E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10070668 |
E.1.2 | Term | Huntington's disease |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To investigate whether Rilmenidine (oral preparation) can safely be taken and is well tolerated by patients suffering from Huntington’s Disease (HD). This will be achieved by regular, periodic assessments of the patients’ physical and mental health monitoring for adverse effects and disease progression. |
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E.2.2 | Secondary objectives of the trial |
Secondary objectives are to assess if there are a) any efficacy effects; and b) any ability to change the biomarker profiles away from that predicted. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Confirmed diagnosis of Huntington’s Disease on the basis of qualifying clinical signs and symptoms as well as a relevant family history, or a CAG repeat of >36 on exon 1 of the htt gene.
2. HD stage 1-3 with a total functioning capacity (TFC) on the Unified Huntington’s Disease Rating Scale 1999 (UHDRS) of at least 5.
3. Able to self care independently and are ambulant.
4. Aged from 18 and 70.
5. Women of childbearing age neither pregnant nor planning to conceive during the period of the study.
6. English speaking and able to provide written, informed consent.
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E.4 | Principal exclusion criteria |
1. An ongoing clinically significant and unstable General Medical Condition (including but not limited to; asthma, chronic obstructive pulmonary disease COPD, unstable ischaemic heart disease IHD, congestive cardiac failure CCF, left bundle branch block LBBB or a cerebral vascular accident CVA) confirmed via past medical history or baseline medical physical examination and investigations.
2. Prescribed anti-hypertensive medication or any drug known to be contraindicated or to have an adverse interaction with Rilmenidine (viz. a Monoamine Oxidase Inhibitor).
3. Known hypersensitivity to Rilmenidine.
4. Ongoing significant mental illness determined by evidence or a history of a psychotic or affective (depression or mania) episode in the six months prior to Baseline Interview assessed using the Diagnostic and Statistical Manual of Mental Disorders criteria (Fourth Edition with Text Revision; American Psychiatric Association).
5. Prescribed typical or atypical anti-psychotic medication (only if being prescribed explicitly for treatment of a psychotic illness).
6. Pregnant or breastfeeding female patients, including those planning to conceive during the period of the trial.
7. Substance (alcohol or illegal/prescription drug) misuse in the six months prior to the baseline assessment.
8. Known co-morbid major neurological disorder (incl. Parkinson’s Disease or dementia), HIV/AIDS or Hepatitis (HBV or HCV).
9. Previous neurosurgery to the brain.
10. Marked clinically adverse abnormalities on laboratory investigations including creatinine clearance <15mg/min or creatinine serum level >177 Umol/l.
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E.5 End points |
E.5.1 | Primary end point(s) |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 4 |
E.8.9.1 | In the Member State concerned months | 7 |
E.8.9.1 | In the Member State concerned days | 29 |
E.8.9.2 | In all countries concerned by the trial years | 4 |
E.8.9.2 | In all countries concerned by the trial months | 7 |
E.8.9.2 | In all countries concerned by the trial days | 29 |