Trial Outcomes & Findings for Historical Case Record Survey of Visual Acuity Data From Patients With Leber's Hereditary Optic Neuropathy (LHON) (NCT NCT02796274)

Last Updated: 2020-01-30

Results Overview

Clinically Relevant Recovery (CRR) is defined as a VA improvement from off-chart (ETDRS) to 5 letters on-chart, or an on-chart improvement of 10 letters.

Recruitment status

COMPLETED

Target enrollment

219 participants

Primary outcome timeframe

12 months

Results posted on

2020-01-30

Participant Flow

Unit of analysis: eyes

Participant milestones

Participant milestones
Measure	LHON Patients Case Record Cohort Historical case record data from all LHON patients fulfilling the prospectively-defined inclusion criteria: 1. Age ≥ 12 years. 2. Onset of symptoms was dated after 1999 and was well documented (at least month of onset of symptoms is known for each eye). 3. At least two VA assessments were available within 5 years of onset of symptoms and prior to idebenone use. 4. Have a genetic diagnosis for LHON for one of the following mtDNA mutations: G11778A, G3460A, or T14484C.
Overall Study STARTED	219 438
Overall Study COMPLETED	219 438
Overall Study NOT COMPLETED	0 0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Historical Case Record Survey of Visual Acuity Data From Patients With Leber's Hereditary Optic Neuropathy (LHON)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	LHON Patients Case Record Cohort n=438 eyes Historical case record data from all LHON patients fulfilling the prospectively-defined inclusion criteria: 1. Age ≥ 12 years. 2. Onset of symptoms was dated after 1999 and was well documented (at least month of onset of symptoms is known for each eye). 3. At least two VA assessments were available within 5 years of onset of symptoms and prior to idebenone use. 4. Have a genetic diagnosis for LHON for one of the following mtDNA mutations: G11778A, G3460A, or T14484C. Actual: 48 (with VA assessment made ≤1 year after the onset of symptoms) out of 219 patients from whom data were collected and entered into the database.
Age, Categorical <=18 years	49 Participants n=219 Participants
Age, Categorical Between 18 and 65 years	169 Participants n=219 Participants
Age, Categorical >=65 years	1 Participants n=219 Participants
Age, Continuous	26.0 years n=219 Participants
Sex: Female, Male Female	44 Participants n=219 Participants
Sex: Female, Male Male	175 Participants n=219 Participants
Region of Enrollment Netherlands	24 participants n=219 Participants
Region of Enrollment Belgium	30 participants n=219 Participants
Region of Enrollment Poland	40 participants n=219 Participants
Region of Enrollment Italy	20 participants n=219 Participants
Region of Enrollment United Kingdom	20 participants n=219 Participants
Region of Enrollment France	29 participants n=219 Participants
Region of Enrollment Germany	56 participants n=219 Participants

PRIMARY outcome

Timeframe: 12 months

Population: Data from a total of 219 LHON patients and 438 eyes were obtained from this study, out of which 96 eyes had a VA assessment made ≤1 year after the onset of symptoms (primary outcome measure).

Clinically Relevant Recovery (CRR) is defined as a VA improvement from off-chart (ETDRS) to 5 letters on-chart, or an on-chart improvement of 10 letters.

Outcome measures

Outcome measures
Measure	LHON Patients Case Record Cohort n=96 eyes Historical case record data from all LHON patients fulfilling the prospectively-defined inclusion criteria: 1. Age ≥ 12 years. 2. Onset of symptoms was dated after 1999 and was well documented (at least month of onset of symptoms is known for each eye). 3. At least two VA assessments were available within 5 years of onset of symptoms and prior to idebenone use. 4. Have a genetic diagnosis for LHON for one of the following mtDNA mutations: G11778A, G3460A, or T14484C.
In Eyes With VA Assessment Made ≤1 Year After Onset of Symptoms: Proportion of Eyes With Clinically Relevant Recovery (CRR) of VA (Measured by Change in ETDRS Letters) From Baseline (BL) or in Which BL VA Better Than 1.0 logMAR Was Maintained at 12 Months	15 ETDRS letters Interval 1.5 to 34.5

Adverse Events

LHON Patients Case Record Cohort

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Vanessa Dos Reis Ferreira

Santhera Pharmaceuticals

Phone: +41 61 906 89 50

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60