Efficacy and Safety of SOM3355 in Huntington's Disease Chorea

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03575676
• Date of registration: 11/06/2018
• Prospective Registration: Yes
• Primary sponsor: SOM Biotech SL
• Public title: Efficacy and Safety of SOM3355 in Huntington's Disease Chorea
• Scientific 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.
• Date of first enrolment: August 8, 2018
• Target sample size: 32
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03575676
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

Spain

Key inclusion & exclusion 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.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Huntington's Chorea

Intervention(s)

Drug: Placebo BID

Drug: SOM3355 200mg BID

Drug: SOM3355 100mg BID

Primary Outcome(s)

UHDRS (Unified Huntington's Disease Rating Scale) Total Maximal Chorea (TMC) score [Time Frame: 6 months]

Secondary Outcome(s)

UHDRS Gait score [Time Frame: 6 months]

UHDRS Total Functional Capacity (TFC) [Time Frame: 6 months]

Patient Global Impression of Change (PGIC) scale [Time Frame: 6 months]

Safety (number of participants with adverse events) [Time Frame: 6 months]

UHDRS Total Motor Score (TMS) [Time Frame: 6 months]

Clinical Global Impression of Change (CGIC) scale [Time Frame: 6 months]

Columbia Suicide Severity Rating Scale (C-SSRS) [Time Frame: 6 months]

UHDRS Functional Assessment [Time Frame: 6 months]

Secondary ID(s)

2018-000203-16

SOMCT02

Source(s) of Monetary Support

Please refer to primary and secondary sponsors