Trial Outcomes & Findings for A Pilot Study for the Evaluation of Minocycline as a Microglia Inhibitor in the Treatment of Diabetic Macular Edema (NCT NCT01120899)
NCT ID: NCT01120899
Last Updated: 2014-10-17
Results Overview
Visual acuity was measured using the Early Treatment Diabetic Retinopathy Study (ETDRS) protocol. Acuity is measured as letters read on an ETDRS eye chart and the letters read equate to Snellen measurements. For example, if a participant reads between 84 and 88 letters, the equivalent Snellen measurement is 20/20. The participant's eye that met the study eye eligibility criteria was selected as the study eye. For cases in which both eyes met the study eye eligibility criteria, the study eye was selected according to the "choice of study eye in cases of bilateral disease" selection criteria outlined in the eligibility criteria. The eye not chosen as the study eye is referred to as the "fellow eye."
COMPLETED
PHASE1/PHASE2
6 participants
6 months
2014-10-17
Participant Flow
Participant milestones
| Measure |
Minocycline
Participants take an oral dose of 100 mg of minocycline twice daily for 24 months.
|
|---|---|
|
Overall Study
STARTED
|
6
|
|
Overall Study
Month 6
|
5
|
|
Overall Study
Month 12
|
3
|
|
Overall Study
Month 18
|
3
|
|
Overall Study
COMPLETED
|
3
|
|
Overall Study
NOT COMPLETED
|
3
|
Reasons for withdrawal
| Measure |
Minocycline
Participants take an oral dose of 100 mg of minocycline twice daily for 24 months.
|
|---|---|
|
Overall Study
Withdrawal by Subject
|
3
|
Baseline Characteristics
A Pilot Study for the Evaluation of Minocycline as a Microglia Inhibitor in the Treatment of Diabetic Macular Edema
Baseline characteristics by cohort
| Measure |
Minocycline
n=6 Participants
Participants take an oral dose of 100 mg of minocycline twice daily for 24 months.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
4 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
2 Participants
n=5 Participants
|
|
Age, Continuous
|
63.8 years
STANDARD_DEVIATION 9.5 • n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
6 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 6 monthsVisual acuity was measured using the Early Treatment Diabetic Retinopathy Study (ETDRS) protocol. Acuity is measured as letters read on an ETDRS eye chart and the letters read equate to Snellen measurements. For example, if a participant reads between 84 and 88 letters, the equivalent Snellen measurement is 20/20. The participant's eye that met the study eye eligibility criteria was selected as the study eye. For cases in which both eyes met the study eye eligibility criteria, the study eye was selected according to the "choice of study eye in cases of bilateral disease" selection criteria outlined in the eligibility criteria. The eye not chosen as the study eye is referred to as the "fellow eye."
Outcome measures
| Measure |
Minocycline
n=5 Eyes
Participants take an oral dose of 100 mg of minocycline twice daily for 24 months.
|
|---|---|
|
Number of Study Eyes Demonstrating an Increase or Decrease in Best-corrected Visual Acuity (BCVA) of 15 or More Early Treatment Diabetic Retinopathy Study (ETDRS) Letters at 6 Months Compared to Baseline
|
1 Eyes
|
SECONDARY outcome
Timeframe: Baseline and 6 MonthsVisual acuity was measured using the Early Treatment Diabetic Retinopathy Study (ETDRS) protocol. Acuity is measured as letters read on an ETDRS eye chart and the letters read equate to Snellen measurements. For example, if a participant reads between 84 and 88 letters, the equivalent Snellen measurement is 20/20. The participant's eye that met the study eye eligibility criteria was selected as the study eye. For cases in which both eyes met the study eye eligibility criteria, the study eye was selected according to the "choice of study eye in cases of bilateral disease" selection criteria outlined in the eligibility criteria. The eye not chosen as the study eye is referred to as the "fellow eye."
Outcome measures
| Measure |
Minocycline
n=5 Eyes
Participants take an oral dose of 100 mg of minocycline twice daily for 24 months.
|
|---|---|
|
Change in BCVA in the Study Eye at 6 Months Compared to Baseline
|
5.8 ETDRS Letters
Standard Deviation 5.4
|
SECONDARY outcome
Timeframe: Baseline and 12 MonthsVisual acuity was measured using the Early Treatment Diabetic Retinopathy Study (ETDRS) protocol. Acuity is measured as letters read on an ETDRS eye chart and the letters read equate to Snellen measurements. For example, if a participant reads between 84 and 88 letters, the equivalent Snellen measurement is 20/20. The participant's eye that met the study eye eligibility criteria was selected as the study eye. For cases in which both eyes met the study eye eligibility criteria, the study eye was selected according to the "choice of study eye in cases of bilateral disease" selection criteria outlined in the eligibility criteria. The eye not chosen as the study eye is referred to as the "fellow eye."
