Pediatric Ulcerative ColitisPhase 1/2NCT04348890
Proof of Concept Trial of Vamorolone in Pediatric Ulcerative Colitis
A Phase 1/2 study testing Vamorolone 4% suspension for oral dosing in people with Pediatric Ulcerative Colitis, run by ReveraGen BioPharma, Inc..
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… Read more about this study
- Who is it for?
- Ages 4 to 17 years
- Conditions the study is for
- Pediatric Ulcerative Colitis
- What is being tested?
- Vamorolone 4% suspension for oral dosing
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 1 Sep 2020to 1 Sep 2022
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
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- 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
About this study
Official title: A Phase I/II Open-Label, Proof-of-Concept Study of Vamorolone in Children and Adolescents With Mild-Moderately Active Ulcerative Colitis
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
- Being tested
- Vamorolone 4% suspension for oral dosing
- Medical subject headings
- Pediatric ulcerative colitis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Vamorolone 4% suspension for oral dosingDrug
vamorolone 6 mg/kg/day orally once daily for 8 weeks.
| Study group | Role | What they receive |
|---|---|---|
| Treatment Arm | Experimental | Not described Receives: Drug: Vamorolone 4% suspension for oral dosing |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 8 weeks
Secondary outcomes (13)
Safety (adverse events)
Measured: From day 1 through 30 days after last dose
Change in osteocalcin, P1NP, CTX
Bone biomarkers
Measured: From baseline to week 8 and week 12
Cushingoid appearance
Physician reported, yes or no
Measured: Week 8 and Week 12
Week 8 response
Decrease in PUCAI score by 20 points or more (lower scores indicate less severe disease activity)
Measured: Baseline to 8 Weeks and 12 Weeks
Change in Mayo Score Stool Frequency subscore
Score ranges from 0-3, with lower scores indicating less severe disease activity
Measured: Baseline to 8 Weeks and 12 Weeks
Change in Mayo rectal bleeding score
Scores range from 0-3, with lower scores indicating less severe disease activity
Measured: Baseline to 8 Weeks and 12 Weeks
Mayo Score Stool Frequency subscore of 0 or 1
Measured: Week 12
Mayo Score Rectal Bleeding Score of 0
Measured: Week 12
Change in fecal calprotectin
Measured: Baseline to Week 8 and Week 12
Change in serum C-reactive protein
Measured: Baseline to Week 8 and Week 12
Change in serum CCL22
Measured: Baseline to Week 8 and Week 12
Change in serum miRNA 146b
Measured: Baseline to Week 8 and Week 12
Change in serum trefoil factor 3
Measured: Baseline to Week 8 and Week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Apr 2020
- Study start1 Sep 2020 (estimated)
- Primary completion1 Sep 2022 (estimated)
- Study completion1 Sep 2022 (estimated)
Why it stopped: Programmatic decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pediatric Ulcerative ColitisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- ReveraGen BioPharma, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Laurie Conklin, MD, Study director, ReveraGen BioPharma
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04348890
- Sponsor's protocol ID
- VBP15 UC-001
- Status last verified
- Sep 2020
- First submitted
- 13 Apr 2020
- Last updated
- 29 Sep 2020
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Sep 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Open-Label, Proof-of-Concept Study of Vamorolone in Children and Adolescents With Mild-Moderately Active Ulcerative Colitis
- ClinicalTrials.gov ID
- NCT04348890
- Acronym / sponsor ID
- VBP15 UC-001
- Conditions
- Pediatric Ulcerative Colitis
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Programmatic decision
- Enrolment
- 0 participants (actual)
- Lead sponsor
- ReveraGen BioPharma, Inc.Industry (pharma, biotech, devices)
- Study start
- 1 Sep 2020 (estimated)
- Primary completion
- 1 Sep 2022 (estimated)
- Study completion
- 1 Sep 2022 (estimated)
- First posted
- 16 Apr 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment Arm | Experimental | Drug: Vamorolone 4% suspension for oral dosing |
Who may qualify
- Ages
- 4 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (8 listed)
- 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.
Key exclusion criteria (13 listed)
- 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:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Vamorolone 4% suspension for oral dosingvamorolone 6 mg/kg/day orally once daily for 8 weeks. | Drug | Treatment Arm |
Primary outcome
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.
13 secondary outcomes
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
Cushingoid appearance
Time frame: Week 8 and Week 12
Week 8 response
Time frame: Baseline to 8 Weeks and 12 Weeks
Change in Mayo Score Stool Frequency subscore
Time frame: Baseline to 8 Weeks and 12 Weeks
Change in Mayo rectal bleeding score
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
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Sep 2020.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Laurie Conklin, MD study director
ReveraGen BioPharma
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Vamorolone 4% suspension for oral dosing in people with Pediatric Ulcerative Colitis, run by ReveraGen BioPharma, Inc..
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 29 Sep 2020. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Clinical Remission (time frame: 8 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Sep 2020.