Efficacy and Safety on SOM3355 in Huntington's Disease Chorea

SOM Innovation Biotech SA139 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

139

Conditions

Huntington Chorea

Interventions

Placebo capsulesDRUG

Treatment was blind for the whole duration of the study.

SOM3355 200 mg capsulesDRUG

Treatment was blind for the whole duration of the study.

SOM3355 300 mg capsulesDRUG

Treatment was blind for the whole duration of the study.

Eligibility

Sex: ALLMin age: 21 Years

Medical Language ↔ Plain English

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.

Locations (23)

Centre Hospitalier Universitaire Angers

Angers, France

Outcomes

Primary Outcomes

Change in Total Maximal Chorea (TMC) Score of the UHDRS® for Subjects Not Taking Neuroleptics During the Trial (mITT - N=122)

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.

Time frame: From baseline to end of maintenance dose (10 weeks of treatment).

Secondary Outcomes

Change in the Clinical Global Impression (CGI) (mITT - N=139)

The key secondary endpoint was the Clinical Global Impression of Change (CGI-C) at Visit 5 (week 10). The key secondary efficacy analysis was conducted on the mITT Population, including 139 subjects. Subjects with a score of 1 (Very much improved), 2 (Much improved), or 3 (Minimally improved) were defined as "Improved", and patients with a score of 4 (No change), 5 (Worse), 6 (Much worse), and 7 (Very much worse) were defined as "Not Improved".

Time frame: From baseline to end of maintenance dose (10 weeks of treatment).

Change in the Patient Global Impression (PGI) (mITT - N=139)

Another relevant secondary endpoint was the Patient Global Impression of Change (PGI-C) at Visit 5 (week 10). The efficacy analysis was conducted on the mITT Population, including 139 subjects. Subjects with a score of 1 (Very much improved), 2 (Much improved), or 3 (Minimally improved) were defined as "Improved", and patients with a score of 4 (No change), 5 (Worse), 6 (Much worse), and 7 (Very much worse) were defined as "Not Improved".

Time frame: From baseline to end of maintenance dose (10 weeks of treatment).

Data from ClinicalTrials.gov

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