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VIrtual STudy in Achondroplasia for the US (VISTA)

Study acronym: VISTA
StatusRecruiting
PhaseNot specified
Started
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Record History

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  1. Minor Study sites: 7 to 8v4
  2. Minor Study sites: 2 to 7v3
  3. Notable Enrollment target: 300 to 170
    • Study sites: 1 to 2
    • Eligibility criteria expanded, about 14 words
    • Study title shortened, about 5 words
    v2
  4. Minor Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
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.
Trial Details
NCT Number NCT06168201
Lead Sponsor BioMarin Pharmaceutical
Collaborators: PicnicHealth
Conditions
(from sponsor)
Achondroplasia
Related terms Achondroplasia
Enrollment 170 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov