Back to resultsProof of Concept Trial of Vamorolone in Pediatric Ulcerative Colitis
Primary Purpose
Pediatric Ulcerative Colitis
Status
Withdrawn
Phase
Phase 1
Locations
Study Type
Interventional
Intervention
Vamorolone 4% suspension for oral dosing
Sponsored by
About this trial
This is an interventional treatment trial for Pediatric Ulcerative Colitis
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
Sites / Locations
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Treatment Arm
Arm Description
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
Safety (adverse events)
Change in osteocalcin, P1NP, CTX
Bone biomarkers
Cushingoid appearance
Physician reported, yes or no
Week 8 response
Decrease in PUCAI score by 20 points or more (lower scores indicate less severe disease activity)
Change in Mayo Score Stool Frequency subscore
Score ranges from 0-3, with lower scores indicating less severe disease activity
Change in Mayo rectal bleeding score
Scores range from 0-3, with lower scores indicating less severe disease activity
Mayo Score Stool Frequency subscore of 0 or 1
Mayo Score Rectal Bleeding Score of 0
Change in fecal calprotectin
Change in serum C-reactive protein
Change in serum CCL22
Change in serum miRNA 146b
Change in serum trefoil factor 3
Full Information
NCT ID
NCT04348890
First Posted
April 13, 2020
Last Updated
September 25, 2020
Sponsor
ReveraGen BioPharma, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT04348890
Brief Title
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
2. Study Status
Record Verification Date
September 2020
Overall Recruitment Status
Withdrawn
Why Stopped
Programmatic decision
Study Start Date
September 1, 2020 (Anticipated)
Primary Completion Date
September 1, 2022 (Anticipated)
Study Completion Date
September 1, 2022 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
ReveraGen BioPharma, Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pediatric Ulcerative Colitis
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
0 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Treatment Arm
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Vamorolone 4% suspension for oral dosing
Intervention Description
vamorolone 6 mg/kg/day orally once daily for 8 weeks.
Primary Outcome Measure Information:
Title
Clinical Remission
Description
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.
Time Frame
8 weeks
Secondary Outcome Measure Information:
Title
Safety (adverse events)
Time Frame
From day 1 through 30 days after last dose
Title
Change in osteocalcin, P1NP, CTX
Description
Bone biomarkers
Time Frame
From baseline to week 8 and week 12
Title
Cushingoid appearance
Description
Physician reported, yes or no
Time Frame
Week 8 and Week 12
Title
Week 8 response
Description
Decrease in PUCAI score by 20 points or more (lower scores indicate less severe disease activity)
Time Frame
Baseline to 8 Weeks and 12 Weeks
Title
Change in Mayo Score Stool Frequency subscore
Description
Score ranges from 0-3, with lower scores indicating less severe disease activity
Time Frame
Baseline to 8 Weeks and 12 Weeks
Title
Change in Mayo rectal bleeding score
Description
Scores range from 0-3, with lower scores indicating less severe disease activity
Time Frame
Baseline to 8 Weeks and 12 Weeks
Title
Mayo Score Stool Frequency subscore of 0 or 1
Time Frame
Week 12
Title
Mayo Score Rectal Bleeding Score of 0
Time Frame
Week 12
Title
Change in fecal calprotectin
Time Frame
Baseline to Week 8 and Week 12
Title
Change in serum C-reactive protein
Time Frame
Baseline to Week 8 and Week 12
Title
Change in serum CCL22
Time Frame
Baseline to Week 8 and Week 12
Title
Change in serum miRNA 146b
Time Frame
Baseline to Week 8 and Week 12
Title
Change in serum trefoil factor 3
Time Frame
Baseline to Week 8 and Week 12
10. Eligibility
Sex
All
Minimum Age & Unit of Time
4 Years
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Laurie Conklin, MD
Organizational Affiliation
ReveraGen BioPharma
Official's Role
Study Director
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Proof of Concept Trial of Vamorolone in Pediatric Ulcerative Colitis
We'll reach out to this number within 24 hrs