Leber's Hereditary Optic Neuropathy (LHON)Phase 1/2LHONNCT01389817
Near-infrared Light-emitting Diode (NIR-LED) Therapy for Leber's Hereditary Optic Neuropathy (LHON)
A Phase 1/2 study testing Near-infrared light-emitting diode (NIR-LED) therapy (Med Light 630 PRO (Medical Devices Inc.)) in people with Leber's Hereditary Optic Neuropathy (LHON), run by Medical College of Wisconsin at 3 sites in United States.
Trial summary
The overall objective of the proposed research is to test the hypothesis that Near-infrared Light-emitting Diode (NIR-LED) therapy will stimulate mitochondrial function, attenuate oxidative stress, and improve cell survival and vision in subjects with Leber's Hereditary Optic Neuropathy (LHON). Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Leber's Hereditary Optic Neuropathy (LHON)
- What is being tested?
- Near-infrared light-emitting diode (NIR-LED) therapy (Med Light 630 PRO (Medical Devices Inc.))
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 3 sitesUnited States
- Study dates
- Jul 2011to May 2013
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 18 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Sex - male and female. Men are affected with visual loss more frequently than women, with a male predominance of ~85%. A minimum of 25% of men and 5% of women at risk for LHON experience visual loss.
- Age - 18 years or older. The onset of visual loss typically occurs between the ages of 15 and 35 years, but otherwise classic LHON has been reported in many individuals both younger and older, with a range of age at onset from 2 to 80 years.
- Diagnosis of LHON or a carrier of the gene mutation. Patient must be evaluated by molecular confirmation, with the 11778 and other mutations of Leber's Hereditary Optic Neuropathy stratified in later data analysis.
- Able \& willing to provide informed consent
Who cannot join
- Has an optic nerve disease other than LHON.
- Significant renal disease, defined as a history of chronic renal failure requiring dialysis or kidney transplant.
- Participation in an investigational trial within 30 days of NIR-LED treatment that involved treatment with any drug that has not received regulatory approval at the time of study entry. (Note: subjects cannot receive another investigational drug while participating in the study during the first 6 months.)
- Major surgery within 28 days prior to participation or major surgery planned during the next 6 months. Major surgery is defined as a surgical procedure that is more extensive than fine needle biopsy/aspiration, placement of a central venous access device, removal/biopsy of a skin lesion, or placement of a peripheral venous catheter.
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Froedtert Memorial Lutheran Hospital
Milwaukee, Wisconsin, 53226
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
The Eye Institute
Milwaukee, Wisconsin, 53226
About this study
The overall objective of the proposed research is to test the hypothesis that Near-infrared Light-emitting Diode (NIR-LED) therapy will stimulate mitochondrial function, attenuate oxidative stress, and improve cell survival and vision in subjects with Leber's Hereditary Optic Neuropathy (LHON).
Read the detailed description
To determine the effects of short term (3 month) near-infrared light NIR-LED therapy on anatomic and functional abnormalities of LHON as assessed by a wide variety of ophthalmologic tests, including visual acuity, optical coherence tomography, pattern electroretinography (pERG) N95 retinal ganglion cell peak, fERG-PhNR (flash ERG - Photopic Negative Response), and fundus photography.
- Being tested
- Near-infrared light-emitting diode (NIR-LED) therapy (Med Light 630 PRO (Medical Devices Inc.))
- Medical subject headings
- Optic Atrophy, Hereditary, Leber
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Near-infrared light-emitting diode (NIR-LED) therapy (Med Light 630 PRO (Medical Devices Inc.))Device
Also called Med Light 630 PRO (Medical Devices Inc.)
Subjects will be exposed to light emitted from a Med Light 630 PRO (Medical Devices Inc.) at a wavelength of 630 nm (+/-15nm) with an exposure of 4 J/cm2. This is accomplished by applying the 50 mW/cm2 LED-generated light to the closed study eye for 80 seconds. Treatments involve application of the LED-generated light for 80 seconds, twice daily.
| Study group | Role | What they receive |
|---|---|---|
| Symptomatic LHON patients. | Experimental | Study Arm 1) symptomatic LHON patients. Male and female LHON patients with treatable bilateral optic atrophy. Treat the worse of the 2 eyes if there is a measurable difference in subjective visual functions (visual acuity, peripheral vision). Receives: Device: Near-infrared light-emitting diode (NIR-LED) therapy (Med Light 630 PRO (Medical Devices Inc.)) |
| Asymptomatic LHON mutation carriers | No intervention | Study Arm 2) asymptomatic LHON mutation carriers. Can be male or female; have some dysfunction, with changes occurring over months. Patients in study arm 2 will not be exposed to NIR-LED, but only undergo diagnostic studies. |
What is being measured
The primary outcome is the main question the study is designed to answer.
N95 Peak Via pERG and fERG -PhNR
The primary comparison will be a paired comparison of pre- and post-treatment retinal ganglion cell N95 pattern electroretinogram (pERG) peaks and fERG - Photopic Negative Response (PhNR). Pairing will be done between a subject's treatment and control eye.
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jul 2011 (estimated)
- Study startJul 2011
- Primary completionMay 2013
- Study completionMay 2013
Why it stopped: Unable to record n95 pERG peak (primary measure), as LHON subjects are unable to focus on target.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Leber's Hereditary Optic Neuropathy (LHON)Studies of the same condition, open and under way.
