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Osteochondrodysplasia – Trials in Disease

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Clinical Trials for Osteochondrodysplasia

Currently, there are 10 ongoing clinical trials focused on osteochondrodysplasia, a group of disorders affecting bone and cartilage development. The majority of these trials specifically target achondroplasia, the most common form of this condition, testing various treatments aimed at improving growth in children. Trials are being conducted across multiple European countries, with participation opportunities for children from infancy through adolescence.

Clinical trial locations

Study on Long-Term Use of Infigratinib for Children with Achondroplasia

This trial investigates the long-term safety and effectiveness of infigratinib in children with achondroplasia. Achondroplasia is a genetic condition affecting bone growth that leads to shorter stature. Infigratinib is a type of medication called a tyrosine kinase inhibitor that works by blocking certain proteins involved in bone development.

Inclusion criteria: Children aged 3 to under 18 years with confirmed achondroplasia who have growth potential are eligible. Participants must be able to swallow oral medication in capsule or tablet form. Those who have completed a previous study with infigratinib can join, as well as treatment-naïve children with at least six months of documented growth measurements. Girls aged 10 and older or those who have started menstruating must have a negative pregnancy test. Participants and their parents or guardians must be willing to attend study visits and follow all procedures.

Exclusion criteria: Children with serious health conditions that could interfere with the study, those currently taking other medications that might affect results, and those unable to follow study procedures cannot participate. Recent surgery, pregnancy, breastfeeding, history of allergic reactions to similar medications, substance abuse history, or recent participation in another clinical trial are also grounds for exclusion.

Study focus: The trial aims to assess how infigratinib impacts height growth over time in children with this condition. Researchers will monitor changes in growth rates, body proportions, weight, and physical abilities. The study will also evaluate quality of life measures, including daily activities and cognitive functions. Various health assessments including physical exams, imaging tests, and safety checks will be conducted throughout the study period.

Investigational drug: Infigratinib is taken orally as capsules or tablets on a daily basis. It selectively inhibits FGFR 1-3 proteins that are involved in bone tissue development and maintenance.

Study on the Effectiveness and Safety of Navepegritide for Adolescents with Achondroplasia

This clinical trial evaluates navepegritide (also known as TransCon CNP) for promoting growth in adolescents with achondroplasia. The study is a double-blind trial, meaning neither participants nor researchers know who receives the actual treatment versus placebo during the 52-week treatment period.

Inclusion criteria: Adolescents aged 12 to under 18 years with clinically diagnosed achondroplasia confirmed by genetic testing are eligible. At least one historical height measurement from medical records, taken between 6 to 15 months before screening, must be available. Parents or legal guardians must be willing and able to administer weekly injections under the skin and follow study rules.

Exclusion criteria: Individuals without achondroplasia, those outside the specified age range, participants unavailable for the full study duration, those with interfering medical conditions, current participants in other trials, those who recently had surgery or plan surgery during the study, and those unable to comply with procedures cannot participate.

Study focus: The primary goal is to evaluate average growth velocity at week 52. Throughout the study, height measurements and changes from baseline height scores will be monitored. Regular health assessments will track any potential side effects to ensure treatment safety.

Investigational drug: Navepegritide is administered as a weekly injection under the skin. It contains c-type natriuretic peptide linked to a carrier molecule to help it work effectively in the body.

Study on the Safety and Effectiveness of TransCon CNP for Children and Adolescents with Achondroplasia

This long-term study evaluates the safety, tolerability, and effectiveness of TransCon CNP in children and adolescents with achondroplasia. The medication contains C-type natriuretic peptide linked to a carrier molecule.

Inclusion criteria: Participants must have achondroplasia and completed a previous TransCon CNP clinical trial. Parents or legal guardians must provide written consent and be willing to administer weekly injections under the skin. Participants must be considered eligible based on safety evaluations from the previous trial.

Exclusion criteria: Those with medical conditions other than achondroplasia that could interfere, serious allergic reactions to study medication ingredients, current participation in another trial, significant heart problems, uncontrolled high blood pressure, cancer history, liver or kidney disease, pregnancy or breastfeeding, drug or alcohol abuse history, or inability to comply with procedures cannot participate.

Study focus: The trial monitors long-term safety through regular assessments including laboratory tests, vital signs, physical examinations, heart monitoring (ECG), and imaging. Growth is measured through height assessments and height Z-scores. Annual growth velocity is calculated, and plasma concentrations of medication components are analyzed. Anti-drug antibodies are monitored to evaluate the body’s response.

Investigational drug: TransCon CNP is given as a weekly injection under the skin, releasing C-type natriuretic peptide in a controlled manner to help regulate bone growth.

Study on the Safety and Effectiveness of TransCon CNP for Infants with Achondroplasia

This trial focuses on infants with achondroplasia, comparing TransCon CNP with placebo over 52 weeks, followed by an open-label extension where all participants may receive the actual medication.

