Trial Outcomes & Findings for Topical Phenylephrine Solution in Preventing Oral Mucosa in Bone Marrow Transplant Patients Receiving Cyclophosphamide and Total Body Radiation Therapy (NCT NCT02434146)

Results Overview

The mucositis AUC will be estimated using the trapezoid method and summarized in terms of means, standard deviation, median and range. This analysis will be performed in both the extended cohort as well as in the historical controls.

Recruitment status

TERMINATED

Study phase

PHASE1/PHASE2

Target enrollment

3 participants

Primary outcome timeframe

Up to 3 months

Results posted on

2019-12-09

Participant Flow

This study enrolled adult participants undergoing hematopoietic stem cell transplantation (SCT) who were to receive cyclophosphamide and TBI as the conditioning regimen. Participants were recruited from the University of Wisconsin Hospitals and Clinics between May 2015 and December 2015.

Participant milestones

Participant milestones
Measure	Supportive Care (Topical Phenylephrine Solution) Patients undergoing a cyclophosphamide and total body irradiation regimen receive topical phenylephrine solution via spray to the oral mucosa 15-20 minutes prior to each cyclophosphamide infusion, 25-30 minutes after the beginning of each cyclophosphamide infusion, and 15-20 minutes prior to each radiation treatment. Topical Phenylephrine Solution: Given topically via spray
Overall Study STARTED	3
Overall Study COMPLETED	3
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Topical Phenylephrine Solution in Preventing Oral Mucosa in Bone Marrow Transplant Patients Receiving Cyclophosphamide and Total Body Radiation Therapy

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Supportive Care (Topical Phenylephrine Solution) n=3 Participants Patients undergoing a cyclophosphamide and total body irradiation regimen receive topical phenylephrine solution via spray to the oral mucosa 15-20 minutes prior to each cyclophosphamide infusion, 25-30 minutes after the beginning of each cyclophosphamide infusion, and 15-20 minutes prior to each radiation treatment. Topical Phenylephrine Solution: Given topically via spray
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	3 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants
Race (NIH/OMB) White	2 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	3 participants n=5 Participants

PRIMARY outcome

Timeframe: Up to 3 months

Population: This study was closed prematurely due to decreased numbers of eligible subjects. The full target accrual number was not achieved, and AUC could not be estimated or analyzed.

The mucositis AUC will be estimated using the trapezoid method and summarized in terms of means, standard deviation, median and range. This analysis will be performed in both the extended cohort as well as in the historical controls.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: Date of onset of grade 2 oral mucositis to the date of the resolution of the grade 2 oral mucositis, assessed up to 3 months

Population: This study was closed prematurely due to decreased numbers of eligible subjects. The full target accrual number was not achieved, and the median duration of oral mucositis could not be analyzed.

If the grade 2 oral mucositis has not been resolved (to a grade \< 2) by the last day of toxicity assessment, then the duration will be censored at the last date of toxicity assessment. Will be analyzed using the Kaplan-Meier method. The median duration of grade 2/grade 3 oral mucositis will be calculated and reported along with the corresponding 95% confidence interval. This analysis will be performed in both the extended cohort as well as in the historical controls.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: Date of onset of grade 3 oral mucositis to the date of resolution to grade < 3 oral mucositis, assessed up to 3 months

Population: This study was closed prematurely due to decreased numbers of eligible subjects. The full target accrual number was not achieved, and the median duration of oral mucositis could not be analyzed.

If the grade 3 oral mucositis has not been resolved (to a grade \< 3) by the last day of toxicity assessment, then the duration will be censored at the last date of toxicity assessment. Will be analyzed using the Kaplan-Meier method. The median duration of grade 2/grade 3 oral mucositis will be calculated and reported along with the corresponding 95% confidence interval. This analysis will be performed in both the extended cohort as well as in the historical controls.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: Up to 3 months

Population: This study was closed prematurely due to decreased numbers of eligible subjects. The full target accrual number was not achieved, and the efficacy response rate could not be estimated.

If a patient experiences no higher than grade 2 oral mucositis, then s/he will be defined as a responder. If a patient experiences grade \>= 3 oral mucositis, s/he will be defined as a non-responder. Specifically, the efficacy response rate will be estimated with a standard error of less than 15% and the length of the 95% confidence interval will be less than 50%. The efficacy response rate will be summarized in tabular format. The Wilson score method will be used to calculate the 95% confidence interval for the efficacy response rate for the extended cohort.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: Up to 3 months

Population: This study was closed prematurely due to decreased numbers of eligible subjects. The full target accrual number was not achieved, and no study conclusions could be made.

Adverse events (AEs) will be presented in the summary tables by preferred term nested within the System Organ Class. Verbatim description, preferred term, and system organ class for all AEs will be contained in the patient data listings. All AEs occurring after enrollment and throughout the study period will be recorded. Each toxicity event will be assigned an attribution: unrelated, unlikely, possibly, probably, or definitely phenylephrine treatment related.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: During the conditioning regimen (radiation and cyclophosphamide treatment), which is anticipated to last 1 week.

Population: This study was closed prematurely due to decreased numbers of eligible subjects. The full target accrual number was not achieved, and MTD could not be determined.

Determine Maximum Tolerated Dose (MTD), the highest dose level of phenylephrine applied to the oral mucosa

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: During the conditioning regimen (radiation and cyclophosphamide treatment), which is anticipated to last 1 week.

Population: This study was closed prematurely due to decreased numbers of eligible subjects. The full target accrual number was not achieved, and the dose of topical phenylephrine solution for a larger follow-up phase II efficacy study could not be recommended.

The dose of topical phenylephrine solution which will be recommended for a larger follow-up phase II efficacy study will be established after the dose cohort at the MTD has been expanded to a total of 12 patients.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: Time between the first date of radiation or cyclophosphamide treatment to the date of the onset of grade 2 oral mucositis, assessed up to 3 months

Population: This study was closed prematurely due to decreased numbers of eligible subjects. The full target accrual number was not achieved, and time to onset of grade 2 oral mucositis could not be analyzed.

Will be analyzed using a parametric (one-parameter exponential) cure rate model. Will be performed in both the extended cohort as well as in the historical controls.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: Time between the first date of radiation or cyclophosphamide treatment to the date of the onset of grade 3 oral mucositis, assessed up to 3 months

Population: This study was closed prematurely due to decreased numbers of eligible subjects. The full target accrual number was not achieved, and time to onset of grade 3 oral mucositis could not be analyzed.

Will be analyzed using a parametric (one-parameter exponential) cure rate model. Will be performed in both the extended cohort as well as in the historical controls.

Outcome measures

Outcome data not reported

Adverse Events

Supportive Care (Topical Phenylephrine Solution)

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Supportive Care (Topical Phenylephrine Solution) n=3 participants at risk Patients undergoing a cyclophosphamide and total body irradiation regimen receive topical phenylephrine solution via spray to the oral mucosa 15-20 minutes prior to each cyclophosphamide infusion, 25-30 minutes after the beginning of each cyclophosphamide infusion, and 15-20 minutes prior to each radiation treatment. Topical Phenylephrine Solution: Given topically via spray
Gastrointestinal disorders Grade 2/3 mucositis	100.0% 3/3 • Adverse event data were collected for 1 year.

Additional Information

Margo L Hoover-Regan

University of Wisconsin Carbone Cancer Center

Phone: 608-263-6200

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place