Oral Chronic Graft-versus-host DiseasePhase 2NCT00686855

Topical Dexamethasone and Tacrolimus for the Treatment of Oral Chronic Graft-Versus-Host Disease

A Phase 2 study testing Tacrolimus and Dexamethasone in people with Oral Chronic Graft-versus-host Disease, run by Brigham and Women's Hospital at 5 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

The purpose of this research study is to determine the effectiveness of topical steroid therapy (with a drug called dexamethasone) and topical tacrolimus therapy for the treatment of oral chronic Graft-Versus-Host Disease (cGVHD) Read more about this study

Who is it for?: Ages 4 years and older
Conditions the study is for: Oral Chronic Graft-versus-host Disease
What is being tested?: Tacrolimus and Dexamethasone
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 46 participants
Where: 5 sitesUnited States
Study dates: Aug 2008to Mar 2013
Phase: Phase 2
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 4 years and older
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

Patients with oral chronic graft-versus-host disease
Patients 4 years of age or older
Stable cGVHD medication regimen for the four weeks prior to study enrollment

Who cannot join

Patients already on topical steroid or tacrolimus therapies

Trial locations and contacts

5 sites in 1 country.

City, postcode or hospital

5 sites in 1 country

United States5 sites

Massachusetts General Hospital
Boston, Massachusetts, 02114
Brigham and Women's Hospital
Boston, Massachusetts, 02115
Children's Hospital Boston
Boston, Massachusetts, 02115
Dana-Farber Cancer Institute
Boston, Massachusetts, 02115
Beth-Israel Deaconess Medical Center
Boston, Massachusetts, 02215

Other trials in United States

About this study

Official title: An Open Label Phase II Randomized Trial of Topical Dexamethasone and Tacrolimus for the Treatment of Oral Chronic Graft-Versus-Host Disease

Read the detailed description

Because no one knows which study option is best, participants will be "randomized" into one of two study groups: topical dexamethasone or topical tacrolimus.
Participants will take the medication by mouth rinse four times a day for 5 minutes at a time. After rinsing they will spit out the medication and will not be able to eat or drink for 15 minutes.
Participants will also take anti-fungal medication (fluconazole) orally once a week.
After two weeks on study treatment, participants will have a blood drawn to monitor tacrolimus levels.
After the final treatment (4 weeks), participants will have the following tests and procedures: clinical examination; questionnaire; blood tests; oral culture; optional tissue biopsy.

Conditions: Oral Chronic Graft-versus-host Disease
Being tested: Tacrolimus, Dexamethasone
Keywords: tacrolimus, dexamethasone, oral rinse

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

TacrolimusDrug
Tacrolimus elixir taken as an oral rinse four times a day for 4 weeks
DexamethasoneDrug
Dexamethasone elixir taken as an oral rinse 4 times a day for 4 weeks

Study groups in NCT00686855
Study group	Role	What they receive
Tacrolimus	Experimental	Tacrolimus Arm Closed to Accrual as of January 2012 Receives: Drug: Tacrolimus
Dexamethasone	Experimental	Not described Receives: Drug: Dexamethasone

What is being measured

The primary outcome is the main question the study is designed to answer.

The Clinical Efficacy of Topical Steroid and Topical Tacrolimus Therapies for the Treatment of Oral cGHVD.
Participants were given a survey at the time of screening and 4 weeks after start of therapy. The participants self-reported three symptoms of oral cGVHD: oral sensitivity, mouth pain, and mouth dryness. Each symptom was given a score ranging from 0-10, with 0 as none and 10 as the worst. Improvement in subjective scores was defined as 3 points or further reduction from pre-treatment to post-treatment assessment.
Measured: Participants were assessed at Baseline and 4 weeks after start of therapy

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Aug 2008Mar 2013

First posted30 May 2008 (estimated)
Study startAug 2008
Primary completionDec 2012
Study completionMar 2013

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 Oral Chronic Graft-versus-host DiseaseStudies of the same condition, open and under way.
More trials run by Brigham and Women's HospitalThe 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 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: None (open label)
Enrolment: 46 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00686855
Sponsor's protocol ID: 08-027
Status last verified: Feb 2014
First submitted: 27 May 2008
Last updated: 20 Mar 2014
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Oral Chronic Graft-versus-host Disease trials

