Achondroplasia Clinical Trial
— OMPR-Ach/HyOfficial title:
Local Registry for Data Collection of Patients With Achondroplasia / Hypochondroplasia for Epidemiological, Care and Research Studies
This registry is a observational, single-center study designed to collect clinical data on patients with achondroplasia and hypochondroplasia.
| Status | Recruiting |
| Enrollment | 200 |
| Est. completion date | December 31, 2036 |
| Est. primary completion date | December 31, 2031 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | N/A and older |
| Eligibility | Inclusion Criteria: - Confirmed diagnosis of achondroplasia/hypochondroplasia - Patients (and/or Parents legal guardian when required) able to provide informed consent Exclusion Criteria: - Absence of diagnosis of achondroplasia/hypochondroplasia - Patients (and/or Parents legal guardian when required) not able to provide informed consent |
| Country | Name | City | State |
|---|---|---|---|
| Italy | Medical Genetics Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico Milan | Milan |
| Lead Sponsor | Collaborator |
|---|---|
| Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico |
Italy,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Registry of Achondroplasia and Hypochondroplasia Patients | Collection of medical information of achondroplasia and hypochndroplasia patients intended for use in future research studies. | 10 years |
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Recruiting |
NCT05353192 -
A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children With Achondroplasia
|
Phase 4 | |
| Completed |
NCT05659719 -
A Study to Learn About Recifercept in Patients With Achondroplasia
|
||
| Active, not recruiting |
NCT04554940 -
A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia
|
Phase 2 | |
| Completed |
NCT01435629 -
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 |
NCT03583697 -
A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia
|
Phase 2 | |
| Completed |
NCT01516229 -
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 |
NCT04265651 -
Study of Infigratinib in Children With Achondroplasia
|
Phase 2 | |
| Recruiting |
NCT05603936 -
Adaption and Testing of the Quality of Life in Short Stature Youth (QoLISSY) Questionnaire for Parents With Children From 0-4
|
||
| Completed |
NCT03780153 -
The Norwegian Adult Achondroplasia Study
|
||
| 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 | |
| Completed |
NCT03875534 -
A Multi-center, Longitudinal, Observational Study of Children With Achondroplasia
|
||
| Terminated |
NCT03794609 -
Observational Study Investigating Clinical & Anthropometric Characteristics of Children With Achondroplasia.
|
||
| 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 | |
| Active, not recruiting |
NCT02724228 -
A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)
|
Phase 2 |