Ignite Creation Date:
2024-05-06 @ 11:29 AM
Last Modification Date:
2024-10-26 @ 12:46 PM
Study NCT ID:
NCT03536559
Status:
TERMINATED
Last Update Posted:
2023-04-03
First Post:
2018-05-02
Brief Title:
Nanocrystalline Gold to Treat Remyelination Failure in Chronic Optic Neuropathy In Multiple Sclerosis
Sponsor:
Clene Nanomedicine
Organization:
Clene Nanomedicine
Study Overview
Official Title:
A Phase 2 Randomized DB-PC Parallel Group Study for the Treatment of Visual Pathway Deficits In Chronic Optic Neuropathy to Assess the Efficacy Safety Tolerability and Pharmacokinetics of CNM-Au8 For Remyelination In Multiple Sclerosis
Status:
TERMINATED
Status Verified Date:
2023-03
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
COVID-19 related enrollment challenges
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
VISIONARY-MS
Brief Summary:
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 to assess the efficacy and safety of CNM-Au8 as a remyelinating therapy in patients with stable RMS The secondary endpoint is Change in Functional Composite Responder Analysis Score from Baseline to Week 24
Detailed 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 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 functional electrophysiological at participating research sites 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 both 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 investigational product IP dose 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 up to a 48-week maximum blinded duration until the last-patient enrolled completes hisher Week 24 study visit per the study scheme in Figure 2 When the last enrolled patient completes his or her Week 24 visit patients enrolled in the Variable Duration Treatment Period will complete the End-of-Study EOS visit at their next scheduled study visit
The primary efficacy outcome measure will be assessed Efficacy will be assessed as an improvement in best-corrected low contrast letter acuity BC-LCLA Safety will be assessed up through the frequency of treatment-emergent adverse events TEAEs serious adverse events SAEs and discontinuations due to adverse events AEs and changes in safety assessments eg vitals ECG C-SSRS
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 eg 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
1 A six-week screening period Screening Period
2 A fixed 24-week double-blind randomized treatment period Fixed Duration Treatment Period
3 A variable-duration double-blind 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 the EOS Visit at their next scheduled study visit
4 A four-week follow-up period Safety Follow-Up Period for patients not continuing in the separate Open-Label Long-Term Safety Extension Study
Following the end of the blinded treatment period all patients who complete the 24-week Fixed Duration Treatment Period may be eligible to receive open-label CNM-Au8 in a separate Open-Label Long-Term Safety Extension Study
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 functional electrophysiological at participating research sites 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 both 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 investigational product IP dose 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 up to a 48-week maximum blinded duration until the last-patient enrolled completes hisher Week 24 study visit per the study scheme in Figure 2 When the last enrolled patient completes his or her Week 24 visit patients enrolled in the Variable Duration Treatment Period will complete the End-of-Study EOS visit at their next scheduled study visit
The primary efficacy outcome measure will be assessed Efficacy will be assessed as an improvement in best-corrected low contrast letter acuity BC-LCLA Safety will be assessed up through the frequency of treatment-emergent adverse events TEAEs serious adverse events SAEs and discontinuations due to adverse events AEs and changes in safety assessments eg vitals ECG C-SSRS
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 eg 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
1 A six-week screening period Screening Period
2 A fixed 24-week double-blind randomized treatment period Fixed Duration Treatment Period
3 A variable-duration double-blind 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 the EOS Visit at their next scheduled study visit
4 A four-week follow-up period Safety Follow-Up Period for patients not continuing in the separate Open-Label Long-Term Safety Extension Study
Following the end of the blinded treatment period all patients who complete the 24-week Fixed Duration Treatment Period may be eligible to receive open-label CNM-Au8 in a separate Open-Label Long-Term Safety Extension Study
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None