Study at a glance

From the registry record, last updated by the sponsor on 20 Mar 2014. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: An Open Label Phase II Randomized Trial of Topical Dexamethasone and Tacrolimus for the Treatment of Oral Chronic Graft-Versus-Host Disease
ClinicalTrials.gov ID: NCT00686855
Acronym / sponsor ID: 08-027
Conditions: Oral Chronic Graft-versus-host Disease
Keywords: tacrolimus, dexamethasone, oral rinse
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 46 participants (actual)
Lead sponsor: Brigham and Women's HospitalUniversity, hospital or other
Collaborators: Dana-Farber Cancer Institute, Boston Children's Hospital, Beth Israel Deaconess Medical Center, Massachusetts General Hospital
Study start: Aug 2008
Primary completion: Dec 2012 (actual)
Study completion: Mar 2013 (actual)
First posted: 30 May 2008 (estimated)
Results: Posted 19 Feb 2014

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Because no one knows which study option is best, participants will be "randomized" into one of two study groups: topical dexamethasone or topical tacrolimus.
Participants will take the medication by mouth rinse four times a day for 5 minutes at a time. After rinsing they will spit out the medication and will not be able to eat or drink for 15 minutes.
Participants will also take anti-fungal medication (fluconazole) orally once a week.
After two weeks on study treatment, participants will have a blood drawn to monitor tacrolimus levels.
After the final treatment (4 weeks), participants will have the following tests and procedures: clinical examination; questionnaire; blood tests; oral culture; optional tissue biopsy.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: None (open label)
Number of arms: 2

Arm	Type	Receives
Tacrolimus	Experimental	Drug: TacrolimusTacrolimus Arm Closed to Accrual as of January 2012
Dexamethasone	Experimental	Drug: Dexamethasone

Who may qualify

Ages: 4 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (3 listed)

Patients with oral chronic graft-versus-host disease
Patients 4 years of age or older
Stable cGVHD medication regimen for the four weeks prior to study enrollment

Key exclusion criteria (1 listed)

Patients already on topical steroid or tacrolimus therapies

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
TacrolimusTacrolimus elixir taken as an oral rinse four times a day for 4 weeks	Drug	Tacrolimus
DexamethasoneDexamethasone elixir taken as an oral rinse 4 times a day for 4 weeks	Drug	Dexamethasone

Primary outcome

The Clinical Efficacy of Topical Steroid and Topical Tacrolimus Therapies for the Treatment of Oral cGHVD.
Time frame: Participants were assessed at Baseline and 4 weeks after start of therapy
Participants were given a survey at the time of screening and 4 weeks after start of therapy. The participants self-reported three symptoms of oral cGVHD: oral sensitivity, mouth pain, and mouth dryness. Each symptom was given a score ranging from 0-10, with 0 as none and 10 as the worst. Improvement in subjective scores was defined as 3 points or further reduction from pre-treatment to post-treatment assessment.

Results and safety

Results posted to the registry on 19 Feb 2014, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Dexamethasone	28	27
Tacrolimus	18	17

Primary outcome: The Clinical Efficacy of Topical Steroid and Topical Tacrolimus Therapies for the Treatment of Oral cGHVD.

Time frame: Participants were assessed at Baseline and 4 weeks after start of therapy. Number. Unit: participants

Measure	Dexamethasone n = 22	Tacrolimus n = 14
Improvement in Oral Sensitivity	15	3
Improvement in Mouth Pain	11	2
Improvement in Mouth Dryness	11	5
Improvement in 1 or more areas	17	7
Improvement in 2 or more areas	13	2
Improvement in all 3 areas	7	1

Population analysed: Of the 46 participants enrolled on the trial, 36 were deemed evaluable for response.

Adverse events

Time frame: Adverse events were collected during the four week peroid participants took the study treatment.. Other adverse events reported at 3.5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Dexamethasone	0 of 28 (0%)	1 of 28 (3.6%)	Not reported
Tacrolimus	1 of 18 (5.6%)	1 of 18 (5.6%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 20 Mar 2014.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

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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.

Nathaniel Treister, DMD principal investigator

Brigham and Women's Hospital

5 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Tacrolimus and Dexamethasone in people with Oral Chronic Graft-versus-host Disease, run by Brigham and Women's Hospital at 5 sites 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 3 inclusion and 1 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 20 Mar 2014. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 sites 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 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?

Yes. Results are posted in the registry (first posted 19 Feb 2014). 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?

The Clinical Efficacy of Topical Steroid and Topical Tacrolimus Therapies for the Treatment of Oral cGHVD. (time frame: Participants were assessed at Baseline and 4 weeks after start of therapy). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00686855.

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 20 Mar 2014.