Niemann Pick DiseasesPhase 1NCT00316498
Saccadic Eye Movements in Patients With Niemann-Pick Type C Disease
A Phase 1 study testing OGT918 in people with Niemann Pick Diseases, run by National Eye Institute (NEI) at 3 sites in 2 countries.
Trial summary
This study is done in conjunction with a trial, conducted at Columbia University College of Physicians and Surgeons in New York and the Royal Manchester Children's Hospital in England, to examine the effectiveness of a new drug called OGT 918 for treating Niemann-Pick Type C (NPC) disease. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Niemann Pick Diseases
- What is being tested?
- OGT918
- How many people
- 30 participants
- Where
- 3 sitesin 2 countries
- Study dates
- 17 Oct 2002to 15 Aug 2007
- Phase
- Phase 1
- 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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients with NPC disease confirmed by abnormal cholesterol esterification and abnormal filipin staining.
- Patients aged 12 and over. Body weight must not be less than 14 kg.
- Patients who can ingest a capsule.
Who cannot join
- Patients younger than 18 who are unable to give informed assent and/or whose legal guardian is unable to provide informed consent.
- Patients aged 18 and over who cannot provide informed consent and/or whose legal guardian is unable to provide witnessed informed consent.
- Fertile patients who do not agree to use adequate contraception throughout the study and for three months after cessation of OGT 918 treatment.
- Patients who cannot tolerate the study procedures or who are unable to travel to the study center as required by this protocol.
- Patients currently undergoing therapy with other investigational agents or patients taking drugs or food supplements which may interfere with gastrointestinal absorption or motility.
- Patients suffering from clinically significant diarrhoea (greater than 3 liquid stools per day for greater than 7 days) without definable cause within 3 months of Screening Visit, or who have a history of significant gastrointestinal disorders.
- Patients with an intercurrent medical condition that would render them unsuitable for the study e.g. HIV, hepatitis infection.
- Patients who in the opinion of the investigator (for whatever reason) are thought to be unsuitable for the study.
Trial locations and contacts
3 sites in 2 countries.
3 sites in 2 countries
United States2 sites
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, 20892
Columbia University
New York, New York, 10032-3784
About this study
Official title: A Phase I/II Randomized, Controlled Study of OGT 918 in Adult and Juvenile Patients With Niemann Pick C Disease
This study is done in conjunction with a trial, conducted at Columbia University College of Physicians and Surgeons in New York and the Royal Manchester Children's Hospital in England, to examine the effectiveness of a new drug called OGT 918 for treating Niemann-Pick Type C (NPC) disease. Patients with this genetic disorder do not transport lipids (fatty substances) in their cells, resulting in problems of the liver, spleen and brain. An early sign of NPC is a reduced ability to move the eyes rapidly up and down or from side to side. These voluntary eye movements are called saccades. Patients in the OGT 918 trial who participate in this sub-study will have their saccadic eye movements measured to see if improvement occurs with OGT 918 treatment.
Patients with Niemann-Pick Type C disease 12 years of age and older who are enrolled in the OGT 918 trial described above may be eligible for this study.
Participants will have both vertical (up and down) and horizontal (side to side) saccadic eye movements measured at two time points before starting treatment with OGT 918 and after 12 months of treatment. For the test, patients sit in a chair with their head positioned as for a regular eye examination (steadied by a chin cup and headrest) and follow with their eyes a series of lights or laser spots moving on a screen at a distance of 1 meter (3 feet). During the test, patients wear either special recording glasses, infrared goggles, or special contact lenses for measuring eye movements. A full eye evaluation lasts about 1 hour, and each eye is evaluated twice. The evaluations are separated in time by at least an hour, and possibly a day.
Read the detailed description
None given, verified by PI.
- Conditions
- Niemann Pick Diseases
- Being tested
- OGT918
- Keywords
- Eye Movement, Niemann Pick C
- Medical subject headings
- Niemann-Pick Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OGT918Drug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Apr 2006 (estimated)
- Study start17 Oct 2002
- Study completion15 Aug 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Niemann Pick DiseasesStudies of the same condition, open and under way.
