Recurrent Aphthous StomatitisPhase 4NCT03690544
Apremilast for RAS
A Phase 4 study testing Apremilast 30mg in people with Recurrent Aphthous Stomatitis, run by Mayo Clinic at 1 site in United States.
Trial summary
Determination of treatment efficacy and safety of Apremilast in patients with RAS Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Recurrent Aphthous Stomatitis
- What is being tested?
- Apremilast 30mg
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- 12 Oct 2018to 14 Jul 2021
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male and female subjects between 18 and 70 years of age
- Oral ulcers that occurred at least monthly in the 6 month period prior to enrollment
- Had at least 2 oral ulcers in the 4 weeks prior to enrollment at baseline
- At least 3 oral ulcers during an ulcer flare
- Patients must be candidates for systemic therapy for the treatment of oral ulcers, those that are considered unsuitable for topical therapy alone based on severity of disease, or whose oral ulcers cannot be adequately controlled with topical therapy.
- Female premenopausal subjects must use one of the approved contraceptive options while taking apremilast and for at least 28 days after administration of the last dose of apremilast
- Patients are able and willing to provide written informed consent after the nature of the study is fully explained.
- No evidence of systemic disease
Who cannot join
- Prior use of apremilast.
- Use of any investigational drug within 4 weeks prior to randomization, or 5 pharmacokinetic/pharmacodynamic half-lives, if known (whichever is longer).
- Having received concomitant immune modulating therapy 12 weeks prior to enrollment, systemic steroids 6 weeks prior to enrollment or topical steroids within 4 weeks prior to enrollment.
- Pregnant women or breast-feeding mothers.
- Systemic or opportunistic fungal infection.
- Known active current or history of recurrent bacterial, viral, fungal, mycobacterial or other infections (tuberculosis and atypical mycobacterial disease, hepatitis B and C and herpes zoster, histoplasmosis, coccidiomycosis) or any major episode of infection requiring hospitalization or treatment with IV or oral antibiotics within 4 weeks of the screening phase.
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- History of positive test for, or any clinical suspicion of, human immunodeficiency virus (HIV), or congenital or acquired immunodeficiency.
- History of depression.
- Malignancy or history of malignancy, except for:
- a - treated (ie, cured) basal cell or squamous cell in situ skin carcinomas; b - treated (ie, cured) cervical intraepithelial neoplasia (CIN) or carcinoma in situ of cervix with no evidence of recurrence within the previous 5 years.
- Other than disease under study, any clinically significant (as determined by the Investigator) cardiac, endocrinologic, pulmonary, neurologic, psychiatric, hepatic, renal, hematologic, immunologic disease, or other major disease that is currently uncontrolled.
- Any condition, including the presence of laboratory abnormalities, which would place the subject at unacceptable risk if he/she were to participate in the study.
- Prior history of suicide attempt at any time in the subject's life time prior to screening or randomization, or major psychiatric illness requiring hospitalization within the last 3 years.
- Active substance abuse or a history of substance abuse within 6 months prior to screening.
- Presence of any of the following vitamin deficiencies - B1, B2, B6, B12, vitamin C, zinc, folate, iron.
- Celiac disease.
- Inflammatory Bowel Disease.
- Genital aphthous ulcers.
- Behçet's disease.
- History of positive patch test for allergic contact stomatitis.
- Positive anti-endomysial or anti-gliadin antibodies.
- A diagnosis of uveitis (current or previous).
- Erythema nodosum-like lesions (current or previous).
- An established diagnosis of a systemic disease (SLE, Reiter's, Sweet's and MAGIC syndrome).
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Pilot Study Evaluating the Efficacy of Apremilast in the Treatment of Subjects With Severe Recurrent Aphthous Stomatitis (RAS)
Determination of treatment efficacy and safety of Apremilast in patients with RAS
Read the detailed description
The study will be a pilot study using apremilast 30mg orally twice daily, for treatment of RAS in males and females between 18 and 70 years old.
Subjects will be recruited from the clinical practice of the Department of Dermatology or Division of Rheumatology at Mayo Clinic Florida. Fifteen males and females with RAS will be enrolled.
The study will consist of 3 phases: a screening phase, a 16 week treatment phase and an 8 week posttreatment observational follow-up phase.
