Infantile Nystagmus SyndromePhase 4NCT01312402
Topical Brinzolamide Ophthalmic Suspension Versus Placebo in the Treatment of Infantile Nystagmus Syndrome
A Phase 4 study testing topical brinzolamide 1% in 5mL ophthalmic medication in people with Infantile Nystagmus Syndrome, run by Akron Children's Hospital at 1 site in United States.
Trial summary
This study is a prospective, single crossover, double-masked, controlled clinical trial that will use topical brinzolamide (Azopt)ophthalmic medication to try to improve the nystagmus and visual consequences of nystagmus in patients with infantile nystagmus syndrome (INS). Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Infantile Nystagmus Syndrome
- What is being tested?
- topical brinzolamide 1% in 5mL ophthalmic medication
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 5 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2011to Mar 2014
- Phase
- Phase 4
- 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
- Age; greater than 12 years old and able to cooperate for full study protocol
- Subject able to understand and sign informed consent
- Subject able to participate in complete ophthalmic and ocular motility evaluation
- Subjects with Infantile Nystagmus Syndrome diagnosed by clinical evaluation and eye movement recordings
- Best-binocular visual acuity in null position 20/50 to 20/200 inclusive using ATS or ETDRS vision testing
- Subject/family able and willing to make the required study visits
- No previous ophthalmic treatment for nystagmus other than for refractive error
Who cannot join
- Any current use of systemic or topical medications (traditional or non-traditional)
- History of ocular surgery, trauma or chronic ocular disease other than amblyopia
- Systemic diseases requiring medication or other treatments that are known to affect the ocular motor system (e.g., depression, seizure disorders, psychosis)
- Behavioral or neurological disorders which interfere with the study
- Physical or mental impairment precluding study compliance
- Participation in any study involving an IND investigational drug within the past year
Show all 9Show fewer
- Individual (female) is pregnant, nursing or planning a pregnancy (The safety of Azopt for use during pregnancy has NOT been determined.)
- Periodicity or aperiodicity of INS present on eye movement recordings
- Allergy to sulfa or other components of Azopt solution
Trial locations and contacts
1 site in 1 country.
About this study
This study is a prospective, single crossover, double-masked, controlled clinical trial that will use topical brinzolamide (Azopt)ophthalmic medication to try to improve the nystagmus and visual consequences of nystagmus in patients with infantile nystagmus syndrome (INS). Subjects will undergo a clinical exam, questionnaire and eye movement recordings on day 1 and then receive either topical Azopt or placebo three times a day in both eyes for days 2,3 and 4 followed on the morning of day 5 by a repeat clinical exam, questionnaire and eye movement recordings. After at least one week, this protocol is repeated with the crossover regimen being taken by the subject. One week after all medications are discontinued, another clinical exam is done before study discharge. The hypothesis is that nystagmus and associated visual symptoms will be improved while on the Azopt compared to the placebo. There will be a total of 5 visits over a 1-2 month period.
Read the detailed description
5 subjects are expected to be enrolled in the study. Each subject will be in the study for approximately 1 month.
Efficacy will be assessed by:
- ETDRS Visual Acuity Testing of Binocular Best Corrected Visual Acuity In The Nystagmus Null Zone
- The Validated Amblyopia \& Strabismus Ocular Motor Questionnaire
- Eye Movement Recording Data Analysis of The Nystagmus Waveform
Safety will be evaluated by:
- Ocular signs and symptoms
- Visual acuity (uncorrected and best corrected)
- Slit lamp exam and Intraocular Pressure
- Systemic signs and symptoms
- Conditions
- Infantile Nystagmus Syndrome
- Being tested
- topical brinzolamide 1% in 5mL ophthalmic medication, Placebo in 5 mL dispenser
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
topical brinzolamide 1% in 5mL ophthalmic medicationDrug
Also called Azopt 1% in 5 mL
1 drop three times a day in both eyes on days 2,3 and 4
Placebo in 5 mL dispenserDrug
Placebo ophthalmic solution in 5 mL dispenser; 1 drop three times a day on days 2,3 and 4
| Study group | Role | What they receive |
|---|---|---|
| Topical Brinzolamide (Azopt) | Active comparator | ophthalmic drop given three times a day Receives: Drug: topical brinzolamide 1% in 5mL ophthalmic medication |
| placebo ophthalmic drop in 5 mL solution | Placebo comparator | masked non-active eye drop (absence of Brinzolamide) Receives: Drug: Placebo in 5 mL dispenser |
What is being measured
The primary outcome is the main question the study is designed to answer.
Best Corrected Binocular Visual Acuity
Early Treatment of Diabetic Retinopathy Study Vision Testing Protocol of Best corrected Binocular Acuity
Measured: 1 week
Secondary outcomes (1)
Visual Function
THe Validated Amblyopia and Visual Function Questionnaire will be administered
Measured: 5 Days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Mar 2011 (estimated)
- Study startJan 2011
- Primary completionMar 2014
- Study completionMar 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (7)Papers the sponsor has linked to this study.
