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NCT Number: NCT06168201

VIrtual STudy in Achondroplasia for the US (VISTA)

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.

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Key information

Conditions

Achondroplasia Bone Diseases Bone Diseases, Developmental Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dwarfism Genetic Diseases, Inborn Musculoskeletal Diseases Osteochondrodysplasias

Age range

0 month–18 year

Sex eligibility

All sexes

Study type

Observational

Primary location

UCSF, Oakland, California, United States

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Primary outcomes

  1. Height

    Time frame: Retrospective data and prospective data for up to 5 years starting from time of study enrolment

    sitting and standing measured in centimetres

  2. Weight

    Time frame: Retrospective data and prospective data for up to 5 years starting from time of study enrolment

    measured in kilograms

  3. Body Mass Index

    Time frame: Retrospective data and prospective data for up to 5 years starting from time of study enrolment

    measured in kg/m2

  4. Head Circumference

    Time frame: Retrospective data and prospective data for up to 5 years starting from time of study enrolment

    measured in centimeters

  5. Annualized growth velocity (AGV)

    Time frame: Retrospective data and prospective data for up to 5 years starting from time of study enrolment

    measured in centimeters/year

  6. Medical diagnoses by age

    Time frame: Retrospective data and prospective data for up to 5 years starting from time of study enrolment

    Clinical examination and/or radiological assessment

  7. 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

  8. Changes in physical functioning

    Time frame: every 6 months starting from time of study enrolment up to 5 years

    assessed using the Mobility and Upper Extremity Patient-Reported Outcomes Measurement Information System (PROMIS) tools

  9. Changes in health-related quality of life (HRQoL)

    Time frame: every 6 months starting from time of study enrolment up to 5 years

    assessed using the Pediatric Quality of Life Inventory (PedsQL)

  10. Adherence to treatment with VOXZOGO™ (vosoritide), if prescribed

    Time frame: every 6 months starting from time of study enrolment up to 5 years

    assessed using a medication adherence questionnaire to capture the number of missed doses in the prior month. Parent(ObsRO) version for all participants enrolled ages 5-13 years

Study contacts

Contact information is provided by the study sponsor or research team.

Trial Specialist

CONTACT

[email protected]

1-800-938-4587

Sponsors and collaborators

Lead sponsor

BioMarin Pharmaceutical

Industry

Collaborators

  • PicnicHealth

Registry information

Acronym: VISTA

Important dates

Study start
2023
Primary completion
2028
Study completion
2028
First posted
Dec 13, 2023
Registry last updated
Jun 30, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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