Efficacy and Safety of SOM3355 in Huntington's Disease Chorea

Phase IIa, Double-blind, Randomized, Placebo-controlled Study of the Efficacy and Safety of SOM3355 in Huntington's Disease (HD) Patients With Chorea Movements.

Phase IIa study to evaluate the efficacy and safety of SOM3355 in chorea movements associated with Huntington's disease

Study Overview

• Status: Completed
• Conditions: Huntington's Chorea
• Intervention / Treatment: Drug: SOM3355 100mg BID; Drug: SOM3355 200mg BID; Drug: Placebo BID

• Study Type: Interventional
• Enrollment (Actual): 32
• Phase: Phase 2

Contacts and Locations

Study Locations

• Spain
  • Barcelona, Spain
    • Hospital Clínic de Barcelona
  • Barcelona, Spain
    • Hospital Universitari Vall d'Hebron
  • Barcelona, Spain
    • Hospital de la Santa Creu i Sant Pau
  • L'Hospitalet de Llobregat, Spain
    • Hospital Universitari de Bellvitge

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Subject is at least 18 years of age at time of consent.
• Diagnosis of HD definite by a movement disorders expert and confirmed by a number of HTT gene CAG repeats equal or greater than 36.
• Female of child bearing potential (FCBP) and non-vasectomized male agree to practice appropriate methods of birth control.
• Ability to walk independently or with minimal assistance.
• UHDRS TMC score equal or greater than 8.
• UHDRS TFC equal or greater than 4.
• Subject has provided written informed consent or through his/her legally authorized representative.

Exclusion Criteria:

• Onset of HD symptoms prior to age 18 (Juvenile forms of HD).
• Non-ambulatory patients.
• A past medical history of clinically significant ECG abnormalities or a family history (grandparents, parents and siblings) of a prolonged QT-interval syndrome.
• Pregnant or breastfeeding female patients, including those planning to conceive during the period of the trial.
• Patients with psychiatric symptoms, or other impairments, that would interfere with their full compliance with the Investigator instructions and testing, unless there is an identified caregiver to support the patient.
• Any surgical or medical condition which might significantly alter the absorption, distribution, metabolism, or excretion of drugs, or which may jeopardize the subject in case of participation in the study. The Investigator should make this determination in consideration of the subject's medical history and/or clinical laboratory test results at screening and baseline.
• Known allergy/sensitivity/intolerance to the study drugs or their excipients.
• Any significant laboratory results which, in the Investigator's opinion, would not be compatible with study participation or represent a risk for the subject while in the study.
• Prescribed anti-hypertensive medication, tetrabenazine, deutetrabenazine or valbenazine within 15 days prior starting the investigational treatment.
• Excluded concomitant medications: any anti-hypertensive medication, tetrabenazine, deutetrabenazine or valbenazine, all typical neuroleptics and all MAO inhibitors
• Subject has a history of alcohol or substance abuse in the previous 12 months.
• Patients with diabetic ketoacidosis or metabolic acidosis.
• Patients with cardiogenic shock, congestive heart failure, pulmonary hypertension due to right-sided heart failure, severe sinus bradycardia, atrioventricular block (grades II and III) or sinoatrial block.
• Subject has participated in an investigational drug or device trial within 30 days prior starting the investigational treatment.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Crossover Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Group A; Administration of SOM3355 100mg BID for 6 weeks, SOM3355 200mg BID for 6 weeks, SOM3355 100mg BID for 6 weeks and placebo BID for 6 weeks.	Drug: SOM3355 100mg BID; Oral; Drug: SOM3355 200mg BID; Oral; Drug: Placebo BID; Oral
Experimental: Group B; Administration of placebo BID for 6 weeks, SOM3355 100mg BID for 6 weeks, SOM3355 200mg BID for 6 weeks and SOM3355 100mg BID for 6 weeks.	Drug: SOM3355 100mg BID; Oral; Drug: SOM3355 200mg BID; Oral; Drug: Placebo BID; Oral

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
UHDRS (Unified Huntington's Disease Rating Scale) Total Maximal Chorea (TMC) score	UHDRS subscore used to measure the effectiveness of SOM3355 on HD chorea.	6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Clinical Global Impression of Change (CGIC) scale	PGIC reflects clinician's belief about the efficacy of treatment.	6 months
Patient Global Impression of Change (PGIC) scale	PGIC reflects a patient's belief about the efficacy of treatment.	6 months
UHDRS Total Functional Capacity (TFC)	A standardized rating scale used to assess capacity to work, handle finances, perform domestic chores and self-care tasks.	6 months
UHDRS Functional Assessment	Functional Assessment should be judged according to the investigator's opinion of capacity to perform the activity rather than the actual performance of this activity.	6 months
UHDRS Gait score	UHDRS subscore used to assess patient's gait.	6 months
UHDRS Total Motor Score (TMS)	Categoric clinical rating scale assessing multiple domains of motor disability in HD.	6 months
Columbia Suicide Severity Rating Scale (C-SSRS)	International validated questionnaire used for suicide assessment.	6 months
Safety (number of participants with adverse events)	The maximum grade for each type of AE will be recorded for each subject, and frequency tables will be presented and reviewed to determine patterns. The relationship of the AE to the study treatment will be taken into consideration. Hypotension and cardiovascular events will be specifically analyzed.	6 months

Collaborators and Investigators

• Sponsor: SOM Innovation Biotech SA

Publications and helpful links

Helpful Links

• SOM Biotech website

Study record dates

Study Major Dates

• Study Start (Actual): August 8, 2018
• Primary Completion (Actual): August 22, 2019
• Study Completion (Actual): August 22, 2019

Study Registration Dates

• First Submitted: June 11, 2018
• First Submitted That Met QC Criteria: June 28, 2018
• First Posted (Actual): July 2, 2018

Study Record Updates

• Last Update Posted (Actual): September 16, 2019
• Last Update Submitted That Met QC Criteria: September 13, 2019
• Last Verified: September 1, 2019

More Information

Terms related to this study

• Keywords: Chorea; Huntington; SOM3355
• Additional Relevant MeSH Terms: Mental Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Neurocognitive Disorders; Genetic Diseases, Inborn; Basal Ganglia Diseases; Movement Disorders; Neurodegenerative Diseases; Dyskinesias; Heredodegenerative Disorders, Nervous System; Dementia; Cognition Disorders; Huntington Disease; Chorea
• Other Study ID Numbers: SOMCT02; 2018-000203-16 (EudraCT Number)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No