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A Study to Test Efficacy, Safety, and Pharmacokinetics of Certolizumab Pegol in Children and Adolescents With Moderately to Severely Active Crohn's Disease

Primary Purpose

Crohn's Disease

Status
Withdrawn
Phase
Phase 3
Locations
Study Type
Interventional
Intervention
Certolizumab pegol
Adalimumab
Placebo
Sponsored by
UCB Biopharma SRL
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Crohn's Disease focused on measuring Certolizumab pegol, Cimzia, Phase 3, Crohn's disease, Pediatric, Kids, OHANA, CD

Eligibility Criteria

6 Years - 17 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Participant must be 6 to 17 years, inclusive, at the time of signing informed consent/assent
  • Participant has been diagnosed with active Crohn's disease (CD) as confirmed by endoscopic examination with/without histological confirmation ≤12 weeks before the Screening Visit
  • Participant has moderately to severely active disease despite current treatment
  • Participant has an inadequate response or intolerance to conventional therapy
  • Participants are certolizumab pegol (CZP) and adalimumab (ADA) naïve

Exclusion Criteria:

  • Participant has had an extensive colonic resection, subtotal or total colectomy, diagnosis of short bowel syndrome or a history of >3 small bowel resections
  • Participant has had a primary failure (ie, lack of response within the first 12 weeks of treatment) to any anti-Tumor necrosis factor-α agent for treatment of Crohn's disease

Sites / Locations

    Arms of the Study

    Arm 1

    Arm 2

    Arm 3

    Arm Type

    Experimental

    Experimental

    Active Comparator

    Arm Label

    Certolizumab pegol low dose arm

    Certolizumab pegol high dose arm

    Adalimumab reference arm

    Arm Description

    Participants randomized to certolizumab pegol (CZP) who weigh ≥17 kg to <40 kg will receive placebo at Week 0 and a loading dose of 200 mg at Weeks 0, 2, and 4, followed by a maintenance dose of 100 mg CZP subcutaneously (sc) every 2 weeks (Q2W). Participants randomized to CZP who weigh ≥40 kg will receive placebo at Week 0 and a loading dose of 400 mg at Weeks 0, 2, and 4, followed by placebo and a maintenance dose of 200 mg CZP sc Q2W.

    Participants randomized to CZP who weigh ≥17 kg to <40 kg will receive placebo at Week 0 and a loading dose of 200 mg at Weeks 0, 2, and 4, followed by a maintenance dose of 200 mg CZP sc Q2W. Participants randomized to CZP who weigh ≥40 kg will receive placebo at Week 0 and a loading dose of 400 mg at Weeks 0, 2, and 4, followed by a maintenance dose of 300 mg CZP sc Q2W.

    Participants randomized to adalimumab who weigh ≥17 kg to <40 kg will receive a loading dose of 80 mg at Week 0 and 40 mg at Week 2, followed by a maintenance dose of 20 mg sc Q2W. Participants randomized to Adalimumab who weigh ≥40 kg will receive a loading dose of 160 mg at Week 0 and 80 mg at Week 2, 40 mg and placebo at week 4 followed by a maintenance dose of 40 mg sc and placebo Q2W.

    Outcomes

    Primary Outcome Measures

    Clinical remission based on total Pediatric Crohn's Disease Activity Index (PCDAI) score ≤10.0 points at Week 26
    Clinical remission is defined as a Pediatric Crohn's Disease Activity Index (PCDAI) score ≤ 10. The Pediatric Crohn's Disease Activity Index (PCDAI) consists of 4 domains (laboratory, height/weight, examination, and history) with several assessments that are converted into a total PCDAI score which can range from 0 to 100 points, with a higher score indicating more severe disease activity.

