E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Wound Healing in Uraemic Calciphylaxis |
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E.1.1.1 | Medical condition in easily understood language |
Deposition of Calcium in the Blood Vessels |
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E.1.1.2 | Therapeutic area | Diseases [C] - Nutritional and Metabolic Diseases [C18] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10051714 |
E.1.2 | Term | Calciphylaxis |
E.1.2 | System Organ Class | 10027433 - Metabolism and nutrition 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 evaluate the effect of SNF472 on top of standard of care on promoting wound healing and other parameters of therapeutic response in haemodialysis patients with calciphylaxis |
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Patients with newly diagnosed calciphylaxis (diagnosed within 3 weeks of study start) 2. Patients who signed the written informed consent to participate in this clinical trial (prior to any clinical trial-related procedures being performed), after reading the Patient Information Sheet and Informed Consent Form (ICF), and who had the opportunity to discuss the clinical trial with the Investigator or designee 3. Males or females aged ≥18 4. Patients on maintenance haemodialysis 5. Patients with a minimum level of pain on VAS scale or on pain-killers stronger than NSAIDs 6. Females of child-bearing potential should use a highly effective contraceptive measure throughout the study AND have a negative serum pregnancy test at entry. Male patients having sexual relationship in which pregnancy can occur should take adequate contraceptive precautions (wear a condom) |
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E.4 | Principal exclusion criteria |
1. Body weight above 150 kg 2. BMI >35 and central(abdominal) ulcers 3. History of bisphosphonate treatment within 12 months before entering into the study 4. Patients being anticoagulated with citrate 5. Severely ill patients without reasonable expectation of survival for > 6 months according to the treating physician 6. Patients with scheduled parathyroidectomy during the run-in or study period 7. Female patients who are either intending to get pregnant or are undergoing treatment to get pregnant, as well as breast-feeding females 8. Participation in another clinical trial with an experimental drug within 90 days prior the inclusion 9. Any psychological, emotional problems, any disorders or resultant therapy that is likely to invalidate informed consent, or limit the ability of the patient to comply with the Clinical Trial Protocol requirements 10. Patients who, in the opinion of the Investigator, are considered unsuitable for any other reason |
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary lesion is the largest one - based on longitudinal and transverse measurements - and the one to be followed through imaging throughout the duration of the study by taking pictures of the lesion - A maximum of 3 lesions - including the primary one - will be thoroughly assessed during the study - Lesion score (Bates Jensen Assessment tool) will be performed during Weeks 1, 2, then 4, 6, 8, 10, 12 and EOS (FU) visit |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Weeks 1, 2, then 4, 6, 8, 10, 12 and EOS (FU) visit |
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E.5.2 | Secondary end point(s) |
- Wound infection, both for the primary and the secondary lesions - Systemic infection - Surgical treatment of the wound - Hospitalisation defined as being admitted for >24 hours - Course of the secondary lesions defined clinically as for the primary - Change in pain from week 0 to 12 according to 100 mm Visual Analog Pain Scale (VAS) - Pain scale done at Screening, weeks 1, 2, 4, 6, 8, 10, 12 and EOS(FU) visit * Change in dose and type of pain medication assessed as a continuous variable. Name, dose and number of pills captured. - Wound QOL score - Death |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1. Pain Score: at Screening, weeks 1, 2, 4, 6, 8, 10, 12 and EOS (FU) visit 2. Wound QoL Score: at Screening (for training purposes) and weeks 1, 6 and 12 |
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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 | Yes |
E.6.4 | Safety | No |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
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) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
E.8.2.4 | Number of treatment arms in the trial | 1 |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
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 | 4 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Germany |
Spain |
United Kingdom |
United States |
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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 | 0 |
E.8.9.1 | In the Member State concerned months | 9 |
E.8.9.1 | In the Member State concerned days | 17 |
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 | 1 |
E.8.9.2 | In all countries concerned by the trial days | 29 |