Ulcerative Colitis
Conditions
Keywords
Upadacitinib, RINVOQ, Ulcerative Colitis, UC, Corticosteroids, Immunosuppressants, Biologic Therapy
Brief summary
Ulcerative colitis (UC) is a type of inflammatory bowel disease that causes inflammation and bleeding from the lining of the rectum and colon (large intestine). This study will assess how safe and effective Upadacitinib is in treating pediatric participants with UC. Adverse events and change in disease activity will be assessed. Upadacitinib (RINVOQ) is a drug approved in adults for moderate- to severely active UC and is being developed for moderate- to severely active UC in pediatric participants. This study is conducted in 2 periods: Period 1 is comprised of two phases: an 8-week open-label induction phase which means that the study doctor and patients know that participants will receive UPA Dose-A (or the adult equivalent based on body weight) followed by a 44-week double-blind maintenance phase meaning that neither the participants nor the study doctors will know which dose of upadacitinib will be given(UPA Dose B or Dose C). Period 2 is a 260 week open-label extension (OLE) of Period 1. Approximately 110 pediatric participants with moderate to severely active UC will be enrolled at up to 100 sites worldwide. Participants will receive upadacitinib oral tablets once daily or oral solution twice daily at approximately the same time each day, with or without food. Participants will be followed up for 30 days after each phase (i.e. after induction, maintenance, OLE) and only if a participant doesn't continue into the next phase. There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, checking for side effects and completing questionnaires.
Interventions
DRUGUpadacitinib
Oral Solution/ Tablets
Sponsors
AbbVie
Study design
Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
2 Years to 17 Years
Healthy volunteers
No
Inclusion criteria
* Active UC with an AMS of 5 to 9 points and endoscopic subscore of 2 to 3. * Demonstrate an inadequate response, loss of response, intolerance, or medical contraindications to corticosteroids, immunosuppressants, and/or biologic therapy.
Exclusion criteria
* Partcipants with previous exposure to JAK inhibitors (e.g., tofacitinib, baricitinib, filgotinib, upadacitinib). * Females who are pregnant, breastfeeding, or considering becoming pregnant during the study and for approximately 30 days after the last dose of study drug.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants Achieving Adapted Mayo score (AMS) Clinical Remission (Period 1) | Week 8 | The Mayo score is a tool designed to measure disease activity for ulcerative colitis. The AMS is a composite of the following subscores: stool frequency subscore (SFS), rectal bleeding subscore (RBS) and endoscopy subscore (MES). AMS ranges from 0 to 9 where higher scores represent more severe disease. Clinical remission is defined as an AMS \< or = 2, with SFS \< or = 1 and not higher than baseline, RBS of 0, and MES \< or = 1. |
| Percentage of Participants Achieving AMS Clinical Remission Among Week 8 Responders per AMS (Period 1) | Week 52 | The Mayo score is a tool designed to measure disease activity for ulcerative colitis. AMS ranges from 0 to 9 where higher scores represent more severe disease. Clinical remission is defined as an AMS ≤ 2, with SFS ≤ 1 and not higher than Baseline, RBS of 0, and MES ≤ 1. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants Achieving Endoscopic Improvement (Period 1) | Week 8 | Endoscopic Improvement is defined as MES \< or = 1. |
| Percentage of Participants Achieving Partial Mayo Score (PMS) Clinical Remission (Period 1) | Week 8 | The Mayo score is a tool designed to measure disease activity for ulcerative colitis. The PMS is a composite of the following subscores: SFS, RBS and physician's global assessment (PGA). The PMS ranges from 0 to 9 with higher scores representing more severe disease. PMS clinical remission is defined as a PMS \< or = 2 and no individual subscore \> 1. |
| Percentage of Participants Achieving AMS Clinical Response (Period 1) | Week 8 | The adapted mayo score (AMS) is a composite of the following subscores: SFS, RBS and MES. AMS clinical response is defined as decrease in AMS by \> or = 2 points and \> or = 30% from baseline with a decrease in RBS of \> or = 1 or an absolute RBS of 0 or 1. |
| Percentage of Participants Achieving Endoscopic Improvement Among Week 8 Responders per AMS (Period 1) | Week 52 | Endoscopic Improvement is defined as MES of \< or = 1. The AMS is a composite of the following subscores: SFS, RBS and MES. |
| Percentage of Participants Achieving PMS Clinical Remission Among Week 8 Responders per AMS (Period 1) | Week 52 | The Mayo score is a tool designed to measure disease activity for ulcerative colitis. The PMS ranges from 0 to 9 with higher scores representing more severe disease. PMS clinical remission is defined as a PMS \< or = 2 and no individual subscore \> 1. The AMS is a composite of the following subscores: SFS, RBS and MES. |
| Percentage of Participants Achieving AMS Clinical Response Among Week 8 Responders per AMS (Period 1) | Week 52 | The AMS is a composite of the following subscores: SFS, RBS and MES. AMS clinical response is defined as decrease in AMS by \> or = 2 points and \> or = 30% from baseline with a decrease in RBS of \> or = 1 or an absolute RBS of 0 or 1. |
| Percentage of Participants Achieving PMS Clinical Response Among Week 8 Clinical Responders per AMS (Period 1) | Week 52 | The AMS is a composite of the following subscores: SFS, RBS and endoscopy MES. PMS clinical response is defined as decrease in PMS by \> or = 2 points and \> or = 30% from baseline with a decrease in RBS \> or = 1 or an absolute RBS of 0 or 1. |
| Percentage of Participants Achieving Corticosteroid-Free AMS Clinical Remission Among Week 8 Responders per AMS (Period 1) | Week 52 | Corticosteroid-free AMS clinical remission is defined as being in AMS clinical remission and free of corticosteroids for \>= 90 days immediately preceeding the timepoint of endpoint assessment. The AMS is a composite of the following subscores: SFS, RBS and MES. |
| Percentage of Participants Achieving AMS Clinical Remission Among Week 8 Remitters per AMS (Period 1) | Week 52 | The AMS is a composite of the following subscores: SFS, RBS and MES. Adapted Mayo score ranges from 0 to 9 where higher scores represent more severe disease. Clinical remission is defined as an AMS \< or = 2, with SFS \< or = 1 and not higher than baseline, RBS of 0, and MES \< or = 1. |
Countries
Australia, Belgium, Brazil, Bulgaria, Canada, Czechia, France, Germany, Greece, Hungary, Israel, Italy, Japan, Mexico, Netherlands, New Zealand, Poland, Puerto Rico, South Korea, Spain, Taiwan, United Kingdom, United States
Contacts
STUDY_DIRECTORABBVIE INC.
AbbVie
Outcome results
None listed