Clinical trial NCT05475483: Efficacy and Safety on SOM3355 in Huntington's Disease Chorea
Official title: 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
Overall status: COMPLETED
Phase: PHASE2
Conditions: Huntington Chorea
Keywords: Huntington
Chorea
SOM3355
Lead sponsor: SOM Innovation Biotech SA
Sponsor class: INDUSTRY
First posted: 2022-07-27
Enrollment: 140
Sex: ALL
Minimum age (years): 21
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: Inclusion Criteria:

1. Males or females ≥21 years old.
2. Patients with a diagnosis of Huntington's Disease determined by a movement disorders expert and confirmed by a number of HTT gene CAG repeats ≥36.
3. UHDRS® Total maximal chorea (TMC) score ≥10.
4. UHDRS® Total Functional Capacity (TFC) ≥7.
5. Able to walk independently or with minimal assistance.
6. Females of child-bearing potential must use a medically accepted effective method of birth control and should not be breast-feeding.
7. In the opinion of the Investigator, the patient must have adequate support to comply with the entire study requirements.
8. Able and willing to provide written informed consent.

Exclusion Criteria:

1. Onset of HD symptoms prior to age of 21 years (juvenile forms of HD).
2. HD patients presenting rigid akinesia.
3. Use of other VMAT2 inhibitors such as tetrabenazine, deutetrabenazine, or valbenazine; and use of other antichoreic treatment such as any neuroleptic, or amantadine, memantine, riluzole.
4. Patients who experienced severe depression or suicide attempt in the last 5 years.
5. Severe untreated or under-treated psychiatric illness such as active suicidal ideation or behavior or depression.
6. Patients with a history of, or current, hypotension, bradycardia, or orthostatic hypotension.
7. Patients with hypertension already treated with more than 2 antihypertensive drugs.
8. Other active clinically significant illness, which could interfere with the study conduct, counter-indicate the study treatment, place the patient at risk during the trial, or compromise their study participation.
9. Any significant serious abnormality in the electrocardiogram (ECG), or a known history of long QTc syndrome.
10. Patients with severe hepatic impairment, or with severe renal impairment, or with any other significant abnormality in the physical examination or clinical laboratory results that, in the Investigator's opinion, would not be compatible with study participation or represent a risk for the patient while in the study.
11. Females who are pregnant or lactating, or who intend to become pregnant during the study period.
12. Patients with allergy under desensitization, with known psoriasis, or a known allergy/hypersensitivity to any ingredients of the trial medication or placebo.
13. History of alcohol or substance abuse in the previous 12 months.
14. Patients participating in any other study, and the use of any investigational therapy.

Primary outcomes:
measure: Change in Total Maximal Chorea (TMC) score of the UHDRS®
timeFrame: Between baseline and end of maintenance dose (after 10 weeks of treatment)
Secondary outcomes:
measure: Change in the Clinical Global Impression (CGI)
timeFrame: Between baseline and end of maintenance dose (after 10 weeks of treatment)
measure: Change in the Patient Global Impression (PGI)
timeFrame: Between baseline and end of maintenance dose (after 10 weeks of treatment)
measure: TMC-response defined as improvement ≥2 in TMC score
timeFrame: Between baseline and end of maintenance dose (after 10 weeks of treatment)
measure: Percentage of change in TMC score
timeFrame: Between baseline and end of maintenance dose (after 10 weeks of treatment)
measure: Change in the Total Motor Score (TMS) of the UHDRS®
timeFrame: Between baseline and end of maintenance dose (after 10 weeks of treatment)
measure: Change in the Gait sub-score of the UHDRS®
timeFrame: Between baseline and end of maintenance dose (after 10 weeks of treatment)
measure: Change in the Dystonia sub-score of the UHDRS®
timeFrame: Between baseline and end of maintenance dose (after 10 weeks of treatment)
measure: Change in the European Quality of Life scale (EQ-5D-5L)
timeFrame: Between baseline and end of maintenance dose (after 10 weeks of treatment)
Other outcomes:
Locations:
city: Angers
country: France
facility: Centre Hospitalier Universitaire Angers
city: Créteil
country: France
facility: CHU Hôpital Henri Mondor (APHP)
city: Lille
country: France
facility: Hôpital Roger Salengro - CHU Lille
city: Strasbourg
country: France
facility: Hopital de Hautepierre
city: Toulouse
country: France
facility: Hôpital Purpan - CHU Toulouse
city: Berlin
country: Germany
facility: Charité - Universitätsmedizin Berlin
city: Münster
country: Germany
facility: George Huntington Institut
city: Taufkirchen
country: Germany
facility: Kbo-Isar-Amper-Klinikum Taufkirchen
city: Ulm
country: Germany
facility: Hospital of University of Ulm
city: Bologna
country: Italy
facility: IRCCS Istituto delle Scienze Neurologiche di Bologna
city: Napoli
country: Italy
facility: Azienda Ospedaliera Universitaria Federico II
city: Roma
country: Italy
facility: IRCCS Casa Sollievo della Sofferenza
city: Roma
country: Italy
facility: Sant'Andrea University Hospital
city: Kraków
country: Poland
facility: Krakowska Akademia Neurologii Sp. z o.o.
city: Poznań
country: Poland
facility: Indywidualna Praktyka Lekarska Daniel Zielonka
city: Barcelona
country: Spain
facility: Hospital de la Santa Creu i Sant Pau
city: Bilbao
country: Spain
facility: Hospital Universitario de Cruces
city: Burgos
country: Spain
facility: Hospital Universitario de Burgos
city: Madrid
country: Spain
facility: Hospital Ramón y Cajal
city: Bern
country: Switzerland
facility: Universitaetsspital Bern - Inselspital
city: Cambridge
country: United Kingdom
facility: Cambridge University Hospitals NHS Foundation Trust Addenbrooke's Hospital
city: Manchester
country: United Kingdom
facility: NIHR Wellcome Trust Manchester Clinical Research Facility
city: Oxford
country: United Kingdom
facility: Oxford University Hospitals NHS Foundation Trust
Study officials:
References: