E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Moderate to severe primary Raynaud’s,
OR patients with Raynaud’s phenomenon secondary to autoimmune diseases, such as scleroderma |
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MedDRA Classification |
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
Primary objectives: 1) Evaluate improvement in patient’s health, compared to during that patient’s placebo usage, when patients with Raynaud’s phenomenon apply the treatment gel, MQX-503, in anticipation of a likely Raynaud’s triggering event. This is determined by: Improvements in the patient’s Raynaud’s Condition Score, which is made up of the following three components: i) Number of Raynaud’s events ii) Duration of Raynaud’s events iii) Pain and symptoms associated with Raynaud’s.
2) Assess the safety, i.e., the frequency and severity of adverse events associated with MQX-503 in the treatment of patients with Raynaud’s phenomenon.
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E.2.2 | Secondary objectives of the trial |
Secondary objectives: 1) Evaluate improvement in patient’s health, compared to during placebo group, when patients with Raynaud’s phenomenon apply the treatment gel, MQX-503, in anticipation of a likely Raynaud’s triggering event, as determined by the Global Assessment Questions and the patient’s Health Assessment Questions.
2) For the patients with scleroderma, evaluate the reduction, compared to placebo patient group, in the emergence of digital ulcers for those who applied MQX-503 at least twice a day.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Outpatients, 18 years to 70 years. 2. Patients with a clinical diagnosis of Raynaud’s phenomenon as determined by a history of cold sensitivity with pallor or cyanosis of the digits or an observed event by the study physician and have a history of at least two Raynaud’s events daily during a typical winter day. 3. Patients who agree to apply the test gels to their fingers as specified in the protocol. 4. Patients who are willing to discontinue current vasodilator therapies two weeks prior to Visit 1 5. Patients who have recently participated in a clinical trial and are willing to wait four weeks prior to Visit 1. 6. Patients who agree not to use any other investigational medications or approved therapies to treat Raynaud’s phenomenon and its symptoms while participating in this study. Such medications include: other dosages forms of nitroglycerin, isosorbide dinitrate, fenoldopam mesylate, milrinone lactate, nifedipine, diltiazem, felodipine, nimodipine, nisoldipine, and verapamil. 7. Negative pregnancy test in fertile women prior to the first study treatment and who agree to use effective contraception throughout the study 8. Patients who are able to give written informed consent and comply with all study requirements
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E.4 | Principal exclusion criteria |
1. Patients who concurrently use any nitrate medication or medications known to interact with Nitroglycerin such as sildenafil, and other treatments for erectile dysfunction. 2. Patients who concurrently use any medication which might interfere with the study medication, specifically calcium channel blockers and the compounds listed above in prohibited concomitant medications. 3. Patients who have a known allergy to Nitroglycerin or common topical gel ingredients. 4. Patients with a history of migraine, cluster or vascular headaches, chronic pain (defined as pain of three hour duration or longer on a daily basis) with greater intensity than the pain associated with Raynaud’s phenomenon or other chronic pain condition in their fingers. 5. Patients who have a history of an unstable medical problem or any current medical condition that, in the judgment of the investigator, would contraindicate the administration of the study medication, interfere with the study evaluations, or interfere with the patient’s ability to comply with the study protocol. 6. Patients with cognitive or language difficulties that would impair completion of the pain assessment instruments. 7. Patients who, within the past three months, have had either a myocardial infarction, uncontrolled congestive heart failure, unstable angina, uncontrolled hypotension, or uncontrolled hypertension (defined as patients not being treated medically to control these conditions). 8. Patients who participated in a study of any investigational drug within four weeks prior to Visit 1. 9. Patients who have screening laboratory values which are 20% or greater of the upper or lower limit of normal or which are considered to be clinically significant to the investigator. 10. Patients who have had major abdominal, thoracic, or vascular surgery within six months of Visit 1. 11. Patients with non-epithelialized skin lesions or interfering skin conditions at time of screening in the area where the gel is to be applied. 12. Pregnant or nursing women. 13. Women of childbearing potential who are unable or unwilling to comply with the contraceptive requirements during the study period.
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E.5 End points |
E.5.1 | Primary end point(s) |
The efficacy of MQX-503 will be assessed, when compared to the placebo group, in terms of the improvement in the patient’s health as measured by decreases in the patient’s RCS which is a composite of the number of Raynaud events in a week, the average symptoms associated with Raynaud’s phenomenon in the fingers for that same week, and the average duration of Raynaud events following application of the treatment gel, when comparing the treatment gels containing the active to those containing the placebo gel. Secondary efficacy measurements will include patient improvement as determined by HAQ and GAQ. The safety of MQX-503 will be assessed from the frequency and severity of adverse events as reported by the patient to the clinic personnel. In addition, for those patients with scleroderma, the percent of patients who develop an emergence of digital ulcers will be compared between the MQX-503 active group and the placebo group. |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | Information not present in EudraCT |
E.6.2 | Prophylaxis | Information not present in EudraCT |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Information not present in EudraCT |
E.6.7 | Pharmacodynamic | Information not present in EudraCT |
E.6.8 | Bioequivalence | Information not present in EudraCT |
E.6.9 | Dose response | Information not present in EudraCT |
E.6.10 | Pharmacogenetic | Information not present in EudraCT |
E.6.11 | Pharmacogenomic | Information not present in EudraCT |
E.6.12 | Pharmacoeconomic | Information not present in EudraCT |
E.6.13 | Others | Information not present in EudraCT |
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) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | Yes |
E.8.1.7 | Other | Yes |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
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 | No |
E.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 8 |
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 | Information not present in EudraCT |
E.8.7 | Trial has a data monitoring committee | Yes |
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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The end of the trial will be the last visit of the last subject undergoing the trial, as provided in the protocol |
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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 | 1 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |