A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia

• Sponsor: Ascendis Pharma A/S (Industry)
• Overall Status: Enrolling by invitation
• CT.gov ID: NCT04085523
• Collaborator: (none)
• Enrollment: 60
• Locations: 15
• Arms: 5
• Anticipated Duration (Months): 71.2
• Patients Per Site: 4
• Patients Per Site Per Month: 0.1

Study Details

Study Description

Brief Summary

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.

Condition or Disease	Intervention/Treatment	Phase
Achondroplasia	Drug: TransCon CNP; Drug: Placebo for TransCon CNP; Drug: sWfI Placebo for TransCon CNP	Phase 2

Study Design

• Study Type: Interventional
• Anticipated Enrollment: 60 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Intervention Model Description: There are 5 cohorts enrolling approximately 60 subjects who will be randomized to receive either TransCon CNP or Placebo in a 3:1 ratioThere are 5 cohorts enrolling approximately 60 subjects who will be randomized to receive either TransCon CNP or Placebo in a 3:1 ratio
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official 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
• Actual Study Start Date: Jun 24, 2020
• Anticipated Primary Completion Date: Jun 1, 2023
• Anticipated Study Completion Date: Jun 1, 2026

Arms and Interventions

Arm	Intervention/Treatment
Other: TransCon CNP 6 mcg TransCon CNP 6 mcg CNP/kg or placebo mimicking TransCon CNP 6 mcg delivered once weekly by subcutaneous injection	Drug: 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.; Drug: Placebo for TransCon CNP; Weekly subcutaneously injection of placebo.; Drug: sWfI Placebo for TransCon CNP; Weekly subcutaneously injection of placebo.
Other: TransCon CNP 20 mcg TransCon CNP 20 mcg CNP/kg or placebo mimicking TransCon CNP 20 mcg delivered once weekly by subcutaneous injection	Drug: 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.; Drug: Placebo for TransCon CNP; Weekly subcutaneously injection of placebo.
Other: TransCon CNP 50 mcg TransCon CNP 50 mcg CNP/kg or placebo mimicking TransCon CNP 50 mcg delivered once weekly by subcutaneous injection	Drug: 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.; Drug: Placebo for TransCon CNP; Weekly subcutaneously injection of placebo.
Other: TransCon CNP 100 mcg TransCon CNP 100 mcg CNP/kg or placebo mimicking TransCon CNP 100 mcg delivered once weekly by subcutaneous injection	Drug: 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.; Drug: Placebo for TransCon CNP; Weekly subcutaneously injection of placebo.
Other: TransCon CNP >100 mcg TransCon CNP >100 mcg CNP/kg delivered once weekly by subcutaneous injection (to be determined after completion of 100 mcg cohort)	Drug: 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.; Drug: Placebo for TransCon CNP; Weekly subcutaneously injection of placebo.

Outcome Measures

Primary Outcome Measures

1. Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability) [52 weeks]

   Safety and tolerability of once weekly TransCon CNP treatment or placebo

2. Annualized height velocity (centimeters/year) at 52 weeks [52 weeks]

   Annualized height velocity measured in centimeters over 52 weeks for TransCon CNP or placebo

Eligibility Criteria

Criteria

• Ages Eligible for Study: 2 Years to 10 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

1. Have received treatment (>3 months) of human growth hormone (hGH) or other medications known to affect stature or body proportionality at any time

2. Have received any dose of medications intended to affect stature or body proportionality within the previous 6 months of Screening Visit

3. Have received any study drug or device intended to affect stature or body proportionality at any time

4. History or presence of injury or disease of the growth plate(s), other than Achondroplasia, that affects growth potential of long bones

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Ascendis Pharma Investigational Site	Little Rock	Arkansas	United States	72211
2	Ascendis Pharma Investigational Site	Aurora	Colorado	United States	80045
3	Ascendis Pharma Investigational Site	Saint Paul	Minnesota	United States	55102
4	Ascendis Pharma Investigational Site	Columbia	Missouri	United States	65212
5	Ascendis Pharma Investigational Site	Buffalo	New York	United States	14203
6	Ascendis Pharma Investigational Site	Houston	Texas	United States	77030
7	Ascendis Pharma Investigational Site	Seattle	Washington	United States	98105
8	Ascendis Pharma Investigational Site	Madison	Wisconsin	United States	53705
9	Ascendis Pharma Investigational Site	Parkville	Victoria	Australia	3052
10	Ascendis Pharma Investigational Site	Linz		Austria	4020
11	Ascendis Pharma Investigational Site	Copenhagen		Denmark	2100
12	Ascendis Pharma Investigational Site	Berlin		Germany	13353
13	Ascendis Pharma Investigational Site	Dublin		Ireland	D01 YC76
14	Ascendis Pharma Investigational Site	Auckland		New Zealand	1023
15	Ascendis Pharma Investigational Site	Coimbra		Portugal	3000-602

Sponsors and Collaborators

• Ascendis Pharma A/S

Investigators

• Study Director: Adebola Giwa, MD, Ascendis Pharma

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Ascendis Pharma A/S
• ClinicalTrials.gov Identifier: NCT04085523
• Other Study ID Numbers: TCC-201
• First Posted: Sep 11, 2019
• Last Update Posted: Aug 1, 2022
• Last Verified: Jul 1, 2022

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Keywords provided by Ascendis Pharma A/S: Achondroplasia; Dwarfism
• Additional relevant MeSH terms: Achondroplasia

Study Results

No Results Posted as of Aug 1, 2022