Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04085523




Registration number
NCT04085523
Ethics application status
Date submitted
9/09/2019
Date registered
11/09/2019
Date last updated
7/03/2024

Titles & IDs
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
Secondary ID [1] 0 0
TCC-201
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Achondroplasia 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - TransCon CNP
Treatment: Drugs - Placebo for TransCon CNP

Experimental: TransCon CNP 6 mcg - TransCon CNP 6 mcg CNP/kg delivered once weekly by subcutaneous injection.

Experimental: TransCon CNP 20 mcg - TransCon CNP 20 mcg CNP/kg delivered once weekly by subcutaneous injection.

Experimental: TransCon CNP 50 mcg - TransCon CNP 50 mcg CNP/kg delivered once weekly by subcutaneous injection.

Experimental: TransCon CNP 100 mcg - TransCon CNP 100 mcg CNP/kg delivered once weekly by subcutaneous injection.

Placebo comparator: Placebo - Placebo mimicking 6, 20, 50, or 100 mcg CNP/kg delivered once weekly by subcutaneous injection.

Experimental: Open-Label Extension Period - Subjects who complete the 52-week blinded treatment period can continue into the 104-week open-label extension period and will receive treatment with TransCon CNP.


Treatment: Drugs: 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.

Treatment: Drugs: Placebo for TransCon CNP
Weekly subcutaneously injection of placebo.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Annualized Height Velocity (cm/Year) After 52 Weeks of Double-blind Treatment
Timepoint [1] 0 0
52 weeks

Eligibility
Key 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
Minimum age
2 Years
Maximum age
10 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
24/06/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/09/2024
Actual
Sample size
Target
Accrual to date
Final
57
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Ascendis Pharma Investigational Site - Parkville
Recruitment postcode(s) [1] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arkansas
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
Minnesota
Country [4] 0 0
United States of America
State/province [4] 0 0
Missouri
Country [5] 0 0
United States of America
State/province [5] 0 0
New York
Country [6] 0 0
United States of America
State/province [6] 0 0
Texas
Country [7] 0 0
United States of America
State/province [7] 0 0
Washington
Country [8] 0 0
United States of America
State/province [8] 0 0
Wisconsin
Country [9] 0 0
Austria
State/province [9] 0 0
Linz
Country [10] 0 0
Denmark
State/province [10] 0 0
Copenhagen
Country [11] 0 0
Germany
State/province [11] 0 0
Berlin
Country [12] 0 0
Ireland
State/province [12] 0 0
Dublin
Country [13] 0 0
New Zealand
State/province [13] 0 0
Auckland
Country [14] 0 0
Portugal
State/province [14] 0 0
Coimbra

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Ascendis Pharma A/S
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Aimee D. Shu, MD
Address 0 0
Ascendis Pharma, Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?




Results publications and other study-related documents

TypeCitations or Other Details
Journal Savarirayan R, Hoernschemeyer DG, Ljungberg M, Zar... [More Details]

Results not provided in https://clinicaltrials.gov/study/NCT04085523