VIrtual STudy in Achondroplasia for the US (VISTA)
ConditionAchondroplasia
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age0-18
SponsorBioMarin Pharmaceutical
About this trial
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
This trial does not accept healthy volunteersQualifiers
Physician diagnosis of achondroplasia
Receiving medical care in the United States
Complete PicnicHealth's onboarding process, including signing informed consent and authorization for medical record retrieval
Disqualifiers
Lack of any medical records
Trial 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
Trial design
Design model
Cohort
Time perspective
Other
Treatments tested in this trial
Not listed
Trial groups
170 Participants
are grouped into 2 trial groupsGroup A: Primary Pediatric Cohort
Group B: Secondary Adolescent and Adult Cohort
Trial outcomes
Primary outcomes
1
Height
sitting and standing measured in centimetres
Time frame
Retrospective data and prospective data for up to 5 years starting from time of study enrolment2
Weight
measured in kilograms
Time frame
Retrospective data and prospective data for up to 5 years starting from time of study enrolment3
Body Mass Index
measured in kg/m2
Time frame
Retrospective data and prospective data for up to 5 years starting from time of study enrolment4
Head Circumference
measured in centimeters
Time frame
Retrospective data and prospective data for up to 5 years starting from time of study enrolmentSponsors and contacts
Click on the lead sponsor to view all of their trials.
BioMarin Pharmaceutical
Lead sponsor
PicnicHealth
Collaborator
Source ClinicalTrials.gov