Phase IIa, double-blind, randomized, placebo-controlled study of the efficacy and safety of SOM3335 in Huntington`s disease (HD) patients with chorea movements.
- Conditions
- Chorea movements associated with Huntington's DiseaseMedDRA version: 20.0 Level: LLT Classification code 10020469 Term: Huntington's chorea System Organ Class: 100000004850Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- EUCTR2018-000203-16-ES
- Lead Sponsor
- SOM Biotech
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Not specified
- Target Recruitment
- 30
- Subject is at least 18 years of age at time of consent.
- Diagnosis of HD definite by a movement disorders expert confirmed by a number of HTT gene CAG repeats equal or greater than 36.
- Female of child bearing potential 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.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 30
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
- Onset of HD symptoms prior to age 18 (Juvenile forms of HD).
- Non-ambulatory patients.
- A past medical history of clinically significant electrocardiogram (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; 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 & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To determine wheter SOM3355 reduces chorea movements associated with Huntington's Disease.;Primary end point(s): Improvement in any active drug period in TMC Score of at least 2 points compared with placebo period.;Timepoint(s) of evaluation of this end point: At the end of study treatment.;<br> Secondary Objective: - Improvement based on Clinical Global Impression of Change (CGIC), on Patient Global Impression of Change (PGIC) as well as Total Functional Capacity (TFC), Functional Assessment, Gait Score and Total Motor Score (TMS) of the Unified Huntington's Disease Rating Scale (UHDRS).<br> - Suicide severity assessment based on Columbia’s Suicide Severity Rating Scale (C-SSRS).<br> - Safety when the drug is administered to HD patients, with special attention to blood pressure (BP) and cardiac function.<br>
- Secondary Outcome Measures
Name Time Method <br> Secondary end point(s): - Clinical Global Impression of Change (CGIC) scale.<br> - Patient Global Impression of Change (PGIC) scale.<br> - Total Functional Capacity (TFC) of the UHDRS.<br> - Functional Assessment of the UHDRS.<br> - Gait score of the UHDRS.<br> - Total Motor Score (TMS) of the UHDRS.<br> - Columbia Suicide Severity Rating Scale (C-SSRS)<br> - Safety: The maximum grade for each type of adverse event (AE) will be recorded for each subject, and frequency tables will be presented and reviewed to determine patterns. Additionally, the relationship of the AE to the study treatment will be taken into consideration. The frequency of AE as hypotension and cardiovascular events will be specifically analyzed.<br> ;Timepoint(s) of evaluation of this end point: At the end of the study.