Efficacy and Safety of SOM3355 in Huntington's Disease Chorea

• Sponsor: SOM Biotech SL
• Information provided by (Responsible Party): SOM Biotech SL

Study Description

Brief Summary:

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

Condition or disease	Intervention/treatment	Phase
Huntington's Chorea	Drug: SOM3355 100mg BID; Drug: SOM3355 200mg BID; Drug: Placebo BID	Phase 2

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 30 participants
• Allocation: Randomized
• Intervention Model: Crossover Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: Phase IIa, Double-blind, Randomized, Placebo-controlled Study of the Efficacy and Safety of SOM3355 in Huntington's Disease (HD) Patients With Chorea Movements.
• Actual Study Start Date: August 8, 2018
• Estimated Primary Completion Date: August 2019
• Estimated Study Completion Date: September 2019

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures :

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

Secondary Outcome Measures :

1. Clinical Global Impression of Change (CGIC) scale [ Time Frame: 6 months ]
   PGIC reflects clinician's belief about the efficacy of treatment.
2. Patient Global Impression of Change (PGIC) scale [ Time Frame: 6 months ]
   PGIC reflects a patient's belief about the efficacy of treatment.
3. UHDRS Total Functional Capacity (TFC) [ Time Frame: 6 months ]
   A standardized rating scale used to assess capacity to work, handle finances, perform domestic chores and self-care tasks.
4. UHDRS Functional Assessment [ Time Frame: 6 months ]
   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.
5. UHDRS Gait score [ Time Frame: 6 months ]
   UHDRS subscore used to assess patient's gait.
6. UHDRS Total Motor Score (TMS) [ Time Frame: 6 months ]
   Categoric clinical rating scale assessing multiple domains of motor disability in HD.
7. Columbia Suicide Severity Rating Scale (C-SSRS) [ Time Frame: 6 months ]
   International validated questionnaire used for suicide assessment.
8. Safety (number of participants with adverse events) [ Time Frame: 6 months ]
   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.

Eligibility Criteria

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

Criteria

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.

Contacts and Locations

Locations

Spain

Hospital Clínic de Barcelona

Barcelona, Spain

Hospital de la Santa Creu i Sant Pau

Barcelona, Spain

Hospital Universitari Vall d'Hebron

Barcelona, Spain

Hospital Universitari de Bellvitge

L'Hospitalet de Llobregat, Spain

Sponsors and Collaborators

SOM Biotech SL

More Information

Additional Information:

SOM Biotech website 

• Responsible Party: SOM Biotech SL
• ClinicalTrials.gov Identifier: NCT03575676 History of Changes
• Other Study ID Numbers: SOMCT02; 2018-000203-16 ( EudraCT Number )
• First Posted: July 2, 2018
• Last Update Posted: March 18, 2019
• Last Verified: March 2019

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by SOM Biotech SL:

Huntington; Chorea; SOM3355

Additional relevant MeSH terms:

Huntington Disease; Chorea; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Dementia; Dyskinesias; Movement Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Cognition Disorders; Neurocognitive Disorders; Mental Disorders; Neurologic Manifestations; Signs and Symptoms