    Secondary Outcome Measures

    Clinical remission (modified) at Week 26
    Clinical remission (modified) is defined as a Pediatric Crohn's Disease Activity Index (PCDAI) score ≤ 10 and the abdominal pain and stool frequency items in the PCDAI having scores of 0. The Pediatric Crohn's Disease Activity Index (PCDAI) consists of 4 domains (laboratory, height/weight, examination, and history) with several assessments that are converted into a PCDAI score which can range from 0 to 100 points, with a higher score indicating more severe disease activity.
    Corticosteroid-free clinical remission at Week 26
    Corticosteroid free clinical remission is defined as not taking corticosteroids after tapering, within 4 weeks prior to Week 26, and total PCDAI score ≤10.0 points and the abdominal pain and stool frequency items in the PCDAI having scores of 0 at Week 26.
    Clinical response at Week 26 and Week 52
    Clinical response is defined as a decrease from Week 0 in Pediatric Crohn's Disease Activity Index (PCDAI) score of ≥ 15 points and a total PCDAI score ≤ 30 points. The Pediatric Crohn's Disease Activity Index (PCDAI) consists of 4 domains (laboratory, height/weight, examination, and history) with several assessments that are converted into a PCDAI score which can range from 0 to 100 points, with a higher score indicating more severe disease activity.
    Endoscopic remission at Week 26
    Endoscopic remission is defined as Simple Endoscopic Score for Crohn's Disease [SES-CD] from 0 to 2, with a higher score indicating more disease activity. SES-CD is based on four endoscopic variables (presence and size of ulcers, proportion of surface covered by ulcers, proportion of surface affected by disease, and presence and severity of stenosis). Each of the four SES-CD variables is scored from 0 to 3, with the sum of the scores for each variable ranging from 0 to 15, except for the presence and extent of stenosis, which ranges from 0 to 11, yielding a total SES-CD score of 0-56.
    Clinical remission based on total PCDAI score ≤10.0 points at Week 52
    Clinical remission is defined as a Pediatric Crohn's Disease Activity Index (PCDAI) score ≤ 10. The Pediatric Crohn's Disease Activity Index (PCDAI) consists of 4 domains (laboratory, height/weight, examination, and history) with several assessments that are converted into a total PCDAI score which can range from 0 to 100 points, with a higher score indicating more severe disease activity.
    Clinical remission (modified) at Week 52
    Clinical remission (modified) is defined as a Pediatric Crohn's Disease Activity Index (PCDAI) score ≤ 10 and the abdominal pain and stool frequency items in the PCDAI having scores of 0. The Pediatric Crohn's Disease Activity Index (PCDAI) consists of 4 domains (laboratory, height/weight, examination, and history) with several assessments that are converted into a PCDAI score which can range from 0 to 100 points, with a higher score indicating more severe disease activity.
    Incidence of serious Treatment Emergent Adverse Event (TEAEs)
    A Serious Treatment Emergent Adverse Event is any untoward medical occurrence that at any dose: Results in death Is life-threatening Requires inpatient hospitalization or prolongation of existing hospitalization Results in persistent disability/incapacity Is a congenital anomaly/birth defect Other important medical events which based on medical or scientific judgement may jeopardize the patients, or may require medical or surgical intervention to prevent any of the above
    Incidence of TEAEs leading to permanent withdrawal of investigational medicinal product
    An Adverse Event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.

    Full Information

    First Posted
    November 19, 2020
    Last Updated
    June 17, 2021
    Sponsor
    UCB Biopharma SRL
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    1. Study Identification

    Unique Protocol Identification Number
    NCT04643483
    Brief Title
    A Study to Test Efficacy, Safety, and Pharmacokinetics of Certolizumab Pegol in Children and Adolescents With Moderately to Severely Active Crohn's Disease
    Official Title
    A Phase 3, Randomized, Double-Blind, Multicenter Study Including an Active Reference Arm to Evaluate Efficacy, Safety, and Pharmacokinetics of Certolizumab Pegol in Children and Adolescents (6 to 17 Years of Age) With Moderately to Severely Active Crohn's Disease
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    June 2021
    Overall Recruitment Status
    Withdrawn
    Why Stopped
    Sponsor decision
    Study Start Date
    June 2021 (Anticipated)
    Primary Completion Date
    September 2023 (Anticipated)
    Study Completion Date
    April 2026 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    UCB Biopharma SRL

    4. Oversight

    Studies a U.S. FDA-regulated Drug Product
    Yes
    Studies a U.S. FDA-regulated Device Product
    No
    Product Manufactured in and Exported from the U.S.
    Yes
    Data Monitoring Committee
    Yes

    5. Study Description

    Brief Summary
    The purpose of the study is to assess efficacy, safety and tolerability of 2 dose regimens of certolizumab pegol

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Crohn's Disease
    Keywords
    Certolizumab pegol, Cimzia, Phase 3, Crohn's disease, Pediatric, Kids, OHANA, CD

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 3
    Interventional Study Model
    Parallel Assignment
    Model Description
    The study will be conducted in 3 parts: Part A will investigate 2 certolizumab pegol (CZP) dosing regimens with the objective of selecting a regimen that provides the highest rate of clinical remission. A cohort of participants will receive adalimumab (ADA) as a reference arm in Part A to assess clinical remission rates at Week 26 in this population. Endoscopic remission at Week 26 will also be evaluated Part B will assess maintenance of clinical remission from Week 26 through Week 52 Part C is an optional open-label extension (OLE) of CZP after Part B, and will evaluate the long-term safety of CZP through Week 156
    Masking
    ParticipantCare ProviderInvestigatorOutcomes Assessor
    Allocation
    Randomized
    Enrollment
    0 (Actual)