- Other trials for Infantile Nystagmus SyndromeStudies 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 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Akron Children's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Richard W. Hertle, Akron Children's Hospital
- Investigators
- Richard W. Hertle, M.D., Principal investigator, Akron Children's HospitalDongsheng Yang, Ph.D., Principal investigator, Akron Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01312402
- Sponsor's protocol ID
- 101109
- Status last verified
- Jun 2014
- First submitted
- 21 Jan 2011
- Last updated
- 25 Jun 2014
- Results posted
- No
- Data monitoring committee
- No
Publications
Dell'Osso LF, Jacobs JB. An expanded nystagmus acuity function: intra- and intersubject prediction of best-corrected visual acuity. Doc Ophthalmol. 2002 May;104(3):249-76. doi: 10.1023/a:1015299930849.
PubMed 12076015Hertle RW, Dell'Osso LF, FitzGibbon EJ, Thompson D, Yang D, Mellow SD. Horizontal rectus tenotomy in patients with congenital nystagmus: results in 10 adults. Ophthalmology. 2003 Nov;110(11):2097-105. doi: 10.1016/S0161-6420(03)00802-9.
PubMed 14597515Hertle RW, Anninger W, Yang D, Shatnawi R, Hill VM. Effects of extraocular muscle surgery on 15 patients with oculo-cutaneous albinism (OCA) and infantile nystagmus syndrome (INS). Am J Ophthalmol. 2004 Dec;138(6):978-87. doi: 10.1016/j.ajo.2004.07.029.
PubMed 15629289Hertle RW, Dell'Osso LF, FitzGibbon EJ, Yang D, Mellow SD. Horizontal rectus muscle tenotomy in children with infantile nystagmus syndrome: a pilot study. J AAPOS. 2004 Dec;8(6):539-48. doi: 10.1016/j.jaapos.2004.08.005.
PubMed 15616501Jacobs JB, Dell'Osso LF, Hertle RW, Acland GM, Bennett J. Eye movement recordings as an effectiveness indicator of gene therapy in RPE65-deficient canines: implications for the ocular motor system. Invest Ophthalmol Vis Sci. 2006 Jul;47(7):2865-75. doi: 10.1167/iovs.05-1233.
PubMed 16799026Jacobs JB, Dell'Osso LF, Wang ZI, Acland GM, Bennett J. Using the NAFX to measure the effectiveness over time of gene therapy in canine LCA. Invest Ophthalmol Vis Sci. 2009 Oct;50(10):4685-92. doi: 10.1167/iovs.09-3387. Epub 2009 May 20.
PubMed 19458334Hertle RW, Yang D, Adkinson T, Reed M. Topical brinzolamide (Azopt) versus placebo in the treatment of infantile nystagmus syndrome (INS). Br J Ophthalmol. 2015 Apr;99(4):471-6. doi: 10.1136/bjophthalmol-2014-305915. Epub 2014 Oct 21.
PubMed 25336575
Study at a glance
From the registry record, last updated by the sponsor on 25 Jun 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Topical Brinzolamide Ophthalmic Suspension Versus Placebo in the Treatment of Infantile Nystagmus Syndrome
- ClinicalTrials.gov ID
- NCT01312402
- Acronym / sponsor ID
- 101109
- Conditions
- Infantile Nystagmus Syndrome
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Akron Children's HospitalUniversity, hospital or other
- Study start
- Jan 2011
- Primary completion
- Mar 2014 (actual)
- Study completion
- Mar 2014 (actual)
- First posted
- 10 Mar 2011 (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 a prospective, single crossover, double-masked, controlled clinical trial that will use topical brinzolamide (Azopt)ophthalmic medication to try to improve the nystagmus and visual consequences of nystagmus in patients with infantile nystagmus syndrome (INS). Subjects will undergo a clinical exam, questionnaire and eye movement recordings on day 1 and then receive either topical Azopt or placebo three times a day in both eyes for days 2,3 and 4 followed on the morning of day 5 by a repeat clinical exam, questionnaire and eye movement recordings. After at least one week, this protocol is repeated with the crossover regimen being taken by the subject. One week after all medications are discontinued, another clinical exam is done before study discharge. The hypothesis is that nystagmus and associated visual symptoms will be improved while on the Azopt compared to the placebo. There will be a total of 5 visits over a 1-2 month period.
Detailed description
5 subjects are expected to be enrolled in the study. Each subject will be in the study for approximately 1 month.
