Proof of Concept Trial of Vamorolone in Pediatric Ulcerative Colitis

Reveragen BioPharma

Status and phase

Withdrawn

Phase 2

Phase 1

Conditions

Pediatric Ulcerative Colitis

Treatments

Drug: Vamorolone 4% suspension for oral dosing

Study type

Interventional

Funder types

Industry

Identifiers

NCT04348890

VBP15 UC-001

Details and patient eligibility

About

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.

Sex

All

Ages

4 to 17 years old

Volunteers

No Healthy Volunteers

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