    8. Arms, Groups, and Interventions

    Arm Title
    Certolizumab pegol low dose arm
    Arm Type
    Experimental
    Arm Description
    Participants randomized to certolizumab pegol (CZP) who weigh ≥17 kg to <40 kg will receive placebo at Week 0 and a loading dose of 200 mg at Weeks 0, 2, and 4, followed by a maintenance dose of 100 mg CZP subcutaneously (sc) every 2 weeks (Q2W). Participants randomized to CZP who weigh ≥40 kg will receive placebo at Week 0 and a loading dose of 400 mg at Weeks 0, 2, and 4, followed by placebo and a maintenance dose of 200 mg CZP sc Q2W.
    Arm Title
    Certolizumab pegol high dose arm
    Arm Type
    Experimental
    Arm Description
    Participants randomized to CZP who weigh ≥17 kg to <40 kg will receive placebo at Week 0 and a loading dose of 200 mg at Weeks 0, 2, and 4, followed by a maintenance dose of 200 mg CZP sc Q2W. Participants randomized to CZP who weigh ≥40 kg will receive placebo at Week 0 and a loading dose of 400 mg at Weeks 0, 2, and 4, followed by a maintenance dose of 300 mg CZP sc Q2W.
    Arm Title
    Adalimumab reference arm
    Arm Type
    Active Comparator
    Arm Description
    Participants randomized to adalimumab who weigh ≥17 kg to <40 kg will receive a loading dose of 80 mg at Week 0 and 40 mg at Week 2, followed by a maintenance dose of 20 mg sc Q2W. Participants randomized to Adalimumab who weigh ≥40 kg will receive a loading dose of 160 mg at Week 0 and 80 mg at Week 2, 40 mg and placebo at week 4 followed by a maintenance dose of 40 mg sc and placebo Q2W.
    Intervention Type
    Drug
    Intervention Name(s)
    Certolizumab pegol
    Intervention Description
    Pharmaceutical form: Solution for injection Route of administration: Subcutaneous Subjects will receive certolizumab pegol in a pre-specified sequence during the Treatment Periods.
    Intervention Type
    Drug
    Intervention Name(s)
    Adalimumab
    Intervention Description
    Pharmaceutical form: Solution for injection Route of administration: Subcutaneous Subjects will receive adalimumab in a pre-specified sequence during the Treatment Periods.
    Intervention Type
    Drug
    Intervention Name(s)
    Placebo
    Intervention Description
    Pharmaceutical form: Solution for injection Route of administration: Subcutaneous Subjects will receive placebo in a pre-specified sequence during the Treatment Periods.
    Primary Outcome Measure Information:
    Title
    Clinical remission based on total Pediatric Crohn's Disease Activity Index (PCDAI) score ≤10.0 points at Week 26
    Description
    Clinical remission is defined as a Pediatric Crohn's Disease Activity Index (PCDAI) score ≤ 10. The Pediatric Crohn's Disease Activity Index (PCDAI) consists of 4 domains (laboratory, height/weight, examination, and history) with several assessments that are converted into a total PCDAI score which can range from 0 to 100 points, with a higher score indicating more severe disease activity.
    Time Frame
    Week 26
    Secondary Outcome Measure Information:
    Title
    Clinical remission (modified) at Week 26
    Description
    Clinical remission (modified) is defined as a Pediatric Crohn's Disease Activity Index (PCDAI) score ≤ 10 and the abdominal pain and stool frequency items in the PCDAI having scores of 0. The Pediatric Crohn's Disease Activity Index (PCDAI) consists of 4 domains (laboratory, height/weight, examination, and history) with several assessments that are converted into a PCDAI score which can range from 0 to 100 points, with a higher score indicating more severe disease activity.
    Time Frame
    Week 26
    Title
    Corticosteroid-free clinical remission at Week 26
    Description
    Corticosteroid free clinical remission is defined as not taking corticosteroids after tapering, within 4 weeks prior to Week 26, and total PCDAI score ≤10.0 points and the abdominal pain and stool frequency items in the PCDAI having scores of 0 at Week 26.
    Time Frame
    Week 26
    Title
    Clinical response at Week 26 and Week 52
    Description
    Clinical response is defined as a decrease from Week 0 in Pediatric Crohn's Disease Activity Index (PCDAI) score of ≥ 15 points and a total PCDAI score ≤ 30 points. The Pediatric Crohn's Disease Activity Index (PCDAI) consists of 4 domains (laboratory, height/weight, examination, and history) with several assessments that are converted into a PCDAI score which can range from 0 to 100 points, with a higher score indicating more severe disease activity.
    Time Frame
    Week 26; Week 52
    Title
    Endoscopic remission at Week 26
    Description
    Endoscopic remission is defined as Simple Endoscopic Score for Crohn's Disease [SES-CD] from 0 to 2, with a higher score indicating more disease activity. SES-CD is based on four endoscopic variables (presence and size of ulcers, proportion of surface covered by ulcers, proportion of surface affected by disease, and presence and severity of stenosis). Each of the four SES-CD variables is scored from 0 to 3, with the sum of the scores for each variable ranging from 0 to 15, except for the presence and extent of stenosis, which ranges from 0 to 11, yielding a total SES-CD score of 0-56.
    Time Frame
    Week 26
    Title
    Clinical remission based on total PCDAI score ≤10.0 points at Week 52
    Description
    Clinical remission is defined as a Pediatric Crohn's Disease Activity Index (PCDAI) score ≤ 10. The Pediatric Crohn's Disease Activity Index (PCDAI) consists of 4 domains (laboratory, height/weight, examination, and history) with several assessments that are converted into a total PCDAI score which can range from 0 to 100 points, with a higher score indicating more severe disease activity.
    Time Frame
    Week 52
    Title
    Clinical remission (modified) at Week 52
    Description
    Clinical remission (modified) is defined as a Pediatric Crohn's Disease Activity Index (PCDAI) score ≤ 10 and the abdominal pain and stool frequency items in the PCDAI having scores of 0. The Pediatric Crohn's Disease Activity Index (PCDAI) consists of 4 domains (laboratory, height/weight, examination, and history) with several assessments that are converted into a PCDAI score which can range from 0 to 100 points, with a higher score indicating more severe disease activity.
    Time Frame
    Week 52
    Title
    Incidence of serious Treatment Emergent Adverse Event (TEAEs)
    Description
    A Serious Treatment Emergent Adverse Event is any untoward medical occurrence that at any dose: Results in death Is life-threatening Requires inpatient hospitalization or prolongation of existing hospitalization Results in persistent disability/incapacity Is a congenital anomaly/birth defect Other important medical events which based on medical or scientific judgement may jeopardize the patients, or may require medical or surgical intervention to prevent any of the above
    Time Frame
    From Baseline to the Safety Follow-Up visit (Week 166)
    Title
    Incidence of TEAEs leading to permanent withdrawal of investigational medicinal product
    Description
    An Adverse Event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.
    Time Frame
    From Baseline to the Safety Follow-Up visit (Week 166)