Outcome measures
| Measure |
Minocycline
n=3 Eyes
Participants take an oral dose of 100 mg of minocycline twice daily for 24 months.
|
|---|---|
|
Change in BCVA in the Study Eye at 12 Months Compared to Baseline
|
8.0 ETDRS Letters
Standard Deviation 8.9
|
SECONDARY outcome
Timeframe: Baseline and 18 MonthsVisual acuity was measured using the Early Treatment Diabetic Retinopathy Study (ETDRS) protocol. Acuity is measured as letters read on an ETDRS eye chart and the letters read equate to Snellen measurements. For example, if a participant reads between 84 and 88 letters, the equivalent Snellen measurement is 20/20.
Outcome measures
| Measure |
Minocycline
n=3 Eyes
Participants take an oral dose of 100 mg of minocycline twice daily for 24 months.
|
|---|---|
|
Change in BCVA in the Study Eye at 18 Months Compared to Baseline
|
9.0 ETDRS letters
Standard Deviation 4.0
|
SECONDARY outcome
Timeframe: Baseline and 24 MonthsVisual acuity was measured using the Early Treatment Diabetic Retinopathy Study (ETDRS) protocol. Acuity is measured as letters read on an ETDRS eye chart and the letters read equate to Snellen measurements. For example, if a participant reads between 84 and 88 letters, the equivalent Snellen measurement is 20/20.
Outcome measures
| Measure |
Minocycline
n=3 Eyes
Participants take an oral dose of 100 mg of minocycline twice daily for 24 months.
|
|---|---|
|
Change in BCVA in the Study Eye at 24 Months Compared to Baseline
|
9.3 ETDRS letters
Standard Deviation 6.8
|
SECONDARY outcome
Timeframe: Baseline and 6 MonthsRetinal thickness was assessed by spectral-domain optical coherence tomography (Cirrus HD-OCT; Carl Zeiss Meditec, Dublin, CA), a non-invasive imaging technique that uses long-wavelength light to capture micrometer-resolution cross-sectional images from biological tissue. The participant's eye that met the study eye eligibility criteria was selected as the study eye. For cases in which both eyes met the study eye eligibility criteria, the study eye was selected according to the "choice of study eye in cases of bilateral disease" selection criteria outlined in the eligibility criteria. The eye not chosen as the study eye is referred to as the "fellow eye."
Outcome measures
| Measure |
Minocycline
n=5 Eyes
Participants take an oral dose of 100 mg of minocycline twice daily for 24 months.
|
|---|---|
|
Percentage Change in Retinal Thickness in the Study Eye at 6 Months Compared to Baseline
|
-8.1 percentage change
Standard Deviation 14.6
|
SECONDARY outcome
Timeframe: Baseline and 12 MonthsRetinal thickness was assessed by spectral-domain optical coherence tomography (Cirrus HD-OCT; Carl Zeiss Meditec, Dublin, CA), a non-invasive imaging technique that uses long-wavelength light to capture micrometer-resolution cross-sectional images from biological tissue.
Outcome measures
| Measure |
Minocycline
n=3 Eyes
Participants take an oral dose of 100 mg of minocycline twice daily for 24 months.
|
|---|---|
|
Percentage Change in Retinal Thickness in the Study Eye at 12 Months Compared to Baseline
|
-12.35 percentage change in retinal thickness
Standard Deviation 1.21
|
SECONDARY outcome
Timeframe: Baseline and 18 MonthsRetinal thickness was assessed by spectral-domain optical coherence tomography (Cirrus HD-OCT; Carl Zeiss Meditec, Dublin, CA), a non-invasive imaging technique that uses long-wavelength light to capture micrometer-resolution cross-sectional images from biological tissue.
Outcome measures
| Measure |
Minocycline
n=3 Eyes
Participants take an oral dose of 100 mg of minocycline twice daily for 24 months.
|
|---|---|
|
Percentage Change in Retinal Thickness in the Study Eye at 18 Months Compared to Baseline
|
-20.09 percentage change in retinal thickness
Standard Deviation 10.65
|
SECONDARY outcome
Timeframe: Baseline and 24 MonthsRetinal thickness was assessed by spectral-domain optical coherence tomography (Cirrus HD-OCT; Carl Zeiss Meditec, Dublin, CA), a non-invasive imaging technique that uses long-wavelength light to capture micrometer-resolution cross-sectional images from biological tissue.