Inclusion criteria: Children younger than 2 years old with clinically diagnosed achondroplasia confirmed by genetic testing are eligible. Parents or caregivers must provide written consent and be willing to give weekly injections under the skin. Infants aged 14 days to 1 year must take daily Vitamin D supplements. Children older than 1 year with low Vitamin D levels must start supplementation before joining. Participants must be ineligible for vosoritide treatment due to unavailability, parental preference, or affordability issues.

Exclusion criteria: Those without achondroplasia, participants outside the age range, individuals with interfering medical conditions, those taking medications affecting study results, recent clinical trial participants, those with allergies to similar treatments, and those unable to comply with procedures and visits are excluded.

Study focus: The trial assesses safety, tolerability, and growth effects over 52 weeks. Researchers monitor growth rates and length/height Z-scores. Additional evaluations include motor and language skill development, X-rays, and MRIs to examine bone growth and spinal development.

Investigational drug: TransCon CNP is administered as a weekly injection under the skin, releasing C-type natriuretic peptide to help regulate bone growth in infants.

Study on the Effectiveness and Safety of Infigratinib for Children Aged 3 to 17 with Achondroplasia

This double-blind study compares infigratinib to placebo over 52 weeks in children aged 3 to under 18 years with achondroplasia who still have growth potential.

Inclusion criteria: Children must be between 3 and under 18 years old with growth potential, meaning they grow more than 1.5 cm annually, are in Tanner stage 4 or less, and have bone age of 13 years or less for girls and 15 years or less for boys. Vosoritide must not be suitable due to safety concerns, unavailability, or family preference. Achondroplasia diagnosis must be confirmed through clinical examination and genetic testing. Participants must have completed at least 26 weeks in the previous PROPEL study, be able to swallow pills, walk and stand unassisted, and attend study visits. Girls aged 10 and older or those menstruating must have a negative pregnancy test. Sexually active participants must use effective birth control.

Exclusion criteria: Those without achondroplasia, outside the age range, or without growth potential cannot participate.

Study focus: The trial evaluates changes in annual height velocity after 52 weeks, monitoring height, body proportions, and other physical parameters. Regular assessments track responses to medication and side effects.

Investigational drug: Infigratinib is taken orally as capsules, targeting specific cellular pathways that affect bone growth to promote normal development.

Study on Long-Term Safety and Effects of Vosoritide in Children with Achondroplasia

This trial examines the long-term safety and effects of BMN 111 (also known as Voxzogo) containing vosoritide in children with achondroplasia who have completed 24 months of previous treatment.

Inclusion criteria: Children who completed 24 months of BMN 111 treatment in a previous study are eligible. Parent or guardian written consent is required, along with participant assent if under 18. Females aged 10 or older or those menstruating must have a negative pregnancy test at baseline. Sexually active participants must use effective contraception. Participants must be willing to perform all study procedures, and caregivers must be trained to administer daily injections (except in France).

Exclusion criteria: Children without achondroplasia, those outside the age range, those with interfering medical conditions, those unable to receive daily injections, recent trial participants, those with medication allergies, and those unable to follow procedures or attend visits cannot participate.

Study focus: Safety is monitored through adverse event tracking, laboratory tests (urinalysis, blood chemistry, hematology), vital signs, physical examinations, heart monitoring, X-rays, and hip assessments. Effectiveness is evaluated by measuring changes in height growth velocity, final adult height, and body proportions including weight, head circumference, and arm and leg lengths.

Investigational drug: Voxzogo contains vosoritide and is given as a daily injection under the skin in dosages of 0.56 mg or 1.2 mg.

Study on the Effects of Navepegritide and Lonapegsomatropin in Children with Achondroplasia

This trial evaluates the combination of navepegritide and lonapegsomatropin compared to navepegritide alone over 156 weeks (approximately three years) in children with achondroplasia.

Inclusion criteria: Children aged 2 to 11 years with clinically diagnosed achondroplasia confirmed by genetic testing showing a heterozygote genotype are eligible. Participants must be able to stand without assistance. Parents, caregivers, or legal guardians must provide written consent and be willing to administer weekly injections under the skin. At least six months of growth and disease history from previous studies or medical records must be available.

Exclusion criteria: Those without achondroplasia, outside the age range, with interfering medical conditions, taking medications affecting results, having previous treatments influencing outcomes, unable to follow procedures, or without parental permission cannot participate.

Study focus: The trial monitors growth and health throughout the 156-week period through assessments at weeks 26, 52, 104, and 156. Measurements include height, body composition, and health indicators. Safety assessments track vital signs, laboratory tests, and physical examinations.

Investigational drugs: Navepegritide (TransCon CNP) and lonapegsomatropin (TransCon hGH) are both administered as weekly injections under the skin. Navepegritide targets pathways influencing bone growth, while lonapegsomatropin mimics natural growth hormone to stimulate growth in bones and tissues.

Study on the Effects of TransCon CNP in Children Aged 2-11 with Achondroplasia

This double-blind trial evaluates TransCon CNP versus placebo for 52 weeks in children aged 2 to 11 years with achondroplasia, followed by an open-label extension.

