AchondroplasiaPhase 2NCT06079398
A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia
A Phase 2 study testing Navepegritide in people with Achondroplasia, run by Ascendis Pharma A/S at 18 sites in 15 countries.
Trial summary
This trial is a Phase 2, multicenter, double-blind, randomized (ratio 2:1 TransCon CNP vs. placebo), placebo-controlled trial, designed to evaluate the safety, tolerability, and efficacy of 100 μg CNP/kg of Navepegritide (TransCon CNP) administered SC once-weekly for 52 weeks in infants with genetically verified… Read more about this study
- Who is it for?
- Ages 0 to 2 years
- Conditions the study is for
- Achondroplasia
- What is being tested?
- Navepegritide
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 72 participants
- Where
- 18 sitesin 15 countries
- Study dates
- 23 Jan 2024to Dec 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 to 2 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Written, signed informed consent by the parent(s)/caregiver(s) of the participant, and as required by the institutional review board/human research ethics committee/independent ethics committee (IRB/HREC/IEC).
- Male or female younger than 2 years of age at the time of randomization; or for open label sentinel participants, at the time of first administration of IMP.
- Clinical diagnosis of achondroplasia (ACH) with genetic confirmation of heterozygous genotype present during screening.
- Parent(s)/caregiver(s) willing to follow the protocol and instructions provided, including being able to administer weekly subcutaneous injections of trial treatment.
- Compliance to daily Vitamin D supplementation for infants aged 14 days to 1 year. All participants older than 1 year of age with serum 25-hydroxyvitamin D (25OHD) measured below lower limit of reference range at screening should start daily Vitamin D supplementation prior to randomization.
- Considered eligible based on the medical history, physical examination, and the results of vital signs, ECG, imaging, and clinical laboratory tests performed during the screening period.
Who cannot join
- Known or suspected hypersensitivity to the investigational product or related products (trehalose, tris[hydroxymethyl]aminomethane, succinate, and polyethylene glycol [PEG]).
- Genetic confirmation of ACH homozygous genotype.
- Premature birth with gestational age < 32 weeks.
- Premature birth with gestational age 32 to 37 weeks, unless time from birth is > 6 months at the time of screening and the child is in good nutritional status, defined as gain in body weight expected for age and diagnosis of ACH, as determined by the Investigator and confirmed with the Medical Monitor.
- Anticipated, as assessed by Investigator and confirmed with Medical Monitor, to undergo surgical intervention during trial participation, including cervicomedullary decompression. Evaluation of immediate risk of requiring cervicomedullary decompression surgery will rely on the following assessments:
- Physical examination (e.g., neurologic findings of clonus, opisthotonus, exaggerated reflexes, dilated facial veins)
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- Evidence of uncontrolled sleep apnea as confirmed by local standard of care assessment (e.g. polysomnography or simple sleep test) performed within 6 months prior to screening.
- MRI performed at screening indicating presence of severe cervicomedullary compression (CMC) or spinal cord damage. Presence of abnormal MRI T2 signal intensity at and immediately above and below the cervicomedullary junction should be considered high risk for requiring surgery and the participant is not eligible for trial participation.
- Common surgeries, such as insertion of grommets, adenoidectomy, tonsillectomy, or myringotomy tube placement are permitted during trial participation.
- Have a growth disorder or medical condition, other than ACH, resulting in short stature or abnormal growth as determined by the Investigator and confirmed with the Medical Monitor.
- Have received any dose of prescription medications and/or investigational medicinal product or device intended to affect stature, growth, or body proportionality (including human growth hormone or vosoritide) at any time.
- Requires or anticipated to require chronic (> 4 weeks) or repeated treatment (more than twice/year) with oral corticosteroids, or high-dose inhaled corticosteroids during trial participation.
- History or presence of injury or disease of the growth plate(s), other than ACH, affecting growth potential of long bones, including Salter-Harris fracture and recent bone-related surgery, as determined by Investigator and confirmed with the Medical Monitor.
- Have a clinically significant finding indicating abnormal cardiac function, including but not limited to:
- Repaired or unrepaired coarctation.
- Moderate or greater complexity congenital heart disease including tetralogy of Fallot, atrioventricular septal defects, truncus arteriosus, total anomalous pulmonary venous return, double outlet right ventricle, or single ventricle heart disease.
- QTcF ≥ 450 msec on screening 12-lead ECG.
