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Looking for participantsPhase2

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

Sponsor: Ascendis Pharma A/S

NCT ID: NCT06732895

View on ClinicalTrials.gov ↗

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.

Achondroplasia

How this trial compares with your answers

Answer 2 more questions to improve match

What we know so far

Condition· Matched your search
Age· Tell us your age for better matching
Gender· Tell us your sex for better matching

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

Continuous PositiveeGFR

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
Inclusion Criteria: * 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. Exclusion Criteria: * 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: * Orthopedic reconstructive surgery for bone lengthening (e.g., procedures for leg bowing such as 8-plate are not exclusionary). * 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 * Otherwise, are considered by the Investigator to be unfit to receive trial treatment or undergo trial related procedures. * Have a clinically significant finding or arrhythmia as determined by the investigator in consultation with the medical monitor 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 hemodynamic stability (such as autonomic dysfunction and orthostatic intolerance). * Known history or presence of the following: * Chronic anemia (iron deficiency anemia that is resolved or 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 more than 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. * Participant with serum 25-hydroxy-vitamin D (25OHD) levels of less than 30 nmol/L (less than 12 ng/mL) at Screening Visit will be excluded. Participants with 25OHD levels between 30-50 nmol/L (12-20 ng/mL) can be randomized provided treatment with Vitamin D supplementation is initiated according to local standards. * 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 and female partners of male participants of childbearing potential not using a highly effective form of contraceptive for the entire trial period and for 90 days after last dose of trial treatment. * Female participants who are pregnant, lactating or breastfeeding.

Where Is This Study? (1 UK site)

Ascendis Investigational Site

Norwich NR4 7UY, United Kingdom

Recruiting

How to Get in Touch

Ascendis Registry Inquiries

Sponsor contact

CONTACT

+4561242484 asnd_registryinquiries@ascendispharma.com
Data sourced from ClinicalTrials.gov · Last verified: 2026-04