AchondroplasiaVISTANCT06168201
VIrtual STudy in Achondroplasia for the US (VISTA)
An observational study of Achondroplasia, run by BioMarin Pharmaceutical at 8 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 0 months to 18 years
- Conditions the study is for
- Achondroplasia
- How many people
- 170 participants
- Where
- 8 sitesUnited States
- Study dates
- 21 Feb 2023to Feb 2028
- How long will I take part?
- 5 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 months to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
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
Who may be able to join
- 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
Who cannot join
- Lack of any medical records
Trial locations and contacts
8 sites in 1 country. Contact the study team or a site near you to ask about taking part.
8 sites in 1 country
United States8 sites, 8 recruiting
- Recruiting
Johns Hopkins University (JHU)
Baltimore, Maryland, 21205
Julie Hoover-Fong
- Recruiting
University of Cincinnati
Cincinnati, Ohio, 45229
Laurie Bailey
- Recruiting
Baylor College of Medicine
Houston, Texas, 77030-2316
Carlos Bacino
- Recruiting
University of Wisconsin
Madison, Wisconsin, 53726
Janet Legare
- Recruiting
UCSF
Oakland, California, 94609
Hind Al Saif
Hind Al Saif
- Recruiting
PicnicHealth
San Francisco, California, 94107
PicnicHealth, hello@picnichealth.com
- Recruiting
Sanford Health
Sioux Falls, South Dakota, 57115
Isum Ward
- Recruiting
Piedmont Health
Statesville, North Carolina, 28625
Samuel Hughes
About this study
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:
Read more
- 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. .
- 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.
- Conditions
- Achondroplasia
- Medical subject headings
- Achondroplasia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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 being measured
The primary outcome is the main question the study is designed to answer.
Height
sitting and standing measured in centimetres
Measured: Retrospective data and prospective data for up to 5 years starting from time of study enrolment
Weight
measured in kilograms
Measured: Retrospective data and prospective data for up to 5 years starting from time of study enrolment
Body Mass Index
measured in kg/m2
Measured: Retrospective data and prospective data for up to 5 years starting from time of study enrolment
Head Circumference
measured in centimeters
Measured: Retrospective data and prospective data for up to 5 years starting from time of study enrolment
Annualized growth velocity (AGV)
measured in centimeters/year
Measured: Retrospective data and prospective data for up to 5 years starting from time of study enrolment
Medical diagnoses by age
Clinical examination and/or radiological assessment
Measured: Retrospective data and prospective data for up to 5 years starting from time of study enrolment
Surgical procedures and/or medical interventions by age
Measured: Retrospective data and prospective data for up to 5 years starting from time of study enrolment
Changes in physical functioning
assessed using the Mobility and Upper Extremity Patient-Reported Outcomes Measurement Information System (PROMIS) tools
Measured: every 6 months starting from time of study enrolment up to 5 years
Changes in health-related quality of life (HRQoL)
assessed using the Pediatric Quality of Life Inventory (PedsQL)
Measured: every 6 months starting from time of study enrolment up to 5 years
Adherence to treatment with VOXZOGO™ (vosoritide), if prescribed
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
Measured: every 6 months starting from time of study enrolment up to 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Dec 2023
- Study start21 Feb 2023
- Primary completionFeb 2028 (estimated)
- Study completionFeb 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AchondroplasiaStudies of the same condition, open and under way.
- More trials run by BioMarin PharmaceuticalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 170 participants (estimated)
- Target duration
- 5 Years
Sponsor and investigators
- Lead sponsor
- BioMarin Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- PicnicHealth
- Responsible party
- Sponsor
- Investigators
- Medical Director, MD, Study director, BioMarin Pharmaceutical
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06168201
- Sponsor's protocol ID
- 111-605
- Acronym
- VISTA
- Status last verified
- Jun 2026
- First submitted
- 2 Nov 2023
- Last updated
- 30 Jun 2026
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- VIrtual STudy in Achondroplasia for the US (VISTA)
- ClinicalTrials.gov ID
- NCT06168201
- Acronym / sponsor ID
- VISTA / 111-605
- Conditions
- Achondroplasia
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 170 participants (estimated)
- Lead sponsor
- BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
- Collaborators
- PicnicHealth
- Study start
- 21 Feb 2023 (actual)
- Primary completion
- Feb 2028 (estimated)
- Study completion
- Feb 2028 (estimated)
- First posted
- 13 Dec 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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:
- 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. .
- 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 design
- Observational model
- Cohort
- Time perspective
- Other
- Target follow-up
- 5 Years
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 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. |
Who may qualify
- Ages
- 0 months to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- 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
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (1 listed)
- Lack of any medical records
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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
Weight
Time frame: Retrospective data and prospective data for up to 5 years starting from time of study enrolment
measured in kilograms
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
Head Circumference
Time frame: Retrospective data and prospective data for up to 5 years starting from time of study enrolment
measured in centimeters
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
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
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
assessed using the Mobility and Upper Extremity Patient-Reported Outcomes Measurement Information System (PROMIS) tools
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)
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Jun 2026.
Related trials for Achondroplasia
Enrolling by invitationPhase 2
Extension Study of Infigratinib in Children With Achondroplasia (ACH)
Achondroplasia
RecruitingPhase 2/3
Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
Achondroplasia
Active, not recruitingPhase 2
A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide in Adolescents (12 - 18 Years of Age) With Achondroplasia.
Achondroplasia
Active, not recruitingPhase 3
Evaluate the Efficacy and Safety of KK8398 in Patients With Achondroplasia（AOBA Study）
Achondroplasia
RecruitingPhase 2
A Study of TYRA-300 in Children With Achondroplasia: BEACH301
Achondroplasia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Medical Director, MD study director
BioMarin Pharmaceutical
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Achondroplasia, run by BioMarin Pharmaceutical at 8 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 30 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Trial Specialist (1-800-938-4587, medinfo@bmrn.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Height (time frame: Retrospective data and prospective data for up to 5 years starting from time of study enrolment). The study lists 10 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Jun 2026.