Research initiatives

Although research is not the principal scope of ERNs, ERN BOND acts in order to facilitate research and clinical trials among members. This section illustrates the research initiatives endorsed by the network.

Call for collaborative clinical research

ERN BOND aims to favour research collaborations among network members to rapidly build up clinical series for rare skeletal disorders, to better delineate the clinical spectrum, natural history, and clinical management of these conditions.

A permanent call of collaborative clinical research is therefore open to clinicians working in the ambit of ERN BOND diseases. The call list will be disseminated through the monthly newsletter.

Clinical Trials & Research Studies

ERN BOND by liaising with relevant stakeholders, collects and disseminate to BOND members the information and activities with the aim to facilitate the good clinical trial conduct for Rare Bone Disorders.

ERN BOND supports also the development of investigator led studies in rare bone disorders, covering both clinical trials of investigational medical products and research studies not involving drugs.

In the past, the thematic group on Clinical Trials supported the development and conduction of a studies involving BOND expert centres to:
– validate the existing paediatric Osteogenesis Imerfecta Quality of Life tools and to,
– ascertain child and parent priorities for research in Osteogenesis Imperfecta.

Two clinical trials, under EU projects, are currently endorsed by ERN BOND and conducted by network members.

  • MCDS-Therapy, an open label phase I/II a trial repurposing carbamazepine (CBZ) for the treatment of children with metaphyseal chondrodysplasia, Schmid type (URL),
  • MOI-A study, a matrix-directed therapy in older adolescents and adults with osteogenesis imperfecta, within the REMEDI4ALL project.

List of clinical trials & research studies involving BOND expert centres (under update)

TitleTrial/study IDConditionsLink
A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With AchondroplasiaNCT04085523AchondroplasiaLink
A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)NCT02724228AchondroplasiaLink
Study to Evaluate Efficacy and Safety of Romosozumab Compared With Bisphosphonates in Children and Adolescents With Osteogenesis ImperfectaNCT05972551Osteogenesis ImperfectaLInk
A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Children With AchondroplasiaNCT05598320AchondroplasiaLink
A Clinical Trial to Investigate Long-term Safety, Tolerability, and Efficacy of Weekly Subcutaneous Doses With TransCon CNP in Children and Adolescents With Achondroplasia (AttaCH)NCT05929807AchondroplasiaLink
A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With AchondroplasiaNCT03197766AchondroplasiaLink
Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Burosumab in Patients Less Than 1 Year of AgeNCT04188964X-linked Hypophosphatemia (XLH)Link
Evaluation of the Safety and Efficacy of Eneboparatide (AZP-3601) in Patients With Chronic Hypoparathyroidism (CALYPSO)NCT05778071hypoparathyroidism (cHP)Link