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Prospective Observational Study on Ustekinumab (Stelara) Assessing Efficacy and Healthcare Resource Utilization in Crohn's Disease (PROSE)

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ClinicalTrials.gov Identifier: NCT03495973
Recruitment Status : Recruiting
First Posted : April 12, 2018
Last Update Posted : July 17, 2018
Sponsor:
Information provided by (Responsible Party):
Janssen-Cilag Ltd.

Brief Summary:
The main purpose of this study is to assess ustekinumab's ability to induce clinical response and remission ("effectiveness") at Week 16 and remission at Week 52 and Week 104 in participants with Crohn's disease (CD). Response and remission will be assessed using clinical parameters. Retention rate at week 52 and 104 will also be studied.

Condition or disease Intervention/treatment
Crohn Disease Drug: Ustekinumab

Study Type : Observational
Estimated Enrollment : 110 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Prospective Observational Study on Stelara Assessing Efficacy and Healthcare Resource Utilization in Crohn's Disease (PROSE)
Actual Study Start Date : October 25, 2017
Estimated Primary Completion Date : September 30, 2020
Estimated Study Completion Date : April 30, 2021

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Crohn's Disease
Drug Information available for: Ustekinumab

Group/Cohort Intervention/treatment
Participants with Crohn's Disease (CD)
Participants with CD will be assessed for the effectiveness of ustekinumab in accordance with national guidelines and routine standard of care. Data will be prospectively collected with the aid of the Swedish inflammatory bowel disease (IBD) registry, SWIBREG and medical records of each participant. Data will also be collected retrospectively from SWIBREG and other databases including national databases such as the participant registry in which cases the national databases will be considered source data.
Drug: Ustekinumab
Participants that are initiated on treatment with ustekinumab and are registered in Swibreg will be recruited into the cohort. All aspects of treatment and clinical management of participants will be in accordance with local clinical practice and applicable local regulations, and at the discretion of the participating physician.




Primary Outcome Measures :
  1. Percentage of Participants with Clinical Response at Week 16, Based on Harvey Bradshaw Index (HBI) [ Time Frame: Week 16 ]
    Clinical response for Crohn's disease (CD) is assessed by Harvey Bradshaw Index (HBI) and is defined as decrease of 3 points in baseline HBI score. HBI consists of clinical parameters: general well-being (0-4, where higher score means lower well-being), abdominal pain (0-3, higher score means more severe pain), number of liquid stools per day, abdominal mass (0-3, where higher score means presence of swelling in the abdomen), and complications (score 1 per item). Total score is the sum of individual parameters. The score ranges from a minimum score of 0 to no pre-specified maximum score as it depends on the number of liquid stools per day and number of complications, where higher scores indicates more severe disease and, score is presented as: less than (<)5 (remission), 5-7 (mild disease), 8-16 (moderate disease), and greater than (>)16 (severe disease).

  2. Percentage of Participants with Clinical Remission at Week 16, Based on HBI [ Time Frame: Week 16 ]
    Clinical remission for CD is assessed by HBI and is defined as HBI less than (<)5. HBI consists of clinical parameters: general well-being (0-4, where higher score means lower well-being), abdominal pain (0-3, higher score means more severe pain), number of liquid stools per day, abdominal mass (0-3, where higher score means presence of swelling in the abdomen), and complications (score 1 per item). Total score is the sum of individual parameters. The score ranges from a minimum score of 0 to no pre-specified maximum score as it depends on the number of liquid stools per day and number of complications, where higher scores indicates more severe disease and, score indicates: less than (<)5 (remission), 5-7 (mild disease), 8-16 (moderate disease), and greater than (>)16 (severe disease).

  3. Percentage of Participants with Clinical Remission at Week 52, Based on HBI [ Time Frame: Week 52 ]
    Clinical remission for CD is assessed by HBI and is defined as HBI <5. HBI consists of clinical parameters: general well-being (0-4, where higher score means lower well-being), abdominal pain (0-3, higher score means more severe pain), number of liquid stools per day, abdominal mass (0-3, where higher score means presence of swelling in the abdomen), and complications (score 1 per item). Total score is the sum of individual parameters. The score ranges from a minimum score of 0 to no pre-specified maximum score as it depends on the number of liquid stools per day and number of complications, where higher scores indicates more severe disease and, score is presented as: less than (<)5 (remission), 5-7 (mild disease), 8-16 (moderate disease), and greater than (>)16 (severe disease).

