Proof of Concept Trial of Vamorolone in Pediatric Ulcerative Colitis

NCT04348890 | Withdrawn Phase 1 DMC FDA Drug

• 1 other identifier: VBP15 UC-001
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Pediatric Ulcerative Colitis

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (13)

• Safety (adverse events)

  From day 1 through 30 days after last dose

• Change in osteocalcin, P1NP, CTX

  From baseline to week 8 and week 12

• Cushingoid appearance

  Week 8 and Week 12

• Week 8 response

  Baseline to 8 Weeks and 12 Weeks

• Change in Mayo Score Stool Frequency subscore

  Baseline to 8 Weeks and 12 Weeks

Study Arms (1)

Treatment Arm

EXPERIMENTAL

Drug: Vamorolone 4% suspension for oral dosing

Interventions

Vamorolone 4% suspension for oral dosing DRUG

vamorolone 6 mg/kg/day orally once daily for 8 weeks.

Treatment Arm

Eligibility Criteria

• Age: 4 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• ReveraGen BioPharma, Inc.: lead

MeSH Terms

Conditions

Pediatric ulcerative colitis

Interventions

VBP15 compound; Suspensions

Intervention Hierarchy (Ancestors)

Colloids; Complex Mixtures; Dosage Forms; Pharmaceutical Preparations

Study Officials

• Laurie Conklin, MD

  ReveraGen BioPharma

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 13, 2020
• First Posted: April 16, 2020
• Study Start: September 1, 2020
• Primary Completion: September 1, 2022
• Study Completion: September 1, 2022
• Last Updated: September 29, 2020

Record last verified: 2020-09

Data Sharing

• IPD Sharing: Will not share