Proof of Concept Trial of Vamorolone in Pediatric Ulcerative Colitis

Pediatric Ulcerative Colitis Clinical Trial

Official title:

A Phase I/II Open-Label, Proof-of-Concept Study of Vamorolone in Children and Adolescents With Mild-Moderately Active Ulcerative Colitis

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT04348890
• Other study ID #: VBP15 UC-001
• Status: Withdrawn
• Phase: Phase 1/Phase 2
• Start date: September 1, 2020
• Est. completion date: September 1, 2022

Study information

• Verified date: September 2020
• Source: ReveraGen BioPharma, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: September 1, 2022
• Est. primary completion date: September 1, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 4 Years to 17 Years

Eligibility

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

Related Conditions & MeSH terms

• Colitis
• Colitis, Ulcerative
• Pediatric Ulcerative Colitis
• Ulcer

Intervention

Drug:
• Vamorolone 4% suspension for oral dosing
vamorolone 6 mg/kg/day orally once daily for 8 weeks.

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
ReveraGen BioPharma, Inc.

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Clinical Remission	Pediatric Ulcerative Colitis Activity Index score <10 and no additional therapy or colectomy). The PUCAI is scored from 0-85, lower numbers indicate less severe disease activity.	8 weeks
Secondary	Safety (adverse events)		From day 1 through 30 days after last dose
Secondary	Change in osteocalcin, P1NP, CTX	Bone biomarkers	From baseline to week 8 and week 12
Secondary	Cushingoid appearance	Physician reported, yes or no	Week 8 and Week 12
Secondary	Week 8 response	Decrease in PUCAI score by 20 points or more (lower scores indicate less severe disease activity)	Baseline to 8 Weeks and 12 Weeks
Secondary	Change in Mayo Score Stool Frequency subscore	Score ranges from 0-3, with lower scores indicating less severe disease activity	Baseline to 8 Weeks and 12 Weeks
Secondary	Change in Mayo rectal bleeding score	Scores range from 0-3, with lower scores indicating less severe disease activity	Baseline to 8 Weeks and 12 Weeks
Secondary	Mayo Score Stool Frequency subscore of 0 or 1		Week 12
Secondary	Mayo Score Rectal Bleeding Score of 0		Week 12
Secondary	Change in fecal calprotectin		Baseline to Week 8 and Week 12
Secondary	Change in serum C-reactive protein		Baseline to Week 8 and Week 12
Secondary	Change in serum CCL22		Baseline to Week 8 and Week 12
Secondary	Change in serum miRNA 146b		Baseline to Week 8 and Week 12
Secondary	Change in serum trefoil factor 3		Baseline to Week 8 and Week 12