Late Infantile Neuronal Ceroid LipofuscinosisNCT01698229
Collection of Cerebrospinal Fluid in Healthy Children
An observational study of Late Infantile Neuronal Ceroid Lipofuscinosis and Batten Disease, run by Weill Medical College of Cornell University at 1 site in United States.
Trial summary
The purpose of this study is to obtain cerebrospinal fluid from healthy children already undergoing a lumbar puncture procedure at New York Presbyterian Hospital, to act as healthy controls when compared to children with late infantile neuronal ceroid lipofuscinosis (LINCL), a fatal neurodegenerative disease. Read more about this study
- Who is it for?
- Ages 2 to 8 yearsHealthy volunteers can take part
- Conditions the study is for
- Late Infantile Neuronal Ceroid Lipofuscinosis, Batten Disease
- What is being tested?
- Group 1
- How many people
- 32 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2011to Dec 2015
- 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 2 to 8 years
- All sexes
- Healthy volunteers accepted
- Child
Study population: Subjects will be healthy controls (children ages 2 years to 8 years) undergoing a lumbar puncture at New York Presbyterian Hospital, for clinical care purposes. They will be inpatients and outpatients.
Who may be able to join
- Males and females, ages 2 yrs to 8 yrs
- Children already undergoing a lumbar puncture procedure for clinical or diagnostic purposes
Who cannot join
- Presence of any significant neurological diseases, as judged by the co-investigators
- Presence of any significant medical conditions including cancer
Trial locations and contacts
1 site in 1 country.
United States1 site
Weill Cornell Medical College / New York Presbyterian Hospital
New York, New York, 10065
About this study
The purpose of this study is to obtain cerebrospinal fluid from healthy children already undergoing a lumbar puncture procedure at New York Presbyterian Hospital, to act as healthy controls when compared to children with late infantile neuronal ceroid lipofuscinosis (LINCL), a fatal neurodegenerative disease.
Read the detailed description
obtain cerebrospinal fluid from healthy children already undergoing a lumbar puncture procedure at New York Presbyterian Hospital, to act as healthy controls when compared to children with late infantile neuronal ceroid lipofuscinosis (LINCL), a fatal neurodegenerative disease.
- Being tested
- Group 1
- Keywords
- Cerebrospinal fluid, Lumbar puncture, Spinal tap, Late infantile neuronal ceroid lipofuscinosis, Batten disease
- Medical subject headings
- Neuronal Ceroid-Lipofuscinoses
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Group 1Other
Collecting a small amount of cerebrospinal fluid (5 mL) from healthy children, ages 2 yrs - 8 yrs, who are already undergoing a lumbar puncture procedure at New York Presbyterian Hospital for clinical or diagnostic purposes.
| Study group | Role | What they receive |
|---|---|---|
| Group 1 | Study cohort is comprised of healthy children, ages 2yrs - 8yrs, who are already undergoing a lumbar puncture procedure at New York Presbyterian Hospital for clinical or diagnostic purposes. Receives: Other: Group 1 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Cerebrospinal fluid (CSF)
The primary study endpoint is the metabolic profiling of 32 samples of cerebrospinal fluid from healthy subjects, in comparison to 32 samples of CSF from children with LINCL, to determine whether there are biomarkers found in the cerebrospinal fluid that are associated with LINCL.
Measured: 1 time point (once)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Oct 2012 (estimated)
- Study startDec 2011
- Primary completionDec 2015
- Study completionDec 2015
Why it stopped: Study interest shifted
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Late Infantile Neuronal Ceroid LipofuscinosisStudies of the same condition, open and under way.
- More trials run by Weill Medical College of Cornell UniversityThe 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
- Time perspective
- Cross-sectional
- Enrolment
- 32 participants (actual)
Sponsor and investigators
- Lead sponsor
- Weill Medical College of Cornell University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Ronald G Crystal, MD, Principal investigator, Weill Medical College of Cornell University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01698229
- Sponsor's protocol ID
- 1111012024
- Status last verified
- May 2016
- First submitted
- 15 Aug 2012
- Last updated
- 4 May 2016
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 May 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Collection of Cerebrospinal Fluid in Healthy Children
- ClinicalTrials.gov ID
- NCT01698229
- Acronym / sponsor ID
- 1111012024
- Keywords
- Cerebrospinal fluid, Lumbar puncture, Spinal tap, Late infantile neuronal ceroid lipofuscinosis, Batten disease
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Study interest shifted
- Enrolment
- 32 participants (actual)
- Lead sponsor
- Weill Medical College of Cornell UniversityUniversity, hospital or other
- Study start
- Dec 2011
- Primary completion
- Dec 2015 (actual)
- Study completion
- Dec 2015 (actual)
- First posted
- 2 Oct 2012 (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 purpose of this study is to obtain cerebrospinal fluid from healthy children already undergoing a lumbar puncture procedure at New York Presbyterian Hospital, to act as healthy controls when compared to children with late infantile neuronal ceroid lipofuscinosis (LINCL), a fatal neurodegenerative disease.
Detailed description
obtain cerebrospinal fluid from healthy children already undergoing a lumbar puncture procedure at New York Presbyterian Hospital, to act as healthy controls when compared to children with late infantile neuronal ceroid lipofuscinosis (LINCL), a fatal neurodegenerative disease.
Study design
- Observational model
- Case only
- Time perspective
- Cross-sectional
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Group 1 | – | Other: Group 1Study cohort is comprised of healthy children, ages 2yrs - 8yrs, who are already undergoing a lumbar puncture procedure at New York Presbyterian Hospital for clinical or diagnostic purposes. |
Who may qualify
- Ages
- 2 to 8 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child
- Study population
- Subjects will be healthy controls (children ages 2 years to 8 years) undergoing a lumbar puncture at New York Presbyterian Hospital, for clinical care purposes. They will be inpatients and outpatients.
Key inclusion criteria (2 listed)
- Males and females, ages 2 yrs to 8 yrs
- Children already undergoing a lumbar puncture procedure for clinical or diagnostic purposes
Key exclusion criteria (2 listed)
- Presence of any significant neurological diseases, as judged by the co-investigators
- Presence of any significant medical conditions including cancer
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Group 1Collecting a small amount of cerebrospinal fluid (5 mL) from healthy children, ages 2 yrs - 8 yrs, who are already undergoing a lumbar puncture procedure at New York Presbyterian Hospital for clinical or diagnostic purposes. | Other | Group 1 |
Primary outcome
Cerebrospinal fluid (CSF)
Time frame: 1 time point (once)
The primary study endpoint is the metabolic profiling of 32 samples of cerebrospinal fluid from healthy subjects, in comparison to 32 samples of CSF from children with LINCL, to determine whether there are biomarkers found in the cerebrospinal fluid that are associated with LINCL.
Results and safety
The study was stopped early (reason given: Study interest shifted). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 May 2016.
- Individual participant dataSharing planned: No
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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.
Ronald G Crystal, MD principal investigator
Weill Medical College of Cornell University
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 Late Infantile Neuronal Ceroid Lipofuscinosis and Batten Disease, run by Weill Medical College of Cornell University 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: stopped early, as of the sponsor's last update on 4 May 2016. 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?
The study was stopped early (reason given: Study interest shifted). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Cerebrospinal fluid (CSF) (time frame: 1 time point (once)). 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 4 May 2016.