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Compares treatment options for Achondroplasia
Official title A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia
ClinicalTrials.gov ID: NCT06079398
What this study is testing
What is Navepegritide?
Navepegritide is an investigational medicine, given as an once-weekly injection under the skin, being studied as a potential treatment for achondroplasia.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 0 to 2
You may be able to join if
- Written, signed informed consent by the parent(s)/caregiver(s) of the participant, and as required by the institutional review board/human research...
- 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...
- 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...
- Compliance to daily Vitamin D supplementation for infants aged 14 days to 1 year. All participants older than 1 year of age with serum...
You likely can't join if
- Known or suspected hypersensitivity to the investigational product or related products (trehalose, tris[hydroxymethyl]aminomethane, succinate, and...
- 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...
- Anticipated, as assessed by Investigator and confirmed with Medical Monitor, to undergo surgical intervention during trial participation, including...
- Physical examination (e.g., neurologic findings of clonus, opisthotonus, exaggerated reflexes, dilated facial veins)
See the full eligibility criteria
- 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...
- 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.
- 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...
- 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...
- Physical examination (e.g., neurologic findings of clonus, opisthotonus, exaggerated reflexes, dilated facial veins)
- 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...
- 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...
- 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...
- 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...
- 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...
The study team makes the final eligibility decision.
Where it's taking place
- Saint Paul, Minnesota, United States
- Houston, Texas, United States
- Madison, Wisconsin, United States
- Parkville, Australia
- Linz, Austria
- Montreal, Canada
- Copenhagen, Denmark
- Helsinki, Finland
- Paris, France
- Berlin, Germany
- Cologne, Germany
- Dublin, Ireland
- Milan, Italy
- Auckland, New Zealand
- Oslo, Norway
- Coimbra, Portugal
- Stockholm, Sweden
- London, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0 years to 2 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Saint Paul, Minnesota, United States; Houston, Texas, United States; Madison, Wisconsin, United States; Parkville, Australia; Linz, Austria; Montreal, Canada and 12 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.