At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Navepegritide (drug), Placebo for navepegritide (drug)
- How long the study runs
- Study runs about 33 months (dates as stated)
- About the drug or intervention
- Navepegritide — drug: Once-weekly subcutaneous injection of 100 µg/kg navepegritide for 52 weeks · Placebo for navepegritide — drug: Once-weekly subcutaneous injection of 100 µg/kg placebo for navepegritide
- Patient visit burden
- Not specified by the sponsor
In plain English
This study is testing a medicine called navepegritide in young people aged 12 to 18 who have achondroplasia, a genetic condition that affects bone growth and causes short stature. The researchers want to find out how well the medicine works and how safe it is. The sponsor is Ascendis Pharma A/S.
Who can take part
- Aged between 12 and 18 at the time of joining the study
- A clinical diagnosis of achondroplasia confirmed by a genetic test (past test results are acceptable)
- A parent or legal guardian willing and able to give the study medicine as a weekly injection under the skin and follow the study rules
- At least one past height measurement (standing) in their medical records, taken 6 to 15 months before screening
- Written consent (and assent from the young person where required) from the participant, parent(s) or legal guardian(s)
- If sexually active, using a highly effective form of contraception during the study and for 90 days after the last dose
Who may not be able to
- Took part in another clinical trial of a treatment in the 3 months before screening (unless no study medicine was given)
- Slowed growth (less than 1.5 cm per year over at least 6 months) or evidence on scans that the growth plates have closed
- Known or suspected allergy to the study medicine or its ingredients (trehalose, tris[hydroxymethyl]aminomethane, succinate, and mPEG)
- Another growth condition or cause of short stature, such as hypochondroplasia, growth hormone deficiency, Turner syndrome, coeliac disease, thyroid problems, or diabetes
- A severe change (mutation) in the FGFR3 gene, such as two variants on the same gene copy, or severe achondroplasia with developmental delay and acanthosis nigricans (darkened, thickened skin)
- Any medicine or surgery at any time meant to affect height, growth, or body proportions
- Needing long-term or repeated treatment with steroid tablets or injections (more than 4 weeks at a time, or more than twice a year), or long-term high-dose inhaled steroids
- Past injury or disease of the growth plates (other than achondroplasia), or bone surgery that could affect growth, such as leg-lengthening surgery (some procedures, like 8-plate surgery for leg bowing, VP shunt, or laminectomy, may be allowed after at least 6 months of healing)
- A bone fracture in the 6 months before screening (2 months for finger or toe fractures and minor buckle fractures)
- Needing surgery during the study that could affect the results or study checks (common operations such as grommets, removing tonsils or adenoids, or ear tubes are allowed)
- Severe untreated sleep apnoea (paused breathing during sleep), or sleep apnoea treatment started within 2 months before screening
- Serious hip problems or certain growth-plate injuries (for example, Salter-Harris fractures)
- Significant heart problems, including certain types of congenital heart disease or a heart rhythm tracing (QTcF) of 450 milliseconds or more at screening
- Conditions affecting blood pressure or circulation, such as autonomic dysfunction or orthostatic intolerance (feeling faint when standing)
- Long-term anaemia, long-term kidney problems, or ongoing illness affecting hydration or nutrition (treated iron deficiency anaemia may be allowed)
- Cancer, now or in the past
- Low vitamin D levels (below 30 nmol/L) at screening; those between 30 and 50 nmol/L can join if they start vitamin D supplements
- Any other condition or situation that, in the researcher's opinion, makes taking part unwise or could affect the study results
- Pregnancy, or breastfeeding
What taking part involves
- • The study medicine, navepegritide, is given as a weekly injection under the skin
- • Parents or legal guardians give the injections and follow the study plan
- • The study compares how well the medicine works and how safe it is — further details on doses, groups, and tests are not stated — ask the trial team
Time commitment: Weekly injections given at home by a parent or guardian, plus study visits and checks — the total length of the study and number of hospital visits are not stated — ask the trial team.
Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.
- Type of study
- Testing a treatment
- Ages
- 12 Years to 17 Years
- Who
- All
- Number of participants
- 24
- Started
- 2024-12-10
- Last checked
- 2026-04
Plain English Summary
What is this study?
- • Testing a new treatment for achondroplasia
- • Phase2 - 24 participants
- • 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
Who can take part?
- • Ages 12 Years to 17 Years
- • Diagnosed with achondroplasia
Where?
- • Norwich - Ascendis Investigational Site
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
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.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 12 Years - 17 Years
- Who can join: All genders
Biomarkers mentioned
Treatment history
Treatments you must have had:
- ✓ been collected between 6 months to 15 months
What the study is looking for
- ✓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...
- ✓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...
Who cannot take part
- ✗Participation (signed agreement to take part) in any interventional clinical trial within 3 months prior to Screening...
- ✗Decreased growth velocity (AGV less than 1.5 cm/year based on measurement over a period of at least 6 months) or...
- ✗Known or suspected hypersensitivity to the IMP or related products (trehalose, tris\[hydroxymethyl\]aminomethane,...
- ✗Severe mutation in the FGFR3 gene, e.g. two variants on the same allele or severe ACH with developmental delay and...
- ✗Known history of presence of injury or disease of the growth plate(s), other than ACH, that affects growth potential...
See the full criteria
Where Is This Study? (1 UK site)
Ascendis Investigational Site
Norwich NR4 7UY, United Kingdom
How to Get in Touch
Ascendis Registry Inquiries
Sponsor contactCONTACT
