achondroplasia
Conditions
Interventions
None listed
Sponsors
Tokuoka Hirofumi
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Documented diagnosis of ACH as per standard of care. 2. Have initiated Voxzogo treatment, or plan to initiate Voxzogo treatment within 3 months, before the age of 2 years, in accordance with local standard clinical practice and local label with full medical record available. 3. Parent(s) or guardian(s) must be willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to performance of any research-related procedure. 4. Have available medical history from the start of Voxzogo treatment, or 6 months of age, whichever is earlier, to enable collection of data elements of interest for the study (including anthropomorphic measurements, medical and surgical outcomes of interest). 5. Participants who are on Voxzogo treatment must be enrolled in 111-604 in parallel and provide consent to share safety information via 111-604.
Exclusion criteria
Exclusion criteria: Currently enrolled in an interventional study (e.g., studies which require medical device use or drug therapy or with protocol required procedures) or prior enrollment in another interventional study(s) or receiving investigational agent(s)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Event rate of ACH-related surgeries of interest | — |
Secondary
| Measure | Time frame |
|---|---|
| Event rate of comorbidities including obstructive or central sleep apnea, foramen magnum stenosis, otitis media Event rate of continuous positive airway pressure (CPAP) and bilevel positive airway pressure (BiPAP) therapy Changes over time in anthropometric measurements including: Age at achievement of gross motor developmental milestones | — |
Contacts
Public ContactSpecialist Trial
BioMarin Pharmaceutical Inc.
Outcome results
None listed