Safety and Efficacy of Efavaleukin Alfa in Participants With Moderately to Severely Active Ulcerative Colitis

  • End date
    Apr 25, 2025
  • participants needed
  • sponsor
Updated on 21 September 2023
tumor necrosis factor
tumour necrosis
janus kinase inhibitor


The main purpose of this study is to evaluate the effect of efavaleukin alfa on induction of clinical remission in participants with moderately to severely active ulcerative colitis (UC).

Participants will be randomized to receive 1 of 3 efavaleukin alfa doses or placebo during a 12-week induction period. Participants who complete the 12-week induction period will have the option to enter an exploratory long-term treatment period for up to 40 weeks (total of up to 52 weeks of treatment) if, in the opinion of the investigator, they may benefit from continued treatment. During the long-term period, participants randomized to efavaleukin alfa will remain on the same efavaleukin alfa blinded dose; participants randomized to placebo who achieved clinical response at week 12 will remain on placebo; and placebo non-responders (ie, participants randomized to placebo who did not achieve clinical response at week 12) will receive efavaleukin alfa in a blinded manner during continued treatment. All participants will complete a safety follow-up visit 6 weeks after their last dose of investigational product.

Condition Ulcerative Colitis
Treatment Placebo, Efavaleukin Alfa
Clinical Study IdentifierNCT04987307
Last Modified on21 September 2023


Yes No Not Sure

Inclusion Criteria

Participant has provided informed consent prior to initiation of any study specific activities or procedures
Men and women aged ≥ 18 to < 80 years at screening visit (≥ 19 to < 80 in South Korea)
Diagnosis of UC established ≥ 3 months prior to enrollment by clinical and endoscopic evidence and corroborated by a histopathology report. If a histopathology report is not available at screening, then additional biopsies may be taken during the screening period for local histopathology analysis to corroborate
Moderately to severely active UC as defined by a modified Mayo score of 5 to 9, with a centrally read endoscopy subscore ≥ 2
Has documentation of
A surveillance colonoscopy (performed according to local standard) within 12 months of day 1 visit for participants with pancolitis of > 8 years duration, or participants with left-sided colitis of > 12 years duration, or participants with primary sclerosing cholangitis
Participants must have demonstrated inadequate response, loss of response, or
At the discretion of the investigator, a colonoscopy (instead of a rectosigmoidoscopy) may be performed as the screening endoscopy for this study
intolerance to at least 1 conventional therapy, biologic therapy, or targeted
For all other participants, up-to-date colorectal cancer surveillance (performed according to local standard). Participants who do not have a colonoscopy report available in source documentation will have a colonoscopy instead of rectosigmoidoscopy performed as the screening endoscopy for the study
Conventional therapy failed participants
small molecule therapy (ie, Janus kinase [JAK]-inhibitor or or S1P
Corticosteroids (corticosteroid-refractory colitis, defined as signs and/or symptoms of active UC despite oral prednisone [or equivalent] at doses of at least 30 mg/day for a minimum of 2 weeks; or corticosteroid-dependent colitis, defined as: an inability to reduce corticosteroids below the equivalent of prednisone 10 mg/day within 3 months of starting corticosteroids without a return of signs and/or symptoms of active UC; or a relapse within 3 months of completing a course of corticosteroids)
modulators), as follows
History of intolerance of corticosteroids (including, but not limited to, Cushing's syndrome, osteopenia/ osteoporosis, hyperglycemia, or neuropsychiatric side-effects, including insomnia, associated with corticosteroid treatment)
Immunomodulators: signs and/or symptoms of persistently active disease despite at least 3 months treatment with one of the following at locally approved doses: oral azathioprine (eg, ≥ 1.5 mg/kg/day) or 6-mercaptopurine (eg, ≥ 0.75 mg/kg/day), or oral azathioprine or 6-mercatopurine within a therapeutic range as judged by thioguanine metabolite testing, or a combination of a thiopurine and allopurinol within a therapeutic range as judged by thioguanine metabolite testing
History of intolerance to at least 1 immunomodulator (including but not limited to nausea/vomiting, abdominal pain, pancreatitis, liver function test abnormalities, and lymphopenia) and have neither failed nor demonstrated an intolerance to a biological medication (anti-tumor necrosis factor [TNF] antibody, anti-integrin antibody, or interleukin [IL]-12/23 antagonists) that is indicated for the treatment of UC
Biologic or targeted small molecule therapy failed participants: those who
demonstrated inadequate response or loss of response or intolerance to
biologic therapy for UC (eg, anti-TNF antibodies or IL-12/23 antagonists
anti-integrin antibodies) or targeted small molecules (eg, JAK inhibitors or
S1P modulators). The therapy used to qualify the participant for entry into
Inadequate response: signs and symptoms of persistently active disease despite induction treatment at the approved induction dosing that was indicated in the product label at the time of use
this category must be approved for the treatment of UC in the country of use
at the time of use. Participants must fulfil one of the following criteria
If receiving any of the following therapies, participants must have stable dosage for
Loss of response: recurrence of signs and symptoms of active disease during approved maintenance dosing following prior clinical benefit (discontinuation despite clinical benefit does not quality as having failed or being intolerant to UC biological therapy, JAK inhibitor, or S1P modulators)
the specified duration
Intolerance: history of intolerance to infliximab, adalimumab, golimumab, vedolizumab, ustekinumab, tofacitinib or other approved biologicals, JAK inhibitors or S1P modulators (including but not limited to infusion-related event, demyelination, congestive heart failure, or any other drug-related adverse event that led to a reduction in dose or discontinuation of the medication)
-aminosalicylates (ASAs), stable dosage for ≥ 2 weeks prior to screening endoscopy
Oral corticosteroids: prednisone ≤ 20 mg/day or its equivalent, stable dose for ≥ 2 weeks prior to screening endoscopy
Budesonide: extended release tablets 9 mg/day [budensonide MMX], stable dose for ≥ 2 weeks prior to screening endoscopy
Conventional immunomodulators: azathioprine, 6-mercaptopurine, methotrexate, stable dosage for ≥ 12 weeks prior to screening endoscopy
Beclomethasone dipropionate: gastro-resistant prolonged-release tablet 5 mg/day, stable dose for >= 2 weeks prior to screening endoscopy

