Trial Outcomes & Findings for Study of Oral Minocycline in Treating Bilateral Cystoid Macular Edema Associated With Retinitis Pigmentosa (NCT NCT02140164)

NCT ID: NCT02140164

Last Updated: 2024-04-17

Results Overview

Three visits (two pre-treatment and one baseline) were conducted prior to the receipt of investigational product (IP) and an average of the OCT measurements at these three visits was used as the pre-treatment value.

• Recruitment status: COMPLETED
• Study phase: PHASE1/PHASE2
• Target enrollment: 7 participants
• Primary outcome timeframe: Pre-treatment and 6 Months
• Results posted on: 2024-04-17

Participant Flow

Five participants, ages 12 and older, with cystoid macular edema (CME) secondary to retinitis pigmentosa (RP) who meet the eligibility criteria will be initially accrued; however, up to an additional five participants may be enrolled to replace participants who may withdraw from the study prior to reaching Month 6

Participant milestones

Measure	Minocycline Oral administration of minocycline Minocycline: Oral dose of 100 mg (or appropriate weight-adjusted pediatric dose) of minocycline twice daily for 12 months.
Overall Study STARTED	7
Overall Study COMPLETED	5
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Measure	Minocycline Oral administration of minocycline Minocycline: Oral dose of 100 mg (or appropriate weight-adjusted pediatric dose) of minocycline twice daily for 12 months.
Overall Study Cystoid Macular Edema (CME) resolved	2

Baseline Characteristics

Study of Oral Minocycline in Treating Bilateral Cystoid Macular Edema Associated With Retinitis Pigmentosa

Baseline characteristics by cohort

Measure	Minocycline n=7 Participants Oral administration of minocycline Minocycline: Oral dose of 100 mg (or appropriate weight-adjusted pediatric dose) of minocycline twice daily for 12 months.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	7 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	27.7 years STANDARD_DEVIATION 8.81 • n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants

PRIMARY outcome

Timeframe: Pre-treatment and 6 Months

Population: Participants receiving investigational product (IP) at the Month 6 visit were included in the primary efficacy analysis.

Three visits (two pre-treatment and one baseline) were conducted prior to the receipt of investigational product (IP) and an average of the OCT measurements at these three visits was used as the pre-treatment value.

Outcome measures

Measure	Minocycline n=5 Participants Oral administration of minocycline Minocycline: Oral dose of 100 mg (or appropriate weight-adjusted pediatric dose) of minocycline twice daily for 12 months.
Change in Cystoid Macular Edema (CME) Based on Optical Coherence Tomography (OCT) Measurements in the Study Eye at 6 Months Compared to the Average of the Pre-treatment Values.	-16.7 microns Standard Deviation 42.16

SECONDARY outcome

Timeframe: Pre-treatment and 12 Months

Three visits (two pre-treatment and one baseline) were conducted prior to the receipt of investigational product (IP) and an average of the OCT measurements at these three visits was used as the pre-treatment value.

Outcome measures

Measure	Minocycline n=5 Participants Oral administration of minocycline Minocycline: Oral dose of 100 mg (or appropriate weight-adjusted pediatric dose) of minocycline twice daily for 12 months.
Change in Cystoid Macular Edema (CME) Based on Optical Coherence Tomography (OCT) Measurements in the Study Eye at 12 Months Compared to the Average of the Pre-treatment Values	-37.3 microns Standard Deviation 52.90

SECONDARY outcome

Timeframe: Pre-Treatment and 6 Months

Population: Participants had non-recordable ERGs; therefore, changes could not be measured.

This outcome measure will not be reported.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Pre-treatment and 12 Months

Population: Participants had non-recordable ERGs; therefore, changes could not be measured.

This outcome measure will not be reported.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Pre-treatment and 6 Months

Three visits (two pre-treatment and one baseline) were conducted prior to the receipt of investigational product (IP) and an average of the microperimetry measurements at these three visits was used as the pre-treatment value.

Outcome measures

Measure	Minocycline n=5 Participants Oral administration of minocycline Minocycline: Oral dose of 100 mg (or appropriate weight-adjusted pediatric dose) of minocycline twice daily for 12 months.
Change in Microperimetry at 6 Months as Compared to the Average of Pre-treatment Values	-1.01 decibels (dB) Standard Deviation 0.36

SECONDARY outcome

Timeframe: Pre-treatment and 12 Months

Three visits (two pre-treatment and one baseline) were conducted prior to the receipt of investigational product (IP) and an average of the microperimetry measurements at these three visits was used as the pre-treatment value.

Outcome measures

Measure	Minocycline n=5 Participants Oral administration of minocycline Minocycline: Oral dose of 100 mg (or appropriate weight-adjusted pediatric dose) of minocycline twice daily for 12 months.
Change in Microperimetry at 12 Months as Compared to the Average of Pre-treatment Values	-1.02 dB Standard Deviation 0.45

SECONDARY outcome

Timeframe: Pre-treatment and 6 Months

Population: Two participants had to switch to HVF 10-2; therefore they were not included in the analysis.

Three visits (two pre-treatment and one baseline) were conducted prior to the receipt of investigational product (IP) and an average of the HVF 30-2 measurements at these three visits was used as the pre-treatment value.

Outcome measures

Measure	Minocycline n=3 Participants Oral administration of minocycline Minocycline: Oral dose of 100 mg (or appropriate weight-adjusted pediatric dose) of minocycline twice daily for 12 months.
Change in Visual Field as Measured by HVF 30-2 Visual Field Testing at 6 Months as Compared to the Average of Pre-treatment Values	-3.62 dB Standard Deviation 5.80

SECONDARY outcome

Timeframe: Pre-treatment and 12 Months

Three visits (two pre-treatment and one baseline) were conducted prior to the receipt of investigational product (IP) and an average of the HVF 30-2 measurements at these three visits was used as the pre-treatment value.

