026 Jun 19. doi: 10.1186/s41687-026-01123-z. Online ahead of print. |
| FG001 | TransCon CNP 20 mcg | TransCon CNP 20 mcg CNP/kg delivered once weekly by subcutaneous injection. TransCon CNP: TransCon CNP drug product is a lyophilized powder in a single-use vial containing either TransCon CNP 3.9 mg CNP-38/vial or TransCon CNP 0.80 mg CNP-38/vial. Prior to use, the lyophilized powder is reconstituted with sterile water for injection and administered by subcutaneous injection via syringe and needle. |
| FG002 | TransCon CNP 50 mcg | TransCon CNP 50 mcg CNP/kg delivered once weekly by subcutaneous injection. TransCon CNP: TransCon CNP drug product is a lyophilized powder in a single-use vial containing either TransCon CNP 3.9 mg CNP-38/vial or TransCon CNP 0.80 mg CNP-38/vial. Prior to use, the lyophilized powder is reconstituted with sterile water for injection and administered by subcutaneous injection via syringe and needle. |
| FG003 | TransCon CNP 100 mcg | TransCon CNP 100 mcg CNP/kg delivered once weekly by subcutaneous injection. TransCon CNP: TransCon CNP drug product is a lyophilized powder in a single-use vial containing either TransCon CNP 3.9 mg CNP-38/vial or TransCon CNP 0.80 mg CNP-38/vial. Prior to use, the lyophilized powder is reconstituted with sterile water for injection and administered by subcutaneous injection via syringe and needle. |
| FG004 | Placebo | Placebo mimicking 6, 20, 50, or 100 mcg CNP/kg delivered once weekly by subcutaneous injection. Placebo for TransCon CNP: Weekly subcutaneously injection of placebo. |
| FG005 | Open-Label Extension Period: TransCon CNP | Participants who completed the 52-week blinded treatment period continued into the 104-week open-label extension period and received treatment with TransCon CNP (navepegritide) doses escalated up to a maximum of 100 mcg/kg delivered once weekly by subcutaneous injection. TransCon CNP: TransCon CNP drug product is a lyophilized powder in a single-use vial containing either TransCon CNP 3.9 mg CNP-38/vial or TransCon CNP 0.80 mg CNP-38/vial. Prior to use, the lyophilized powder is reconstituted with sterile water for injection and administered by subcutaneous injection via syringe and needle. |
| COMPLETED |
|
| NOT COMPLETED |
|
| Open-Label Period (Weeks 52 to 156) |
|
|
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | TransCon CNP 6 mcg | TransCon CNP 6 mcg CNP/kg delivered once weekly by subcutaneous injection. TransCon CNP: TransCon CNP drug product is a lyophilized powder in a single-use vial containing either TransCon CNP 3.9 mg CNP-38/vial or TransCon CNP 0.80 mg CNP-38/vial. Prior to use, the lyophilized powder is reconstituted with sterile water for injection and administered by subcutaneous injection via syringe and needle. |
| BG001 | TransCon CNP 20 mcg | TransCon CNP 20 mcg CNP/kg delivered once weekly by subcutaneous injection. TransCon CNP: TransCon CNP drug product is a lyophilized powder in a single-use vial containing either TransCon CNP 3.9 mg CNP-38/vial or TransCon CNP 0.80 mg CNP-38/vial. Prior to use, the lyophilized powder is reconstituted with sterile water for injection and administered by subcutaneous injection via syringe and needle. |
| BG002 | TransCon CNP 50 mcg | TransCon CNP 50 mcg CNP/kg delivered once weekly by subcutaneous injection. TransCon CNP: TransCon CNP drug product is a lyophilized powder in a single-use vial containing either TransCon CNP 3.9 mg CNP-38/vial or TransCon CNP 0.80 mg CNP-38/vial. Prior to use, the lyophilized powder is reconstituted with sterile water for injection and administered by subcutaneous injection via syringe and needle. |
| BG003 | TransCon CNP 100 mcg | TransCon CNP 100 mcg CNP/kg delivered once weekly by subcutaneous injection. TransCon CNP: TransCon CNP drug product is a lyophilized powder in a single-use vial containing either TransCon CNP 3.9 mg CNP-38/vial or TransCon CNP 0.80 mg CNP-38/vial. Prior to use, the lyophilized powder is reconstituted with sterile water for injection and administered by subcutaneous injection via syringe and needle. |
| BG004 | Placebo | Placebo mimicking 6, 20, 50, or 100 mcg CNP/kg delivered once weekly by subcutaneous injection. Placebo for TransCon CNP: Weekly subcutaneously injection of placebo. |
| BG005 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Height | Mean | Standard Deviation | cm |
| |||||||||||||||
| Height SDS | Height expressed in Standard Deviation Score (SDS) is derived using CDC 2000 (USA)/Kuczmarski method. A Height SDS of 0 represents the population mean or average. A lower SDS means the value is closer to the average and a higher SDS means the value is further from the average. A negative SDS indicates below average height and a positive SDS indicates above average height. | Mean | Standard Deviation | standard deviation score |
| ||||||||||||||
| Weight | Mean | Standard Deviation | kg |
| |||||||||||||||
| Body Mass Index (BMI) | Mean | Standard Deviation | kg/m^2 |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Annualized Height Velocity (cm/Year) After 52 Weeks of Double-blind Treatment | The primary efficacy analysis compared the difference in the primary efficacy endpoint between the TransCon CNP treatment group and the pooled placebo group using an ANCOVA model with the annualized height velocity (AHV) at Week 52 as the response variable, treatment (dose groups and placebo) and sex as factors, baseline age and baseline height SDS as the covariates, and based on the Full Analysis Set. | Posted | Least Squares Mean | 95% Confidence Interval | cm/year | 52 weeks |
|
|
|
|
From Week 0 to Week 52 for double-blind treatment period and up to Week 156 for OLE Period