Efficacy will be assessed by:
- ETDRS Visual Acuity Testing of Binocular Best Corrected Visual Acuity In The Nystagmus Null Zone
- The Validated Amblyopia \& Strabismus Ocular Motor Questionnaire
- Eye Movement Recording Data Analysis of The Nystagmus Waveform
Safety will be evaluated by:
- Ocular signs and symptoms
- Visual acuity (uncorrected and best corrected)
- Slit lamp exam and Intraocular Pressure
- Systemic signs and symptoms
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Topical Brinzolamide (Azopt) | Active comparator | Drug: topical brinzolamide 1% in 5mL ophthalmic medicationophthalmic drop given three times a day |
| placebo ophthalmic drop in 5 mL solution | Placebo comparator | Drug: Placebo in 5 mL dispensermasked non-active eye drop (absence of Brinzolamide) |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Age; greater than 12 years old and able to cooperate for full study protocol
- Subject able to understand and sign informed consent
- Subject able to participate in complete ophthalmic and ocular motility evaluation
- Subjects with Infantile Nystagmus Syndrome diagnosed by clinical evaluation and eye movement recordings
- Best-binocular visual acuity in null position 20/50 to 20/200 inclusive using ATS or ETDRS vision testing
Key exclusion criteria (9 listed)
- Any current use of systemic or topical medications (traditional or non-traditional)
- History of ocular surgery, trauma or chronic ocular disease other than amblyopia
- Systemic diseases requiring medication or other treatments that are known to affect the ocular motor system (e.g., depression, seizure disorders, psychosis)
- Behavioral or neurological disorders which interfere with the study
- Physical or mental impairment precluding study compliance
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 |
|---|---|---|
| topical brinzolamide 1% in 5mL ophthalmic medicationAlso: Azopt 1% in 5 mL1 drop three times a day in both eyes on days 2,3 and 4 | Drug | Topical Brinzolamide (Azopt) |
| Placebo in 5 mL dispenserPlacebo ophthalmic solution in 5 mL dispenser; 1 drop three times a day on days 2,3 and 4 | Drug | placebo ophthalmic drop in 5 mL solution |
Primary outcome
Best Corrected Binocular Visual Acuity
Time frame: 1 week
Early Treatment of Diabetic Retinopathy Study Vision Testing Protocol of Best corrected Binocular Acuity
1 secondary outcome
Visual Function
Time frame: 5 Days
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 25 Jun 2014.
- Background: PubMed 12076015Dell'Osso LF, Jacobs JB. An expanded nystagmus acuity function: intra- and intersubject prediction of best-corrected visual acuity. Doc Ophthalmol. 2002 May;104(3):249-76. doi: 10.1023/a:1015299930849.
- Background: PubMed 14597515Hertle RW, Dell'Osso LF, FitzGibbon EJ, Thompson D, Yang D, Mellow SD. Horizontal rectus tenotomy in patients with congenital nystagmus: results in 10 adults. Ophthalmology. 2003 Nov;110(11):2097-105. doi: 10.1016/S0161-6420(03)00802-9.
- Background: PubMed 15629289Hertle RW, Anninger W, Yang D, Shatnawi R, Hill VM. Effects of extraocular muscle surgery on 15 patients with oculo-cutaneous albinism (OCA) and infantile nystagmus syndrome (INS). Am J Ophthalmol. 2004 Dec;138(6):978-87. doi: 10.1016/j.ajo.2004.07.029.
- Background: PubMed 15616501Hertle RW, Dell'Osso LF, FitzGibbon EJ, Yang D, Mellow SD. Horizontal rectus muscle tenotomy in children with infantile nystagmus syndrome: a pilot study. J AAPOS. 2004 Dec;8(6):539-48. doi: 10.1016/j.jaapos.2004.08.005.
- Background: PubMed 16799026Jacobs JB, Dell'Osso LF, Hertle RW, Acland GM, Bennett J. Eye movement recordings as an effectiveness indicator of gene therapy in RPE65-deficient canines: implications for the ocular motor system. Invest Ophthalmol Vis Sci. 2006 Jul;47(7):2865-75. doi: 10.1167/iovs.05-1233.
- Background: PubMed 19458334Jacobs JB, Dell'Osso LF, Wang ZI, Acland GM, Bennett J. Using the NAFX to measure the effectiveness over time of gene therapy in canine LCA. Invest Ophthalmol Vis Sci. 2009 Oct;50(10):4685-92. doi: 10.1167/iovs.09-3387. Epub 2009 May 20.
- Derived from this study: PubMed 25336575Hertle RW, Yang D, Adkinson T, Reed M. Topical brinzolamide (Azopt) versus placebo in the treatment of infantile nystagmus syndrome (INS). Br J Ophthalmol. 2015 Apr;99(4):471-6. doi: 10.1136/bjophthalmol-2014-305915. Epub 2014 Oct 21.
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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.
Richard W. Hertle, M.D. principal investigator
Akron Children's Hospital
Dongsheng Yang, Ph.D. principal investigator
Akron Children's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing topical brinzolamide 1% in 5mL ophthalmic medication in people with Infantile Nystagmus Syndrome, run by Akron Children's Hospital 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 7 inclusion and 9 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 25 Jun 2014. 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. Some participants in this study may receive a placebo. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
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.
What does the primary outcome measure?
Best Corrected Binocular Visual Acuity (time frame: 1 week). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01312402, which also lists 7 linked publications.
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 25 Jun 2014.