E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Generalized Pustular Psoriasis |
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E.1.1.1 | Medical condition in easily understood language |
Generalized Pustular Psoriasis |
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E.1.1.2 | Therapeutic area | Diseases [C] - Skin and Connective Tissue Diseases [C17] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10037159 |
E.1.2 | Term | Psoriasis pustular |
E.1.2 | System Organ Class | 100000004858 |
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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 long-term safety and efficacy of BI 655130 in patients with GPP, who have completed previous BI 655130 trials and are qualified for entry in this trial |
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E.2.2 | Secondary objectives of the trial |
The primary endpoint is the occurrence of treatment emergent adverse events (TEAEs) up to week 252 of maintenance treatment |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Male or female patients who have completed the treatment period without premature discontinuation in the previous BI 655130 trial (1368-0013 or 1368-0027) and are willing and able to continue treatment in the current trial 2. Women of childbearing potential must be ready and able to use highly effective methods of birth control per ICH M3 (R2) that result in a low failure rate of less than 1% per year when used consistently and correctly. A list of contraception methods meeting these criteria is provided in Section 4.2.2.3 as well as in the patient information. Note: A woman is considered of childbearing potential, i.e. fertile, following menarche and until becoming postmenopausal unless permanently sterile. Permanent sterilisation methods include hysterectomy, bilateral salpingectomy and bilateral oophorectomy. Tubal ligation is not a method of permanent sterilization. A postmenopausal state is defined as no menses for 12 months without an alternative medical cause. 3. Signed and dated written informed consent for the current trial 1368-0025, in accordance with ICH-GCP and local legislation prior to admission to the current trial |
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E.4 | Principal exclusion criteria |
1. Evidence of flare symptoms of moderate/severe intensity at screening 2. Treatment with any restricted medication as specified in Table 4.2.2.1:1, or any drugs considered by the investigator likely to interfere with the safe conduct of the study since the last visit of the previous BI 651330 trial and during the screening period for the current trial, with exception of methotrexate, cyclosporine, or retinoids started following rescue treatment for GPP flare in trial 1368-0027. 3. Severe, progressive, or uncontrolled hepatic disease, defined as >3- fold Upper Limit of Normal (ULN) elevation in AST or ALT or alkaline phosphatase, or >2-fold ULN elevation in total bilirubin. 4. Patients with congestive heart disease, as assessed by the investigator. 5. Relevant chronic or acute infections including human immunodeficiency virus (HIV) or viral hepatitis. A patient can be rescreened if the patient was treated and is cured from acute infection. 6. Active or Latent tuberculosis (TB): - Patients with active tuberculosis should be excluded - Patients will be screened with Interferon Gamma Release Assay (IGRA) such as QuantiFERON®-TB-Gold Plus or T-spot®. Patients with positive IGRA (indicating active or latent tuberculosis) are excluded unless they have completed treatment for active or latent tuberculosis per investigator discretion, at the time of screening. - Patients with indeterminate QuantiFERON®-TB-Gold Plus or invalid/borderline T-spot® may be retested with IGRA (once) or Tuberculin Skin test (TST). - TST or any alternative test/procedure (as per local standards) to rule out TB can be performed if IGRA is not available or indeterminate. A TST reaction ≥10mm (≥5mm if receiving ≥15mg/d prednisone or other immunosuppressant) is considered positive. Patients with a positive TST are excluded unless they have completed treatment as above. 7. History of allergy/hypersensitivity to a systemically administered trial medication agent or its excipients. 8. Any documented active or suspected malignancy at screening, except appropriately treated basal cell carcinoma of the skin, squamous cell carcinoma of the skin or in situ carcinoma of uterine cervix. 9. Women who are pregnant, nursing, or who plan to become pregnant while in the trial. Women who stop nursing before the study drug administration do not need to be excluded from participating; they should refrain from breastfeeding up to 16 weeks after the study drug administration (see Section 4.2.2). 10. Major surgery (major according to the investigator's assessment) performed since the last visit of previous BI 655130 trial or planned during the current trial, e.g. hip replacement, aneurysm removal, stomach ligation), as assessed by the investigator. 11. Evidence of a current or previous disease, medical condition (including chronic alcohol or drug abuse or any condition) other than GPP, surgical procedure, psychiatric or social problems, medical examination finding (including vital signs and electrocardiogram [ECG]), or laboratory value at the screening outside the reference range that in the opinion of the investigator is clinically significant and would make the study participant unreliable to adhere to the protocol, comply with all study visits/procedures or to complete the trial, compromise the safety of the patient or compromise the quality of the data. 12. Patients with a confirmed active infection with SARS-CoV-2 (as confirmed by PCR test). A patient may be enrolled once recovered from SARS-CoV-2 infection according to the investigator's assessment. 13. Presence of acute demyelinating neuropathy |
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E.5 End points |
E.5.1 | Primary end point(s) |
1) occurrence of treatment emergent adverse events (TEAEs) up to week 252 of maintenance treatment |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
1) The reoccurrence of a GPP flare defined by GPPGA (as defined in Section 3.1) 2) Time to first achievement of a GPPGA score of 0 or 1 (Patients received flare rescue Treatment) 3) A GPPGA pustulation sub-score of 0 indicating no visible pustules, by visit (Patients received flare rescue treatment) 4) Change from baseline in Psoriasis Symptom Scale (PSS) score, by visit (Patients received flare rescue treatment) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1) 252 weeks 2) 252 weeks 3) 252 weeks 4) 252 weeks |
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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 | 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 | Yes |
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.8.2.4 | Number of treatment arms in the trial | 1 |
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 | 6 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 14 |
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 |
Argentina |
Chile |
China |
Japan |
Malaysia |
Mexico |
Philippines |
Thailand |
Tunisia |
United States |
Viet Nam |
Russian Federation |
Turkey |
Belgium |
France |
Germany |
Italy |
Spain |
Korea, Republic of |
Taiwan |
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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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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 8 |
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 | 23 |