- 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
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Enrolment
- 30 participants
Sponsor and investigators
- Lead sponsor
- National Eye Institute (NEI)
- Sponsor type
- US National Institutes of Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00316498
- Sponsor's protocol ID
- 030011
- Other IDs
- 03-EI-0011
- Status last verified
- 15 Aug 2007
- First submitted
- 19 Apr 2006
- Last updated
- 2 Jul 2017
- Results posted
- No
Publications
Platt FM, Neises GR, Reinkensmeier G, Townsend MJ, Perry VH, Proia RL, Winchester B, Dwek RA, Butters TD. Prevention of lysosomal storage in Tay-Sachs mice treated with N-butyldeoxynojirimycin. Science. 1997 Apr 18;276(5311):428-31. doi: 10.1126/science.276.5311.428.
PubMed 9103204
Study at a glance
From the registry record, last updated by the sponsor on 2 Jul 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Randomized, Controlled Study of OGT 918 in Adult and Juvenile Patients With Niemann Pick C Disease
- ClinicalTrials.gov ID
- NCT00316498
- Acronym / sponsor ID
- 030011
- Conditions
- Niemann Pick Diseases
- Keywords
- Eye Movement, Niemann Pick C
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants
- Lead sponsor
- National Eye Institute (NEI)US National Institutes of Health
- Study start
- 17 Oct 2002
- Study completion
- 15 Aug 2007
- First posted
- 20 Apr 2006 (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.
This study is done in conjunction with a trial, conducted at Columbia University College of Physicians and Surgeons in New York and the Royal Manchester Children's Hospital in England, to examine the effectiveness of a new drug called OGT 918 for treating Niemann-Pick Type C (NPC) disease. Patients with this genetic disorder do not transport lipids (fatty substances) in their cells, resulting in problems of the liver, spleen and brain. An early sign of NPC is a reduced ability to move the eyes rapidly up and down or from side to side. These voluntary eye movements are called saccades. Patients in the OGT 918 trial who participate in this sub-study will have their saccadic eye movements measured to see if improvement occurs with OGT 918 treatment.
Patients with Niemann-Pick Type C disease 12 years of age and older who are enrolled in the OGT 918 trial described above may be eligible for this study.
Participants will have both vertical (up and down) and horizontal (side to side) saccadic eye movements measured at two time points before starting treatment with OGT 918 and after 12 months of treatment. For the test, patients sit in a chair with their head positioned as for a regular eye examination (steadied by a chin cup and headrest) and follow with their eyes a series of lights or laser spots moving on a screen at a distance of 1 meter (3 feet). During the test, patients wear either special recording glasses, infrared goggles, or special contact lenses for measuring eye movements. A full eye evaluation lasts about 1 hour, and each eye is evaluated twice. The evaluations are separated in time by at least an hour, and possibly a day.
Detailed description
None given, verified by PI.
Study design
- Primary purpose
- Treatment
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Patients with NPC disease confirmed by abnormal cholesterol esterification and abnormal filipin staining.
- Patients aged 12 and over. Body weight must not be less than 14 kg.
- Patients who can ingest a capsule.
Key exclusion criteria (8 listed)
- Patients younger than 18 who are unable to give informed assent and/or whose legal guardian is unable to provide informed consent.
- Patients aged 18 and over who cannot provide informed consent and/or whose legal guardian is unable to provide witnessed informed consent.
- Fertile patients who do not agree to use adequate contraception throughout the study and for three months after cessation of OGT 918 treatment.
- Patients who cannot tolerate the study procedures or who are unable to travel to the study center as required by this protocol.
- Patients currently undergoing therapy with other investigational agents or patients taking drugs or food supplements which may interfere with gastrointestinal absorption or motility.
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 |
|---|---|---|
| OGT918 | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Jul 2017.
- Background: PubMed 9103204Platt FM, Neises GR, Reinkensmeier G, Townsend MJ, Perry VH, Proia RL, Winchester B, Dwek RA, Butters TD. Prevention of lysosomal storage in Tay-Sachs mice treated with N-butyldeoxynojirimycin. Science. 1997 Apr 18;276(5311):428-31. doi: 10.1126/science.276.5311.428.
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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.
The record lists no contact. Use the official record to reach the sponsor.
3 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing OGT918 in people with Niemann Pick Diseases, run by National Eye Institute (NEI) at 3 sites in 2 countries.
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 3 inclusion and 8 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: finished, as of the sponsor's last update on 2 Jul 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States, United Kingdom. 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.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00316498, which also lists 1 linked publication.
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 2 Jul 2017.