- History or presence of a condition impacting hemodynamic stability (such as autonomic dysfunction and orthostatic intolerance).
- History or presence of the following:
- Chronic anemia.
- Chronic renal insufficiency.
- Chronic or recurrent illness that can affect hydration or volume status, including conditions associated with decreased nutritional intake or increased volume loss.
- History or presence of malignant disease.
- Any disease or condition that, in the opinion of the Investigator, may make the participant unlikely to fully complete the trial, not adhering to trial procedures, may confound interpretation of trial results, or may present undue risk from receiving trial treatment. This could include family situations, comorbid conditions, or medications that might impact safety or be considered confounding.
Trial locations and contacts
18 sites in 15 countries. Contact the study team or a site near you to ask about taking part.
18 sites in 15 countries
United States3 sites, 3 recruiting
- Recruiting
Ascendis Investigational Site
Houston, Texas, 77030
- Recruiting
Ascendis Investigational Site
Madison, Wisconsin, 53705
- Recruiting
Ascendis Investigational Site
Saint Paul, Minnesota, 55102
Germany2 sites, 2 recruiting
- Recruiting
Ascendis Investigational Site
Berlin, 13353
- Recruiting
Ascendis Investigational Site
Cologne, 50931
Australia1 site, 1 recruiting
- Recruiting
Ascendis Investigational Site
Parkville, 3052
Austria1 site, 1 recruiting
- Recruiting
Ascendis Investigational Site
Linz, 4020
Canada1 site, 1 recruiting
- Recruiting
Ascendis Investigational Site
Montreal, H3T 1C5
Denmark1 site, 1 recruiting
- Recruiting
Ascendis Investigational Site
Copenhagen, 2100
Finland1 site, 1 recruiting
- Recruiting
Ascendis Investigational Site
Helsinki, 00029
France1 site, 1 recruiting
- Recruiting
Ascendis Investigational Site
Paris, 75743
Ireland1 site, 1 recruiting
- Recruiting
Ascendis Investigational Site
Dublin, D01 YC76
New Zealand1 site, 1 recruiting
- Recruiting
Ascendis Investigational Site
Auckland, 1023
Portugal1 site, 1 recruiting
- Recruiting
Ascendis Investigational Site
Coimbra, 3000-602
Sweden1 site, 1 recruiting
- Recruiting
Ascendis Investigational Site
Stockholm, 17176
United Kingdom1 site, 1 recruiting
- Recruiting
Ascendis Investigational Site
London, SE1 7EH
About this study
Official title: A Phase 2, Multicenter, Double-Blind, Randomized, Placebo-controlled Trial, Evaluating Safety, Tolerability, and Efficacy of Subcutaneous Doses of TransCon CNP Administered Once Weekly for 52 Weeks in Infants (0 to <2 Years of Age) With Achondroplasia Followed by an Open Label Extension (OLE) Period
This trial is a Phase 2, multicenter, double-blind, randomized (ratio 2:1 TransCon CNP vs. placebo), placebo-controlled trial, designed to evaluate the safety, tolerability, and efficacy of 100 μg CNP/kg of Navepegritide (TransCon CNP) administered SC once-weekly for 52 weeks in infants with genetically verified heterozygous ACH, aged 0 to < 2 years at the time of randomization.
