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.
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.
- Together4RD, is an agile multi-stakeholder initiative aimed at supporting ERNs to collaborate with stakeholders to pursue opportunities that will address unmet medical needs of people living with rare diseases.
- RealiseD: the project aims to tackle these issues by developing new tools, methods, and systems that make clinical trials for rare diseases more efficient, accessible, and patient-focused.
List of clinical trials & research studies involving BOND expert centres (under update)
| Title | Trial/study ID | Conditions | Link |
|---|---|---|---|
| A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia | NCT04085523 | Achondroplasia | Link |
| A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH) | NCT02724228 | Achondroplasia | Link |
| Study to Evaluate Efficacy and Safety of Romosozumab Compared With Bisphosphonates in Children and Adolescents With Osteogenesis Imperfecta | NCT05972551 | Osteogenesis Imperfecta | LInk |
| A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Children With Achondroplasia | NCT05598320 | Achondroplasia | Link |
| 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) | NCT05929807 | Achondroplasia | Link |
| A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia | NCT03197766 | Achondroplasia | Link |
| Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Burosumab in Patients Less Than 1 Year of Age | NCT04188964 | X-linked Hypophosphatemia (XLH) | Link |
| Evaluation of the Safety and Efficacy of Eneboparatide (AZP-3601) in Patients With Chronic Hypoparathyroidism (CALYPSO) | NCT05778071 | hypoparathyroidism (cHP) | Link |
| An Interventional Study to Evaluate the Impact of Blood Flow Restriction Training on Muscle, Bone, and Quality of Life in Adults With Osteogenesis Imperfecta Type I | NCT07478224 | Osteogenesis Imperfecta (OI) | Link |
| Prospective Clinical Assessment Study in Children With Achondroplasia (ACH) | NCT04035811 | Achondroplasia | Link |
| An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia | NCT03424018 | Achondroplasia | Link |
| Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia (CANOPY-HCH-3) | NCT06455059 | Hypochondroplasia | Link |
| A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia | NCT06079398 | Osteogenesis Imperfecta (OI) | Link |
| To Assess the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva (Progress) | NCT05090891 | Fibrodysplasia Ossificans Progressiva (FOP) | Link |
| Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ENPP1 Deficiency | NCT04686175 | Autosomal recessive hypophosphatemic rickets type 2 (ARHR2) | Link |
| ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency | NCT06462547 | Autosomal recessive hypophosphatemic rickets type 2 (ARHR2) | Link |
| A First-in-human Study of KK8123 in Adults With X-linked Hypophosphatemia | NCT06525636 | X-linked Hypophosphatemia | Link |
| A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide in Adolescents (12 - 18 Years of Age) With Achondroplasia. (teACH) | NCT06732895 | Achondroplasia | Link |
| Extension Study of Infigratinib in Children With Achondroplasia (ACH) | NCT05145010 | Achondroplasia | Link |
| A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia (PROPEL3) | NCT06164951 | Achondroplasia | Link |
| A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature | NCT06382155 | Idiopathic Short Stature | Link |
| A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment | NCT06668805 | Noonan Syndrome | Link |
Call for collaborative clinical research
Do you have a research idea in the field of rare skeletal disorders?
ERN BOND is inviting its members to submit new collaborative proposals to help expand clinical knowledge and improve patient care across Europe.
Funded by the European Union
“Funded by the European Union. Views and opinions expressed are however those of the author(s) only and do not necessarily reflect those of the European Union or HaDEA. Neither the European Union nor the granting authority can be held responsible for them.”
