Clinical trial NCT03886246: Effisayil™ ON: A Study to Test Long-term Treatment With Spesolimab in People With Generalized Pustular Psoriasis Who Took Part in a Previous Study
Official 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)
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
Conditions: Generalized Pustular Psoriasis
Lead sponsor: Boehringer Ingelheim
Sponsor class: INDUSTRY
First posted: 2019-03-22
Enrollment: 132
Sex: ALL
Minimum age (years): 12
Maximum age (years): 75
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This study is open to people with generalized pustular psoriasis (GPP). People can only take part if they have completed treatment in a previous study with spesolimab (1368-0013 or 1368-0027).

The goal of this study is to find out how well people with GPP tolerate long-term treatment with spesolimab. The study also tests whether spesolimab helps improve GPP symptoms and how quickly the symptoms improve after a flare-up.

Every participant gets spesolimab for almost 5 years (252 weeks). Depending on their symptoms and whether they had a GPP flare during the previous trial, they get spesolimab every few weeks. When participants have a GPP flare during this trial, they get spesolimab as an infusion into a vein.

Participants visit their doctors regularly. During these visits, the doctors collect information on any health problems of the participants. To assess the study endpoints, doctors regularly check participants' skin.


Eligibility 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.

Primary outcomes:
measure: Occurrence of treatment emergent adverse events (TEAEs) up to week 252 of maintenance treatment
timeFrame: Up to 252 Weeks
Secondary outcomes:
measure: The reoccurrence of a GPP flare defined by GPPGA
timeFrame: Up to 252 Weeks
measure: Time to first achievement of a GPPGA score of 0 or 1 (Patients received flare rescue Treatment)
timeFrame: Up to 252 Weeks
measure: A GPPGA pustulation sub-score of 0 indicating no visible pustules, by visit
timeFrame: Up to 252 Weeks
measure: Change from baseline in Psoriasis Symptom Scale (PSS) score, by visit
timeFrame: Up to 252 Weeks
Other outcomes:
Locations:
city: Auburn Hills
country: United States
facility: Oakland Hills Dermatology
state: Michigan
city: New York
country: United States
facility: Icahn School of Medicine at Mount Sinai
state: New York
city: Caba
country: Argentina
facility: Hospital Italiano de Buenos Aires
city: Bruxelles
country: Belgium
facility: Brussels - UNIV Saint-Luc
city: Vitacura
country: Chile
facility: Clínica Dermacross S.A.
city: Guangzhou
country: China
facility: Sun yet-sen Memorial Hospital, Sun yet-sen Univesity
city: Hangzhou
country: China
facility: The Second Affiliated Hospital Zhejiang University School of Medicine
city: Shanghai
country: China
facility: Shanghai Skin Disease Hospital
city: Shanghai
country: China
facility: Huashan Hospital, Fudan University
city: Xi'an
country: China
facility: Second Affiliated Hospital of Xi'an JiaoTong University
city: Bordeaux
country: France
facility: HOP Saint-André
city: Nice
country: France
facility: HOP l'Archet
city: Paris
country: France
facility: HOP Saint-Louis
city: Reims
country: France
facility: HOP Robert Debré
city: Bad Bentheim
country: Germany
facility: Fachklinik Bad Bentheim
city: Berlin
country: Germany
facility: Charite Universitätsmedizin Berlin KöR
city: Bonn
country: Germany
facility: Universitätsklinikum Bonn AöR
city: Frankfurt am Main
country: Germany
facility: Universitätsklinikum Frankfurt
city: München
country: Germany
facility: Klinikum der Universität München - Campus Innenstadt
city: Münster
country: Germany
facility: Westfälische Wilhelms-Universität Münster
city: Oldenburg
country: Germany
facility: Klinikum Oldenburg AöR
city: Rozzano (MI)
country: Italy
facility: Istituto Clinico Humanitas
city: Aichi, Nagoya
country: Japan
facility: Nagoya City University Hospital
city: Ibaraki, Inashiki-gun
country: Japan
facility: Tokyo Medical University Ibaraki Medical Center
city: Saitama, Iruma-gun
country: Japan
facility: Saitama Medical University Hospital
city: Tokyo, Hachioji
country: Japan
facility: Tokyo Medical University Hachioji Medical Center
city: Seoul
country: Korea, Republic of
facility: Severance Hospital
city: George Town
country: Malaysia
facility: Hospital Pulau Pinang-Pulau Pinang-21953
city: Ipoh
country: Malaysia
facility: Hospital Raja Permaisuri Bainun
city: Johor Bahru
country: Malaysia
facility: Hospital Sultanah Aminah
city: Johor Bahru
country: Malaysia
facility: Hospital Sultan Ismail
city: Kota Kinabalu
country: Malaysia
facility: Queen Elizabeth Hospital-Kota Kinabalu-40735
city: Kuala Lumpur
country: Malaysia
facility: Hospital Kuala Lumpur
city: Kuching, Sarawak
country: Malaysia
facility: Sarawak General Hospital
city: Muar
country: Malaysia
facility: Hospital Pakar Sultanah Fatimah
city: Selangor
country: Malaysia
facility: Hospital Selayang
city: Monterrey
country: Mexico
facility: Hospital Universitario Dr Jose Eleuterio Gonzalez
city: Davao City
country: Philippines
facility: Southern Philippines Medical Center -Davao-62091
city: Iloilo City, Iloilo
country: Philippines
facility: Iloilo Doctors Hospital
city: Makati City
country: Philippines
facility: Center for Skin Research, Testing and Product Development
city: Chelyabinsk
country: Russian Federation
facility: SBHI Chelyabinsk Reg.Clin.Derma.Dispen.
city: Kazan
country: Russian Federation
facility: LLC "Medical Center Azbuka Zdorovia"
city: Kirov
country: Russian Federation
facility: FSBEI HE "Kirov State Medical University"
city: Saratov
country: Russian Federation
facility: Saratov State Med.Univ.n.a.Razumovskogo
city: St. Petersburg
country: Russian Federation
facility: LLC Skin Disease Clinic of Pier Volkenstein, St. Petersburg
city: Singapore
country: Singapore
facility: Singapore General Hospital
city: Esplugues Del Llobregat
country: Spain
facility: Hospital Sant Joan de Déu
city: Linkou
country: Taiwan
facility: Chang Gung Medical Foundation (CGMF) - Linkou Bran
city: Taipei
country: Taiwan
facility: National Taiwan University Hospital
city: Bangkok
country: Thailand
facility: Institute of Dermatology
city: Bangkok
country: Thailand
facility: Ramathibodi Hospital
city: Sousse
country: Tunisia
facility: Farhat Hached Hospital
city: Tunisia
country: Tunisia
facility: Hedi Chaker Hospital, Department of Dermatology
city: Tunis
country: Tunisia
facility: La Rabta Hospital
city: Istanbul
country: Turkey
facility: Marmara Universitesi Pendik Egitim ve Arastirma Hastanesi
city: Ha Noi
country: Vietnam
facility: National Hospital of Dermatology and Venereology
city: Ho Chi Minh
country: Vietnam
facility: HCMC Hospital of Dermato-Venereology-Ho Chi Minh-66092
Study officials:
References: