Collection and Storage of Human Biospecimens for Research Into Rare Diseases and Medical Conditions

NCT02365376 | Unknown

• 1 other identifier: BMN 000-901
• Study Type: observational
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

The objective of this protocol is to enable collection of biospecimens to facilitate current and future multidisciplinary research in rare genetic disorders and medical conditions.

Conditions

Rare Genetic Disorders

Outcome Measures

Primary Outcomes (1)

• continuous collection of donated study subject samples

  unknown, continuous sample collection

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Individuals diagnosed with rare genetic diseases or medical conditions actively being investigated by BioMarin.

You may qualify if:

• Willing and able to provide written, signed informed consent
• Willing and able to comply with all study procedures

You may not qualify if:

• Use of any investigational product or investigational medical device within 30 days prior to screening, except as part of a BioMarin-sponsored study
• Concurrent disease or condition that would interfere with study participation or safety.

Sponsors & Collaborators

• BioMarin Pharmaceutical: lead

Study Sites (1)

Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

RECRUITING

Central Study Contacts

Evelyn Wang

CONTACT

415-506-6818; EWang@bmrn.com

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 11, 2015
• First Posted: February 18, 2015
• Study Start: June 1, 2013
• Last Updated: February 18, 2015

Record last verified: 2015-02

Locations

US (1)