A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide in Adolescents (12 - 18 Years of Age) With Achondroplasia.

NCT06732895 | Recruiting Phase 2 FDA Drug

• 2 other identifiers: ASND00452024-514208-15-00
• Study Type: interventional
• Target: 24
• Locations: 5 countries
• Sites: 5

Brief Summary

The purpose of this clinical trial is to evaluate efficacy and safety of once weekly subcutaneous (SC) doses of navepegritide 100 μg/kg compared to placebo (inactive drug) in adolescents aged 12 to 18 years with Achondroplasia. What will be measured is Annualized Growth Velocity after a 52-week treatment period.

Conditions

Achondroplasia

Outcome Measures

Primary Outcomes (1)

• Annualized Growth Velocity

  cm per year

  52 weeks

Secondary Outcomes (1)

• Height Z-score

  52 weeks

Study Arms (2)

Navepegritide

EXPERIMENTAL

Once weekly double-blinded treatment with SC injection of 100 µg/kg of navepegritide for 52 weeks

Drug: Navepegritide

Placebo for Navepegritide

PLACEBO COMPARATOR

Once weekly double-blinded treatment with SC injection of 100 µg/kg of placebo for navepegritide for 52 weeks

Drug: Placebo for navepegritide

Interventions

Navepegritide DRUG

Once-weekly subcutaneous injection of 100 µg/kg navepegritide for 52 weeks

Navepegritide

Placebo for navepegritide DRUG

Once-weekly subcutaneous injection of 100 µg/kg placebo for navepegritide

Placebo for Navepegritide

Eligibility Criteria

• Age: 12 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Written, signed informed consent and/or assent of the participant, participant parent(s) or legal guardian(s) of the participant, and as required by the institutional review board/human research ethics committee/independent ethics committee (IRB/HREC/IEC). For participants who are below the age of consent, a written assent will be obtained in accordance with applicable requirements as required by IRB/HREC/IEC. Upon reaching the legal age of consent, depending on applicable requirements, these participants will be asked to give their own written consent.
• Male or female, between 12 (inclusive) and 18 years of age at the time of randomization
• Clinical diagnosis of ACH with documented genetic confirmation available. Documentation of historic test results are acceptable for proof of diagnosis.
• Parent(s)/legal guardian(s) willing and able to administer weekly SC injections of IMP and to follow the protocol.
• At least one historical standing height measurement available from medical records. The measurement must have been collected between 6 months to 15 months prior to the time of screening.

You may not qualify if:

• Participation (signed informed consent) in any interventional clinical trial within 3 months prior to Screening unless no doses of IMP was given.
• Decreased growth velocity (AGV less than 1.5 cm/year based on measurement over a period of at least 6 months) or radiological evidence of growth plate closure.
• Known or suspected hypersensitivity to the IMP or related products (trehalose, tris\[hydroxymethyl\]aminomethane, succinate, and mPEG).
• Have a growth disorder or medical condition other than ACH that results in short stature, or abnormal growth such as SADDAN, hypochondroplasia, growth hormone deficiency, Turner syndrome, pseudo-ACH, inflammatory bowel disease, celiac disease, hypothyroidism, hyperthyroidism, or diabetes mellitus.
• Severe mutation in the FGFR3 gene, e.g. two variants on the same allele or severe ACH with developmental delay and acanthosis nigricans, are not eligible for trial participation.
• Have received any dose of prescription medications and/or IMP (placebo treatment only is allowed, if documented) or surgical intervention intended to affect stature, growth, or body proportionality at any time.
• Requires, or anticipated to require, chronic (more than 4 weeks) or repeated treatment (more than twice/year and less than 3 weeks/year) with systemic corticosteroids during participation in the trial. Chronic use of high dose inhaled corticosteroids is not allowed.
• Known history of presence of injury or disease of the growth plate(s), other than ACH, that affects growth potential of long bones.
• Known history of any bone-related surgery affecting growth potential of long bones, such as:
• Ventriculoperitoneal (VP) shunt and laminectomy with full recovery are allowed with minimum of 6 months of bone healing.
• Bone fracture within 6 months prior to screening (within 2 months for fracture of digits and buckle fractures).
• Clinically significant findings at Screening, such as:
• Expected to require surgical intervention during participation in the trial that may significantly affect trial parameters (confounding of safety events) or would prevent the participant from performing trial procedures. Common surgeries, such as insertion of grommets, adenoidectomy, tonsillectomy, or myringotomy tube placement, are permitted.
• Severe untreated sleep apnea or newly initiated sleep apnea treatment (e.g., Continuous Positive Airway Pressure \[CPAP\] in the previous 2 months prior to Screening.
• MS disease, such as Salter-Harris fractures or clinical and/or radiographic evidence of severe hip pathology

Sponsors & Collaborators

• Ascendis Pharma A/S: lead

Study Sites (5)

Ascendis Investigational Site

Montreal, H3T 1C5, Canada

RECRUITING

Ascendis Investigational Site

Copenhagen, 2100, Denmark

RECRUITING

Ascendis Investigational Site

Paris, 75743, France

RECRUITING

Ascendis Investigational Site

Dublin, D01 YC76, Ireland

RECRUITING

Ascendis Investigational Site

Norwich, NR4 7UY, United Kingdom

RECRUITING

MeSH Terms

Conditions

Achondroplasia

Condition Hierarchy (Ancestors)

Dwarfism; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Osteochondrodysplasias; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Medical Director, MD

  Ascendis Pharma A/S

  STUDY DIRECTOR

Central Study Contacts

Ascendis Registry Inquiries

CONTACT

+4561242484; asnd_registryinquiries@ascendispharma.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 10, 2024
• First Posted: December 13, 2024
• Study Start: December 10, 2024
• Primary Completion (Estimated): August 1, 2027
• Study Completion (Estimated): September 1, 2027
• Last Updated: April 21, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

DK (1); FR (1); IE (1); GB (1); CA (1)