The screening phase will consist of: obtaining informed consent, demographic information, medical history, inclusion and exclusion criteria, prior and concomitant medication use, adverse events; collecting vital signs and weight; performing complete physical examination and limited physical examination; obtaining hematology, serum chemistry, urinalysis, pregnancy test and providing contraception education.
During the 16-week treatment phase, all subjects receive apremilast.
All subjects who complete the active treatment phase are to enter the 8-week posttreatment observational follow-up phase.
- Conditions
- Recurrent Aphthous Stomatitis
- Being tested
- Apremilast 30mg
- Medical subject headings
- Stomatitis, Aphthous
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Apremilast 30mgDrug
Apremilast is an oral small-molecule inhibitor of phosphodiesterase (PDE) 4 that works intracellularly to modulate a network of pro-inflammatory and anti-inflammatory mediators. PDE 4 is a cyclic adenosine monophosphate (cAMP)-specific PDE and the dominant PDE in inflammatory cells. PDE4 inhibition elevates intracellular cAMP levels, which in turn down-regulates the inflammatory response by modulating the expression of TNF-alfa, IL-23, IL-17 and other inflammatory cytokines. Cyclic AMP also modulates levels of anti-inflammatory cytokines such as IL-10. Apremilast has immunomodulatory activity and, therefore, has the potential to be effective in the treatment of RAS.
| Study group | Role | What they receive |
|---|---|---|
| Single Arm | Experimental | Apremilast 30mg orally twice daily for 16 weeks, sixteen weeks on active study. Post treatment follow-up period of 8 weeks, in the Treatment of Subjects with Severe Recurrent Aphthous Stomatitis (RAS) Receives: Drug: Apremilast 30mg |
What is being measured
The primary outcome is the main question the study is designed to answer.
Duration of RAS Lesions
The total length of time (duration) subjects experienced RAS lesions. Measured in weeks
Measured: 24 weeks
Change in Number of RAS Lesions
Number of participants with fewer oral ulcers at Week 24 compared to Baseline
Measured: baseline, 24 weeks
Duration of the Remission Period Between Ulcer Episodes
The length of time of remission of RAS lesions experienced by the subjects. As measured in months.
Measured: 24 weeks
Secondary outcomes (3)
Adverse Events
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Measured: 24 weeks
Discontinuation of Study Participants
Number of subjects who prematurely discontinue treatment with apremilast due to any adverse event.
Measured: 24 weeks
Change in Visual Analog Scale Pain Score (VAS) From Baseline to 16 Weeks and Baseline to 24 Weeks.
The Visual Analog Scale (VAS) for Pain is a validated tool used to measure pain. A 100mm horizontal line anchored by "no pain" (score of 0) and "pain as bad as it could be" (score of 100).
Measured: baseline, 16 weeks, 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Oct 2018
- Study start12 Oct 2018
- Primary completion14 Jul 2021
- Study completion14 Jul 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Recurrent Aphthous StomatitisStudies of the same condition, open and under way.
- More trials run by Mayo ClinicThe sponsor's other registered studies.
- 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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Apremilast 30mg orally twice daily for 16 weeks
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mayo Clinic
- Sponsor type
- University, hospital or other
- Collaborators
- Celgene
- Responsible party
- Principal investigator, Alison J. Bruce, Mayo Clinic
- Investigators
- Alison J Bruce, Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03690544
- Sponsor's protocol ID
- 18-002914
- Status last verified
- Jun 2022
- First submitted
- 4 Sep 2018
- Last updated
- 14 Jul 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- 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 14 Jul 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study Evaluating the Efficacy of Apremilast in the Treatment of Subjects With Severe Recurrent Aphthous Stomatitis (RAS)
- ClinicalTrials.gov ID
- NCT03690544
- Acronym / sponsor ID
- 18-002914
- Conditions
- Recurrent Aphthous Stomatitis
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Mayo ClinicUniversity, hospital or other
- Collaborators
- Celgene
- Study start
- 12 Oct 2018 (actual)
- Primary completion
- 14 Jul 2021 (actual)
- Study completion
- 14 Jul 2021 (actual)
- First posted
- 1 Oct 2018 (actual)
- Results
- Posted 14 Jul 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Determination of treatment efficacy and safety of Apremilast in patients with RAS
Detailed description
The study will be a pilot study using apremilast 30mg orally twice daily, for treatment of RAS in males and females between 18 and 70 years old.