  4. Percentage of Participants with Clinical Remission at Week 104, Based on HBI [ Time Frame: Week 104 ]
    Clinical remission for CD is assessed by HBI and is defined as HBI <5. HBI consists of clinical parameters: general well-being (0-4, where higher score means lower well-being), abdominal pain (0-3, higher score means more severe pain), number of liquid stools per day, abdominal mass (0-3, where higher score means presence of swelling in the abdomen), and complications (score 1 per item). Total score is the sum of individual parameters. The score ranges from a minimum score of 0 to no pre-specified maximum score as it depends on the number of liquid stools per day and number of complications, where higher scores indicates more severe disease and, score is presented as: less than (<)5 (remission), 5-7 (mild disease), 8-16 (moderate disease), and greater than (>)16 (severe disease).


Secondary Outcome Measures :
  1. Percentage of Participants with Clinical Response at Week 52 and 104, Based on HBI [ Time Frame: Week 52 and 104 ]
    Clinical response for CD is assessed by HBI and is defined as decrease of 3 points in baseline HBI score. HBI consists of clinical parameters: general well-being (0-4, where higher score means lower well-being), abdominal pain (0-3, higher score means more severe pain), number of liquid stools per day, abdominal mass (0-3, where higher score means presence of swelling in the abdomen), and complications (score 1 per item). Total score is the sum of individual parameters. The score ranges from a minimum score of 0 to no pre-specified maximum score as it depends on the number of liquid stools per day and number of complications, where higher scores indicates more severe disease and, score is presented as: less than (<)5 (remission), 5-7 (mild disease), 8-16 (moderate disease), and greater than (>)16 (severe disease).

  2. Percentage of Participants with Clinical Remission at Week 52 and 104 who are Remitters at 16 Weeks [ Time Frame: Week 52 and 104 ]
    Clinical remission for CD is assessed by HBI and is defined as HBI <5. HBI consists of clinical parameters: general well-being (0-4, where higher score means lower well-being), abdominal pain (0-3, higher score means more severe pain), number of liquid stools per day, abdominal mass (0-3, where higher score means presence of swelling in the abdomen), and complications (score 1 per item). Total score is the sum of individual parameters. The score ranges from a minimum score of 0 to no pre-specified maximum score as it depends on the number of liquid stools per day and number of complications, where higher scores indicates more severe disease and, score is presented as: less than (<)5 (remission), 5-7 (mild disease), 8-16 (moderate disease), and greater than (>)16 (severe disease).

  3. Percentage of Participants with Clinical Remission at Week 104 who are Remitters at 52 Weeks [ Time Frame: Week 104 ]
    Clinical remission for CD is assessed by HBI and is defined as HBI <5. HBI consists of clinical parameters: general well-being (0-4, where higher score means lower well-being), abdominal pain (0-3, higher score means more severe pain), number of liquid stools per day, abdominal mass (0-3, where higher score means presence of swelling in the abdomen), and complications (score 1 per item). Total score is the sum of individual parameters. The score ranges from a minimum score of 0 to no pre-specified maximum score as it depends on the number of liquid stools per day and number of complications, where higher scores indicates more severe disease and, score is presented as: less than (<)5 (remission), 5-7 (mild disease), 8-16 (moderate disease), and greater than (>)16 (severe disease).

  4. Percentage of Participants with Clinical Remission at Week 52 and 104 who are Responders at 16 Weeks [ Time Frame: Week 52 and 104 ]
    Clinical remission for CD is assessed by HBI and is defined as HBI <5. Responders are defined as the participants with decrease of 3 points in baseline HBI score. HBI consists of clinical parameters: general well-being (0-4, where higher score means lower well-being), abdominal pain (0-3, higher score means more severe pain), number of liquid stools per day, abdominal mass (0-3, where higher score means presence of swelling in the abdomen), and complications (score 1 per item). Total score is the sum of individual parameters. The score ranges from a minimum score of 0 to no pre-specified maximum score as it depends on the number of liquid stools per day and number of complications, where higher scores indicates more severe disease.

  5. Percentage of Participants in Clinical Remission Overtime [ Time Frame: Up to Week 104 ]
    Clinical remission for CD is assessed by HBI and is defined as HBI <5. HBI consists of clinical parameters: general well-being (0-4, where higher score means lower well-being), abdominal pain (0-3, higher score means more severe pain), number of liquid stools per day, abdominal mass (0-3, where higher score means presence of swelling in the abdomen), and complications (score 1 per item). Total score is the sum of individual parameters. The score ranges from a minimum score of 0 to no pre-specified maximum score as it depends on the number of liquid stools per day and number of complications, where higher scores indicates more severe disease.

  6. Percentage of Participants with Corticosteroid Free Remission at Week 52 and 104 [ Time Frame: Week 52 and 104 ]
    Participants on corticosteroids at baseline will be analyzed according to the percentage of participants in remission at week 52 and 104 without corticosteroids to assess corticosteroids free remission.

