Phase IIb, Randomized, Double-blind, Placebo-controlled Study in Parallel Groups Assessing the Efficacy and Safety of Two Doses of SOM3355 in Patients Suffering From Huntington's Disease With Choreic Movements
Overview
- Phase
- Phase 2
- Intervention
- Placebo capsules
- Conditions
- Huntington Chorea
- Sponsor
- SOM Innovation Biotech SA
- Enrollment
- 139
- Locations
- 23
- Primary Endpoint
- Change in Total Maximal Chorea (TMC) Score of the UHDRS® for Subjects Not Taking Neuroleptics During the Trial (mITT - N=122)
- Status
- Completed
- Last Updated
- 8 months ago
Overview
Brief Summary
Phase IIb, randomized, double-blind, placebo-controlled study in parallel groups assessing the efficacy and safety of two doses of SOM3355 in patients suffering from Huntington's Disease with choreic movements.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Males or females ≥21 years old, a diagnosis of Huntington's Disease determined by a movement disorders expert and confirmed by a number of HTT gene CAG repeats ≥36, a UHDRS® Total maximal chorea (TMC) score ≥10, and a UHDRS® Total Functional Capacity (TFC) ≥7.
Exclusion Criteria
- •Onset of HD symptoms prior to age of 21 years (juvenile forms of HD), HD patients presenting rigid akinesia, and use of other VMAT2 inhibitors such as tetrabenazine, deutetrabenazine, or valbenazine, or other antichoreic treatment such as any neuroleptic, or amantadine, memantine, riluzole.
Arms & Interventions
Placebo
Placebo capsules were administered twice daily (BID) for at least 9 weeks at maintenance dose.
Intervention: Placebo capsules
SOM3355 400 mg/day
SOM3355 200 mg capsules were administered twice daily (BID) for at least 9 weeks at maintenance dose.
Intervention: SOM3355 200 mg capsules
SOM3355 600 mg/day
SOM3355 300 mg capsules were administered twice daily (BID) for at least 8 weeks at maintenance dose.
Intervention: SOM3355 300 mg capsules
Outcomes
Primary Outcomes
Change in Total Maximal Chorea (TMC) Score of the UHDRS® for Subjects Not Taking Neuroleptics During the Trial (mITT - N=122)
Time Frame: From baseline to end of maintenance dose (10 weeks of treatment).
Pre-defined analysis of the primary efficacy endpoint (change in TMC score from baseline to the end of maintenance dose) performed with the 122 subjects of the mITT not taking neuroleptics during the trial. The TMC is part of the motor assessment of the Unified Huntington's Disease Rating Scale (UHDRS) and measures chorea in 7 different body parts, including the face, oral-buccal-lingual region, trunk, and each limb independently. The TMC score is the sum of the individual scores, ranging from 0 to 28. A decrease in TMC scores indicates improvement in chorea symptoms.
Secondary Outcomes
- Change in the Clinical Global Impression (CGI) (mITT - N=139)(From baseline to end of maintenance dose (10 weeks of treatment).)
- Change in the Patient Global Impression (PGI) (mITT - N=139)(From baseline to end of maintenance dose (10 weeks of treatment).)