Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 1 of 6 for:    vamorolone
Previous Study | Return to List | Next Study

Proof of Concept Trial of Vamorolone in Pediatric Ulcerative Colitis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04348890
Recruitment Status : Withdrawn (Programmatic decision)
First Posted : April 16, 2020
Last Update Posted : September 29, 2020
Sponsor:
Information provided by (Responsible Party):
ReveraGen BioPharma, Inc.

Tracking Information
First Submitted Date  ICMJE April 13, 2020
First Posted Date  ICMJE April 16, 2020
Last Update Posted Date September 29, 2020
Estimated Study Start Date  ICMJE September 1, 2020
Estimated Primary Completion Date September 1, 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 15, 2020)
Clinical Remission [ Time Frame: 8 weeks ]
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.
Original Primary Outcome Measures  ICMJE
 (submitted: April 13, 2020)
Clinical Remission [ Time Frame: 8 weeks ]
Pediatric Ulcerative Colitis Activity Index score <10 and no additional therapy or colectomy)
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: April 15, 2020)
  • Safety (adverse events) [ Time Frame: From day 1 through 30 days after last dose ]
  • Change in osteocalcin, P1NP, CTX [ Time Frame: From baseline to week 8 and week 12 ]
    Bone biomarkers
  • Cushingoid appearance [ Time Frame: Week 8 and Week 12 ]
    Physician reported, yes or no
  • Week 8 response [ Time Frame: Baseline to 8 Weeks and 12 Weeks ]
    Decrease in PUCAI score by 20 points or more (lower scores indicate less severe disease activity)
  • Change in Mayo Score Stool Frequency subscore [ Time Frame: Baseline to 8 Weeks and 12 Weeks ]
    Score ranges from 0-3, with lower scores indicating less severe disease activity
  • Change in Mayo rectal bleeding score [ Time Frame: Baseline to 8 Weeks and 12 Weeks ]
    Scores range from 0-3, with lower scores indicating less severe disease activity
  • Mayo Score Stool Frequency subscore of 0 or 1 [ Time Frame: Week 12 ]
  • Mayo Score Rectal Bleeding Score of 0 [ Time Frame: Week 12 ]
  • Change in fecal calprotectin [ Time Frame: Baseline to Week 8 and Week 12 ]
  • Change in serum C-reactive protein [ Time Frame: Baseline to Week 8 and Week 12 ]
  • Change in serum CCL22 [ Time Frame: Baseline to Week 8 and Week 12 ]
  • Change in serum miRNA 146b [ Time Frame: Baseline to Week 8 and Week 12 ]
  • Change in serum trefoil factor 3 [ Time Frame: Baseline to Week 8 and Week 12 ]
Original Secondary Outcome Measures  ICMJE
 (submitted: April 13, 2020)
  • Safety (adverse events) [ Time Frame: From day 1 through 30 days after last dose ]
  • Change in osteocalcin, P1NP, CTX [ Time Frame: From baseline to week 8 and week 12 ]
    Bone biomarkers
  • Cushingoid appearance [ Time Frame: Week 8 and Week 12 ]
    Physician reported, yes or no
  • Week 8 response [ Time Frame: Baseline to 8 Weeks and 12 Weeks ]
    Decrease in PUCAI score by 20 points or more
  • Mayo Score Stool Frequency subscore [ Time Frame: Baseline to 8 Weeks and 12 Weeks ]
  • Mayo rectal bleeding score change [ Time Frame: Baseline to 8 Weeks and 12 Weeks ]
  • Mayo Score Stool Frequency subscore of 0 or 1 [ Time Frame: Week 12 ]
  • Mayo Score Rectal Bleeding Score of 0 [ Time Frame: Week 12 ]
  • Change in fecal calprotectin [ Time Frame: Baseline to Week 8 and Week 12 ]
  • Change in serum C-reactive protein [ Time Frame: Baseline to Week 8 and Week 12 ]
  • Change in serum CCL22 [ Time Frame: Baseline to Week 8 and Week 12 ]
  • Change in serum miRNA 146b [ Time Frame: Baseline to Week 8 and Week 12 ]
  • Change in serum trefoil factor 3 [ Time Frame: Baseline to Week 8 and Week 12 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Proof of Concept Trial of Vamorolone in Pediatric Ulcerative Colitis
Official Title  ICMJE A Phase I/II Open-Label, Proof-of-Concept Study of Vamorolone in Children and Adolescents With Mild-Moderately Active Ulcerative Colitis
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Pediatric Ulcerative Colitis
Intervention  ICMJE Drug: Vamorolone 4% suspension for oral dosing
vamorolone 6 mg/kg/day orally once daily for 8 weeks.
Study Arms  ICMJE Experimental: Treatment Arm
Intervention: Drug: Vamorolone 4% suspension for oral dosing
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Withdrawn
Actual Enrollment  ICMJE
 (submitted: September 25, 2020)
0
Original Estimated Enrollment  ICMJE
 (submitted: April 13, 2020)
20
Estimated Study Completion Date  ICMJE September 1, 2022
Estimated Primary Completion Date September 1, 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 4 Years to 17 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Not Provided
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04348890
Other Study ID Numbers  ICMJE VBP15 UC-001
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party ReveraGen BioPharma, Inc.
Study Sponsor  ICMJE ReveraGen BioPharma, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Laurie Conklin, MD ReveraGen BioPharma
PRS Account ReveraGen BioPharma, Inc.
Verification Date September 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP