Vedolizumab for Chronic pouchitis | Pouchitis

Inclusion criteria

• {"criterion_text":"- In the opinion of the investigator, the subject, parent, or legal guardian is capable of understanding and complying with protocol requirements."}
• {"criterion_text":"- The subject, parent or legal guardian has signed and dated a written, informed consent form and subject assent form and any required privacy authorization before the initiation of any study procedures."}
• {"criterion_text":"- The subject is aged 2 to 17 years, inclusive, at the time of screening and first dose."}
• {"criterion_text":"- The subject weighs ≥10 kg at the time of screening and first dose."}
• {"criterion_text":"- The subject has a history of proctocolectomy and IPAA as treatment for ulcerative colitis (UC), Crohn’s disease (CD), familial adenomatous polyposis, or other underlying conditions, such as Hirschsprung's disease, for which construction of a puch was medically indicated, completed at least 1 year before the screening visit."}
• {"criterion_text":"- \"The subject has active chronic pouchitis, defined by an mPDAI score ≥5 assessed using the average of subject-reported clinical symptoms from 3 days during a 5-day period immediately before screening endoscopy (ie, video pouchoscopy with biopsy) or bowel preparation for endoscopy, and a minimum mPDAI endoscopic subscore of 2 (outside the staple or suture line) and either: a) Has had ≥1 previous episodes of pouchitis within 1 year before the screening visit, with symptoms lasting at least 4 weeks, treated with ≥2 weeks of antibiotic or otherprescription therapy (ie, other antibiotics, probiotics, immunomodulators, or anti-TNFs within 1 year before screening). or b) Has had an inadequate response with, or lost response to, or is intolerant to antibiotic therapy (ie, requiring maintenance antibiotic therapy taken continuously for ≥4 weeks immediately before the baseline endoscopy visit or not able to receive or continue antibiotic treatment due to intolerance or other contraindication).\""}
• {"criterion_text":"- \"A male subject who is sexually active with a female partner of childbearing potential agrees to use a barrier method of contraception (ie, condom with spermicide)* from signing of parental informed consent/subject assent throughout the duration of the study and for 18 weeks after last dose. The female partner of a male subject should also be advised to use a highly effective method of contraception.\""}
• {"criterion_text":"- A female subject of childbearing potential who is sexually active with a male partner agrees to use a highly effective method of contraception* from signing of parental informed consent/subject assent throughout the duration of the study and for 18 weeks after last dose."}

Exclusion criteria

• {"criterion_text":"- The subject has symptoms believed to be predominantly due to irritable pouch syndrome."}
• {"criterion_text":"- The subject has isolated cuffitis."}
• {"criterion_text":"- The subject is found to have dysplasia at the screening endoscopy."}
• {"criterion_text":"- The subject has any prior exposure to approved or investigational anti-integrins including, but not limited to natalizumab, efalizumab, etrolizumab, or AMG 181, or mucosal addressin cell adhesion molecule-1 (MAdCAM-1) antagonists, or rituximab as a treatment for pouchitis."}
• {"criterion_text":"- The subject has received either (1) an investigational biologic agent (other than those listed in Exclusion Criterion #1) within 60 days or 5 half-lives before screening (whichever is longer); or (2) an approved biologic or biosimilar agent for inflammatory bowel disease within 2 weeks before the first dose of study drug or at any time during the screening period"}
• {"criterion_text":"- The subject has mechanical complications of the pouch (ie, pouch stricture or pouch fistula)."}
• {"criterion_text":"- The subject has a diverting stoma."}
• {"criterion_text":"- The subject currently requires or has a planned surgical intervention during the study."}

Primary endpoints

• {"endpoint_text":"- 01. The primary efficacy endpoint is clinical (mPDAI) remission (defined as an mPDAI score <5 and a reduction of overall mPDAI score by ≥2 points from baseline) assessed at W14.","definition_or_measurement_approach":"Defined as an mPDAI score <5 and a reduction of overall mPDAI score by ≥2 points from baseline, assessed at Week 14."}

