AchondroplasiaPhase 2COACHNCT06433557
A Phase 2 Clinical Trial to Evaluate Efficacy, Safety, and Tolerability of Navepegritide in Combination With Lonapegsomatropin in Children With Achondroplasia
A Phase 2 study testing Combination of Navepegritide and Lonapegsomatropin administered as two separate s.c. injections in people with Achondroplasia, run by Ascendis Pharma Growth Disorders A/S at 3 sites in 3 countries.
Trial summary
This proof-of-concept trial is being conducted to evaluate the efficacy, safety and tolerability of combination treatment with navepegritide and lonapegsomatropin administered as separate subcutaneous (SC) injections once weekly in children with achondroplasia (ACH) aged 2 to 11 years. Read more about this study
- Who is it for?
- Ages 2 to 11 years
- Conditions the study is for
- Achondroplasia
- What is being tested?
- Combination of Navepegritide and Lonapegsomatropin administered as two separate s.c. injections
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 22 participants
- Where
- 3 sitesin 3 countries
- Study dates
- 26 Jul 2024to Nov 2027
- 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 2 to 11 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Written, signed informed consent and/or assent (if applicable) by the parent(s) or legal representative(s) of the participant, and as required by the IRB/HREC/IEC.
- Male or female between 2 to 11 years of age (inclusive) at the time of Visit 1.
- Clinical diagnosis of ACH with genetic confirmation of heterozygote genotype present at Visit 1. Documentation of historic test results are acceptable for proof of diagnosis.
- Able to stand without assistance.
- Parent(s)/caregiver(s)/legal guardian(s) willing and able to administer weekly SC injections of IMP and comply with all protocol requirements.
- At least 6 months of growth and disease history from TCC-NHS-01 or TCC-201 or comparable growth and disease history available from medical records.
- No intracranial pathology as confirmed by brain MRI (historical MRI obtained within 2 years prior to screening allowable).
Who cannot join
- Participation in any interventional clinical trial within three months prior to screening (except TCC-201 or ASND0039).
- Closed epiphysis at screening.
- History of or suspected hypersensitivity to the IMP or related products.
- Findings on fundoscopy at screening consistent with intracranial hypertension, papilledema, or evidence of any other retinal disease for which GH therapy is contraindicated.
- Have a growth disorder or medical condition other than ACH that results in short stature or abnormal growth such as severe ACH with developmental delay and acanthosis nigricans (SADDAN), hypochondroplasia, GHD, Turner syndrome, pseudoachondroplasia, inflammatory bowel disease, celiac disease, hypothyroidism, hyperthyroidism, pre-diabetes, or diabetes mellitus.
- Have received any dose of prescription and/or investigational medications or device intended to affect stature, growth, or body proportionality at any time prior to screening.
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- Receiving concurrent treatment with any agent that might influence growth or interfere with GH secretion or action:
- Inhaled corticosteroid therapy at a dose of >400 µg/day of inhaled budesonide or equivalent for more than 28 consecutive days total over the course of 12 months prior to screening.
- Require, or anticipated to require, chronic (>4 weeks) or repeated treatment (more than twice/year and >3 weeks/year) with systemic corticosteroids during participation in the trial.
- Currently using or have used within 12 months prior to screening any sex steroids (for example estrogen), non-steroidal anabolic agents (for example, oxandrolone) or gonadotropin-releasing hormone (GnRH) analogues treatment.
- Treatment for attention-deficit hyperactive disorder (ADHD) such as methylphenidate.
- Known history or 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 Orthopaedic reconstructive surgery for bone lengthening (procedures for leg bowing such as 8-plate are not exclusionary).
- Cervicomedullary decompression surgery within 6 months prior to Screening or with anticipated need for repeat decompression surgery during the time of the trial.
- Evidence at screening consistent with severe cervicomedullary junction compression based on clinical and/or radiologic findings that indicate immediate surgical intervention is required.
- Ventriculoperitoneal shunt and laminectomy with full recovery within 6 months prior to Screening.
- Salter-Harris fracture within 6 months prior to screening (within 2 months for fracture of digits and buckle fractures).
- Clinically significant musculoskeletal disease, such as Salter-Harris fractures or clinical and/or radiographic evidence of severe hip pathology.
- Planned or expected surgical intervention during trial participation that may significantly affect trial parameters (confounding of safety events) or would prevent the participant from performing trial procedures. Minimally invasive surgeries such as insertion of grommets, adenoidectomy, tonsillectomy, or myringotomy tube placement, are permitted during the trial.