- Conditions
- Achondroplasia
- Being tested
- Navepegritide, Placebo for Navepegritide
- Medical subject headings
- Achondroplasia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NavepegritideDrug
Once-weekly subcutaneous injection of 100 µg/kg Navepegritide
Placebo for NavepegritideDrug
Once-weekly subcutaneous injection of 100 µg/kg placebo for Navepegritide
| Study group | Role | What they receive |
|---|---|---|
| Navepegritide | Experimental | Once weekly double-blinded treatment with SC injection of 100 µg/kg of Navepegritide for 52 weeks Receives: 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 Receives: Drug: Placebo for Navepegritide |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the safety and tolerability of Navepegritide
Incidence of treatment emergent adverse events (TEAEs)
Measured: 52 weeks
To evaluate the effect of Navepegritide on growth
Change from baseline to 52 weeks in length/height Z-score
Measured: 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Oct 2023
- Study start23 Jan 2024
- Primary completionDec 2027 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AchondroplasiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 72 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ascendis Pharma A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, MD, Study director, Ascendis Pharma A/S
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06079398
- Sponsor's protocol ID
- ASND0030
- Status last verified
- Apr 2026
- First submitted
- 6 Oct 2023
- Last updated
- 8 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Multicenter, Double-Blind, Randomized, Placebo-controlled Trial, Evaluating Safety, Tolerability, and Efficacy of Subcutaneous Doses of TransCon CNP Administered Once Weekly for 52 Weeks in Infants (0 to <2 Years of Age) With Achondroplasia Followed by an Open Label Extension (OLE) Period
- ClinicalTrials.gov ID
- NCT06079398
- Acronym / sponsor ID
- ASND0030
- Conditions
- Achondroplasia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 72 participants (estimated)
- Lead sponsor
- Ascendis Pharma A/SIndustry (pharma, biotech, devices)
- Study start
- 23 Jan 2024 (actual)
- Primary completion
- Dec 2027 (estimated)
- Study completion
- Dec 2028 (estimated)
- First posted
- 12 Oct 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This trial is a Phase 2, multicenter, double-blind, randomized (ratio 2:1 TransCon CNP vs. placebo), placebo-controlled trial, designed to evaluate the safety, tolerability, and efficacy of 100 μg CNP/kg of Navepegritide (TransCon CNP) administered SC once-weekly for 52 weeks in infants with genetically verified heterozygous ACH, aged 0 to < 2 years at the time of randomization.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Navepegritide | Experimental | Drug: NavepegritideOnce weekly double-blinded treatment with SC injection of 100 µg/kg of Navepegritide for 52 weeks |
| Placebo for Navepegritide | Placebo comparator | Drug: Placebo for NavepegritideOnce weekly double-blinded treatment with SC injection of 100 µg/kg of Placebo for Navepegritide for 52 weeks |
Who may qualify
- Ages
- 0 to 2 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Written, signed informed consent by the parent(s)/caregiver(s) of the participant, and as required by the institutional review board/human research ethics committee/independent ethics committee (IRB/HREC/IEC).
- Male or female younger than 2 years of age at the time of randomization; or for open label sentinel participants, at the time of first administration of IMP.
- Clinical diagnosis of achondroplasia (ACH) with genetic confirmation of heterozygous genotype present during screening.
- Parent(s)/caregiver(s) willing to follow the protocol and instructions provided, including being able to administer weekly subcutaneous injections of trial treatment.
- Compliance to daily Vitamin D supplementation for infants aged 14 days to 1 year. All participants older than 1 year of age with serum 25-hydroxyvitamin D (25OHD) measured below lower limit of reference range at screening should start daily Vitamin D supplementation prior to randomization.
Key exclusion criteria (24 listed)
- Known or suspected hypersensitivity to the investigational product or related products (trehalose, tris[hydroxymethyl]aminomethane, succinate, and polyethylene glycol [PEG]).
- Genetic confirmation of ACH homozygous genotype.
- Premature birth with gestational age < 32 weeks.
- Premature birth with gestational age 32 to 37 weeks, unless time from birth is > 6 months at the time of screening and the child is in good nutritional status, defined as gain in body weight expected for age and diagnosis of ACH, as determined by the Investigator and confirmed with the Medical Monitor.
- Anticipated, as assessed by Investigator and confirmed with Medical Monitor, to undergo surgical intervention during trial participation, including cervicomedullary decompression. Evaluation of immediate risk of requiring cervicomedullary decompression surgery will rely on the following assessments:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NavepegritideOnce-weekly subcutaneous injection of 100 µg/kg Navepegritide | Drug | Navepegritide |
| Placebo for NavepegritideOnce-weekly subcutaneous injection of 100 µg/kg placebo for Navepegritide | Drug | Placebo for Navepegritide |
Primary outcome
To evaluate the safety and tolerability of Navepegritide
Time frame: 52 weeks
Incidence of treatment emergent adverse events (TEAEs)
To evaluate the effect of Navepegritide on growth
Time frame: 52 weeks
Change from baseline to 52 weeks in length/height Z-score
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Apr 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Medical Director, MD study director
Ascendis Pharma A/S
18 sites in 15 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Navepegritide in people with Achondroplasia, run by Ascendis Pharma A/S at 18 sites in 15 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 24 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 8 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 18 sites in United States, Germany, Australia, Austria, Canada and 10 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Ascendis Registry Inquiries (+45 61242484, asnd_registryinquiries@ascendispharma.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To evaluate the safety and tolerability of Navepegritide (time frame: 52 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Apr 2026.