Clinical trial NCT04348890: Proof of Concept Trial of Vamorolone in Pediatric Ulcerative Colitis
Official title: A Phase I/II Open-Label, Proof-of-Concept Study of Vamorolone in Children and Adolescents With Mild-Moderately Active Ulcerative Colitis
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE1
PHASE2
Conditions: Pediatric Ulcerative Colitis
Lead sponsor: ReveraGen BioPharma, Inc.
Sponsor class: INDUSTRY
First posted: 2020-04-16
Enrollment: 0
Sex: ALL
Minimum age (years): 4
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: 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

Primary outcomes:
measure: Clinical Remission
timeFrame: 8 weeks
Secondary outcomes:
measure: Safety (adverse events)
timeFrame: From day 1 through 30 days after last dose
measure: Change in osteocalcin, P1NP, CTX
timeFrame: From baseline to week 8 and week 12
measure: Cushingoid appearance
timeFrame: Week 8 and Week 12
measure: Week 8 response
timeFrame: Baseline to 8 Weeks and 12 Weeks
measure: Change in Mayo Score Stool Frequency subscore
timeFrame: Baseline to 8 Weeks and 12 Weeks
measure: Change in Mayo rectal bleeding score
timeFrame: Baseline to 8 Weeks and 12 Weeks
measure: Mayo Score Stool Frequency subscore of 0 or 1
timeFrame: Week 12
measure: Mayo Score Rectal Bleeding Score of 0
timeFrame: Week 12
measure: Change in fecal calprotectin
timeFrame: Baseline to Week 8 and Week 12
measure: Change in serum C-reactive protein
timeFrame: Baseline to Week 8 and Week 12
measure: Change in serum CCL22
timeFrame: Baseline to Week 8 and Week 12
measure: Change in serum miRNA 146b
timeFrame: Baseline to Week 8 and Week 12
measure: Change in serum trefoil factor 3
timeFrame: Baseline to Week 8 and Week 12
Other outcomes:
Locations:
Study officials:
affiliation: ReveraGen BioPharma
name: Laurie Conklin, MD
role: STUDY_DIRECTOR
References: