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Trial record 1 of 6 for:    vamorolone
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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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04348890
Recruitment Status : Not yet recruiting
First Posted : April 16, 2020
Last Update Posted : April 17, 2020
Sponsor:
Information provided by (Responsible Party):
ReveraGen BioPharma, Inc.

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.

Condition or disease Intervention/treatment Phase
Pediatric Ulcerative Colitis Drug: Vamorolone 4% suspension for oral dosing Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase I/II Open-Label, Proof-of-Concept Study of Vamorolone in Children and Adolescents With Mild-Moderately Active Ulcerative Colitis
Estimated Study Start Date : September 1, 2020
Estimated Primary Completion Date : September 1, 2022
Estimated Study Completion Date : September 1, 2022

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Treatment Arm Drug: Vamorolone 4% suspension for oral dosing
vamorolone 6 mg/kg/day orally once daily for 8 weeks.




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. Safety (adverse events) [ Time Frame: From day 1 through 30 days after last dose ]
  2. Change in osteocalcin, P1NP, CTX [ Time Frame: From baseline to week 8 and week 12 ]
    Bone biomarkers

  3. Cushingoid appearance [ Time Frame: Week 8 and Week 12 ]
    Physician reported, yes or no

  4. 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)

  5. 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

  6. 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

  7. Mayo Score Stool Frequency subscore of 0 or 1 [ Time Frame: Week 12 ]
  8. Mayo Score Rectal Bleeding Score of 0 [ Time Frame: Week 12 ]
  9. Change in fecal calprotectin [ Time Frame: Baseline to Week 8 and Week 12 ]
  10. Change in serum C-reactive protein [ Time Frame: Baseline to Week 8 and Week 12 ]
  11. Change in serum CCL22 [ Time Frame: Baseline to Week 8 and Week 12 ]
  12. Change in serum miRNA 146b [ Time Frame: Baseline to Week 8 and Week 12 ]
  13. Change in serum trefoil factor 3 [ Time Frame: Baseline to Week 8 and Week 12 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   4 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04348890


Contacts
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Contact: Laurie Conklin, MD 240-672-0295 laurie.conklin@reveragen.com

Sponsors and Collaborators
ReveraGen BioPharma, Inc.
Investigators
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Study Director: Laurie Conklin, MD ReveraGen BioPharma
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Responsible Party: ReveraGen BioPharma, Inc.
ClinicalTrials.gov Identifier: NCT04348890    
Other Study ID Numbers: VBP15 UC-001
First Posted: April 16, 2020    Key Record Dates
Last Update Posted: April 17, 2020
Last Verified: April 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Colitis
Colitis, Ulcerative
Ulcer
Gastroenteritis
Gastrointestinal Diseases
Digestive System Diseases
Colonic Diseases
Intestinal Diseases
Pathologic Processes
Inflammatory Bowel Diseases