Secondary endpoints

• {"endpoint_text":"- 01.Clinical (mPDAI) remission (defined as an mPDAI score <5 and a reduction of overall mPDAI score by ≥2 points from baseline) assessed at W34.","definition_or_measurement_approach":"Defined as mPDAI <5 and reduction ≥2 points from baseline, assessed at Week 34."}
• {"endpoint_text":"- 02. PDAI remission (defined as PDAI score <7 and a decrease in PDAI score by ≥3 points from baseline) at W14.","definition_or_measurement_approach":"Defined as PDAI score <7 and decrease ≥3 points from baseline, assessed at Week 14."}
• {"endpoint_text":"- 03. PDAI remisssion (defined as PDAI score <7 and a decrease in PDAI score by >3 points from baseline) at W34","definition_or_measurement_approach":"Defined as PDAI score <7 and decrease >3 points from baseline, assessed at Week 34."}
• {"endpoint_text":"- 04. Clinical (mPDAI) response (defined as a decrease in mPDAI score by ≥2 points from baseline) at W14","definition_or_measurement_approach":"Defined as decrease in mPDAI by ≥2 points from baseline, assessed at Week 14."}
• {"endpoint_text":"- 05. Clinical (mPDAI) response (defined as a decrease in mPDAI score by >2 points fom baseline) at W34.","definition_or_measurement_approach":"Defined as decrease in mPDAI by >2 points from baseline, assessed at Week 34."}
• {"endpoint_text":"- 06. Change from baseline in mPDAI clinical symptom subscore at W14.","definition_or_measurement_approach":"Change from baseline in mPDAI clinical symptom subscore measured at Week 14."}
• {"endpoint_text":"- 07. Change from baseline in mPDAI total score at W34","definition_or_measurement_approach":"Change from baseline in total mPDAI score measured at Week 34."}
• {"endpoint_text":"- 08. Change from baseline in PDAI total score and PDAI clinical symptoms, endoscopic, and histologic subscores at W14","definition_or_measurement_approach":"Change from baseline in PDAI total and subscores (clinical, endoscopic, histologic) assessed at Week 14."}
• {"endpoint_text":"- 09. Change from baseline in PDAI total score and PDAI clinical symptoms, endoscopic and histologic subscores at W34.","definition_or_measurement_approach":"Change from baseline in PDAI total and subscores (clinical, endoscopic, histologic) assessed at Week 34."}
• {"endpoint_text":"- 10. Index scores, visual analogue scale (VAS), and changes from baseline for each in the EQ-5D-Y Proxy Version 1.0 at W14 and W34.","definition_or_measurement_approach":"EQ-5D-Y Proxy Version 1.0 index scores and VAS and changes from baseline measured at Weeks 14 and 34."}

Methods

• Brochures and printed visit guides targeted to parents/caregivers and pediatric patients (country-specific brochures and parent letters available for multiple Member States).
• Parent letters and GP/HCP letters sent to healthcare professionals to support site referrals (country-specific HCP/GP letters present).
• Short informational videos for patients/parents (country-specific video materials listed in recruitment documents).
• Patient-facing materials such as patient cards, appointment/visit passports, and stickers distributed at sites.
• Digital app/eDiary and web back-up tools for subject reporting and study visit support (subject-facing screen reports and app materials listed).

Belgium

Earliest CTIS Part Ii Submission Date

05-06-2024

Latest Decision Or Authorization Date

27-06-2024

Processing Time Days

22

Number Of Sites

1

Number Of Participants

1

Croatia

Earliest CTIS Part Ii Submission Date

19-06-2024

Latest Decision Or Authorization Date

05-07-2024

Processing Time Days

16

Number Of Sites

1

Number Of Participants

2

Czechia

Earliest CTIS Part Ii Submission Date

06-06-2024

Latest Decision Or Authorization Date

03-07-2024

Processing Time Days

27

Number Of Sites

1

Number Of Participants

2

Greece

Earliest CTIS Part Ii Submission Date

10-06-2024

Latest Decision Or Authorization Date

04-07-2024

Processing Time Days

24

Number Of Sites

1

Number Of Participants

1

Italy

Earliest CTIS Part Ii Submission Date

03-04-2024

Latest Decision Or Authorization Date

26-06-2024

Processing Time Days

84

Number Of Sites

6

Number Of Participants

10

Poland

Earliest CTIS Part Ii Submission Date

28-05-2024

Latest Decision Or Authorization Date

02-07-2024

Processing Time Days

35

Number Of Sites

1

Number Of Participants

5

Spain

Earliest CTIS Part Ii Submission Date

20-05-2024

Latest Decision Or Authorization Date

02-07-2024

Processing Time Days

43

Number Of Sites

2

Number Of Participants

4

Primary sponsor

Full Name

Takeda Development Center Americas Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

QPS

Name

PPD Development LP

Name

PPD Global Ltd.

Name

PPD Global Central Labs

Name

Almac Clinical Services Limited

Responsibilities

Secondary packaging, labelling, distribution, returns and destruction of kits

Third parties

• {"country":"United States","full_name":"QPS","duties_or_roles":"","organisation_type":"Industry"}
• {"country":"France","full_name":"Quipment","duties_or_roles":"Quipment will manage the supply, setup/inspection/safety check, storage, logistics, pick & pack, shipping, verification of calibration, provide access to Quipsite, technical support","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Signant Health Global LLC","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom (Northern Ireland)","full_name":"Almac Clinical Services Limited","duties_or_roles":"Almac will secondary package and label the kits along with the distribution and returns and destruction.","organisation_type":"Pharmaceutical company"}
• {"country":"Canada","full_name":"Alimentiv Inc.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Acelabio (US) Inc.","duties_or_roles":"Embedding, processing, cutting and digitalization of samples","organisation_type":"Pharmaceutical company"}
• {"country":"Greece","full_name":"PPD Global Ltd.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"Belgium","full_name":"PPD Global Central Labs","duties_or_roles":"","organisation_type":"Pharmaceutical company"}