Rare Genetic DisordersNCT02365376
Collection and Storage of Human Biospecimens for Research Into Rare Diseases and Medical Conditions
An observational study of Rare Genetic Disorders, run by BioMarin Pharmaceutical at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Rare Genetic Disorders
- How many people
- Not given
- Where
- 1 siteUnited States
- Study dates
- Jun 2013to not given
- Phase
- Observational
- Are there results?
- None posted in the registry
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Individuals diagnosed with rare genetic diseases or medical conditions actively being investigated by BioMarin.
Who may be able to join
- Willing and able to provide written, signed informed consent
- Willing and able to comply with all study procedures
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site, 1 recruiting
- Recruiting
Lurie Children's Hospital of Chicago
Chicago, Illinois
About this study
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
What is being measured
The primary outcome is the main question the study is designed to answer.
continuous collection of donated study subject samples
Measured: unknown, continuous sample collection
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Feb 2015 (estimated)
- Study startJun 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Rare Genetic DisordersStudies 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
- Case only
Sponsor and investigators
- Lead sponsor
- BioMarin Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02365376
- Sponsor's protocol ID
- BMN 000-901
- Status last verified
- Feb 2015
- First submitted
- 11 Feb 2015
- Last updated
- 18 Feb 2015
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Feb 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Collection and Storage of Human Biospecimens for Research Into Rare Diseases and Medical Conditions
- ClinicalTrials.gov ID
- NCT02365376
- Acronym / sponsor ID
- BMN 000-901
- Conditions
- Rare Genetic Disorders
- Study type
- Observational
- Recruitment status
- Unknown status
- Lead sponsor
- BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
- Study start
- Jun 2013
- First posted
- 18 Feb 2015 (estimated)
- 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.
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 design
- Observational model
- Case only
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Individuals diagnosed with rare genetic diseases or medical conditions actively being investigated by BioMarin.
Key inclusion criteria (2 listed)
- Willing and able to provide written, signed informed consent
- Willing and able to comply with all study procedures
Key exclusion criteria (2 listed)
- 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.
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
continuous collection of donated study subject samples
Time frame: unknown, continuous sample collection
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Feb 2015.
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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. This study is not recruiting at the moment.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Rare Genetic Disorders, run by BioMarin Pharmaceutical at 1 site 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 2 inclusion and 2 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: status not confirmed, as of the sponsor's last update on 18 Feb 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Evelyn Wang (415-506-6818, EWang@bmrn.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
continuous collection of donated study subject samples (time frame: unknown, continuous sample collection). 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 18 Feb 2015.