Last updated on January 2020

Nanocrystalline Gold to Treat Remyelination Failure in Chronic Optic Neuropathy In Multiple Sclerosis

Brief description of study

The objective of this trial is to assess the efficacy and safety of CNM-Au8 as a remyelinating treatment for vision-impairing MS lesions in participants who have chronic vision impairment as a result of Relapsing Remitting Multiple Sclerosis. The primary endpoint is the improvement in low contrast vision as measured by low contrast letter acuity after 24 weeks of treatment. The secondary endpoint is the improvement in remyelination as measured by visual evoked potentials after 24 weeks of treatment.

Detailed Study Description

This is a randomized, double-blind, parallel group, placebo-controlled study of the efficacy, safety, and pharmacokinetics of CNM-Au8 in stable RRMS patients who have Chronic Optic Neuropathy (residual deficits of conduction velocity in the visual pathway) evidence by low contrast letter acuity deficits at Screening.

Patients will be screened over a 6-week period. Patients who meet the inclusion criteria and none of the exclusionary criteria will be enrolled into the clinical study.

All enrolled patients will have their visual baseline established in both eyes by electrophysiological, functional, and morphological tests.

For each patient, the eye with the worst Baseline LCLA score will be considered as the affected eye. The other eye will be considered as the fellow eye. If both eyes have the same LCLA score at Baseline, then one eye will be randomly selected by the statistician to assess as the designated affected eye. Efficacy endpoints will be assessed in the affected and the fellow eyes.

Patients will be randomized to one of three groups: placebo, or one of two doses of CNM-Au8. All patients will receive their randomized oral treatment daily over at least 24 consecutive weeks during the Fixed Duration Treatment Period. The study will also have a blinded Variable Duration Treatment Period for up to an additional 24-weeks (48-week maximum blinded duration) until the last-patient enrolled completes his/her Week 24 study visit. The first enrolled patients will remain in the study until either a maximum of 48-weeks or until the last-enrolled patient reaches their 24-week visit, whichever comes first. When the last enrolled patient completes his or her 24-Week visit, treatment will end for the patients enrolled in the Variable Duration Treatment Period with their next scheduled study visit.

The primary efficacy outcome measure will be assessed based upon each patient's Week 24 study visit. The study will remain blinded until the study database is locked.

All patients who are discontinued from treatment will complete the End-of-Study (EOS) assessment.

At the end of the Variable Duration Treatment Period, patients will complete an EOS assessment and then may choose either to exit the study, or receive open-label CNM-Au8 in a separate Open-Label Safety Extension Study.

An independent DSMB will be responsible for monitoring the safety of the study on a quarterly basis and ad hoc at the request of the DSMB or the Sponsor (e.g., in the event of unexpected SAEs) to review data throughout the Fixed Duration Treatment Period and the Variable Duration Treatment Period. The DSMB may make recommendations on the conduct of the study, including study termination. Appropriate procedures will be detailed in a DSMB Charter that will define disclosure of any findings along with patient- and study-stopping criteria.

There will be four study periods followed by a separate Open-Label Safety Extension Study:

  1. A six-week screening period (Screening Period);
  2. A fixed 24-week blinded, randomized treatment period (Fixed Duration Treatment Period);
  3. A variable-duration, blinded treatment period (Variable Duration Treatment Period) where patients continue the previously randomized treatment for up to an additional 24-weeks (total blinded duration of 48-weeks). This period will end for all patients when the last-enrolled patient reaches his or her 24-week visit (LP-24Wk) at which time patients in the Variable Duration Treatment Period will complete their next scheduled study visit;
  4. A four-week follow-up period (End-Of-Study Assessment) for patients not continuing in the separate Open-Label Safety Extension Study.

Following the end of the blinded treatment period, patients will be given the option to enter a separate Open-Label Safety Extension Study.

Clinical Study Identifier: NCT03536559

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Westmead Hospital

Westmead, Australia
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Recruitment Status: Open

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