Trial Outcomes & Findings for Safety and Efficacy of Nitrogen Mustard in Treatment of Mycosis Fungoides (NCT NCT00168064)

NCT ID: NCT00168064

Last Updated: 2012-10-31

Results Overview

The ratio of the response rate of the patients treated with the PG formulation to the response rate of the patients treated with the AP formulation. Skin response determined by at least a 50% reduction from baseline in the Composite Assessment of Index Lesion Severity (CAILS) following up to 12 months of treatment

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 260 participants
• Primary outcome timeframe: Assessment made at Day 1 and every subsequent visit during treatment
• Results posted on: 2012-10-31

Participant Flow

Participant milestones

Measure	PG -Mechlorethamine (Nitrogen Mustard) 0.02% PG Gel Study formulation of Mechlorethamine-MCH (Nitrogen Mustard) 0.02% Gel	AP- Mechlorethamine 0.02% Compounded in Aquaphor Compounded Mechlorethamine-MCH (Nitrogen Mustard) in Aquaphor 0.02%
Overall Study STARTED	130	130
Overall Study COMPLETED	81	86
Overall Study NOT COMPLETED	49	44

Reasons for withdrawal

Measure	PG -Mechlorethamine (Nitrogen Mustard) 0.02% PG Gel Study formulation of Mechlorethamine-MCH (Nitrogen Mustard) 0.02% Gel	AP- Mechlorethamine 0.02% Compounded in Aquaphor Compounded Mechlorethamine-MCH (Nitrogen Mustard) in Aquaphor 0.02%
Overall Study Adverse Event	26	22
Overall Study Withdrawal by Subject	3	4
Overall Study Lack of Efficacy	4	4
Overall Study Lost to Follow-up	4	3
Overall Study Non-compliance	2	3
Overall Study Concurrent Illness	4	3
Overall Study Subject's Best interest	2	2
Overall Study Various	4	3

Baseline Characteristics

Safety and Efficacy of Nitrogen Mustard in Treatment of Mycosis Fungoides

Baseline characteristics by cohort

Measure	PG -Mechlorethamine-MCH (Nitrogen Mustard) 0.02% PG Gel n=130 Participants Study formulation of mechlorethamine-MCH (Nitrogen Mustard) 0.02% Gel	AP- Mechlorethamine-MCH (NM) 0.02% Compounded in Aquaphor n=130 Participants Compounded mechlorethamine-MCH (Nitrogen Mustard)in Aquaphor 0.02%	Total n=260 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	93 Participants n=5 Participants	86 Participants n=7 Participants	179 Participants n=5 Participants
Age, Categorical >=65 years	37 Participants n=5 Participants	43 Participants n=7 Participants	80 Participants n=5 Participants
Age Continuous	54.7 years STANDARD_DEVIATION 14.20 • n=5 Participants	56.7 years STANDARD_DEVIATION 14.34 • n=7 Participants	55.7 years STANDARD_DEVIATION 14.28 • n=5 Participants
Sex: Female, Male Female	53 Participants n=5 Participants	53 Participants n=7 Participants	106 Participants n=5 Participants
Sex: Female, Male Male	77 Participants n=5 Participants	77 Participants n=7 Participants	154 Participants n=5 Participants
Region of Enrollment United States	130 participants n=5 Participants	130 participants n=7 Participants	260 participants n=5 Participants

PRIMARY outcome

Timeframe: Assessment made at Day 1 and every subsequent visit during treatment

Population: ITT

The ratio of the response rate of the patients treated with the PG formulation to the response rate of the patients treated with the AP formulation. Skin response determined by at least a 50% reduction from baseline in the Composite Assessment of Index Lesion Severity (CAILS) following up to 12 months of treatment

Outcome measures

Measure	PG- Mechlorethamine-MCH (Nitrogen Mustard) 0.02% Gel n=130 Participants Study formulation of mechlorethamine-MCH (Nitrogen Mustard) 0.02% Gel	AP- Aquaphor Formulation Mechlorethamine-MCH (NM) 0.02% n=130 Participants Mechlorethamine-MCH (Nitrogen Mustard) compounded in Aquaphor 0.02%
Ratio of Response Rates Based on CAILS	76 percentage of participants	62 percentage of participants

SECONDARY outcome

Timeframe: Assessment made at Day 1 and every subsequent visit during treatment

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline to end of therapy

Assessment of lesion distribution and severity. A responder analysis was performed on whether subject achieved at least 50% improvement on scale. This had to be confirmed on at least one visit at least 4 weeks apart.