Outcome measures
| Measure |
Minocycline
n=3 Eyes
Participants take an oral dose of 100 mg of minocycline twice daily for 24 months.
|
|---|---|
|
Percentage Change in Retinal Thickness in the Study Eye at 24 Months Compared to Baseline
|
-20.83 percentage change in retinal thickness
Standard Deviation 4.62
|
SECONDARY outcome
Timeframe: 6 MonthsFluorescein angiography (FA) images from both eyes in each participant were obtained via a standard digital imaging system (OIS, Sacramento, CA) at baseline and at Month 6, Month 12, Month 18, and Month 24. Three retinal specialists independently graded the area of late fluorescein leakage (at approximately 10 minutes)in each eye using a region-of-interest tool in an image analysis software package (NIH ImageJ, Bethesda, MD). The participant's eye that met the study eye eligibility criteria was selected as the study eye. For cases in which both eyes met the study eye eligibility criteria, the study eye was selected according to the "choice of study eye in cases of bilateral disease" selection criteria outlined in the eligibility criteria. The eye not chosen as the study eye is referred to as the "fellow eye."
Outcome measures
| Measure |
Minocycline
n=5 Eyes
Participants take an oral dose of 100 mg of minocycline twice daily for 24 months.
|
|---|---|
|
Number of Study Eyes Demonstrating a Decrease in the Area of Late Leakage, as Measured by Fluorescein Angiography (FA), at 6 Months Compared to Baseline
|
5 Eyes
|
SECONDARY outcome
Timeframe: 12 MonthsFluorescein angiography (FA) images from both eyes in each participant were obtained via a standard digital imaging system (OIS, Sacramento, CA) at baseline and at Month 6, Month 12, Month 18, and Month 24. Three retinal specialists independently graded the area of late fluorescein leakage (at approximately 10 minutes)in each eye using a region-of-interest tool in an image analysis software package (NIH ImageJ, Bethesda, MD). The participant's eye that met the study eye eligibility criteria was selected as the study eye. For cases in which both eyes met the study eye eligibility criteria, the study eye was selected according to the "choice of study eye in cases of bilateral disease" selection criteria outlined in the eligibility criteria. The eye not chosen as the study eye is referred to as the "fellow eye."
Outcome measures
| Measure |
Minocycline
n=3 Eyes
Participants take an oral dose of 100 mg of minocycline twice daily for 24 months.
|
|---|---|
|
Number of Study Eyes Demonstrating a Decrease in the Area of Late Leakage, as Measured by Fluorescein Angiography (FA), at 12 Months Compared to Baseline
|
3 Eyes
|
SECONDARY outcome
Timeframe: 18 MonthsFluorescein angiography (FA) images from both eyes in each participant were obtained via a standard digital imaging system (OIS, Sacramento, CA) at baseline and at Month 6, Month 12, Month 18, and Month 24. Three retinal specialists independently graded the area of late fluorescein leakage (at approximately 10 minutes)in each eye using a region-of-interest tool in an image analysis software package (NIH ImageJ, Bethesda, MD). The participant's eye that met the study eye eligibility criteria was selected as the study eye. For cases in which both eyes met the study eye eligibility criteria, the study eye was selected according to the "choice of study eye in cases of bilateral disease" selection criteria outlined in the eligibility criteria. The eye not chosen as the study eye is referred to as the "fellow eye."
Outcome measures
| Measure |
Minocycline
n=3 Eyes
Participants take an oral dose of 100 mg of minocycline twice daily for 24 months.
|
|---|---|
|
Number of Study Eyes Demonstrating a Decrease in the Area of Late Leakage, as Measured by Fluorescein Angiography (FA), at 18 Months Compared to Baseline
|
3 Eyes
|
SECONDARY outcome
Timeframe: 24 MonthsFluorescein angiography (FA) images from both eyes in each participant were obtained via a standard digital imaging system (OIS, Sacramento, CA) at baseline and at Month 6, Month 12, Month 18, and Month 24. Three retinal specialists independently graded the area of late fluorescein leakage (at approximately 10 minutes)in each eye using a region-of-interest tool in an image analysis software package (NIH ImageJ, Bethesda, MD). The participant's eye that met the study eye eligibility criteria was selected as the study eye. For cases in which both eyes met the study eye eligibility criteria, the study eye was selected according to the "choice of study eye in cases of bilateral disease" selection criteria outlined in the eligibility criteria. The eye not chosen as the study eye is referred to as the "fellow eye."
