NCT06168201 VIrtual STudy in Achondroplasia for the US (VISTA)
| NCT ID | NCT06168201 |
| Status | Recruiting |
| Phase | — |
| Sponsor | BioMarin Pharmaceutical |
| Condition | Achondroplasia |
| Study Type | OBSERVATIONAL |
| Enrollment | 170 participants |
| Start Date | 2023-02-21 |
| Primary Completion | 2028-02 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 170 participants in total. It began in 2023-02-21 with a primary completion date of 2028-02.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
This is an observational study of individuals with achondroplasia in the United States. The primary study population consists of pediatric individuals treated and untreated with VOXZOGO™. Study enrollment started in February 2023. The projected total duration of the study is approximately 5 years at minimum from start of study recruitment in February 2023, with the duration of individual prospective follow-up differing depending on the time of enrollment. The study duration may be extended based on decisions by the study sponsor. 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.
Eligibility Criteria
Inclusion Criteria: * Physician diagnosis of achondroplasia * 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: * Lack of any medical records
Contact & Investigator
Medical Director, MD
STUDY DIRECTOR
BioMarin Pharmaceutical
Frequently Asked Questions
Who can join the NCT06168201 clinical trial?
This trial is open to participants of all sexes, aged 0 Months or older, up to 18 Years, studying Achondroplasia. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT06168201 currently recruiting?
Yes, NCT06168201 is actively recruiting participants. Contact the research team at medinfo@bmrn.com for enrollment information.
Where is the NCT06168201 trial being conducted?
This trial is being conducted at Oakland, United States, San Francisco, United States, Baltimore, United States, Statesville, United States and 4 additional locations.
Who is sponsoring the NCT06168201 clinical trial?
NCT06168201 is sponsored by BioMarin Pharmaceutical. The principal investigator is Medical Director, MD at BioMarin Pharmaceutical. The trial plans to enroll 170 participants.