EffisayilTM ON: A study to test long-term treatment with spesolimab in people with generalized pustular psoriasis who took part in a previous study

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Boehringer Ingelheim International GmbH, Boehringer Ingelheim Espana S.A.

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Skin and Connective Tissue Diseases [C17]

Trial duration

27 May 2019 → ongoing

Decision date (initial)

2024-02-19

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

External identifiers

EU CT number

2023-509216-28-00

EudraCT number

2018-003080-56

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety, Therapy

To evaluate the long-term safety and efficacy of spesolimab in patients with GPP, who have completed previous spesolimab trials and are qualified for entry in this trial

Conditions and MedDRA coding

Generalized Pustular Psoriasis

Study design 3 periods

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. Male or female patients who have completed the treatment period without premature discontinuation in the previous spesolimab 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 the patient, parent(s) (or patient’s legal guardian) 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 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 8

1. Evidence of flare symptoms of moderate/severe intensity at screening
2. Treatment with any restricted medication, 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.
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 re-screened 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. Further criteria apply.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Occurrence of treatment emergent adverse events (TEAEs) up to week 252 of maintenance treatment

Secondary endpoints 4

1. The reoccurrence of a GPP flare defined by GPPGA
2. Time to first achievement of a GPPGA score of 0 or 1 in patients who received flare rescue treatment
3. A GPPGA pustulation sub-score of 0 indicating no visible pustules, by visit in patients who received flare rescue treatment
4. Change from baseline in Psoriasis Symptom Scale (PSS) score, by visit in patients who received flare rescue treatment

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Boehringer Ingelheim International GmbH

Sponsor organisation

Boehringer Ingelheim International GmbH

Address

Binger Strasse 173

City

Ingelheim Am Rhein

Postcode

55216

Country

Germany

Scientific contact point

Organisation

Boehringer Ingelheim International GmbH

Contact name

CT Disclosure & Data Transparency

Public contact point

Organisation

Boehringer Ingelheim International GmbH

Contact name

CT Disclosure & Data Transparency

Boehringer Ingelheim Espana S.A.

Sponsor organisation

Boehringer Ingelheim Espana S.A.

Address

Carrer Prat De La Riba 50, Sant Cugat Del Valles Sant Cugat Del Valles

City

Barcelona

Postcode

08174

Country

Spain

Scientific contact point

Organisation

Boehringer Ingelheim Espana S.A.

Contact name

CT Disclosure & Data Transparency

Public contact point

Organisation

Boehringer Ingelheim Espana S.A.

Contact name

CT Disclosure & Data Transparency

Sponsor responsibilities

Article 77 compliance

Boehringer Ingelheim International GmbH

Contact point sponsor

Boehringer Ingelheim International GmbH

Article 77 implementation

Boehringer Ingelheim International GmbH

Locations

5 EU/EEA countries · 13 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 58 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

4 submissions — initial application plus substantial / non-substantial modifications