  7. Participant's Time off Corticosteroids [ Time Frame: Up to week 104 ]
    Duration for which participants were off from corticosteroids will be determined.

  8. Percentage of Participants in Every 12 weeks/8 weeks (q12 w/q8 w) maintenance groups at Week 16 [ Time Frame: Week 16 ]
    Percentage of participants in q12 w/q8 w maintenance groups will be determined at Week 16.

  9. Percentage of Participants on Initial q12 w Maintenance Dose Escalated from q12 w to q8 w in Year 1 and 2 [ Time Frame: Year 1 and 2 ]
    Percentage of participants on initial q12 maintenance dose escalated from q12 w to q8 w in Year 1 and 2 will be determined.

  10. Percentage of Participants with Dose Optimization [ Time Frame: Year 1 and 2 ]
    Percentage of participants with dose optimized to other dose than 90 milligram (mg) q8 w/q12 w in maintenance in Year 1 and 2 will be determined.

  11. Average Maintenance Dose per Month [ Time Frame: Week 52 and 104 ]
    Average maintenance dose (in mg) taken per month will be determined.

  12. Mean Change from Baseline in Fecal Calprotectin (fCal) Levels at Week 16, 52 and 104 [ Time Frame: Baseline, Week 16, 52 and 104 ]
    Mean change from baseline in fCal (inflammatory biomarker) levels will be determined.

  13. Mean Change from Baseline in C-reactive Protein (CRP) Levels at Week 16, 52 and 104 [ Time Frame: Baseline, Week 16, 52 and 104 ]
    Mean change from baseline in C-reactive protein (CRP) (inflammatory biomarker) levels will be determined.

  14. Percentage of Participants with fCal Less Than (<)250 milligram per kilogram (mg/kg) [ Time Frame: Week 16, 52 and 104 ]
    Percentage of participants with fCal <250 mg/kg will be determined.

  15. Percentage of Participants with fCal levels <250 mg/kg at Week 16, 52 and 104 [ Time Frame: Week 16, 52 and 104 ]
    Percentage of participants with fCal levels <250 mg/kg at Week 16, 52 and 104 will be determined in total and amongst participants with fCal levels greater than (>)250 mg/kg at baseline.

  16. Percentage of Participants with Normalization of Plasma-CRP (P-CRP) [ Time Frame: Week 16, 52 and 104 ]
    Percentage of participants with normalization of P-CRP in in participants with abnormal P-CRP at baseline will be reported. Normalization of P-CRP is defined as the high-sensitive (Hs)-CRP level <3 milligram per Liter (mg/L) or CRP below threshold of abnormal CRP concentration.

  17. Change from Baseline in Short Health Scale (SHS) Score at Week 16, 52 and 104 [ Time Frame: Baseline, Week 16, 52 and 104 ]
    Quality of life will be assessed by SHS score. SHS is a health related quality of life (HRQoL) questionnaire in which the participants rate the disease impact on 4 important aspects of subjective health (symptoms, function, worry, and general well-being). Responses are scored on 100-mm visual analogue scales and presented as individual scores for each of the four questions. The scores are then added for a total score. Total score ranges from 0 (low disease activity) to 100 (high disease activity).

  18. Change from Baseline in EuroQoL 5-Dimension 5-Level Health Status (EQ-5D-5L) Questionnaire at Week 16, 52 and 104 [ Time Frame: Baseline, Week 16, 52 and 104 ]
    Quality of life will be assessed by EQ-5D-5L score. The EQ-5D-5L descriptive system comprises following 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each of 5 dimension is divided into 5 levels of perceived problems (Level 1: no problem, Level 2: slight problems, Level 3: moderate problems, Level 4: severe problems, and Level 5: extreme problems). The participant selects an answer for each of 5 dimensions considering response that best matches his or her health "today". The descriptive system can be represented as a health state.

  19. Change from Baseline in EQ-5D-5L Visual Analog Scale (VAS) at Week 16, 52 and 104 [ Time Frame: Baseline, Week 16, 52 and 104 ]
    The EQ-VAS self-rating scale records respondent's own assessment of his or her overall health status at time of completion, on a scale of 0 (worst imaginable health) to 100 (best imaginable health).

  20. Change from Baseline in the Presence of Extra Intestinal Manifestations at Week 16, 52 and 104 [ Time Frame: Baseline, Week 16, 52 and 104 ]
    Change in the presence of extra intestinal manifestations, that is arthralgia, peripheral arthritis, anchylosing spondylitis, uveitis, erythema nodosum, pyoderma gangrenosum, primary sclerosing cholangitis, psoriatic arthritis and psoriasis will be determined.

  21. Healthcare Resource: Number of Imaging Procedures [ Time Frame: Up to week 104 ]
    Number of imaging procedures completed as a part of healthcare resources used in participants will be reported.