Outcome measures

Measure	PG- Mechlorethamine-MCH (Nitrogen Mustard) 0.02% Gel n=130 Participants Study formulation of mechlorethamine-MCH (Nitrogen Mustard) 0.02% Gel	AP- Aquaphor Formulation Mechlorethamine-MCH (NM) 0.02% n=130 Participants Mechlorethamine-MCH (Nitrogen Mustard) compounded in Aquaphor 0.02%
Percent of Participants Achieving at Least 50% Improvement of Severity Weighted Assessment Tool (SWAT)	61 Percent of participants	60 Percent of participants

Adverse Events

PG -Mechlorethamine-MCH (Nitrogen Mustard) 0.02% PG Gel

Serious events: 0 serious events

Other events: 108 other events

Deaths: 0 deaths

AP- Mechlorethamine-MCH (NM) 0.02% Compounded in Aquaphor

Serious events: 0 serious events

Other events: 99 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	PG -Mechlorethamine-MCH (Nitrogen Mustard) 0.02% PG Gel n=128 participants at risk Study formulation of mechlorethamine-MCH (Nitrogen Mustard) 0.02% Gel	AP- Mechlorethamine-MCH (NM) 0.02% Compounded in Aquaphor n=127 participants at risk Compounded mechlorethamine-MCH (Nitrogen Mustard)in Aquaphor 0.02%
Skin and subcutaneous tissue disorders skin irritation	25.0% 32/128 • Number of events 32 • adverse event data were collected over 12 months of the patient's participation There were a total of 130 participants randomized to each treatment arm. However, 2 from the PG group and 3 from the AP group did not receive drug and are not included in the safety population.	14.2% 18/127 • Number of events 18 • adverse event data were collected over 12 months of the patient's participation There were a total of 130 participants randomized to each treatment arm. However, 2 from the PG group and 3 from the AP group did not receive drug and are not included in the safety population.
Skin and subcutaneous tissue disorders pruritus	19.5% 25/128 • Number of events 25 • adverse event data were collected over 12 months of the patient's participation There were a total of 130 participants randomized to each treatment arm. However, 2 from the PG group and 3 from the AP group did not receive drug and are not included in the safety population.	15.7% 20/127 • Number of events 20 • adverse event data were collected over 12 months of the patient's participation There were a total of 130 participants randomized to each treatment arm. However, 2 from the PG group and 3 from the AP group did not receive drug and are not included in the safety population.
Skin and subcutaneous tissue disorders erythema	17.2% 22/128 • Number of events 22 • adverse event data were collected over 12 months of the patient's participation There were a total of 130 participants randomized to each treatment arm. However, 2 from the PG group and 3 from the AP group did not receive drug and are not included in the safety population.	14.2% 18/127 • Number of events 18 • adverse event data were collected over 12 months of the patient's participation There were a total of 130 participants randomized to each treatment arm. However, 2 from the PG group and 3 from the AP group did not receive drug and are not included in the safety population.
Skin and subcutaneous tissue disorders dermatitis contact	14.8% 19/128 • Number of events 19 • adverse event data were collected over 12 months of the patient's participation There were a total of 130 participants randomized to each treatment arm. However, 2 from the PG group and 3 from the AP group did not receive drug and are not included in the safety population.	15.0% 19/127 • Number of events 19 • adverse event data were collected over 12 months of the patient's participation There were a total of 130 participants randomized to each treatment arm. However, 2 from the PG group and 3 from the AP group did not receive drug and are not included in the safety population.
Skin and subcutaneous tissue disorders skin hyperpigmentation	5.5% 7/128 • Number of events 7 • adverse event data were collected over 12 months of the patient's participation There were a total of 130 participants randomized to each treatment arm. However, 2 from the PG group and 3 from the AP group did not receive drug and are not included in the safety population.	7.1% 9/127 • Number of events 9 • adverse event data were collected over 12 months of the patient's participation There were a total of 130 participants randomized to each treatment arm. However, 2 from the PG group and 3 from the AP group did not receive drug and are not included in the safety population.
Respiratory, thoracic and mediastinal disorders upper respiratory tract infection	8.6% 11/128 • Number of events 11 • adverse event data were collected over 12 months of the patient's participation There were a total of 130 participants randomized to each treatment arm. However, 2 from the PG group and 3 from the AP group did not receive drug and are not included in the safety population.	7.9% 10/127 • Number of events 10 • adverse event data were collected over 12 months of the patient's participation There were a total of 130 participants randomized to each treatment arm. However, 2 from the PG group and 3 from the AP group did not receive drug and are not included in the safety population.
Infections and infestations folliculitis	5.5% 7/128 • Number of events 7 • adverse event data were collected over 12 months of the patient's participation There were a total of 130 participants randomized to each treatment arm. However, 2 from the PG group and 3 from the AP group did not receive drug and are not included in the safety population.	3.9% 5/127 • Number of events 5 • adverse event data were collected over 12 months of the patient's participation There were a total of 130 participants randomized to each treatment arm. However, 2 from the PG group and 3 from the AP group did not receive drug and are not included in the safety population.

Additional Information

H. Jeffrey Wilkins, MD

Yaupon Therapeutics

Phone: 4843247935

Email: hjwilkins@yaupontherapeutics.com

Results disclosure agreements

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