VIrtual STudy in Achondroplasia for the US (VISTA)

Part of paid clinical trials in Oakland, California.

Sponsor
BioMarin Pharmaceutical
Study ID
NCT06168201
Status
Recruiting
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Conditions

  • Achondroplasia

Eligibility Criteria

Sex
ALL
Age
0 Months - 18 Years
Healthy Volunteers
Not accepted

Study Details

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.

Key Dates

Start date
Feb 21, 2023
Status verified
Dec 2025
Primary completion
Feb 29, 2028
Completion
Feb 29, 2028

Study Design

Enrollment
170 participants (estimated)

Arms

  • Arm: Primary Pediatric Cohort
    ≤13 years old at time of enrollment. This is an observational study.
  • Arm: Secondary Adolescent and Adult Cohort
    ≥14 years old at time of enrollment. This is an observational study.

Primary Outcome Measure

Height [ Time Frame: Retrospective data and prospective data for up to 5 years starting from time of study enrolment ]

Central Contacts

Locations (7)

FacilityCityStateZIPSite coordinators
UCSFOaklandCalifornia94609
Hind Al Saif
Hind Al Saif (PRINCIPAL_INVESTIGATOR)
PicnicHealthSan FranciscoCalifornia94107
PicnicHealth
[email protected]
Piedmont HealthStatesvilleNorth Carolina28625
Samuel Hughes
University of CincinnatiCincinnatiOhio45229
Laurie Bailey
Sanford HealthSioux FallsSouth Dakota57105
Isum Ward
Baylor College of MedicineHoustonTexas77030-2316
Carlos Bacino
University of WisconsinMadisonWisconsin53726
Janet Legare

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