MedDataX Logo

Trial Outcomes & Findings for Treatment of Oral Premalignant Lesions With 5-ALA PDT (NCT NCT00571974)

NCT ID: NCT00571974

Last Updated: 2015-03-24

Results Overview

The traditional 3+3 dose escalation design was employed. Three cohorts were enrolled at 3 subjects per cohort, and treated with escalating radiant exposures of 6, 7, or 8 J/cm2. In each cohort, the number of dose-limiting toxicities (DLTs) were observed. Dose escalation rules were the same as those provided by Storer 1989.

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

29 participants

Primary outcome timeframe

Day 2

Results posted on

2015-03-24

Participant Flow

Subjects were identified in the head and neck oncology clinic and enrolled into the study within the time period of January 1, 2007 to November 18, 2009.

Screen failure did meet incl./excl. criteria

Participant milestones

Participant milestones
Measure
Phase I
To determine the maximum tolerated energy density of the Pulse Dye Laser operated at 585 nm with a pulse time of 1.5 ms (PDL-585), when used in combination with 5-aminolevulinic acid (5-ALA) applied topically to the premalignant lesion. The maximum tolerated energy density will be called the Maximum Tolerated Dose (MTD).
Phase II
During Phase II, to assess the treatment efficacy of PDL-585 when used at the MTD in combination with 5-ALA applied topically to the premalignant lesion. Treatment efficacy will be assessed by means of the 90-day Objective Response rate observed among study subjects treated at the MTD.
Phase I
STARTED
9
0
Phase I
COMPLETED
9
0
Phase I
NOT COMPLETED
0
0
Phase II
STARTED
0
21
Phase II
COMPLETED
0
17
Phase II
NOT COMPLETED
0
4

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Treatment of Oral Premalignant Lesions With 5-ALA PDT

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Phase I
n=11 Participants
To determine the maximum tolerated energy density of the Pulse Dye Laser operated at 585 nm with a pulse time of 1.5 ms (PDL-585), when used in combination with 5-aminolevulinic acid (5-ALA) applied topically to the premalignant lesion. The maximum tolerated energy density will be called the Maximum Tolerated Dose (MTD).
Phase II
n=18 Participants
During Phase II, to assess the treatment efficacy of PDL-585 when used at the MTD in combination with 5-ALA applied topically to the premalignant lesion. Treatment efficacy will be assessed by means of the 90-day Objective Response rate observed among study subjects treated at the MTD.
Total
n=29 Participants
Total of all reporting groups
Age, Customized
<=18 years
0 participants
n=5 Participants
0 participants
n=7 Participants
0 participants
n=5 Participants
Age, Customized
Between 18 and 80 years
11 participants
n=5 Participants
18 participants
n=7 Participants
29 participants
n=5 Participants
Age, Customized
>=80 years
0 participants
n=5 Participants
0 participants
n=7 Participants
0 participants
n=5 Participants
Sex: Female, Male
Female
7 Participants
n=5 Participants
7 Participants
n=7 Participants
14 Participants
n=5 Participants
Sex: Female, Male
Male
4 Participants
n=5 Participants
11 Participants
n=7 Participants
15 Participants
n=5 Participants

PRIMARY outcome

Timeframe: Day 2

Population: Three cohorts were enrolled at 3 subjects per cohort, and treated with escalating radiant exposures (laser doses) of 6, 7, or 8 J/cm2. In each cohort, the number of dose-limiting toxicities (DLTs) were observed. The highest radiant light dose attained that did not produce more than 2 DLTs was declared to be the maximum tolerated dose (MTD).

The traditional 3+3 dose escalation design was employed. Three cohorts were enrolled at 3 subjects per cohort, and treated with escalating radiant exposures of 6, 7, or 8 J/cm2. In each cohort, the number of dose-limiting toxicities (DLTs) were observed. Dose escalation rules were the same as those provided by Storer 1989.

