VIrtual STudy in Achondroplasia for the US (VISTA) (VISTA)

December 11, 2025 updated by: BioMarin Pharmaceutical

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
170

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • United States
    • California
      • Oakland, California, United States, 94609
        • Recruiting
        • UCSF
        • Contact:
          • Hind Al Saif
        • Principal Investigator:
          • Hind Al Saif
      • San Francisco, California, United States, 94107
    • North Carolina
      • Statesville, North Carolina, United States, 28625
        • Recruiting
        • Piedmont Health
        • Contact:
          • Samuel Hughes
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Recruiting
        • University of Cincinnati
        • Contact:
          • Laurie Bailey
    • South Dakota
      • Sioux Falls, South Dakota, United States, 57105
        • Recruiting
        • Sanford Health
        • Contact:
          • Isum Ward
    • Texas
      • Houston, Texas, United States, 77030-2316
        • Recruiting
        • Baylor College of Medicine
        • Contact:
          • Carlos Bacino
    • Wisconsin
      • Madison, Wisconsin, United States, 53726
        • Recruiting
        • University of Wisconsin
        • Contact:
          • Janet Legare

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

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

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Primary Pediatric Cohort
≤13 years old at time of enrollment. This is an observational study.
Secondary Adolescent and Adult Cohort
≥14 years old at time of enrollment. This is an observational study.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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
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
measured in kilograms
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
measured in kg/m2
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
measured in centimeters
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
measured in centimeters/year
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
Clinical examination and/or radiological assessment
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
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
assessed using the Mobility and Upper Extremity Patient-Reported Outcomes Measurement Information System (PROMIS) tools
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
assessed using the Pediatric Quality of Life Inventory (PedsQL)
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
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
every 6 months starting from time of study enrolment up to 5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
BioMarin Pharmaceutical

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
PicnicHealth

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Medical Director, MD, BioMarin Pharmaceutical

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
February 21, 2023

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
February 1, 2028

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
February 1, 2028

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
November 2, 2023

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 10, 2023

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
December 13, 2023

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
December 18, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 11, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
December 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • 111-605

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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