Efficacy and Safety on SOM3355 in Huntington's Disease Chorea

Phase 2

Active, not recruiting

• Conditions: Huntington Chorea
• Interventions: Drug: SOM3355 capsules; Drug: Placebo capsules

• Registration Number: NCT05475483
• Lead Sponsor: SOM Innovation Biotech SA

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2022-07-22
• Study First Submitted QC: 2022-07-26
• Study First Posted: 2022-07-27
• Study Start: 2022-08-02
• Status Verified: 2024-04
• Last Update Submitted: 2024-04-10
• Last Update Posted: 2024-04-11
• Primary Completion: 2024-06
• Study Completion: 2024-06

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 129

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
SOM3355 300 mg BID	SOM3355 capsules	Administration of SOM3355 in up-titration for 3 weeks up to the maintenance dose of 300 mg BID (twice daily) administered for 7 additional weeks, and down-titration for 2 weeks.
Placebo BID	Placebo capsules	Administration of matching Placebo BID (twice daily) for 12 weeks.
SOM3355 200 mg BID	SOM3355 capsules	Administration of SOM3355 in up-titration for 2 weeks up to the maintenance dose of 200 mg BID (twice daily) administered for 8 additional weeks, and down-titration for 2 weeks.

Primary Outcome Measures

Name	Time	Method
Change in Total Maximal Chorea (TMC) score of the UHDRS®	Between baseline and end of maintenance dose (after 10 weeks of treatment)	Total Maximal Chorea score of the Unified Huntington Disease Rating Scale is 0-28 and decrease means improvement.

Secondary Outcome Measures

Name	Time	Method
Change in the Clinical Global Impression (CGI)	Between baseline and end of maintenance dose (after 10 weeks of treatment)
TMC-response defined as improvement ≥2 in TMC score	Between baseline and end of maintenance dose (after 10 weeks of treatment)
Percentage of change in TMC score	Between baseline and end of maintenance dose (after 10 weeks of treatment)
Change in the Total Motor Score (TMS) of the UHDRS®	Between baseline and end of maintenance dose (after 10 weeks of treatment)	Total Motor Score is 0-124 and decrease means improvement.
Change in the Patient Global Impression (PGI)	Between baseline and end of maintenance dose (after 10 weeks of treatment)
Change in the Gait sub-score of the UHDRS®	Between baseline and end of maintenance dose (after 10 weeks of treatment)
Change in the Dystonia sub-score of the UHDRS®	Between baseline and end of maintenance dose (after 10 weeks of treatment)
Change in the European Quality of Life scale (EQ-5D-5L)	Between baseline and end of maintenance dose (after 10 weeks of treatment)

Trial Locations

Locations (23)

Hopital de Hautepierre; 🇫🇷 Strasbourg, France

Hôpital Purpan - CHU Toulouse; 🇫🇷 Toulouse, France

IRCCS Istituto delle Scienze Neurologiche di Bologna; 🇮🇹 Bologna, Italy

Sant'Andrea University Hospital; 🇮🇹 Roma, Italy

Azienda Ospedaliera Universitaria Federico II; 🇮🇹 Napoli, Italy

IRCCS Casa Sollievo della Sofferenza; 🇮🇹 Roma, Italy

Krakowska Akademia Neurologii Sp. z o.o.; 🇵🇱 Kraków, Poland

Hospital Ramón y Cajal; 🇪🇸 Madrid, Spain

Centre Hospitalier Universitaire Angers; 🇫🇷 Angers, France

CHU Hôpital Henri Mondor (APHP); 🇫🇷 Créteil, France

Hôpital Roger Salengro - CHU Lille; 🇫🇷 Lille, France

George Huntington Institut; 🇩🇪 Münster, Germany

Kbo-Isar-Amper-Klinikum Taufkirchen; 🇩🇪 Taufkirchen, Germany

Hospital of University of Ulm; 🇩🇪 Ulm, Germany

Hospital Universitario de Cruces; 🇪🇸 Bilbao, Spain

Indywidualna Praktyka Lekarska Daniel Zielonka; 🇵🇱 Poznań, Poland

NIHR Wellcome Trust Manchester Clinical Research Facility; 🇬🇧 Manchester, United Kingdom

Hospital de la Santa Creu i Sant Pau; 🇪🇸 Barcelona, Spain

Hospital Universitario de Burgos; 🇪🇸 Burgos, Spain

Universitaetsspital Bern - Inselspital; 🇨🇭 Bern, Switzerland

Cambridge University Hospitals NHS Foundation Trust Addenbrooke's Hospital; 🇬🇧 Cambridge, United Kingdom

Oxford University Hospitals NHS Foundation Trust; 🇬🇧 Oxford, United Kingdom

Charité - Universitätsmedizin Berlin; 🇩🇪 Berlin, Germany