A Participant-mediated Observational Virtual Registry of Children With Achondroplasia in the United States

Achondroplasia Clinical Trial

— VISTA  

Official title:

A Participant-mediated Observational Virtual Registry of Children With Achondroplasia in the United States

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06168201
• Other study ID #: 111-605
• Status: Recruiting
• Start date: February 21, 2023
• Est. completion date: February 2028

Study information

• Verified date: December 2023
• Source: BioMarin Pharmaceutical
• Contact: Trial Specialist
• Phone: 1-800-938-4587
• Email: medinfo[@]bmrn.com
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial Summary

This is an observational study of a pediatric cohort with achondroplasia in the United States. This cohort consists of both individuals treated and untreated with VOXZOGO™. Study enrollment started in February 2023. The projected total duration of the study is approximately 5 years, 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:

Participant-mediated access to electronic health records 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

The primary study population will include 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)

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 300
• Est. completion date: February 2028
• Est. primary completion date: February 2028
• Accepts healthy volunteers: No
• Gender: All
• Age group: 0 Months to 18 Years

Eligibility

Inclusion Criteria:

• Physician diagnosis of achondroplasia

• Age at time of enrollment:

• 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

Related Conditions & MeSH terms

• Achondroplasia

Locations

Country	Name	City	State
United States	PicnicHealth	San Francisco	California

Sponsors (2)

Lead Sponsor	Collaborator
BioMarin Pharmaceutical	PicnicHealth

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Height	sitting and standing measured in centimetres	Retrospective data and prospective data for up to 5 years starting from time of study enrolment
Primary	Weight	measured in kilograms	Retrospective data and prospective data for up to 5 years starting from time of study enrolment
Primary	Body Mass Index	measured in kg/m2	Retrospective data and prospective data for up to 5 years starting from time of study enrolment
Primary	Head Circumference	measured in centimeters	Retrospective data and prospective data for up to 5 years starting from time of study enrolment
Primary	Annualized growth velocity (AGV)	measured in centimeters/year	Retrospective data and prospective data for up to 5 years starting from time of study enrolment
Primary	Medical diagnoses by age	Clinical examination and/or radiological assessment	Retrospective data and prospective data for up to 5 years starting from time of study enrolment
Primary	Surgical procedures and/or medical interventions by age		Retrospective data and prospective data for up to 5 years starting from time of study enrolment
Primary	Changes in physical functioning	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
Primary	Changes in health-related quality of life (HRQoL)	assessed using the Pediatric Quality of Life Inventory (PedsQL)	every 6 months starting from time of study enrolment up to 5 years
Primary	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.	every 6 months starting from time of study enrolment up to 5 years