Outcome measures

Measure	Minocycline n=3 Participants Oral administration of minocycline Minocycline: Oral dose of 100 mg (or appropriate weight-adjusted pediatric dose) of minocycline twice daily for 12 months.
Change in Visual Field as Measured by HVF 30-2 Visual Field Testing at 12 Months as Compared to the Average of Pre-treatment Values	1.69 dB Standard Deviation 5.52

SECONDARY outcome

Timeframe: Baseline and 12 Months

Visual Acuity was measured by a certified tester using an electronic visual acuity testing machine based on the Early Treatment Diabetic Retinopathy Study (ETDRS) method.

Outcome measures

Measure	Minocycline n=5 eyes Oral administration of minocycline Minocycline: Oral dose of 100 mg (or appropriate weight-adjusted pediatric dose) of minocycline twice daily for 12 months.
Number of Study Eyes Achieving a 15-letter or More Worsening in Electronic Visual Acuity (EVA) at 12 Months as Compared to Baseline	0 eyes

SECONDARY outcome

Timeframe: Study Duration, up to 16 Months

Population: All participants were included in the safety analysis.

Outcome measures

Measure	Minocycline n=7 Participants Oral administration of minocycline Minocycline: Oral dose of 100 mg (or appropriate weight-adjusted pediatric dose) of minocycline twice daily for 12 months.
Number of Ocular Adverse Events	3 adverse events

SECONDARY outcome

Timeframe: Study Duration, up to 16 Months

Population: All participants were included in the safety analysis.

Outcome measures

Measure	Minocycline n=7 Participants Oral administration of minocycline Minocycline: Oral dose of 100 mg (or appropriate weight-adjusted pediatric dose) of minocycline twice daily for 12 months.
Number of Non-ocular Adverse Events	12 adverse events

SECONDARY outcome

Timeframe: Study Duration, up to 16 Months

Population: All participants were included in the safety analysis.

Outcome measures

Measure	Minocycline n=7 Participants Oral administration of minocycline Minocycline: Oral dose of 100 mg (or appropriate weight-adjusted pediatric dose) of minocycline twice daily for 12 months.
Number of Severe Adverse Events	0 adverse events

Adverse Events

Minocycline

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Minocycline n=7 participants at risk Oral administration of minocycline Minocycline: Oral dose of 100 mg (or appropriate weight-adjusted pediatric dose) of minocycline twice daily for 12 months.
Infections and infestations Nasopharyngitis	14.3% 1/7 • Number of events 1 • Study Duration, up to 16 months Adverse events were collected on and after the baseline visit, and when investigational product (IP) began.
Nervous system disorders Headache	14.3% 1/7 • Number of events 1 • Study Duration, up to 16 months Adverse events were collected on and after the baseline visit, and when investigational product (IP) began.
Respiratory, thoracic and mediastinal disorders Dyspnoea	14.3% 1/7 • Number of events 1 • Study Duration, up to 16 months Adverse events were collected on and after the baseline visit, and when investigational product (IP) began.
Infections and infestations Influenza	14.3% 1/7 • Number of events 1 • Study Duration, up to 16 months Adverse events were collected on and after the baseline visit, and when investigational product (IP) began.
Infections and infestations Upper respiratory tract infection	14.3% 1/7 • Number of events 1 • Study Duration, up to 16 months Adverse events were collected on and after the baseline visit, and when investigational product (IP) began.
Psychiatric disorders Depression	14.3% 1/7 • Number of events 1 • Study Duration, up to 16 months Adverse events were collected on and after the baseline visit, and when investigational product (IP) began.
Psychiatric disorders Attention deficit/hyperactivity disorder	14.3% 1/7 • Number of events 1 • Study Duration, up to 16 months Adverse events were collected on and after the baseline visit, and when investigational product (IP) began.
Infections and infestations Conjunctivitis	14.3% 1/7 • Number of events 1 • Study Duration, up to 16 months Adverse events were collected on and after the baseline visit, and when investigational product (IP) began.
Gastrointestinal disorders Vomiting	14.3% 1/7 • Number of events 1 • Study Duration, up to 16 months Adverse events were collected on and after the baseline visit, and when investigational product (IP) began.
Infections and infestations Vulvovaginal mycotic infection	14.3% 1/7 • Number of events 1 • Study Duration, up to 16 months Adverse events were collected on and after the baseline visit, and when investigational product (IP) began.
Infections and infestations Pharyngitis streptococcal	14.3% 1/7 • Number of events 1 • Study Duration, up to 16 months Adverse events were collected on and after the baseline visit, and when investigational product (IP) began.
Endocrine disorders Hypothyroidism	14.3% 1/7 • Number of events 1 • Study Duration, up to 16 months Adverse events were collected on and after the baseline visit, and when investigational product (IP) began.
Eye disorders Visual acuity reduced	14.3% 1/7 • Number of events 2 • Study Duration, up to 16 months Adverse events were collected on and after the baseline visit, and when investigational product (IP) began.
Investigations Blood potassium increased	14.3% 1/7 • Number of events 1 • Study Duration, up to 16 months Adverse events were collected on and after the baseline visit, and when investigational product (IP) began.

Additional Information

Catherine Cukras, MD, PhD, Principal Investigator, NEI

National Institutes of Health

Phone: 301-435-5061

Email: cukrasc@nei.nih.gov

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place