- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02365376
Collection and Storage of Human Biospecimens for Research Into Rare Diseases and Medical Conditions
February 11, 2015 updated by: BioMarin Pharmaceutical
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.
Study Overview
Status
Unknown
Conditions
Study Type
Observational
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
- Name: Evelyn Wang
- Phone Number: 415-506-6818
- Email: EWang@bmrn.com
Study Locations
-
-
Illinois
-
Chicago, Illinois, United States
- Recruiting
- Lurie Children's Hospital of Chicago
-
-
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
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Sampling Method
Non-Probability Sample
Study Population
Individuals diagnosed with rare genetic diseases or medical conditions actively being investigated by BioMarin.
Description
Inclusion Criteria:
- Willing and able to provide written, signed informed consent
- Willing and able to comply with all study procedures
Exclusion Criteria:
- 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.
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
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
continuous collection of donated study subject samples
Time Frame: unknown, continuous sample collection
|
unknown, continuous sample collection
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
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
June 1, 2013
Study Registration Dates
First Submitted
February 11, 2015
First Submitted That Met QC Criteria
February 11, 2015
First Posted (Estimate)
February 18, 2015
Study Record Updates
Last Update Posted (Estimate)
February 18, 2015
Last Update Submitted That Met QC Criteria
February 11, 2015
Last Verified
February 1, 2015
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- BMN 000-901
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.
Clinical Trials on Rare Genetic Disorders
-
Rady Pediatric Genomics & Systems Medicine InstituteEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsActive, not recruitingGenetic Diseases | Genetic Syndrome | Mendelian DisordersUnited States
-
Karolinska InstitutetKarolinska University HospitalEnrolling by invitationRare Diseases | Genetic Disease | Chromosome AbnormalitySweden
-
Eunice Kennedy Shriver National Institute of Child...National Institutes of Health Clinical Center (CC); National Institute of Neurological... and other collaboratorsActive, not recruitingGenetic DisorderUnited States
-
Charite University, Berlin, GermanyGerman Federal Ministry of Education and ResearchCompleted
-
International Rett Syndrome FoundationBaylor College of Medicine; Children's Hospital of Philadelphia; University of... and other collaboratorsRecruitingNervous System Diseases | Neurologic Manifestations | Neurobehavioral Manifestations | Genetic Diseases, X-Linked | Intellectual Disability | Neurodevelopmental Disorders | Neurologic Disorder | Rett Syndrome | Genetic Disease | Rett Syndrome, AtypicalUnited States
-
Mayo ClinicNational Cancer Institute (NCI)RecruitingGenetic DisorderUnited States
-
YehCompletedFertility Disorders | Genetic Disorder | Reproductive Disorder
-
Rhythm Pharmaceuticals, Inc.CompletedObesity | Genetic Obesity | Obesity Due to Melanocortin 4 Receptor DeficiencyUnited States, United Kingdom, Germany, Netherlands, France, Canada, Greece, Israel, Spain
-
Eunice Kennedy Shriver National Institute of Child...CompletedObesity | Developmental Delay | Mental Retardation | Genetic DisorderUnited States, Canada
-
Spark TherapeuticsCompletedHematologic Diseases | Hemophilia A | Blood Coagulation Disorders, Inherited | Coagulation Protein Disorders | Hemorrhagic Disorders | Genetic Diseases, Inborn | Genetic Diseases, X-Linked | Blood Coagulation Disorder | Factor VIII DeficiencyUnited States, Australia, Thailand, Canada