VIrtual STudy in Achondroplasia for the US (VISTA)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Achondroplasia. Basic parameters: 0 months — 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Overview
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.
Primary outcome measures
- Height [Time frame: Retrospective data and prospective data for up to 5 years starting from time of study enrolment]
- Weight [Time frame: Retrospective data and prospective data for up to 5 years starting from time of study enrolment]
- Body Mass Index [Time frame: Retrospective data and prospective data for up to 5 years starting from time of study enrolment]
- Head Circumference [Time frame: Retrospective data and prospective data for up to 5 years starting from time of study enrolment]
- Annualized growth velocity (AGV) [Time frame: Retrospective data and prospective data for up to 5 years starting from time of study enrolment]
- Medical diagnoses by age [Time frame: Retrospective data and prospective data for up to 5 years starting from time of study enrolment]
- Surgical procedures and/or medical interventions by age [Time frame: Retrospective data and prospective data for up to 5 years starting from time of study enrolment]
- Changes in physical functioning [Time frame: every 6 months starting from time of study enrolment up to 5 years]
- Changes in health-related quality of life (HRQoL) [Time frame: every 6 months starting from time of study enrolment up to 5 years]
- Adherence to treatment with VOXZOGO™ (vosoritide), if prescribed [Time frame: every 6 months starting from time of study enrolment up to 5 years]
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
United States · 8 centers
- UCSF — Oakland
- PicnicHealth — San Francisco
- Johns Hopkins University (JHU) — Baltimore
- Piedmont Health — Statesville
- University of Cincinnati — Cincinnati
- Sanford Health — Sioux Falls
- Baylor College of Medicine — Houston
- University of Wisconsin — Madison
Identifiers
NCT: NCT06168201 · 111-605