  22. Healthcare Resource: Number of Laboratory Test [ Time Frame: Up to Week 104 ]
    Number of Laboratory tests completed as a part of healthcare resources used in participants will be reported.

  23. Healthcare Resource: Number of Surgical Procedures [ Time Frame: Up to Week 104 ]
    Number of surgical procedures completed as a part of healthcare resources used in participants will be reported.

  24. Healthcare Resource: Number of Dual Energy X-ray Absorptiometry (DEXA) Scans [ Time Frame: Up to Week 104 ]
    Number of DEXA scans performed as a part of healthcare resources used will be reported.

  25. Healthcare Resource: Number of Participants with Endoscopies Performed [ Time Frame: Up to Week 104 ]
    Number of participants with endoscopies performed as a part of healthcare resources used will be reported.

  26. Healthcare Resource: Number of Days of Hospitalization [ Time Frame: Up to Week 104 ]
    Number of days of hospitalization (defined as number of days from the day of admission to discharge) as a part of healthcare resources used will be reported.

  27. Healthcare Resource: Number of Sick-Leaves [ Time Frame: Up to Week 104 ]
    Number of sick-leaves taken by participants as a part of healthcare resources used will be reported.

  28. Healthcare Resource: Number of Participants Using Concomitant Treatment [ Time Frame: Up to Week 104 ]
    Number of participants using concomitant treatment as a part of healthcare resources used will be reported.

  29. Healthcare Resource: Number of Health Care Visits [ Time Frame: Up to Week 104 ]
    Number of health care visits to nurses, dieticians and doctors as a part of healthcare resources used will be reported.

  30. Number of Participants with Malignancy [ Time Frame: Up to Week 104 ]
    Number of participants with malignancy developed during the study will be reported.

  31. Number of Participants Requiring Antibiotics for any Infection Occurred During the Study [ Time Frame: Up to Week 104 ]
    Number of participants requiring antibiotics for any infection occurred during the study will be reported.



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Participants with a confirmed Crohn's disease (CD) aged over 18 years treated with ustekinumab.
Criteria

Inclusion Criteria:

  • Participant has a diagnosis of CD confirmed by investigator
  • Participants with active CD who have had an inadequate response with, lost response to, or were intolerant to either conventional therapy or a biologic therapy or have medical contraindications to such therapies
  • The participant must sign informed consent before enrollment in the study. The consent form permits extraction of data from SWIBREG at baseline and during participation of the study and linkages to the Patient Registry (Patientregistret), SCB's registry USA for occupational details (Longitudinell integrationsdatabas for sjukforsakrings- och arbetsmarknadsstudi er), the Social Insurance registry on long-term sick leave (Forsakringskassans register gallande sjukskrivningar) and the Prescription registry (Lakemedelsregistret)
  • The participant must have been initiated on treatment with ustekinumab within the last 2 weeks (that is including those who already discontinued) or started on ustekinumab treatment less than (<) 3 months ago if treatment start was documented in SWIBREG within 2 weeks of onset and has a record of Harvey Bradshaw Index (HBI) when starting ustekinumab

Exclusion Criteria:

  • The participant is enrolled in a clinical trial (randomized or non-randomized) in which the treatment of CD is dictated by a study protocol. If the participant is participating in another observational study (non-interventional), the participant may be included in this observational study
  • Received an investigational drug (including investigational vaccines) or used an invasive investigational medical device within 60 days before the start of the study or the first data collection time point
  • Participants who started ustekinumab despite contraindications for use
  • Participants with planned stop of treatment with ustekinumab within 24 months from onset of treatment, that is bridging to other treatment or pregnancy planned

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03495973


Contacts
Contact: Study Contact 844-434-4210 JNJ.CT@sylogent.com

Locations
Sweden
Universitetssjukhuset Örebro Recruiting
Örebro, Sweden, SE-70185
Sponsors and Collaborators
Janssen-Cilag Ltd.
Investigators
Study Director: Janssen-Cilag Ltd. Clinical Trial Janssen-Cilag Ltd.

Additional Information:
Responsible Party: Janssen-Cilag Ltd.
ClinicalTrials.gov Identifier: NCT03495973     History of Changes
Other Study ID Numbers: CR108430
CNTO1275CRD4006 ( Other Identifier: Janssen-Cilag Ltd. )
First Posted: April 12, 2018    Key Record Dates
Last Update Posted: July 17, 2018
Last Verified: July 2018

Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Crohn Disease
Inflammatory Bowel Diseases
Gastroenteritis
Gastrointestinal Diseases
Digestive System Diseases
Intestinal Diseases
Ustekinumab
Dermatologic Agents