Achondroplasia Clinical Trial
Official title:
The Norwegian Adult Achondroplasia Study
The study aims to investigate the Norwegian adult achondroplasia population regarding degree and extent of symptoms and clinical manifestations and how this population manages in daily life, including demographics, physical function, and work participation.
The present study aims to investigate the degree and extent of spinal stenosis, chronic pain, sleep apnoea, impaired hearing, cardiovascular risk factors and body composition in Norwegian adults with achondroplasia. The study will also explore these factors' impact on physical function, ability to perform daily activities, work participation and needs for assistive devices and social benefits. The study is conducted in collaboration with the Norwegian Restricted Growth Association, and TRS National Resource Centre for Rare Disorders, Sunnaas Rehabilitation Hospital, Lovisenberg Hospital and Oslo University Hospital. ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Recruiting |
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|
Phase 4 | |
| Recruiting |
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Registry for Patients With Achondroplasia / Hypochondroplasia (OMPR-Ach/Hy)
|
||
| Completed |
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|
||
| Active, not recruiting |
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A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia
|
Phase 2 | |
| Completed |
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A Survey Collecting Data on Adult Height in Patients With Achondroplasia Treated With Somatropin
|
N/A | |
| Enrolling by invitation |
NCT06164951 -
A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia
|
Phase 3 | |
| Completed |
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Special Survey for Long Term Application
|
N/A | |
| Completed |
NCT03872531 -
Lifetime Impact Study for Achondroplasia
|
||
| Active, not recruiting |
NCT05598320 -
A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Children With Achondroplasia
|
Phase 2/Phase 3 | |
| Terminated |
NCT05813314 -
Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants
|
Phase 1 | |
| Recruiting |
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Study of Infigratinib in Children With Achondroplasia
|
Phase 2 | |
| Recruiting |
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Adaption and Testing of the Quality of Life in Short Stature Youth (QoLISSY) Questionnaire for Parents With Children From 0-4
|
||
| Active, not recruiting |
NCT04085523 -
A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia
|
Phase 2 | |
| Enrolling by invitation |
NCT05929807 -
A Clinical Trial to Investigate Long-term Safety, Tolerability, and Efficacy of Weekly Subcutaneous Doses With TransCon CNP in Children and Adolescents With Achondroplasia
|
Phase 2/Phase 3 | |
| Not yet recruiting |
NCT06433557 -
A Phase 2 Clinical Trial to Evaluate Efficacy, Safety, and Tolerability of Navepegritide in Combination With Lonapegsomatropin in Children With Achondroplasia
|
Phase 2 | |
| Completed |
NCT03875534 -
A Multi-center, Longitudinal, Observational Study of Children With Achondroplasia
|
||
| Terminated |
NCT03794609 -
Observational Study Investigating Clinical & Anthropometric Characteristics of Children With Achondroplasia.
|
||
| Completed |
NCT03583697 -
A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia
|
Phase 2 | |
| Active, not recruiting |
NCT03989947 -
An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia
|
Phase 2 | |
| Active, not recruiting |
NCT05246033 -
A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Multiple Subcutaneous Doses of TransCon CNP Administered Once Weekly in Children With Achondroplasia
|
Phase 2 |