Outcome measures
| Measure |
Minocycline
n=3 Eyes
Participants take an oral dose of 100 mg of minocycline twice daily for 24 months.
|
|---|---|
|
Number of Study Eyes Demonstrating a Decrease in the Area of Late Leakage, as Measured by Fluorescein Angiography (FA), at 24 Months Compared to Baseline
|
3 Eyes
|
SECONDARY outcome
Timeframe: 6 MonthsFluorescein angiography (FA) images from both eyes in each participant were obtained via a standard digital imaging system (OIS, Sacramento, CA) at baseline and at Month 6, Month 12, Month 18, and Month 24. Three retinal specialists independently graded the area of late fluorescein leakage (at approximately 10 minutes)in each eye using a region-of-interest tool in an image analysis software package (NIH ImageJ, Bethesda, MD). The participant's eye that met the study eye eligibility criteria was selected as the study eye. For cases in which both eyes met the study eye eligibility criteria, the study eye was selected according to the "choice of study eye in cases of bilateral disease" selection criteria outlined in the eligibility criteria. The eye not chosen as the study eye is referred to as the "fellow eye."
Outcome measures
| Measure |
Minocycline
n=5 Eyes
Participants take an oral dose of 100 mg of minocycline twice daily for 24 months.
|
|---|---|
|
Number of Fellow Eyes Demonstrating a Decrease in the Area of Late Leakage, as Measured by Fluorescein Angiography (FA), at 6 Months Compared to Baseline
|
4 Eyes
|
SECONDARY outcome
Timeframe: 12 MonthsFluorescein angiography (FA) images from both eyes in each participant were obtained via a standard digital imaging system (OIS, Sacramento, CA) at baseline and at Month 6, Month 12, Month 18, and Month 24. Three retinal specialists independently graded the area of late fluorescein leakage (at approximately 10 minutes)in each eye using a region-of-interest tool in an image analysis software package (NIH ImageJ, Bethesda, MD). The participant's eye that met the study eye eligibility criteria was selected as the study eye. For cases in which both eyes met the study eye eligibility criteria, the study eye was selected according to the "choice of study eye in cases of bilateral disease" selection criteria outlined in the eligibility criteria. The eye not chosen as the study eye is referred to as the "fellow eye."
Outcome measures
| Measure |
Minocycline
n=3 Eyes
Participants take an oral dose of 100 mg of minocycline twice daily for 24 months.
|
|---|---|
|
Number of Fellow Eyes Demonstrating a Decrease in the Area of Late Leakage, as Measured by Fluorescein Angiography (FA), at 12 Months Compared to Baseline
|
1 Eyes
|
SECONDARY outcome
Timeframe: 18 MonthsFluorescein angiography (FA) images from both eyes in each participant were obtained via a standard digital imaging system (OIS, Sacramento, CA) at baseline and at Month 6, Month 12, Month 18, and Month 24. Three retinal specialists independently graded the area of late fluorescein leakage (at approximately 10 minutes)in each eye using a region-of-interest tool in an image analysis software package (NIH ImageJ, Bethesda, MD). The participant's eye that met the study eye eligibility criteria was selected as the study eye. For cases in which both eyes met the study eye eligibility criteria, the study eye was selected according to the "choice of study eye in cases of bilateral disease" selection criteria outlined in the eligibility criteria. The eye not chosen as the study eye is referred to as the "fellow eye."
Outcome measures
| Measure |
Minocycline
n=3 Eyes
Participants take an oral dose of 100 mg of minocycline twice daily for 24 months.
|
|---|---|
|
Number of Fellow Eyes Demonstrating a Decrease in the Area of Late Leakage, as Measured by Fluorescein Angiography (FA), at 18 Months Compared to Baseline
|
2 Eyes
|
SECONDARY outcome
Timeframe: 24 MonthsFluorescein angiography (FA) images from both eyes in each participant were obtained via a standard digital imaging system (OIS, Sacramento, CA) at baseline and at Month 6, Month 12, Month 18, and Month 24. Three retinal specialists independently graded the area of late fluorescein leakage (at approximately 10 minutes)in each eye using a region-of-interest tool in an image analysis software package (NIH ImageJ, Bethesda, MD). The participant's eye that met the study eye eligibility criteria was selected as the study eye. For cases in which both eyes met the study eye eligibility criteria, the study eye was selected according to the "choice of study eye in cases of bilateral disease" selection criteria outlined in the eligibility criteria. The eye not chosen as the study eye is referred to as the "fellow eye."
