Treatment of Huntington´s disease with OSU 6162 - a pilot study

Phase 1

• Conditions: Patients with Huntington´s disease

• Registration Number: EUCTR2009-015926-13-SE
• Lead Sponsor: Sahlgrenska University Hospital

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2009-12-04
• Last Update Posted: 9 May 2016

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 24

Inclusion Criteria

1. Subjects who have Huntington´s disease diagnosed in a clinical investigation and this has been confirmed in a molecular genetic test showing a CAG repeat length in the range of 39 - 50 repeats.
2. In the total functional capacity scale (TFC) the patients will have between 3 and 13 points indicating that the disease has not reached the two most severe stages. TFC consists of estimations on 5 variables; engagement in occupation, capacity to handle financial affairs, capacity to manage domestic responsibilities, capacity to perform activities of daily living, and in which setting the care of the patient can be provided. Each item can be given 0 to 2 or 3 points according to the importance of the item. Zero points are given when the patient has no or very poor function on a specific item. The sum of all items gives an estimation of the functional capacity of the patient. TFC is the best measurement available to evaluate the progress of all aspects of the diseases (motor, behavioral and cognitive).
3. Signed informed consent by patients.
4. If the general cognitive function of the patient is considered to be mildly impaired an informed consent will also be signed by a close relative living together with the patie

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

Exclusion Criteria

1. Significant co-morbidity and alcohol or drug abuse.
2. Pregnant women.
3. Women of childbearing age not on contraceptives.
4. Patients with a disease so severe that a legal representative is necessary.
5. Sick sinus syndrome; resting heart rate below 50 beats per minute; congestive heart failure classified as functional Class III or IV by the New York Heart Association; myocardial infarction within six months of randomization; a prolonged QTc interval at screen or pretreatment (defined as a QTc interval of> 450 msec for males or> 470 msec for females); atrio-ventricular conduction block; other clinically significant heart conditions which would negatively impact on the patient completing the study.
6. Clinically significant liver and/or an elevation in either total bilirubin, alkaline phosphatase, LDH, SGOT above the laboratory reference. Clinically significant renal disease and/or an elevation in serum creatinine above the laboratory reference.
7. Presence of active neoplastic disease.
8. Patients who have had electroconvulsive therapy within 90 days.
9. Patients with any surgical or medical condition which, in the judgment of the clinical investigator, might interfere with the absorption, distribution, metabolism or excretion of the drug.
10. Patients who are using drugs capable of inducing hepatic enzyme metabolism (e.g., barbiturates, rifampicin, carbamazepine, phenytoin, primidone) within the previous
4 weeks (or 5 half lives of inducing agent, whichever is longer) before the enrollment in this study.
11. Patients with a medical history of seizures.
12. Unstable therapies are not allowed but stable therapies are allowed. A stable therapy is defined as having started at least 4 weeks before the study and continued to be unchanged during the study period. Examples of stable therapies on neuroleptics are: haloperidol or risperidon (highest allowed dose 3 mg/daily); antidepressant therapy such as citalopram (highest allowed dose 40 mg/daily), mirtazapin (higest allowed dose 45 mg daily) or sertraline (higest allowed dose 100 mg/daily). Other stable therapies with hypnotics and anxiolytics are also allowed if they are given at doses recommend by the manufactures. Analgetics such as NSAID, acetyl salicylic acid and paracetamol are permitted.
13. Use of acute or chronic medications for other medical conditions are allowed based on clinical judgment. Occasional use of over-the-counter (OTC) medications is allowed at the investigator's discretion. All concomitant medications, whether OTC or prescription, are required to be noted on the Concomitant Medication from.
14. Tetrabenazine is not permitted during the study and the wash out period before the study is 4 weeks.
15. Suicidal patients according to the clinical estimation or more than 2 points on item 9 of the Beck Depression Inventroy (BDI) scale are excluded.
16. Abnormal laboratory parameters such as: Hemoglobine, white blood cells count, electrolytes, TSH, T4, B12, folic acid. All levels above the laboratory references level are considered abnormal.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The study will be conducted in order to investigate the effect of OSU 6162 on Huntington´s disease and the safety of this product in 24 patients (men and women) who have Huntington´s disease diagnosed in a clinical investigation and confirmed in a molecular genetic test. .;Secondary Objective: There is no secondary objective;Primary end point(s): Primary endpoint is to investigate the therapeutic effects of OSU 6162 as measured by the motor, cognitive, behavior and functional protocol used in Registry 2005 which is a slight modification from the Unified Huntington Disease Rating Scale (UHDRS 1996). The Trail Making test A will also be used.