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    6 Years
    Maximum Age & Unit of Time
    17 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    Inclusion Criteria: Participant must be 6 to 17 years, inclusive, at the time of signing informed consent/assent Participant has been diagnosed with active Crohn's disease (CD) as confirmed by endoscopic examination with/without histological confirmation ≤12 weeks before the Screening Visit Participant has moderately to severely active disease despite current treatment Participant has an inadequate response or intolerance to conventional therapy Participants are certolizumab pegol (CZP) and adalimumab (ADA) naïve Exclusion Criteria: Participant has had an extensive colonic resection, subtotal or total colectomy, diagnosis of short bowel syndrome or a history of >3 small bowel resections Participant has had a primary failure (ie, lack of response within the first 12 weeks of treatment) to any anti-Tumor necrosis factor-α agent for treatment of Crohn's disease
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    UCB Cares
    Organizational Affiliation
    001 844 599 2273 (UCB)
    Official's Role
    Study Director

    12. IPD Sharing Statement

    Plan to Share IPD
    Yes
    IPD Sharing Plan Description
    Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a prespecified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.
    IPD Sharing Time Frame
    Data from this study may be requested by qualified researchers six months after product approval in the US and/or Europe or global development is discontinued, and 18 months after trial completion
    IPD Sharing Access Criteria
    Qualified researchers may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal.
    IPD Sharing URL
    https://www.Vivli.org

    Learn more about this trial

    A Study to Test Efficacy, Safety, and Pharmacokinetics of Certolizumab Pegol in Children and Adolescents With Moderately to Severely Active Crohn's Disease

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