Trial Outcomes & Findings for An Open Label Phase II Trial of Topical Sirolimus for the Treatment of Refractory Oral Chronic Graft-versus-Host-Disease (NCT NCT02123966)

Last Updated: 2018-07-24

Results Overview

The sensitivity score is one question on a self-reported assessment tool from the NIH consensus documents. The question asks: "Your mouth sensitivity at its WORST" with a score 0-10, 10 being the worst. Response was defined as a 3 point reduction in sensitivity score from pre-to-post treatment.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

10 participants

Primary outcome timeframe

Pre treatment and after the 28 day (4 weeks) cycle of treatment

Results posted on

2018-07-24

Participant Flow

Participants were enrolled from July 2014 to November 2016.

Participant milestones

Participant milestones
Measure	Topical Sirolimus + Steroid A four week supply of topical sirolimus will be dispensed. The treatment instructions will be to rinse and spit (NOT swallow) with one teaspoon (5 mL) of sirolimus solution 0.1mg/mL, combined with one teaspoon (5 mL) of steroid solution (dexamethasone 0.1%, clobetasol 0.05%, or budesonide 0.03%, based on the ongoing topical steroid prescription at the time of study enrollment) four times a day for 5 minutes at a time, and not to eat or drink for 15 minutes afterwards.
Overall Study STARTED	10
Overall Study COMPLETED	9
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Topical Sirolimus + Steroid A four week supply of topical sirolimus will be dispensed. The treatment instructions will be to rinse and spit (NOT swallow) with one teaspoon (5 mL) of sirolimus solution 0.1mg/mL, combined with one teaspoon (5 mL) of steroid solution (dexamethasone 0.1%, clobetasol 0.05%, or budesonide 0.03%, based on the ongoing topical steroid prescription at the time of study enrollment) four times a day for 5 minutes at a time, and not to eat or drink for 15 minutes afterwards.
Overall Study Intercurrent Illness	1

Baseline Characteristics

An Open Label Phase II Trial of Topical Sirolimus for the Treatment of Refractory Oral Chronic Graft-versus-Host-Disease

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Topical Sirolimus + Steroid n=10 Participants A four week supply of topical sirolimus will be dispensed. The treatment instructions will be to rinse and spit (NOT swallow) with one teaspoon (5 mL) of sirolimus solution 0.1mg/mL, combined with one teaspoon (5 mL) of steroid solution (dexamethasone 0.1%, clobetasol 0.05%, or budesonide 0.03%, based on the ongoing topical steroid prescription at the time of study enrollment) four times a day for 5 minutes at a time, and not to eat or drink for 15 minutes afterwards.
Age, Continuous	67 years n=99 Participants
Sex: Female, Male Female	4 Participants n=99 Participants
Sex: Female, Male Male	6 Participants n=99 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=99 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	8 Participants n=99 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=99 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=99 Participants
Race (NIH/OMB) Asian	1 Participants n=99 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=99 Participants
Race (NIH/OMB) Black or African American	0 Participants n=99 Participants
Race (NIH/OMB) White	9 Participants n=99 Participants
Race (NIH/OMB) More than one race	0 Participants n=99 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=99 Participants
Region of Enrollment United States	10 Participants n=99 Participants

PRIMARY outcome

Timeframe: Pre treatment and after the 28 day (4 weeks) cycle of treatment

Population: The analysis population is comprised of all treated participants.

Outcome measures

Outcome measures
Measure	Topical Sirolimus + Steroid n=10 Participants A four week supply of topical sirolimus will be dispensed. The treatment instructions will be to rinse and spit (NOT swallow) with one teaspoon (5 mL) of sirolimus solution 0.1mg/mL, combined with one teaspoon (5 mL) of steroid solution (dexamethasone 0.1%, clobetasol 0.05%, or budesonide 0.03%, based on the ongoing topical steroid prescription at the time of study enrollment) four times a day for 5 minutes at a time, and not to eat or drink for 15 minutes afterwards.
Percentage of Participants With a Subjective Sensitivity Score Response	20 percentage of participants Interval 3.7 to 50.7

SECONDARY outcome

Timeframe: Pre treatment and after the 28 day (4 weeks) cycle of treatment

Population: Data was not accessible and therefore the analysis was not conducted.

Oral health related quality of life will be assessed before and after topical sirolimus therapy using the 14-item Oral Health Impact Profile instrument that measures subject's perceptions of the impact of oral conditions on their well-being.

Outcome measures

Outcome data not reported

Adverse Events

Sirolimus

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Nathaniel S. Treister, DMD, DMSc

Dana-Farber Cancer Institute

Phone: 617-632-2806

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place