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Leber's Hereditary Optic NeuropathyPhase 2RHODOSNCT00747487

Study to Assess Efficacy,Safety and Tolerability of Idebenone in the Treatment of Leber's Hereditary Optic Neuropathy

A Phase 2 study testing Idebenone in people with Leber's Hereditary Optic Neuropathy, run by Santhera Pharmaceuticals at 3 sites in 3 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This study is meant to assess the effectiveness of idebenone on visual function measures in patients with Leber's Hereditary Optic Neuropathy over a 6 months period. Read more about this study

Who is it for?
Ages 14 to 65 years
Conditions the study is for
Leber's Hereditary Optic Neuropathy
What is being tested?
Idebenone
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
85 participants
Where
3 sitesin 3 countries
Study dates
Nov 2007to Feb 2010
Phase
Phase 2
Are there results?
None posted in the registry

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 14 to 65 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Age > or = 14 years and < 65 years
  • Impaired visual acuity in at least one eye due to LHON
  • Onset of visual loss due to LHON lies five years or less prior to Baseline
  • Confirmation of either G11778A, T14484C or G3460A LHON mtDNA mutations at >60% in blood
  • No explanation for the visual failure besides LHON
  • Body weight ≥ 45 kg
  • Negative urine pregnancy test at Screening and at Baseline (women of childbearing potential).

Who cannot join

  • Treatment with Coenzyme Q10 or idebenone within 1 month prior to Baseline
  • Pregnancy and/or breast-feeding
  • Weekly alcohol intake 35 units (men) or 24 units (women)
  • Current drug abuse
  • Clinically significant abnormalities of clinical haematology or biochemistry including, but not limited to, elevations greater than 2 times the upper limit of normal of AST, ALT or creatinine
  • Participation in another clinical trial of any investigational drug within 3 months prior to Baseline
  • Other factor that, in the investigator's opinion, excludes the patient from entering the study

Trial locations and contacts

3 sites in 3 countries.

3 sites in 3 countries

Canada1 site

  • Unité de recherche clinique Ophtalmologie- Hopital Notre-Dame

    Montreal, Quebec, H2L 4M1

Other trials in Canada

Germany1 site

  • Klinikum der Universität München - Grosshadern, Neurologische Klinik und Poliklinik

    Munich, 81377

Other trials in Germany

United Kingdom1 site

  • Clinical Research Facility, 4th Floor Leazes Wing, Royal Victoria Infirmary

    Newcastle upon Tyne, NE1 4LP

Other trials in United Kingdom

About this study

Official title: A Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Patients With Leber's Hereditary Optic Neuropathy

This study is meant to assess the effectiveness of idebenone on visual function measures in patients with Leber's Hereditary Optic Neuropathy over a 6 months period.

Read the detailed description

The study involves 6 clinic visits.

Being tested
Idebenone, Placebo
Keywords
Leber, LHON, Leber's Hereditary Optic Neuropathy, Idebenone
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.

  • IdebenoneDrug

    Idebenone 900 mg/day

  • PlaceboDrug

    Placebo

Study groups in NCT00747487
Study groupRoleWhat they receive
1ExperimentalIdebenone
Receives: Drug: Idebenone
2Placebo comparatorPlacebo
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Best recovery of logMAR visual acuity between baseline and Week 24 in either right or left eye

    Measured: 24 weeks

Secondary outcomes (12)

  1. Change in the patient's best logMAR visual acuity between baseline and week 24

    Measured: 24 weeks

  2. Change in scotoma area in both eyes

    Measured: Day -1, Week 4, Week 12, Week 24

  3. Change in optic nerve fibre layer thickness in both eyes

    Measured: Day -1, Week 4, Week 12, Week 24

  4. Colour contrast sensitivity in both eyes (in a subset of patients)

    Measured: Day -1, Week 4, Week 12, Week 24

  5. logMAR visual acuity as a continuous variable in both eyes

    Measured: Screening, Day -1, Week 4, Week 12, Week 24, Week 28

  6. Clinical Global Impression of Change

    Measured: Week 4, Week 12 and Week 24

  7. Change in Health-Related Quality of Life (HRQOL)

    Measured: Day -1, Week 4, Week 12, Week 24

  8. Change in self-reported general energy levels

    Measured: Day -1, Week 4, Week 12, Week 24, Week 28

  9. Proportion of patients in which visual acuity in the initially least affected eye does not deteriorate to 1.0 log MAR or more ( in LHON patients with eye still less affected than 0.5 logMAR at trial entry)

    Measured: 24 weeks

  10. Plasma levels of idebenone matched to measures of efficacy and safety

    Measured: 24 weeks

  11. • Best visual acuity at Week 24 (best eye at Week 24) compared to best visual acuity at Baseline (best eye at Baseline)

    Measured: 24 weeks

  12. • Count of eyes/ patients for which the visual acuity improves between baseline and week 24

    Measured: 24 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted5 Sep 2008 (estimated)
  2. Study startNov 2007
  3. Primary completionFeb 2010
  4. Study completionFeb 2010

Resources

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 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
85 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00747487
Sponsor's protocol ID
SNT-II-003
Acronym
RHODOS
Status last verified
May 2013
First submitted
4 Sep 2008
Last updated
27 May 2013
Results posted
No
Data monitoring committee
Yes

Publications

  • Klopstock T, Yu-Wai-Man P, Dimitriadis K, Rouleau J, Heck S, Bailie M, Atawan A, Chattopadhyay S, Schubert M, Garip A, Kernt M, Petraki D, Rummey C, Leinonen M, Metz G, Griffiths PG, Meier T, Chinnery PF. A randomized placebo-controlled trial of idebenone in Leber's hereditary optic neuropathy. Brain. 2011 Sep;134(Pt 9):2677-86. doi: 10.1093/brain/awr170. Epub 2011 Jul 25.

    PubMed 21788663

  • Rudolph G, Dimitriadis K, Buchner B, Heck S, Al-Tamami J, Seidensticker F, Rummey C, Leinonen M, Meier T, Klopstock T. Effects of idebenone on color vision in patients with leber hereditary optic neuropathy. J Neuroophthalmol. 2013 Mar;33(1):30-6. doi: 10.1097/WNO.0b013e318272c643.

    PubMed 23263355

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Idebenone in people with Leber's Hereditary Optic Neuropathy, run by Santhera Pharmaceuticals at 3 sites in 3 countries.

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 7 inclusion and 7 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: finished, as of the sponsor's last update on 27 May 2013. 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 Canada, Germany, United Kingdom. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

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?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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?

Best recovery of logMAR visual acuity between baseline and Week 24 in either right or left eye (time frame: 24 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00747487, which also lists 2 linked publications.

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 27 May 2013.

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