Outcome measures

Outcome measures
Measure
Phase I
n=9 Participants
Participants in the Phase I part of the study.
Maximum Tolerated Dose
8 J/cm2

PRIMARY outcome

Timeframe: Day 90

Population: The Simon two-stage minimax design with 9 subjects in the first stage and 8 subjects in the second stage, yielding 17 subjects overall.

The response rate was quantified by examination by an experienced head and neck surgeon and classified as follows: significant response (SR) was one where the lesion had greater than 75% resolution, partial response (PR) was one in which the lesion was reduced in size by at least 25%, and no response (NR) was one where the lesion was reduced by less than 25% in size.

Outcome measures

Outcome measures
Measure
Phase I
n=17 Participants
Participants in the Phase I part of the study.
The Objective Response Rate is the Number of Participants With Significant Response (SR), Partial Response (PR) or No Response (NR).
Participants with Significant Response (SR)
7 participants
The Objective Response Rate is the Number of Participants With Significant Response (SR), Partial Response (PR) or No Response (NR).
Participants with Partial Response (PR)
9 participants
The Objective Response Rate is the Number of Participants With Significant Response (SR), Partial Response (PR) or No Response (NR).
Participants with no Response (NR)
1 participants

Adverse Events

Phase I

Serious events: 0 serious events
Other events: 9 other events
Deaths: 0 deaths

Phase II

Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
Phase I
n=9 participants at risk
To determine the maximum tolerated energy density of the Pulse Dye Laser operated at 585 nm with a pulse time of 1.5 ms (PDL-585), when used in combination with 5-aminolevulinic acid (5-ALA) applied topically to the premalignant lesion. The maximum tolerated energy density will be called the Maximum Tolerated Dose (MTD).
Phase II
n=21 participants at risk
During Phase II, to assess the treatment efficacy of PDL-585 when used at the MTD in combination with 5-ALA applied topically to the premalignant lesion. Treatment efficacy will be assessed by means of the 90-day Objective Response rate observed among study subjects treated at the MTD.
Nervous system disorders
Pain
22.2%
2/9 • Number of events 2 • 1 year
9.5%
2/21 • Number of events 2 • 1 year
General disorders
Dry mouth
11.1%
1/9 • Number of events 1 • 1 year
0.00%
0/21 • 1 year
General disorders
Bad breath
11.1%
1/9 • Number of events 1 • 1 year
0.00%
0/21 • 1 year
General disorders
Insomnia
11.1%
1/9 • Number of events 1 • 1 year
0.00%
0/21 • 1 year
Psychiatric disorders
Depression
11.1%
1/9 • Number of events 1 • 1 year
0.00%
0/21 • 1 year
General disorders
Swollen lip
11.1%
1/9 • Number of events 1 • 1 year
0.00%
0/21 • 1 year
General disorders
Redness on chin
11.1%
1/9 • Number of events 1 • 1 year
0.00%
0/21 • 1 year
General disorders
Taste altered
11.1%
1/9 • Number of events 1 • 1 year
0.00%
0/21 • 1 year
General disorders
Burning sensation
11.1%
1/9 • Number of events 1 • 1 year
0.00%
0/21 • 1 year
General disorders
Sensitive to cold
11.1%
1/9 • Number of events 1 • 1 year
0.00%
0/21 • 1 year
General disorders
Sensitive
11.1%
1/9 • Number of events 1 • 1 year
0.00%
0/21 • 1 year
General disorders
Raw
11.1%
1/9 • Number of events 1 • 1 year
0.00%
0/21 • 1 year
General disorders
Soreness
11.1%
1/9 • Number of events 1 • 1 year
0.00%
0/21 • 1 year
General disorders
Swelling
11.1%
1/9 • Number of events 1 • 1 year
0.00%
0/21 • 1 year

Additional Information

Gal Shafirstein, D.Sc.

Roswell Park Cancer Institute

Phone: 716-845-4025

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place