- Severe untreated sleep apnoea or newly initiated sleep apnoea treatment (e.g., Continuous Positive Airway Pressure [CPAP] in the previous 2 months prior to screening).
- Clinically significant finding or arrhythmia as determined by the investigator that indicates abnormal cardiac function or conduction that includes, but is not exclusive 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.
- QT corrected using Fridericia's correction (QTcF) ≥ 450 msec at screening.
- Known history or presence of condition that impacts haemodynamic stability (such as autonomic dysfunction and orthostatic intolerance).
- Known history or presence at screening of the following:
- Chronic anaemia (iron deficiency anaemia that is resolved or considered adequately treated in the Investigator's opinion is allowed).
- Chronic renal insufficiency defined as estimated glomerular filtration rate (eGFR) according to the revised bedside Schwartz equation less than <60 mL/min/1.73 m2 for >3 months.
- Chronic or recurrent illness that can affect hydration or volume status, including conditions associated with decreased nutritional intake or increased volume loss.
- Known history or presence of malignant disease.
- Hepatic transaminases (aspartate aminotransferase (AST) or alanine transferase (ALT)) greater than 3x upper limit of normal (ULN) at screening.
- Serum 25-hydroxy-vitamin D (25OHD) level of <30 nmol/L (<12 ng/mL) at screening.
- Participants with 25OHD levels between 30-50 nmol/L (12-20 ng/mL) may be enrolled provided treatment with Vitamin D supplementation is initiated.
- Any disease or condition that, in the opinion of the Investigator, may make the participant unlikely to fully complete the trial, may confound interpretation of trial results, or may present undue risk from receiving trial treatment. This could include family situations, complications or manifestations, or medications that might impact safety or be considered confounding.
- Sexually active male and female participants who are unwilling or unable to use a highly effective form of contraception for the entire trial period and for 90 days after last dose of trial treatment.
- Female participants who are pregnant, lactating or breastfeeding.
Trial locations and contacts
3 sites in 3 countries.
3 sites in 3 countries
United Kingdom1 site
Ascendis Pharma Investigational Site
London, W1W 5AH
About this study
Official title: A Phase 2, Open-Label, Single-Arm, 156-week Trial to Investigate the Efficacy, Safety and Tolerability of Combined Once Weekly Navepegritide and Lonapegsomatropin in Children With Achondroplasia
This proof-of-concept trial is being conducted to evaluate the efficacy, safety and tolerability of combination treatment with navepegritide and lonapegsomatropin administered as separate subcutaneous (SC) injections once weekly in children with achondroplasia (ACH) aged 2 to 11 years.
- Conditions
- Achondroplasia
- Being tested
- Combination of Navepegritide and Lonapegsomatropin administered as two separate s.c. injections
- Medical subject headings
- Achondroplasia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Combination of Navepegritide and Lonapegsomatropin administered as two separate s.c. injectionsDrug
For navepegritide, a once weekly s.c. dose of 100 μg CNP/kg. For lonapegsomatropin, a once weekly s.c. dose of lonapegsomatropin 0.30 mg hGH/kg as starting dose. Treatment duration of up to 156 weeks.
| Study group | Role | What they receive |
|---|---|---|
| Combination of Navepegritide and Lonapegsomatropin | Experimental | Not described Receives: Drug: Combination of Navepegritide and Lonapegsomatropin administered as two separate s.c. injections |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annualized growth velocity
cm per year, compared to navepegritide alone
Measured: Week 52
Treatment-emergent adverse events (TEAEs).
safety profile of navepegritide and lonapegsomatropin
Measured: Throughout the trial for 156 weeks
Secondary outcomes (1)
Annualized growth velocity
cm per year
Measured: From start until 156 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 May 2024
- Study start26 Jul 2024
- Primary completion14 Nov 2025
- Study completionNov 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 22 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ascendis Pharma Growth Disorders 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
- NCT06433557
- Sponsor's protocol ID
- ASND0042
- Acronym
- COACH
- Status last verified
- Feb 2026
- First submitted
- 23 May 2024
- Last updated
- 18 Feb 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
McDonnell CM, Irving M, Nolting LA, Abdelrahman SG, Dalby LW, Ikle JM, Jensen SM, Komirenko AS, Ominsky MS, Shu AD, Hove HB. Navepegritide combined with lonapegsomatropin for the treatment of children with achondroplasia: 52-week results from the phase 2 COACH trial. Eur J Endocrinol. 2026 Jun 1;194(6):745-755. doi: 10.1093/ejendo/lvag082.