Subjects will be recruited from the clinical practice of the Department of Dermatology or Division of Rheumatology at Mayo Clinic Florida. Fifteen males and females with RAS will be enrolled.
The study will consist of 3 phases: a screening phase, a 16 week treatment phase and an 8 week posttreatment observational follow-up phase.
The screening phase will consist of: obtaining informed consent, demographic information, medical history, inclusion and exclusion criteria, prior and concomitant medication use, adverse events; collecting vital signs and weight; performing complete physical examination and limited physical examination; obtaining hematology, serum chemistry, urinalysis, pregnancy test and providing contraception education.
During the 16-week treatment phase, all subjects receive apremilast.
All subjects who complete the active treatment phase are to enter the 8-week posttreatment observational follow-up phase.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Apremilast 30mg orally twice daily for 16 weeks
| Arm | Type | Receives |
|---|---|---|
| Single Arm | Experimental | Drug: Apremilast 30mgApremilast 30mg orally twice daily for 16 weeks, sixteen weeks on active study. Post treatment follow-up period of 8 weeks, in the Treatment of Subjects with Severe Recurrent Aphthous Stomatitis (RAS) |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Male and female subjects between 18 and 70 years of age
- Oral ulcers that occurred at least monthly in the 6 month period prior to enrollment
- Had at least 2 oral ulcers in the 4 weeks prior to enrollment at baseline
- At least 3 oral ulcers during an ulcer flare
- Patients must be candidates for systemic therapy for the treatment of oral ulcers, those that are considered unsuitable for topical therapy alone based on severity of disease, or whose oral ulcers cannot be adequately controlled with topical therapy.
Key exclusion criteria (24 listed)
- Prior use of apremilast.
- Use of any investigational drug within 4 weeks prior to randomization, or 5 pharmacokinetic/pharmacodynamic half-lives, if known (whichever is longer).
- Having received concomitant immune modulating therapy 12 weeks prior to enrollment, systemic steroids 6 weeks prior to enrollment or topical steroids within 4 weeks prior to enrollment.
- Pregnant women or breast-feeding mothers.
- Systemic or opportunistic fungal infection.
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 |
|---|---|---|
| Apremilast 30mgApremilast is an oral small-molecule inhibitor of phosphodiesterase (PDE) 4 that works intracellularly to modulate a network of pro-inflammatory and anti-inflammatory mediators. PDE 4 is a cyclic adenosine monophosphate (cAMP)-specific PDE and the dominant PDE in inflammatory cells. PDE4 inhibition elevates intracellular cAMP levels, which in turn down-regulates the inflammatory response by modulating the expression of TNF-alfa, IL-23, IL-17 and other inflammatory cytokines. Cyclic AMP also modulates levels of anti-inflammatory cytokines such as IL-10. Apremilast has immunomodulatory activity and, therefore, has the potential to be effective in the treatment of RAS. | Drug | Single Arm |
Primary outcome
Duration of RAS Lesions
Time frame: 24 weeks
The total length of time (duration) subjects experienced RAS lesions. Measured in weeks
Change in Number of RAS Lesions
Time frame: baseline, 24 weeks
Number of participants with fewer oral ulcers at Week 24 compared to Baseline
Duration of the Remission Period Between Ulcer Episodes
Time frame: 24 weeks
The length of time of remission of RAS lesions experienced by the subjects. As measured in months.
3 secondary outcomes
Adverse Events
Time frame: 24 weeks
Discontinuation of Study Participants
Time frame: 24 weeks
Change in Visual Analog Scale Pain Score (VAS) From Baseline to 16 Weeks and Baseline to 24 Weeks.
Time frame: baseline, 16 weeks, 24 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Jul 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Mayo Clinic Clinical TrialsLink provided by the sponsor.
- 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.
Alison J Bruce principal investigator
Mayo Clinic
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Apremilast 30mg in people with Recurrent Aphthous Stomatitis, run by Mayo Clinic at 1 site in United States.
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 24 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: finished, as of the sponsor's last update on 14 Jul 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
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 phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 14 Jul 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Duration of RAS Lesions (time frame: 24 weeks). The study lists 3 primary outcomes in all. 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 14 Jul 2022.