Programmatic decision

This is a Phase I/II, multi-center, open-label proof-of-concept study of vamorolone. Twenty participants with a flare of mild or moderately active Ulcerative Colitis (defined as a Pediatric Ulcerative Colitis Activity Index [PUCAI] 10-60) will be enrolled and receive vamorolone 6 mg/kg/day orally once daily for 8 weeks.

Inclusion Criteria:

• Subject has provided written informed consent/Health Insurance Portability and Accountability Act (HIPAA) authorization prior to any study-related procedures;
• Subject has current mild to moderately active ulcerative colitis, defined as a PUCAI score of 10-60.
• Subject is ≥ 4 years old and <18 years old at the time of enrollment.
• Subject has had 1) a colonoscopy demonstrating endoscopic and histologic inflammation, and/or; 2) a fecal calprotectin > 250 mcg/g, in the preceding 1 month.
• Subject is willing and able to comply with scheduled visits, study drug administration plan, and study procedures.
• Subject has not started a new immunomodulator or biologic in the preceding 2 months.
• If subject is taking an immunomodulator and/or biologic, the dose has not been changed in the last 2 months
• Subject has a positive varicella IgG titer, or history of at least 2 documented varicella vaccines

Exclusion Criteria:

• Subject is currently being treated or has received previous treatment with oral or rectal glucocorticoids (including budesonide) in the past month

• Subject has an allergy or hypersensitivity to the study medication or to any of its constituents

• Subject has previous or ongoing medical condition, medical history, physical findings or laboratory abnormalities that could affect safety, make it unlikely that treatment and follow-up will be correctly completed or impair the assessment of study results, in the opinion of the Investigator

• Subject is taking any other investigational drug currently or has taken any other investigational drug within 3 months prior to the start of study treatment

• Clinically significant abnormal biochemical and hematological parameters, including:

  • Neutrophil count < 1000 cells/mm3
  • Platelet count ≤ 130 cells/mm3
  • Creatinine ≥ 1.2 x the upper limit of normal
  • Alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) ≥ 2x the upper limit of normal
  • Conjugated bilirubin greater than 1.2. mg/dL

• Has active infection with enteric pathogens (including C. difficile)

• Has a positive PPD, Quantiferon Gold, or Interferon-gamma assay

• Is pregnant or breast-feeding