- More trials run by Medical College of WisconsinThe 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
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Medical College of Wisconsin
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Harry T Whelan, MD, Medical College of Wisconsin
- Investigators
- Harry T Whelan, MD, Principal investigator, Medical College of Wisconsin
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01389817
- Sponsor's protocol ID
- PRO14842
- Acronym
- LHON
- Status last verified
- Sep 2014
- First submitted
- 6 Jul 2011
- Last updated
- 29 Sep 2014
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Sep 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Near-infrared Light-emitting Diode (NIR-LED) Therapy for Leber's Hereditary Optic Neuropathy (LHON)
- ClinicalTrials.gov ID
- NCT01389817
- Acronym / sponsor ID
- LHON / PRO14842
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Unable to record n95 pERG peak (primary measure), as LHON subjects are unable to focus on target.
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Medical College of WisconsinUniversity, hospital or other
- Study start
- Jul 2011
- Primary completion
- May 2013 (actual)
- Study completion
- May 2013 (actual)
- First posted
- 8 Jul 2011 (estimated)
- Results
- Posted 29 Sep 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The overall objective of the proposed research is to test the hypothesis that Near-infrared Light-emitting Diode (NIR-LED) therapy will stimulate mitochondrial function, attenuate oxidative stress, and improve cell survival and vision in subjects with Leber's Hereditary Optic Neuropathy (LHON).
Detailed description
To determine the effects of short term (3 month) near-infrared light NIR-LED therapy on anatomic and functional abnormalities of LHON as assessed by a wide variety of ophthalmologic tests, including visual acuity, optical coherence tomography, pattern electroretinography (pERG) N95 retinal ganglion cell peak, fERG-PhNR (flash ERG - Photopic Negative Response), and fundus photography.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Symptomatic LHON patients. | Experimental | Device: Near-infrared light-emitting diode (NIR-LED) therapy (Med Light 630 PRO (Medical Devices Inc.))Study Arm 1) symptomatic LHON patients. Male and female LHON patients with treatable bilateral optic atrophy. Treat the worse of the 2 eyes if there is a measurable difference in subjective visual functions (visual acuity, peripheral vision). |
| Asymptomatic LHON mutation carriers | No intervention | –Study Arm 2) asymptomatic LHON mutation carriers. Can be male or female; have some dysfunction, with changes occurring over months. Patients in study arm 2 will not be exposed to NIR-LED, but only undergo diagnostic studies. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Sex - male and female. Men are affected with visual loss more frequently than women, with a male predominance of ~85%. A minimum of 25% of men and 5% of women at risk for LHON experience visual loss.
- Age - 18 years or older. The onset of visual loss typically occurs between the ages of 15 and 35 years, but otherwise classic LHON has been reported in many individuals both younger and older, with a range of age at onset from 2 to 80 years.
- Diagnosis of LHON or a carrier of the gene mutation. Patient must be evaluated by molecular confirmation, with the 11778 and other mutations of Leber's Hereditary Optic Neuropathy stratified in later data analysis.
- Able \& willing to provide informed consent
Key exclusion criteria (4 listed)
- Has an optic nerve disease other than LHON.
- Significant renal disease, defined as a history of chronic renal failure requiring dialysis or kidney transplant.
- Participation in an investigational trial within 30 days of NIR-LED treatment that involved treatment with any drug that has not received regulatory approval at the time of study entry. (Note: subjects cannot receive another investigational drug while participating in the study during the first 6 months.)
- Major surgery within 28 days prior to participation or major surgery planned during the next 6 months. Major surgery is defined as a surgical procedure that is more extensive than fine needle biopsy/aspiration, placement of a central venous access device, removal/biopsy of a skin lesion, or placement of a peripheral venous catheter.
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 |
|---|---|---|
| Near-infrared light-emitting diode (NIR-LED) therapy (Med Light 630 PRO (Medical Devices Inc.))Also: Med Light 630 PRO (Medical Devices Inc.)Subjects will be exposed to light emitted from a Med Light 630 PRO (Medical Devices Inc.) at a wavelength of 630 nm (+/-15nm) with an exposure of 4 J/cm2. This is accomplished by applying the 50 mW/cm2 LED-generated light to the closed study eye for 80 seconds. Treatments involve application of the LED-generated light for 80 seconds, twice daily. | Device | Symptomatic LHON patients. |
Primary outcome
N95 Peak Via pERG and fERG -PhNR
Time frame: 12 months
The primary comparison will be a paired comparison of pre- and post-treatment retinal ganglion cell N95 pattern electroretinogram (pERG) peaks and fERG - Photopic Negative Response (PhNR). Pairing will be done between a subject's treatment and control eye.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Sep 2014.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Harry T Whelan, MD principal investigator
Medical College of Wisconsin
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Near-infrared light-emitting diode (NIR-LED) therapy (Med Light 630 PRO (Medical Devices Inc.)) in people with Leber's Hereditary Optic Neuropathy (LHON), run by Medical College of Wisconsin at 3 sites 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 4 inclusion and 4 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 29 Sep 2014. 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. 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 combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 29 Sep 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
N95 Peak Via pERG and fERG -PhNR (time frame: 12 months). 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 29 Sep 2014.