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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

Margo L. Hoover-Regan

This phase I/II trial studies the safety of topical phenylephrine solution and to see how well it works in preventing oral mucosa (mouth sores) in bone marrow transplant patients receiving cyclophosphamide and total body radiation therapy. Topical phenylephrine solution may prevent or lessen the severity of oral mucosa in patients receiving cyclophosphamide and total body radiation prior to undergoing a bone marrow transplant.

PRIMARY OBJECTIVES:

I. To evaluate the safety and tolerability of phenylephrine (topical phenylephrine solution) applied topically to the oral mucosa of bone marrow transplant patients treated with cyclophosphamide plus total body irradiation.

II. To determine the maximum tolerated dose (MTD) and recommended phase II dose of phenylephrine applied topically to the oral mucosa of bone marrow transplant patients treated with cyclophosphamide plus total body irradiation.

III. To evaluate the efficacy of phenylephrine applied topically to the oral mucosa in preventing and/or decreasing severity of oral mucositis in bone marrow transplant patients treated with cyclophosphamide plus total body irradiation.

OUTLINE: This is a phase I, dose-escalation study of topical phenylephrine solution followed by a phase II study.

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.

After completion of study treatment, patients are followed up weekly for 2 weeks and then at 3 months.

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

A Phase I/IIa Safety and Efficacy Study of Topical Phenylephrine Applied to Oral Mucosa in Bone Marrow Transplant Patients Receiving Cytoxan Plus Total Body Irradiation

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.

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.

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.

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.

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.

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

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.

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.

University of Wisconsin, Madison

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

Supportive Care (Topical Phenylephrine Solution)

Age, Categorical

Sex: Female, Male

Ethnicity (NIH/OMB)

Race (NIH/OMB)

United States

Region of Enrollment

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.

Margo L Hoover-Regan

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.

Area Under the Curve (AUC) for the Oral Mucositis Severity

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.

Duration of Grade 2 Oral Mucositis

Duration of Grade 3 Oral Mucositis

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.

Efficacy Response Rate for Preventing Oral Mucositis With Sufficient Accuracy

Incidence of Adverse Events, Graded According to the Common Terminology Criteria for Adverse Events Version 4.0

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.

Maximum Tolerated Dose (MTD), Defined as the Highest Dose Level of Phenylephrine Applied to the Oral Mucosa Where 0/3, 0/6, or 1/6 Patients Experience a Dose-limiting Toxicity

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.

Recommended Phase IIa Dose

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.

Time to Onset of Grade 2 Oral Mucositis

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.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
Topical Phenylephrine Solution in Preventing Oral Mucosa in Bone Marrow Transplant Patients Receiving Cyclophosphamide and Total Body Radiation Therapy	NCT02434146	Neoplasm Oral Mucosa Transplant-Rela...	Topical Phenyle...	18 Years -	University of Wisconsin, Madison
Topical Phenylephrine Solution in Preventing Oral Mucosa in Bone Marrow Transplant Patients Receiving Cyclophosphamide and Total Body Radiation Therapy	NCT02434146	Neoplasm Oral Mucosa Transplant-Rela...	Topical Phenyle...	18 Years -	University of Wisconsin, Madison

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