Effisayil™ ON: A Study to Test Long-term Treatment With Spesolimab in People With Generalized Pustular Psoriasis Who Took Part in a Previous Study

• Register: ClinicalTrials.gov
• Last refreshed on: 15 January 2024
• Main ID: NCT03886246
• Date of registration: 21/03/2019
• Prospective Registration: Yes
• Primary sponsor: Boehringer Ingelheim
• Public title: Effisayil™ ON: A Study to Test Long-term Treatment With Spesolimab in People With Generalized Pustular Psoriasis Who Took Part in a Previous Study
• Scientific title: Effisayil™ ON: An Open-label, Long Term Extension Study to Assess the Safety and Efficacy of Spesolimab Treatment in Patients With Generalized Pustular Psoriasis (GPP)
• Date of first enrolment: May 27, 2019
• Target sample size: 131
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT03886246
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Argentina, Belgium, Chile, China, France, Germany, Italy, Japan, Korea, Republic of, Malaysia, Mexico, Philippines, Russian Federation, Singapore, Spain, Taiwan, Thailand, Tunisia, Turkey, United States, Vietnam

Key inclusion & exclusion criteria

Inclusion Criteria:

- Male or female patients who have completed the treatment period without premature
discontinuation in the previous spesolimab trial and are willing and able to continue
treatment in the current trial

- 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.

- Signed and dated written informed consent and assent for the current trial 1368-0025,
in accordance with ICH-GCP and local legislation prior to admission to the current
trial

Exclusion Criteria:

- Evidence of flare symptoms of moderate/severe intensity at screening.

- Treatment with any restricted medication as specified in the protocol, or any drugs
considered by the investigator likely to interfere with the safe conduct of the study
since the last visit of the previous spesolimab trial and during the screening period
for the current trial, with the exception of methotrexate, cyclosporine, or retinoids
started following rescue treatment for GPP flare in trial 1368-0027.

- Severe, progressive, or uncontrolled hepatic disease, defined as >3- fold Upper Limit
of Normal (ULN) elevation in Aspartate Transaminase (AST) or Alanine Aminotransferase
(ALT) or alkaline phosphatase, or >2- fold ULN elevation in total bilirubin.

- Patients with congestive heart disease, as assessed by the investigator.

- Relevant chronic or acute infections including human immunodeficiency virus (HIV) or
viral hepatitis. A patient can be re-screened if the patient was treated and is cured
from acute infection.

- 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.

- History of allergy/hypersensitivity to a systemically administered trial medication
agent or its excipients.

- 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.

Further exclusion criteria apply.



Age minimum: 12 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Generalized Pustular Psoriasis

Intervention(s)

Drug: Spesolimab

Primary Outcome(s)

Occurrence of treatment emergent adverse events (TEAEs) up to week 252 of maintenance treatment [Time Frame: Up to 252 Weeks]

Secondary Outcome(s)

A GPPGA pustulation sub-score of 0 indicating no visible pustules, by visit [Time Frame: Up to 252 Weeks]

The reoccurrence of a GPP flare defined by GPPGA [Time Frame: Up to 252 Weeks]

Change from baseline in Psoriasis Symptom Scale (PSS) score, by visit [Time Frame: Up to 252 Weeks]

Time to first achievement of a GPPGA score of 0 or 1 (Patients received flare rescue Treatment) [Time Frame: Up to 252 Weeks]

Secondary ID(s)

2018-003080-56

1368-0025

Source(s) of Monetary Support

Please refer to primary and secondary sponsors