Clinical trial NCT06168201: VIrtual STudy in Achondroplasia for the US (VISTA)
Official title: VIrtual STudy in Achondroplasia for the US (VISTA)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Achondroplasia
Lead sponsor: BioMarin Pharmaceutical
Sponsor class: INDUSTRY
Collaborators: name: PicnicHealth
First posted: 2023-12-13
Enrollment: 170
Sex: ALL
Minimum age (years): 0
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The primary study population will include individuals with achondroplasia regardless of their treatment status with VOXZOGO™. Whilst individuals may change status from untreated to treated during the prospective period of the study (or vice versa), for the purposes of recruitment targets they will only be counted once, based on their treated status at the point of enrollment

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

Primary outcomes:
measure: Height
timeFrame: Retrospective data and prospective data for up to 5 years starting from time of study enrolment
measure: Weight
timeFrame: Retrospective data and prospective data for up to 5 years starting from time of study enrolment
measure: Body Mass Index
timeFrame: Retrospective data and prospective data for up to 5 years starting from time of study enrolment
measure: Head Circumference
timeFrame: Retrospective data and prospective data for up to 5 years starting from time of study enrolment
measure: Annualized growth velocity (AGV)
timeFrame: Retrospective data and prospective data for up to 5 years starting from time of study enrolment
measure: Medical diagnoses by age
timeFrame: Retrospective data and prospective data for up to 5 years starting from time of study enrolment
measure: Surgical procedures and/or medical interventions by age
timeFrame: Retrospective data and prospective data for up to 5 years starting from time of study enrolment
measure: Changes in physical functioning
timeFrame: every 6 months starting from time of study enrolment up to 5 years
measure: Changes in health-related quality of life (HRQoL)
timeFrame: every 6 months starting from time of study enrolment up to 5 years
measure: Adherence to treatment with VOXZOGO™ (vosoritide), if prescribed
timeFrame: every 6 months starting from time of study enrolment up to 5 years
Secondary outcomes:
Other outcomes:
Locations:
city: Oakland
country: United States
facility: UCSF
state: California
city: San Francisco
country: United States
facility: PicnicHealth
state: California
Study officials:
affiliation: BioMarin Pharmaceutical
name: Medical Director, MD
role: STUDY_DIRECTOR
References: