|
Main
|
|
Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
|
Register:
|
ClinicalTrials.gov |
|
Last refreshed on:
|
20 November 2023 |
|
Main ID: |
NCT04085523 |
|
Date of registration:
|
09/09/2019 |
|
Prospective Registration:
|
Yes |
|
Primary sponsor: |
|
|
Public title:
|
A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia
|
|
Scientific title:
|
ACcomplisH: A Phase 2, Multicenter, Double-blind, Randomized, Placebo-controlled, Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Subcutaneous Doses of TransCon CNP Administered Once Weekly for 52 Weeks in Prepubertal Children With Achondroplasia Followed by an Open-Label Extension Period |
|
Date of first enrolment:
|
June 24, 2020 |
|
Target sample size:
|
57 |
|
Recruitment status: |
Active, not recruiting |
|
URL:
|
https://clinicaltrials.gov/ct2/show/NCT04085523 |
|
Study type:
|
Interventional |
|
Study design:
|
Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
|
|
Phase:
|
Phase 2
|
|
|
Countries of recruitment
|
|
Australia
|
Austria
|
Denmark
|
Germany
|
Ireland
|
New Zealand
|
Portugal
|
United States
|
|
Contacts
|
|
Name:
|
Silas Gylvin, MD |
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
Ascendis Pharma |
| | |
|
Key inclusion & exclusion criteria
|
Inclusion Criteria:
1. Clinical diagnosis of ACH with genetic confirmation
2. Age between 2 to 10 years old (inclusive) at Screening Visit
3. Prepubertal (Stage 1 breasts for girls or testicular volume < 4ml for boys) at
Screening Visit
4. Able to stand without assistance
5. Caregiver willing and able to administer subcutaneous injections of study drug
Exclusion Criteria:
1. Clinically significant findings at Screening that:
- are expected to require surgical intervention during participation in the trial
or
- are musculoskeletal in nature, such as Salter-Harris fractures and severe hip
pain or
- otherwise are considered by investigator or Medical Monitor/Medical Expert to
make a participant unfit to receive study drug or undergo trial related
procedures
2. Have received treatment (>3 months) of human growth hormone (hGH) or other medications
known to affect stature or body proportionality at any time
3. Have received any dose of medications intended to affect stature or body
proportionality within the previous 6 months of Screening Visit
4. Have received any study drug or device intended to affect stature or body
proportionality at any time
5. History or presence of injury or disease of the growth plate(s), other than
Achondroplasia, that affects growth potential of long bones
Age minimum:
2 Years
Age maximum:
10 Years
Gender:
All
|
|
Health Condition(s) or Problem(s) studied
|
|
Achondroplasia
|
|
Intervention(s)
|
|
Drug: Placebo for TransCon CNP
|
|
Drug: TransCon CNP
|
|
Primary Outcome(s)
|
|
Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability)
[Time Frame: 52 weeks]
|
|
Annualized height velocity (centimeters/year) at 52 weeks
[Time Frame: 52 weeks]
|
|
Source(s) of Monetary Support
|
|
Please refer to primary and secondary sponsors
|
|
Results
|
|
Results available:
|
|
|
Date Posted:
|
|
|
Date Completed:
|
|
|
URL:
|
|
|
|