Trial · NCT02774005
Study to Assess the Efficacy and Safety of Raxone in LHON Patients
NCT02774005, Phase 4, Completed, Santhera Pharmaceuticals; results first posted 2023-04-21
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Study to Assess the Efficacy and Safety of Raxone in LHON Patients. Trials, record NCT02774005. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02774005
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study to Assess the Efficacy and Safety of Raxone in LHON Patients
- NCT number
- NCT02774005
- Status
- Completed
- Phases
- Phase 4
- Enrollment
- 199 participants, actual
- Lead sponsor
- Santhera Pharmaceuticals
- Conditions
- Leber's Hereditary Optic Neuropathy (LHON)
- Interventions
- Idebenone
- First posted
- 2016-05-16
- Last updated
- 2023-04-21
- Results first posted
- 2023-04-21
Source ClinicalTrials.gov
Registered outcomes3
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Proportion (Number) of Eyes With Clinically Relevant Recovery of Visual Acuity From Baseline | 12 months | Proportion (number) of eyes with clinically relevant recovery of visual acuity (VA) from Baseline or in which Baseline VA better than 1.0 logMAR was maintained at Month 12 in patients treated with Raxone® ≤1 year after the onset of symptoms, compared to matching external natural history control group |
| Secondary | Components of the Primary Endpoint: Proportion of Eyes With Clinically Relevant Recovery (CRR) of VA From Baseline at Month 12 Compared to Matching External National History (NH) Control Group | 12 months | CRR is defined as: * Improvement from "off-chart" (the equivalent of Counting fingers, Hand motion, Light perception or No light perception) Visual Acuity to at least 1.6 logMAR value, OR * Improvement of at least 0.2 logMAR value within "on-chart" Visual Acuity A patient had a CRR if at least one eye had CRR. logMAR = Logarithm of the minimum angle of resolution |
| Secondary | Components of the Primary Endpoint: Proportion of Eyes in Which Baseline Visual Acuity (VA) Better Than 1.0 logMAR Was Maintained at Month 12 (Clinically Relevant Stabilization) Compared to Matching External NH Control Group | 12 months | For proportion of eyes in which baseline VA better than 1.0 logMAR was maintained at Month 12 (CRS) compared to matching external NH control group only patients having VA \< 1.0 at baseline are taking into account. Clinically relevant stabilization (CRS) was defined as maintenance of VA \<1.0 logMAR in eyes with VA \<1.0 logMAR at Baseline. A patient had a CRS if at least one eye had CRS. logMAR = Logarithm of the minimum angle of resolution |
Source ClinicalTrials.gov
Outcomes3
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Proportion (Number) of Eyes With Clinically Relevant Recovery of Visual Acuity From Baseline | 12 months | In the Month 12 dataset with eyes ≤ 1 year after the onset of symptoms at Baseline, used in the evaluation of the primary endpoint, there were 80 patients in the LEROS mITT who contributed 142 eyes for the evaluation. | eyes | COUNT_OF_UNITS |
| SECONDARY | Components of the Primary Endpoint: Proportion of Eyes With Clinically Relevant Recovery (CRR) of VA From Baseline at Month 12 Compared to Matching External National History (NH) Control Group | 12 months | In the Month 12 dataset with eyes ≤ 1 year after the onset of symptoms at Baseline, used in the evaluation of the primary endpoint, there were 80 patients in the LEROS mITT who contributed 142 eyes for the evaluation. | eyes | COUNT_OF_UNITS |
| SECONDARY | Components of the Primary Endpoint: Proportion of Eyes in Which Baseline Visual Acuity (VA) Better Than 1.0 logMAR Was Maintained at Month 12 (Clinically Relevant Stabilization) Compared to Matching External NH Control Group | 12 months | eyes | COUNT_OF_UNITS |
Source ClinicalTrials.gov
Results by group3
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Proportion (Number) of Eyes With Clinically Relevant Recovery of Visual Acuity From Baseline | Raxone | 60 | eyes | ||
| Components of the Primary Endpoint: Proportion of Eyes With Clinically Relevant Recovery (CRR) of VA From Baseline at Month 12 Compared to Matching External National History (NH) Control Group | Raxone | 47 | eyes | ||
Components of the Primary Endpoint: Proportion of Eyes in Which Baseline Visual Acuity (VA) Better Than 1.0 logMAR Was Maintained at Month 12 (Clinically Relevant Stabilization) Compared to Matching External NH Control Group | Raxone | 20 | eyes |
Source ClinicalTrials.gov
Participant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Raxone | 199 | |
| Overall Study | Safety Population | Raxone | 198 | |
| Overall Study | Intent-to-Treat (ITT) Population | Raxone | 196 | |
| Overall Study | Modified Intent-to-Treat (mITT) Population | Raxone | 181 | |
| Overall Study | COMPLETED | Raxone | 138 | |
| Overall Study | NOT COMPLETED | Raxone | 61 |
Source ClinicalTrials.gov
Adverse events319
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Total | Blood and lymphatic system disorders | Raxone | 3 | 198 | 3 |
| other | Anaemia | Blood and lymphatic system disorders | Raxone | 2 | 198 | 2 |
| other | Splenomegaly | Blood and lymphatic system disorders | Raxone | 1 | 198 | 1 |
| other | Total | Cardiac disorders | Raxone | 2 | 198 | 2 |
| other | Coronary artery disease | Cardiac disorders | Raxone | 1 | 198 | 1 |
| other | Tachycardia | Cardiac disorders | Raxone | 1 | 198 | 1 |
| other | Total | Congenital, familial and genetic disorders | Raxone | 1 | 198 | 1 |
| other | Hereditary optic atrophy | Congenital, familial and genetic disorders | Raxone | 1 | 198 | 1 |
| other | Total | Ear and labyrinth disorders | Raxone | 6 | 198 | 6 |
| other | Ear pain | Ear and labyrinth disorders | Raxone | 3 | 198 | 3 |
1 to 10 of 319Source ClinicalTrials.gov
See all 319 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Raxone | 1 | 198 |
| Total, other adverse events | Raxone | 148 | 198 |
| Total, serious adverse events | Raxone | 27 | 198 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 38428428 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02774005
GET https://conductscience.org/api/trials/v1/NCT02774005/events?page=2