A study to assess the safety and efficacy of a new medicine SBT-020 in patients with Early Stage Huntington’s Disease.

Phase 1

Conditions: Huntington's Disease
MedDRA version: 20.0Level: PTClassification code 10070668Term: Huntington's diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Registration Number: EUCTR2016-003730-25-NL

Lead Sponsor: Stealth Bio Therapeutics Inc

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 24

Inclusion Criteria

1.Male or female patient with a DNA confirmed diagnosis (CAG expansion of 36 or more repeats in the HTT gene) of HD
2.At least 18 years of age
3.Unified Huntington’s Disease Rating Scale (UHDRS) Total Motor Score (TMS) of 5 or more
4.Unified Huntington’s Disease Rating Scale (UHDRS) Total Functional Capacity Score (TFC) of 7 or more
5.?PCr of at least 32.4 seconds, measured by dynamic 31P-MRS of the calf muscles.
6.Absence of evidence of any significant active or chronic disease (apart from HD), following a detailed medical and surgical history, a complete physical examination including vital signs, 12-lead ECG, haematology, blood chemistry, and urinalysis, that might interfere with the study activities or patient’s safety by participating in the study, as judged by the investigator. Psychiatric comorbidities to HD (such as a major depressive disorder), are allowed under the scrutiny of the investigator.
7.Agrees to refrain from making any new, major life-style changes (e.g. starting a new diet or changing exercise pattern)
8.Must agree to use adequate methods of contraception. Female subjects of childbearing potential must use two adequate forms of contraception, one of which must be a barrier method for the duration of the study and for 30 days after the last dose. Male subjects with a partner of childbearing potential must use two adequate forms of contraception, one of which must be a barrier method, for the duration of the study and for 30 days after the last dose.
9.Able to participate and willing to give written informed consent and to comply with the study restrictions.

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 24
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1.Positive test for drugs of abuse, such as metamphetamines and cocaine, at screening or pre-dose, except those prescribed by a physician for treatment of intercurrent medical issues due to HD.
2.History (within 3 months of screening) of alcohol consumption exceeding 2 standard drinks per day on average. Alcohol consumption will be prohibited during study confinement and at least 24 hours before screening and before each scheduled visit.
3.History of active malignancy within the last 5 years, with the exception of localized or in situ carcinoma (e.g., skin basal or squamous cell carcinoma).
4.Positive Hepatitis B surface antigen (HBsAg), Hepatitis C antibody (HCV Ab), or human immunodeficiency virus antibody (HIV Ab) at screening.
5.Aspartate transaminase (AST), alanine transaminase (ALT), gamma glutamyl transferase (GGT) or total bilirubin levels >1.5 times the upper limit of normal at screening.
6.eGFR < 60 mL/min (calculated by the Modification of Diet in Renal Disease equation) at Screening.
7.Clinically significant abnormalities, as judged by the Investigator, in laboratory test results (including hepatic and renal panels, complete blood count, chemistry panel and urinalysis). In the case of uncertain or questionable results, tests performed during screening may be repeated before randomization to confirm eligibility or judged to be clinically irrelevant for HD patients.
8.Participation in an investigational drug or device study within 3 months prior to screening.
9.Loss or donation of blood over 500 mL within three months (males) or four months (females) prior to screening.
10.Concomitant disease or condition that could interfere with, or for which the treatment of might interfere with, the conduct of the study, or that would, in the opinion of the Investigator, pose an unacceptable risk to the subject in this study.
11.Presence of any contraindication to have MRI scans performed (e.g. claustrophobia, pacemaker, vascular clips etc.). MRI contraindications for both the 7 Tesla (part 1 and 2) and 3 T (part 2 only) MRIs will be assessed, using MRI contra-indications questionnaires and, if necessary, a plain radiograph
12.Unwillingness to refrain from smoking more than half pack cigarettes per day (i.e. 10 units).
13.Specific cardiac abnormalities on the resting ECG at screening: QTcF> 450 or < 300 msec, evidence of atrial fibrillation, atrial flutter, complete branch block, Wolf-Parkinson-White Syndrome, or cardiac pacemaker.
14.Any confirmed significant allergic reactions (urticaria or anaphylaxis) against any drug, or multiple drug allergies (non-active hay fever is acceptable).
15.Unwillingness or inability to comply with the study protocol for any other reason.