Inclusion criteria: Children aged 2 to 11 years with clinically diagnosed achondroplasia confirmed by genetic proof are eligible. Participants must be able to stand without help. Parents or legal guardians must provide written consent and be willing to administer weekly injections under the skin. At least six months of growth and disease history from the ACHieve study or medical records must be available. Eligibility is confirmed through medical history, physical examination, vital signs, heart tests (ECG), and laboratory tests during screening.

Exclusion criteria: Those without achondroplasia, outside the age range, unable to follow procedures, with interfering medical conditions, taking medications affecting outcomes, recent trial participants, pregnant or planning pregnancy, and those with medication allergies are excluded.

Study focus: The primary measure is annualized growth velocity at week 52. Secondary evaluations include changes in height Z-score from baseline. Throughout the 52-week period, growth monitoring evaluates TransCon CNP efficacy, followed by an open-label extension phase.

Investigational drug: TransCon CNP is administered as a weekly injection under the skin, releasing C-type natriuretic peptide to help regulate bone growth and development.

Study on the Long-Term Safety and Effectiveness of Vosoritide for Children with Achondroplasia

This study assesses the long-term safety and effectiveness of BMN 111 (Voxzogo containing vosoritide) in children with achondroplasia who completed Study 111-301.

Inclusion criteria: Children who completed Study 111-301 are eligible. Parent or guardian consent is required, along with participant assent if applicable. Upon reaching legal age, personal consent is needed. Females aged 10 or older or those menstruating must have a negative pregnancy test at baseline and be willing to undergo additional testing. Sexually active participants must agree to contraception use. Participants must be willing and able to perform all study procedures.

Exclusion criteria: Those with interfering medical conditions, taking growth-affecting medications, having growth-impacting surgery, recent trial participants, those with medication allergies, serious health issue history, and those unable to follow instructions or attend visits cannot participate.

Study focus: The trial monitors height growth velocity annually to assess effectiveness. Safety is tracked through adverse events, vital signs changes, physical examinations, ECG, and echocardiograms. Imaging and hip monitoring evaluate bone structure and quality. Blood tests monitor hormone levels (FSH and LH) for participants over 8 years. Quality of life is assessed using questionnaires like QoLISSY and PedsQL, with functional independence evaluated through Wee-FIM assessments.

Investigational drug: Voxzogo contains vosoritide and is administered as an injection in dosages of 0.4 mg, 0.56 mg, and 1.2 mg, with specific dosing determined by the study protocol.

Study on the Safety and Effects of SAR442501 for Children with Achondroplasia

This trial investigates SAR442501, a humanized monoclonal antibody derivative targeting fibroblast growth factor receptor 3, in children with achondroplasia. The medication is provided as a powder mixed into solution for injection under the skin.

Inclusion criteria: Participants must have achondroplasia with a confirmed FGFR3 gene mutation. Both males and females aged at least 2 years are eligible. Participants and/or their parent or legal representative must be willing and able to perform all study procedures. Parents or legal representatives must provide signed informed consent, and participants must provide assent when applicable.

Exclusion criteria: Participants with osteochondrodysplasia affecting bone and cartilage development cannot participate.

Study focus: The study assesses safety and tolerability while monitoring for adverse events during the treatment-emergent period. Various changes are evaluated including growth velocity, body segment ratios, and health-related quality of life. Pharmacokinetic parameters such as plasma concentration, maximum plasma concentration, and time to reach maximum concentration are assessed. Pharmacodynamic parameters including collagen X biomarker levels and other bone-related markers are also evaluated.

Investigational drug: SAR442501 is administered through injection under the skin, targeting specific pathways that influence bone growth and development. It is classified as a growth modulator aimed at improving bone growth in affected individuals.

Summary

The ten ongoing clinical trials for osteochondrodysplasia demonstrate a concentrated research effort focused primarily on achondroplasia, the most common form of this bone growth disorder. Several key trends emerge from these studies.

Geographically, trials are widely distributed across Europe, with Spain, France, Germany, Ireland, Denmark, and Italy hosting multiple studies. This broad distribution provides accessibility for families across different regions. Spain and Ireland each host five trials, making them particularly active research centers for this condition.

The investigational treatments fall into several categories. TransCon CNP (navepegritide) appears most frequently, being tested across multiple age groups from infants to adolescents in various trial designs. Infigratinib, a tyrosine kinase inhibitor, is being evaluated in two separate studies for children aged 3 to 17 years. Vosoritide (BMN 111/Voxzogo) is the subject of long-term safety studies in children who have already received prior treatment. Additionally, combination therapy approaches are being explored, such as the trial combining navepegritide with lonapegsomatropin.

Age coverage is comprehensive, spanning from infancy (under 2 years) through adolescence (up to 18 years). This range ensures that potential treatments are being evaluated across all pediatric developmental stages. Most trials focus on evaluating growth velocity as the primary outcome measure, with additional assessments of safety, body proportions, and quality of life.

Administration methods are predominantly weekly injections under the skin, though infigratinib is taken orally. This variety in delivery methods may offer families different options based on their preferences and circumstances. Study durations vary considerably, from 52-week initial treatment periods to long-term extension studies lasting several years, providing both short-term efficacy data and crucial long-term safety information.

Ongoing Clinical Trials on Osteochondrodysplasia

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