Outcome measures
| Measure |
Minocycline
n=3 Eyes
Participants take an oral dose of 100 mg of minocycline twice daily for 24 months.
|
|---|---|
|
Number of Fellow Eyes Demonstrating a Decrease in the Area of Late Leakage, as Measured by Fluorescein Angiography (FA), at 24 Months Compared to Baseline
|
1 Eyes
|
Adverse Events
Minocycline
Serious adverse events
| Measure |
Minocycline
n=6 participants at risk
Participants take an oral dose of 100 mg of minocycline twice daily for 24 months.
|
|---|---|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Laryngeal cancer
|
16.7%
1/6 • Number of events 1 • 18 months
|
Other adverse events
| Measure |
Minocycline
n=6 participants at risk
Participants take an oral dose of 100 mg of minocycline twice daily for 24 months.
|
|---|---|
|
Gastrointestinal disorders
Abdominal pain upper
|
16.7%
1/6 • Number of events 1 • 18 months
|
|
Eye disorders
Blepharitis
|
16.7%
1/6 • Number of events 1 • 18 months
|
|
Investigations
Blood albumin decreased
|
16.7%
1/6 • Number of events 1 • 18 months
|
|
Investigations
Blood glucose increased
|
33.3%
2/6 • Number of events 2 • 18 months
|
|
Investigations
Blood magnesium decreased
|
16.7%
1/6 • Number of events 1 • 18 months
|
|
Investigations
Blood thyroid stimulating hormone abnormal
|
16.7%
1/6 • Number of events 1 • 18 months
|
|
Gastrointestinal disorders
Diarrhoea
|
33.3%
2/6 • Number of events 5 • 18 months
|
|
Nervous system disorders
Dizziness
|
16.7%
1/6 • Number of events 1 • 18 months
|
|
Nervous system disorders
Dysgeusia
|
16.7%
1/6 • Number of events 1 • 18 months
|
|
General disorders
Fatigue
|
16.7%
1/6 • Number of events 1 • 18 months
|
|
Infections and infestations
Gastroenteritis viral
|
16.7%
1/6 • Number of events 1 • 18 months
|
|
Investigations
Glycosylated haemoglobin increased
|
16.7%
1/6 • Number of events 1 • 18 months
|
|
Investigations
Haemoglobin decreased
|
33.3%
2/6 • Number of events 2 • 18 months
|
|
Nervous system disorders
Headache
|
16.7%
1/6 • Number of events 1 • 18 months
|
|
Metabolism and nutrition disorders
Hypercholesterolaemia
|
16.7%
1/6 • Number of events 1 • 18 months
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
16.7%
1/6 • Number of events 1 • 18 months
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
16.7%
1/6 • Number of events 1 • 18 months
|
|
Infections and infestations
Nasopharyngitis
|
16.7%
1/6 • Number of events 1 • 18 months
|
|
Renal and urinary disorders
Nephrolithiasis
|
16.7%
1/6 • Number of events 1 • 18 months
|
|
General disorders
Oedema peripheral
|
16.7%
1/6 • Number of events 1 • 18 months
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
16.7%
1/6 • Number of events 1 • 18 months
|
|
Cardiac disorders
Palpitations
|
16.7%
1/6 • Number of events 1 • 18 months
|
|
Skin and subcutaneous tissue disorders
Rash
|
16.7%
1/6 • Number of events 1 • 18 months
|
|
Skin and subcutaneous tissue disorders
Skin ulcer
|
16.7%
1/6 • Number of events 1 • 18 months
|
|
Infections and infestations
Viral infection
|
16.7%
1/6 • Number of events 1 • 18 months
|
|
Reproductive system and breast disorders
Vulvovaginal pruritus
|
16.7%
1/6 • Number of events 2 • 18 months
|
|
Investigations
White blood cell count increased
|
16.7%
1/6 • Number of events 1 • 18 months
|
|
Eye disorders
Meibomianitis
|
16.7%
1/6 • Number of events 1 • 18 months
|
|
Gastrointestinal disorders
Abdominal discomfort
|
16.7%
1/6 • Number of events 1 • 18 months
|
|
Infections and infestations
Tinea pedis
|
16.7%
1/6 • Number of events 1 • 18 months
|
|
Investigations
Blood pressure increased
|
16.7%
1/6 • Number of events 1 • 18 months
|
|
Skin and subcutaneous tissue disorders
Pruritis
|
16.7%
1/6 • Number of events 2 • 18 months
|
|
Skin and subcutaneous tissue disorders
Skin hyperpigmentation
|
33.3%
2/6 • Number of events 3 • 18 months
|
Additional Information
Catherine Cukras, MD, PhD, Principal Investigator, NEI
National Institutes of Health
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place