Study to evaluate the efficacy and safety of oral PF-06651600 and PF-06700841 in subjects with moderate to severe CD

 

  • Title: Study to evaluate the efficacy and safety of oral PF-06651600 and PF-06700841 in subjects with moderate to severe CD
  • Study drug: PF-06651600, PF-06700841
  • Sponsor: Pfizer
  • Participating centers and contact
    • UniversitätsSpital Zürich
    • Karolina Loch (Nursing Specialist, Study Coordinator Gastroenterology IBD)
    • karolina.loch@usz.ch
  • Target population: Moderate to severe CD
  • Primary outcome:
    • 1. Proportion of subjects achieving clinically meaningful endoscopic improvement (reduction of more than or equal to 3 points from baseline in Simplified Endoscopic Activity Score for Crohn's Disease (SES-CD) at Week 12 [ Time Frame: 12 weeks ]
    • 2. Number of subjects with adverse events [ Time Frame: Baseline of extension period (Week 12) to week 68 ]
    • 3. Number of subjects with serious adverse events [ Time Frame: Baseline of extension period (Week 12) to week 68 ]
    • 4. Number of subjects with electrocardiogram findings of potential clinical importance [ Time Frame: Baseline of extension period (Week 12) to week 68 ]
    • 5. Number of subjects with laboratory tests findings of potential clinical importance [ Time Frame: Baseline of extension period (Week 12) to week 68 ]
    • 6. Number of subjects withdrawal due to adverse events [ Time Frame: Baseline of extension period (Week 12) to week 68 ]
    • 7. Number of subjects with vital signs findings of potential clinical importance [ Time Frame: Baseline of extension period (Week 12) to week 68 ]
  • Inclusion criteria:
    • 1. Male and/or female subjects 18 years to 75 years of age
    • 2. Documented diagnosis of ileal, ileocolonic, or colonic CD with a minimum disease duration of 3 months, as determined by endoscopic and histopathology assessment.
    • 3. Endoscopic confirmation of active disease with total SES CD total score of at least 7. For isolated ileal disease, SES CD total score should be at least 4.
    • 4. An average daily liquid/soft stool frequency (SF) greater than or equal to 2.5 or daily abdominal pain (AP) greater than or equal to 2.0.
    • 5. Must have inadequate response to, loss of response to, or intolerance to at least one conventional therapy for CD:
    • Steroids; Immunosuppressants (azathioprine [AZA], 6 MP, or methotrexate [MTX]); Anti TNF inhibitors (infliximab, adalimumab,certolizumab); Anti integrin inhibitors (eg, vedolizumab); Anti IL 12/23 inhibitor (ustekinumab).
    • 6. Subjects currently receiving the following treatment for CD are eligible providing they have been on stable doses as described below:
      • Oral corticosteroids (prednisone or equivalent up to 25 mg/day; budesonide up to 9 mg/day). Stable dose for at least 2 weeks prior to baseline. If oral corticosteroids have been recently discontinued, they must have been stopped at least 2 weeks prior to baseline. Decreases in steroid use due to AEs are allowed.
      • Oral 5 ASA or sulfasalazine are allowed providing that the dose is stable for at least 4 weeks prior to baseline.
      • Crohn's disease related antibiotics are allowed providing that the dose is stable for at least 4 weeks prior to baseline. If antibiotics are stopped prior to baseline, they must be discontinued at least 4 days prior to baseline.
  • Exclusion criteria:
    • 1. Diagnosis of indeterminate colitis, microscopic colitis, ischemic colitis, infectious colitis, radiation colitis, diverticular disease, ulcerative colitis (UC), or clinical findings suggestive of UC.
    • 2. Presence of active (draining) fistulae or intra abdominal or perineal abscesses.
    • 3. Strictures with obstructive symptoms.
    • 4. Short bowel syndrome.
    • 5. History of bowel perforation requiring surgical intervention within the past 12 months.
    • 6. Previous bowel surgery resulting in an existing stoma. Subjects who have a j pouch are excluded, as a j pouch can result in a stoma.
    • 7. History of bowel surgery within 6 months prior to baseline.
    • 8. Subjects displaying clinical signs of fulminant colitis or toxic megacolon.
    • 9. Subjects with primary sclerosing cholangitis.
    • 10. Subjects with evidence of colonic adenomas, dysplasia or neoplasia.
    • 11. Abnormal findings on the chest x ray film such as presence of tuberculosis (TB), general infections, heart failure, or malignancy.
    • 12. Any history of either untreated or inadequately treated latent or active TB infection, current treatment for active or latent TB infection or evidence of currently active TB by chest x ray, residing with or frequent close contact with individual(s) with active TB.
    • 13. Subjects receiving the following therapies within the time period described below or expected to receive any of these therapies during the study period:
      • a. >9 mg/day of oral budesonide or >25 mg/day of prednisone or equivalent oral systemic corticosteroid dose within 2 weeks prior to baseline.
      • b. IV, IM (parenteral), or topical (rectal) treatment of 5 ASA or corticosteroid enemas/suppositories within 2 weeks prior to baseline.
      • c. Azathioprine, 6 mercaptopurine, or methotrexate within 2 weeks prior to baseline.
      • d. Anti TNF inhibitors (or biosimilars thereof) as described below:
        • Infliximab within 8 weeks prior to baseline;
        • Adalimumab within 8 weeks prior to baseline;
        • Certolizumab within 8 weeks prior to baseline;
      • e. Anti integrin inhibitors (eg, vedolizumab) within 8 weeks prior to baseline.
      • f. Ustekinumab within 8 weeks prior to baseline.
      • g. Interferon therapy within 8 weeks prior to baseline.
      • h. Subjects with prior treatment with lymphocyte depleting agents/therapies within 1 year prior to baseline (eg, CamPath[alemtuzumab], alkylating agents [eg, cyclophosphamide or chlorambucil], total lymphoid irradiation, etc).
      • i. Subjects who have received rituximab or other selective B lymphocyte depleting agents within 1 year prior to baseline.
      • j.Subjects previously receiving leukocyte apheresis, including selective lymphocyte, monocyte, or granulocyte apheresis, or plasma exchange within 6 months prior to baseline.
      • k. Other marketed immunosuppressants or biologics with immunomodulatory properties within 3 months prior to baseline.
      • l. Subjects who have received other JAK inhibitors within 3 months prior to baseline.
      • m. Subjects who have not responded to or have been intolerant of other JAK inhibitors.
      • n. Other investigational procedures(s) or product(s), such as immunosuppressants used in transplantation (eg, mycophenolate mofetil, cyclosporine, rapamycin, or tacrolimus) or live (attenuated) vaccine within 30 days prior to baseline.
    • 14. Subjects with history of thrombotic event(s), including deep venous thrombosis (DVT), and known inherited conditions that predispose to hypercoagulability.
  • More information: https://www.clinicaltrials.gov/ct2/show/NCT03395184?term=Pfizer&cond=PF-06651600&cntry=CH&draw=2&rank=1
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