Exclusion Criteria

Diagnosis of Crohn's disease, inflammatory bowel disease unclassified (indeterminate colitis), microscopic colitis, ischemic colitis, or clinical findings suggestive of Crohn's disease
Evidence of toxic megacolon, fulminant colitis, intra-abdominal abscess, or stricture/stenosis within the small bowel or colon
Participant has had extensive surgery for UC (for example, subtotal colectomy), or is likely to require surgery for the treatment of UC during the study
Currently receiving or had treatment within 12 months prior to screening with T cell depleting agents (eg, antithymocyte globulin, Campath)
Participant has received any of the following prescribed medication or therapy within the specified time period
Anti TNF antibodies (eg, infliximab, adalimumab, golimumab) < 8 weeks prior to screening rectosigmoidoscopy
Anti integrin antibodies (eg, vedolizumab) < 8 weeks prior to screening rectosigmoidoscopy
IL 12/23 antagonist (eg, ustekinumab) < 8 weeks prior to screening rectosigmoidoscopy
JAK inhibitors (eg, tofacitinib) < 4 weeks prior to screening rectosigmoidoscopy
Any other commercially approved biologic agent or targeted small molecule < 8 weeks prior to screening rectosigmoidoscopy or < 5 half lives prior to screening rectosigmoidoscopy, whichever is longer
Immunomodulatory medications, including oral cyclosporine, intravenous cyclosporine, tacrolimus, sirolimus, mycophenolate mofetil, thalidomide < 4 weeks prior to screening rectosigmoidoscopy
Any investigational biologic therapy within 8 weeks prior to screening rectosigmoidoscopy or < 5 half-lives prior to screening rectosigmoidoscopy, whichever is longer
Has used apheresis (eg, Adacolumnâ apheresis) < 2 weeks prior to screening rectosigmoidoscopy
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