Neurogenetics

At CIC Neurosciences, we conduct three types of studies: therapeutic studies (both industry-sponsored and academic), and pathophysiological studies aimed at unraveling the fundamental mechanisms of diseases in order to advance our understanding and improve patient care.

Industrial therapeutic studies

Academic therapeutic studies

Pathophysiological Studies

LEAP2MONO

A Phase 3, multicenter, international, randomized, double-blind, double-dummy, active-controlled study to evaluate the efficacy and safety of venglustat

🎯 : Evaluer l’efficacité du venglustat comparé au Cerezyme chez l’adulte et l’enfant (>= 12 to <18 ans) GD3

⏳: Currently being added

PIVOT HD

A Phase 2b, randomized, double-blind extension study to evaluate the long-term safety and efficacy of PTC518 in participants with Huntington’s disease

🎯: To evaluate the long-term safety and pharmacodynamic effects of PTC518 in patients with Huntington's disease

📝: Currently being monitored

VICO (Huntington's disease)

An open-label Phase 1/2a trial to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of multiple escalating doses of VO659 administered intrathecally to patients with type 1 or 3 spinocerebellar ataxia or Huntington’s disease

🎯: To evaluate the safety and tolerability of intrathecal bolus administration of multiple increasing doses of VO659 in patients with clinically diagnosed SCA1, SCA3, and HD

⏳: Currently being added

At CIC Neurosciences, we conduct three types of studies: therapeutic studies (both industry-sponsored and academic), and pathophysiological studies aimed at unraveling the fundamental mechanisms of diseases in order to advance our understanding and improve patient care.

Industrial therapeutic studies

LEAP2MONO

A Phase 3, multicenter, international, randomized, double-blind, double-dummy, active-controlled study to evaluate the efficacy and safety of venglustat

🎯 : Evaluer l’efficacité du venglustat comparé au Cerezyme chez l’adulte et l’enfant (>= 12 to <18 ans) GD3

⏳: Currently being added

PIVOT HD

A Phase 2b, randomized, double-blind extension study to evaluate the long-term safety and efficacy of PTC518 in participants with Huntington’s disease

🎯: To evaluate the long-term safety and pharmacodynamic effects of PTC518 in patients with Huntington's disease

📝: Currently being monitored

VICO (Huntington's disease)

An open-label Phase 1/2a trial to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of multiple escalating doses of VO659 administered intrathecally to patients with type 1 or 3 spinocerebellar ataxia or Huntington’s disease

🎯: To evaluate the safety and tolerability of intrathecal bolus administration of multiple increasing doses of VO659 in patients with clinically diagnosed SCA1, SCA3, and HD

⏳: Currently being added