Analysis was performed on safety analysis set that included all randomized participants who received at least one dose of trial drug. Participants were analyzed according to trial treatment as treated. Adverse Events were reported as per MedDRA version 24.1 for double-blind treatment period and MedDRA version 26.0 for OLE period.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | TransCon CNP 6 mcg | TransCon CNP 6 mcg CNP/kg delivered once weekly by subcutaneous injection. TransCon CNP: TransCon CNP drug product is a lyophilized powder in a single-use vial containing either TransCon CNP 3.9 mg CNP-38/vial or TransCon CNP 0.80 mg CNP-38/vial. Prior to use, the lyophilized powder is reconstituted with sterile water for injection and administered by subcutaneous injection via syringe and needle. | 0 | 10 | 1 | 10 | 9 | 10 |
| EG001 | TransCon CNP 20 mcg | TransCon CNP 20 mcg CNP/kg delivered once weekly by subcutaneous injection. TransCon CNP: TransCon CNP drug product is a lyophilized powder in a single-use vial containing either TransCon CNP 3.9 mg CNP-38/vial or TransCon CNP 0.80 mg CNP-38/vial. Prior to use, the lyophilized powder is reconstituted with sterile water for injection and administered by subcutaneous injection via syringe and needle. | 0 | 11 | 0 | 11 | 11 | 11 |
| EG002 | TransCon CNP 50 mcg | TransCon CNP 50 mcg CNP/kg delivered once weekly by subcutaneous injection. TransCon CNP: TransCon CNP drug product is a lyophilized powder in a single-use vial containing either TransCon CNP 3.9 mg CNP-38/vial or TransCon CNP 0.80 mg CNP-38/vial. Prior to use, the lyophilized powder is reconstituted with sterile water for injection and administered by subcutaneous injection via syringe and needle. | 0 | 10 | 1 | 10 | 10 | 10 |
| EG003 | TransCon CNP 100 mcg | TransCon CNP 100 mcg CNP/kg delivered once weekly by subcutaneous injection. TransCon CNP: TransCon CNP drug product is a lyophilized powder in a single-use vial containing either TransCon CNP 3.9 mg CNP-38/vial or TransCon CNP 0.80 mg CNP-38/vial. Prior to use, the lyophilized powder is reconstituted with sterile water for injection and administered by subcutaneous injection via syringe and needle. | 0 | 11 | 0 | 11 | 10 | 11 |
| EG004 | Placebo | Placebo mimicking 6, 20, 50, or 100 mcg CNP/kg delivered once weekly by subcutaneous injection. Placebo for TransCon CNP: Weekly subcutaneously injection of placebo. | 0 | 15 | 0 | 15 | 14 | 15 |
| EG005 | Open-Label Extension Period: TransCon CNP | Participants who completed the 52-week blinded treatment period continued into the 104-week open-label extension period and received treatment with TransCon CNP (navepegritide) doses escalated up to a maximum of 100 mcg/kg delivered once weekly by subcutaneous injection. TransCon CNP: TransCon CNP drug product is a lyophilized powder in a single-use vial containing either TransCon CNP 3.9 mg CNP-38/vial or TransCon CNP 0.80 mg CNP-38/vial. Prior to use, the lyophilized powder is reconstituted with sterile water for injection and administered by subcutaneous injection via syringe and needle. | 0 | 57 | 2 | 57 | 57 | 57 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Febrile Convulsion | Nervous system disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Obstructive sleep apnoea syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Tonsillar hypertrophy | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Headache | Nervous system disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Injection Site Reaction | General disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Ear Pain | Ear and labyrinth disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Abdominal Pain Upper | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Snoring | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
| |
| Otitis media | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
| |
| Vitamin D deficiency | Metabolism and nutrition disorders | MedDRA 24.1 | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Otitis media acute | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Pharyngitis streptococcal | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Conjunctivitis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Obstructive sleep apnoea syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Sleep apnoea syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Tonsillar hypertrophy | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Injection site pain | General disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Injection site swelling | General disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Knee deformity | Musculoskeletal and connective tissue disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Hypoacusis | Ear and labyrinth disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Middle ear effusion | Ear and labyrinth disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Lymphadenopathy | Blood and lymphatic system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Skin abrasion | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Seasonal allergy | Immune system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Vitamin D decreased | Investigations | MedDRA 26.0 | Systematic Assessment |
|
Not provided
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Ascendis Pharma | +45 61161658 | Asnd_registryinquiries@ascendispharma.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Oct 11, 2022 | Dec 27, 2023 | SAP_001.pdf |
Not provided
| 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 |
Not provided
Not provided
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| ANCOVA |
| = 0.7022 |
| Superiority |
| ANCOVA model using treatment (dose groups and pooled placebo) and sex as fixed effects, and baseline age and baseline height SDS as the covariates. | ANCOVA | = 0.0849 | Superiority |
| ANCOVA model using treatment (dose groups and pooled placebo) and sex as fixed effects, and baseline age and baseline height SDS as the covariates. | ANCOVA | = 0.0218 | Superiority |