E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
moderate to severe palmoplantar pustular psoriasis |
|
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10037159 |
E.1.2 | Term | Psoriasis pustular |
|
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 |
To evaluate efficacy of efalizumab in patients with moderate to severe palmoplantar pustular psoriasis |
|
E.2.2 | Secondary objectives of the trial |
To collect safety data for efalizumab in patients with moderate to severe palmoplantar pustular psoriasis |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Adult patient with (at least 6 months or longer) moderate to severe palmoplantar pustular psoriasis with or without psoriasis at other sites 2. PGA rating of moderate (4), severe (5) or very severe (6) for palmoplantar pustular psoriasis with at least ten fresh pustules at screening visit and baseline 3. Age 18 to 75 years 4. Body weight £125 kg 5. Naïve to efalizumab treatment 6. Discontinuation of any systemic psoriasis treatment prior to commencement of the study treatment. An appropriate washout period is required for these agents (e.g. for cyclosporin, corticosteroids, methotrexate, retinoids, MMF, thioguanine, hydroxyurea, sirolimus, azathioprine, and 6-MP); and for any systemic immunosuppressive treatment applied for psoriasis. The specific wash out requirements must be followed for each systemic therapy and a wash out period of at least one month prior to receiving the first dose of study drug (Study Day 0 = SD 0) is required, if not indicated otherwise. Application of PUVA treatment must have been discontinued one month prior to receiving the first dose of study drug (SD 0); biologic agents must not have been applied within three months prior to receiving the first dose of study drug (SD 0) 7. Discontinuation of all high potency topical corticosteroid treatments for psoriasis at least 14 days prior to receiving the first dose of study drug (SD 0) 8. Discontinuation of any investigational drug or treatment prior to commencement of the study treatment. A washout period of six months is required for these agents prior to receiving the first dose of study drug (SD 0) 9. Treatment regimens of b blockers, ACE inhibitors, antimalarial drugs, quinidine, interferon, or lithium stable for at least 28 days prior to receiving the first dose of study drug (SD 0) 10. No required vaccination (e.g., tetanus, booster, influenza vaccine) at least 14 days prior to receiving the first dose of study drug (SD 0). 11. Willingness to hold sun exposure reasonably constant and to avoid use of tanning booths or other UV light sources during the study 12. Willingness to participate in photo documentation 13. For women of childbearing potential: Use of an acceptable method of contraception (implants, oral contraceptives, intrauterine device, sterilization or sterilized partner) to prevent pregnancy and agreement to continue to practice an acceptable method of contraception for the duration of their participation in the study and up to 3 months after the last dose of efalizumab 14. Agreement to participate in the study 15. Signed informed consent |
|
E.4 | Principal exclusion criteria |
1. Guttate, erythrodermic or chronic plaque psoriasis as sole or predominant form of psoriasis 2. History of severe allergic or anaphylactic reactions to humanised monoclonal antibodies 3. History of or ongoing uncontrolled bacterial, viral, fungal, or atypical mycobacterial infection. This includes diagnoses that required more than 2 weeks of therapy, such as endocarditis and osteomyelitis that have been treated in the past 6 months. In addition, if the patient is currently receiving antibiotics, antivirals or antifungals for an infection or for suppression or prophylaxis for any diagnosis, the patient will be excluded 4. History of opportunistic infections (e.g., systemic fungal infections, parasites) 5. History of or ongoing active tuberculosis (TB) or other serious infections 6. History of clinically significant thrombocytopenia, bleeding disorders or hemolytic anemia 7. Previous exposure to efalizumab 8. Application of any biologic agent within 3 months prior to receiving the first dose of study drug (SD 0) 9. Application of systemic treatments within 3 months prior to receiving the first dose of study drug (SD 0) 10. Application of any systemic immunosuppressive treatment applied for any condition other than psoriasis within one month prior to receiving the first dose of study drug (SD0) 11. UV/PUVA treatment within 1 month prior to receiving first dose of study drug (SD 0) 12. Application of any investigational drug or treatment less than six months ago prior to receiving the first dose of study drug (SD 0) 13. Application of live or killed virus or bacteria vaccines within 14 days prior to receiving the first dose of study drug (SD 0) 14. Presence of malignancy within the past 5 years, including lymphoproliferative disorders. Patients with a history of fully resolved basal cell or squamous cell skin cancer may be enrolled 15. Diagnosis of hepatic cirrhosis, regardless of cause or severity 16. Hospital admission for cardiac disease, stroke, or pulmonary disease within the last year 17. History of drug abuse within the last 5 years 18. History of seropositivity for human immunodeficiency virus (HIV) 19. History of seropositivity for hepatitis B or C virus 20. WBC count <4,000µ/L or >14,000/µL 21. Hematocrit (HCT) <30% or hemoglobin (Hgb) level <11 g/dL 22. Platelet count <150,000 cells/µL 23. Hepatic enzymes >3 times the upper limit of normal 24. Serum creatinine >2 times the upper limit of normal 25. Pregnancy or lactation 26. Any medical condition that, in the judgment of the investigator, would jeopardize the patient’s safety following exposure to study drug 27. Patient unwilling or unable to follow the protocol requirements |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Primary endpoint: Number of fresh pustules (relative change at week 12 compared to baseline) Secondary efficacy outcome measures include: - The proportion of patients who achieve a PGA rating of absence of disease (1), very mild disease (2), or mild disease (3) at Day 84 - The proportion of patients who achieve a PGA rating of absence of disease (1), very mild disease (2), or mild disease (3) at Day 42 - The change from Day 0 to Day 84 in the following measures: DLQI, PAGA, PP-PASI, PASI |
|
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 | Information not present in EudraCT |
E.8.1.2 | Open | Information not present in EudraCT |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Information not present in EudraCT |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | Information not present in EudraCT |
E.8.1.7 | Other | Information not present in EudraCT |
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.8.3 |
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 | 4 |
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 | 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
|
End of trial is the last visit (follow up) of the last subject |
|
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 | 6 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial months | 6 |