Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
RECRUITING
170 participants
OBSERVATIONAL
2023-02-21
2028-02-29
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Data will be collected in two formats for the primary pediatric study population:
1. Participant-mediated access to electronic health records(including medical imaging, when available) which will enable retrospective and prospective collection of secondary data reflecting real-life treatment use and clinical care. .
2. Primary data collection of Clinical Outcome Assessments (COAs) and questionnaire data.
Data will be collected in the following format for the adult cohort:
\- Participant-mediated access to electronic health records (including medical imaging, when available) which will enable retrospective and prospective collection of secondary data reflecting real-life treatment use and clinical care.
The primary study population will include approximately 150 pediatric individuals with achondroplasia regardless of their treatment status with VOXZOGO™. Individuals may change status from untreated to treated during the prospective period of the study (or vice versa) however they will only be counted once, based on their treated status at the point of enrollment.
The secondary study population will include 20 adolescent and adult participants.
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
COHORT
OTHER
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Primary Pediatric Cohort
≤13 years old at time of enrollment. This is an observational study.
No interventions assigned to this group
Secondary Adolescent and Adult Cohort
≥14 years old at time of enrollment. This is an observational study.
No interventions assigned to this group
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* Age at time of enrollment:
Primary Pediatric Cohort: ≤13 years old Secondary Adolescent and Adult Cohort: ≥14 years old
* Receiving medical care in the United States
* Complete PicnicHealth's onboarding process, including signing informed consent and authorization for medical record retrieval
Exclusion Criteria
0 Months
18 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
PicnicHealth
UNKNOWN
BioMarin Pharmaceutical
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Medical Director, MD
Role: STUDY_DIRECTOR
BioMarin Pharmaceutical
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
UCSF
Oakland, California, United States
PicnicHealth
San Francisco, California, United States
Piedmont Health
Statesville, North Carolina, United States
University of Cincinnati
Cincinnati, Ohio, United States
Sanford Health
Sioux Falls, South Dakota, United States
Baylor College of Medicine
Houston, Texas, United States
University of Wisconsin
Madison, Wisconsin, United States
Countries
Review the countries where the study has at least one active or historical site.
Central Contacts
Reach out to these primary contacts for questions about participation or study logistics.
Facility Contacts
Find local site contact details for specific facilities participating in the trial.
Hind Al Saif
Role: primary
PicnicHealth
Role: primary
Samuel Hughes
Role: primary
Laurie Bailey
Role: primary
Isum Ward
Role: primary
Carlos Bacino
Role: primary
Janet Legare
Role: primary
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
111-605
Identifier Type: -
Identifier Source: org_study_id