PubMed 42144862
Study at a glance
From the registry record, last updated by the sponsor on 18 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Open-Label, Single-Arm, 156-week Trial to Investigate the Efficacy, Safety and Tolerability of Combined Once Weekly Navepegritide and Lonapegsomatropin in Children With Achondroplasia
- ClinicalTrials.gov ID
- NCT06433557
- Acronym / sponsor ID
- COACH / ASND0042
- Conditions
- Achondroplasia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 22 participants (actual)
- Lead sponsor
- Ascendis Pharma Growth Disorders A/SIndustry (pharma, biotech, devices)
- Study start
- 26 Jul 2024 (actual)
- Primary completion
- 14 Nov 2025 (actual)
- Study completion
- Nov 2027 (estimated)
- First posted
- 29 May 2024 (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 proof-of-concept trial is being conducted to evaluate the efficacy, safety and tolerability of combination treatment with navepegritide and lonapegsomatropin administered as separate subcutaneous (SC) injections once weekly in children with achondroplasia (ACH) aged 2 to 11 years.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Combination of Navepegritide and Lonapegsomatropin | Experimental | Drug: Combination of Navepegritide and Lonapegsomatropin administered as two separate s.c. injections |
Who may qualify
- Ages
- 2 to 11 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- Written, signed informed consent and/or assent (if applicable) by the parent(s) or legal representative(s) of the participant, and as required by the IRB/HREC/IEC.
- Male or female between 2 to 11 years of age (inclusive) at the time of Visit 1.
- Clinical diagnosis of ACH with genetic confirmation of heterozygote genotype present at Visit 1. Documentation of historic test results are acceptable for proof of diagnosis.
- Able to stand without assistance.
- Parent(s)/caregiver(s)/legal guardian(s) willing and able to administer weekly SC injections of IMP and comply with all protocol requirements.
Key exclusion criteria (36 listed)
- Participation in any interventional clinical trial within three months prior to screening (except TCC-201 or ASND0039).
- Closed epiphysis at screening.
- History of or suspected hypersensitivity to the IMP or related products.
- Findings on fundoscopy at screening consistent with intracranial hypertension, papilledema, or evidence of any other retinal disease for which GH therapy is contraindicated.
- Have a growth disorder or medical condition other than ACH that results in short stature or abnormal growth such as severe ACH with developmental delay and acanthosis nigricans (SADDAN), hypochondroplasia, GHD, Turner syndrome, pseudoachondroplasia, inflammatory bowel disease, celiac disease, hypothyroidism, hyperthyroidism, pre-diabetes, or diabetes mellitus.
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 |
|---|---|---|
| Combination of Navepegritide and Lonapegsomatropin administered as two separate s.c. injectionsFor navepegritide, a once weekly s.c. dose of 100 μg CNP/kg. For lonapegsomatropin, a once weekly s.c. dose of lonapegsomatropin 0.30 mg hGH/kg as starting dose. Treatment duration of up to 156 weeks. | Drug | Combination of Navepegritide and Lonapegsomatropin |
Primary outcome
Annualized growth velocity
Time frame: Week 52
cm per year, compared to navepegritide alone
Treatment-emergent adverse events (TEAEs).
Time frame: Throughout the trial for 156 weeks
safety profile of navepegritide and lonapegsomatropin
1 secondary outcome
Annualized growth velocity
Time frame: From start until 156 weeks
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 18 Feb 2026.
- Derived from this study: PubMed 42144862McDonnell CM, Irving M, Nolting LA, Abdelrahman SG, Dalby LW, Ikle JM, Jensen SM, Komirenko AS, Ominsky MS, Shu AD, Hove HB. Navepegritide combined with lonapegsomatropin for the treatment of children with achondroplasia: 52-week results from the phase 2 COACH trial. Eur J Endocrinol. 2026 Jun 1;194(6):745-755. doi: 10.1093/ejendo/lvag082.
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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. This study is not recruiting at the moment.
Medical Director, MD study director
Ascendis Pharma A/S
3 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Combination of Navepegritide and Lonapegsomatropin administered as two separate s.c. injections in people with Achondroplasia, run by Ascendis Pharma Growth Disorders A/S at 3 sites in 3 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 7 inclusion and 36 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: under way, not looking for participants, as of the sponsor's last update on 18 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Denmark, Ireland, United Kingdom. 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. 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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Annualized growth velocity (time frame: Week 52). 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?
On the ClinicalTrials.gov record for NCT06433557, which also lists 1 linked publication.
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 18 Feb 2026.