HypochondroplasiaPhase 3HCH UpliftMonoNCT07917143
A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide Versus Placebo in Children and Adolescents With Hypochondroplasia
A Phase 3 study testing Navepegritide in people with Hypochondroplasia, run by Ascendis Pharma A/S.
Trial summary
This trial is being conducted to evaluate the efficacy, safety and tolerability of treatment with navepegritide subcutaneous (SC) injections once weekly in children and adolescents with hypochondroplasia aged 2 to 17 years. Read more about this study
- Who is it for?
- Ages 2 to 17 years
- Conditions the study is for
- Hypochondroplasia
- 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
- 81 participants
- Where
- No sites listed yet
- Study dates
- Nov 2026to Oct 2029
- Phase
- Phase 3
- 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 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 1. Age 2 to <18 years at randomization
- 2. Clinical diagnosis of HCH with documented genetic confirmation
- 3. Height measurement available from medical records
Who cannot join
- 1. Evidence of growth plate closure
- 2. Prior treatment with certain medication(s) which potentially could have affected growth
- 3. History of any surgery intended to affect stature, growth, or body proportionality except procedures for leg bowing
- 4. Unable to stand without assistance
- 5. Known or suspected medical conditions other than hypochondroplasia that could affect growth
- 6. Known history or presence of injury of the growth plates that affects growth potential of long bones
- 7. Known history or presence of malignant disease
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: A Phase 3, Multicenter, Randomized, Double-blind, Placebo-controlled Trial Evaluating the Efficacy and Safety of Once-weekly Navepegritide Versus Placebo in Children and Adolescents With Hypochondroplasia
This trial is being conducted to evaluate the efficacy, safety and tolerability of treatment with navepegritide subcutaneous (SC) injections once weekly in children and adolescents with hypochondroplasia aged 2 to 17 years.
- Conditions
- Hypochondroplasia
- Being tested
- Navepegritide, Placebo for Navepegritide
- Medical subject headings
- Hypochondroplasia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NavepegritideDrug
Administered weekly as s.c. injection. Treatment duration of up to 104 weeks
Placebo for NavepegritideDrug
Administered weekly as s.c. injection. Treatment duration of up to 52 weeks of placebo for navepegritide followed by 52 weeks of navepegritide.
| Study group | Role | What they receive |
|---|---|---|
| Once weekly navepegritide sc. | Experimental | Not described Receives: Drug: Navepegritide |
| Once weekly placebo for navepegritide sc. | Placebo comparator | Not described Receives: Drug: Placebo for Navepegritide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annual Growth Velocity (AGV)
To evaluate the AGV, cm per year, compared to placebo
Measured: Week 52
Secondary outcomes (4)
CDC-based height Z-score
To evaluate the change from baseline in CDC-based height Z-score
Measured: Week 52
Upper to lower body segment ratio
Change from baseline in upper to lower body segment ratio
Measured: Week 52
Quality of Life questionnaire
Change from baseline in health related quality of life Quality of Life in Short Stature (QoLISSY)
Measured: Week 52
Treatment-emergent Adverse Events (AE)
Number of events
Measured: Throughout trial duration 104 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Nov 2026
- Study startNov 2026 (estimated)
- Primary completionOct 2028 (estimated)
- Study completionOct 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HypochondroplasiaStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 81 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
- NCT07917143
- Sponsor's protocol ID
- ASND0051
- Acronym
- HCH UpliftMono
- Status last verified
- Oct 2026
- First submitted
- 30 Oct 2026
- Last updated
- 4 Nov 2026
- Results posted
- No
- 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 4 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Multicenter, Randomized, Double-blind, Placebo-controlled Trial Evaluating the Efficacy and Safety of Once-weekly Navepegritide Versus Placebo in Children and Adolescents With Hypochondroplasia
- ClinicalTrials.gov ID
- NCT07917143
- Acronym / sponsor ID
- HCH UpliftMono / ASND0051
- Conditions
- Hypochondroplasia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 81 participants (estimated)
- Lead sponsor
- Ascendis Pharma A/SIndustry (pharma, biotech, devices)
- Study start
- Nov 2026 (estimated)
- Primary completion
- Oct 2028 (estimated)
- Study completion
- Oct 2029 (estimated)
- First posted
- 4 Nov 2026 (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 being conducted to evaluate the efficacy, safety and tolerability of treatment with navepegritide subcutaneous (SC) injections once weekly in children and adolescents with hypochondroplasia aged 2 to 17 years.
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 |
|---|---|---|
| Once weekly navepegritide sc. | Experimental | Drug: Navepegritide |
| Once weekly placebo for navepegritide sc. | Placebo comparator | Drug: Placebo for Navepegritide |
Who may qualify
- Ages
- 2 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- 1. Age 2 to <18 years at randomization
- 2. Clinical diagnosis of HCH with documented genetic confirmation
- 3. Height measurement available from medical records
Key exclusion criteria (7 listed)
- 1. Evidence of growth plate closure
- 2. Prior treatment with certain medication(s) which potentially could have affected growth
- 3. History of any surgery intended to affect stature, growth, or body proportionality except procedures for leg bowing
- 4. Unable to stand without assistance
- 5. Known or suspected medical conditions other than hypochondroplasia that could affect growth
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 |
|---|---|---|
| NavepegritideAdministered weekly as s.c. injection. Treatment duration of up to 104 weeks | Drug | Once weekly navepegritide sc. |
| Placebo for NavepegritideAdministered weekly as s.c. injection. Treatment duration of up to 52 weeks of placebo for navepegritide followed by 52 weeks of navepegritide. | Drug | Once weekly placebo for navepegritide sc. |
Primary outcome
Annual Growth Velocity (AGV)
Time frame: Week 52
To evaluate the AGV, cm per year, compared to placebo
4 secondary outcomes
CDC-based height Z-score
Time frame: Week 52
Upper to lower body segment ratio
Time frame: Week 52
Quality of Life questionnaire
Time frame: Week 52
Treatment-emergent Adverse Events (AE)
Time frame: Throughout trial duration 104 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 4 Nov 2026.
- Individual participant dataSharing planned: No
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Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia
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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
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Navepegritide in people with Hypochondroplasia, run by Ascendis Pharma A/S.
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 3 inclusion and 7 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: opening soon, as of the sponsor's last update on 4 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
Annual Growth Velocity (AGV) (time frame: Week 52). 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 4 Nov 2026.