Trial Outcomes & Findings for Miltefosine to Treat Mucocutaneous Leishmaniasis (NCT NCT01050907)

Last Updated: 2020-09-30

Results Overview

Percent of participants with clinical cure of all lesions. Ulcerated CL lesions were measured for the longest diameter and perpendicular width of ulceration; non-ulcerated lesions were measured for length and width of the raised area. A healed lesion was 100% reduction in lesion area (0x0); a cured lesion was a lesion healed at the Month 7 visit. For subjects with ML, an Ear, Nose, and Throat specialist examined the nasal and oral mucosa. Each site (nasal skin, nasal mucosa, palate, pharynx, larynx) was evaluated for signs of disease (erythema, edema, infiltration, erosion) and graded on a scale: 0=no disease, 1=mild disease, 2=moderate disease, 3=severe disease. Max score was 60 = poor outcome. Clinical response measured as a composite score, the mucosal severity score, which was the sum of the severity scores for each clinical sign at each clinical site of disease. A healed lesion had a score of 0 in absolute value (0% of the entrance score), and clinical cure was lesion is healed.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

4 participants

Primary outcome timeframe

Week 6, Month 3, Month 7, and Month 13

Results posted on

2020-09-30

Participant Flow

Participant milestones

Participant milestones
Measure	Miltefosine Miltefosine: 2.5 mg/kg/day for 28 days
Overall Study STARTED	4
Overall Study COMPLETED	4
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Miltefosine to Treat Mucocutaneous Leishmaniasis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Miltefosine n=4 Participants Miltefosine: 2.5 mg/kg/day for 28 days
Age, Categorical <=18 years	1 Participants n=93 Participants
Age, Categorical Between 18 and 65 years	3 Participants n=93 Participants
Age, Categorical >=65 years	0 Participants n=93 Participants
Sex: Female, Male Female	2 Participants n=93 Participants
Sex: Female, Male Male	2 Participants n=93 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants
Race (NIH/OMB) Asian	1 Participants n=93 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants
Race (NIH/OMB) Black or African American	1 Participants n=93 Participants
Race (NIH/OMB) White	2 Participants n=93 Participants
Race (NIH/OMB) More than one race	0 Participants n=93 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants
Region of Enrollment United States	4 participants n=93 Participants

PRIMARY outcome

Timeframe: Week 6, Month 3, Month 7, and Month 13

Population: The evaluable population included all subjects who received daily doses of investigational product for at least 25 of the total of 28 days, had lesion measurements at Month 7 for cutaneous leishmaniasis or the month 13 visit for mucosal leishmaniasis, and who did not receive rescue medications to treat leishmaniasis at any time during the study.

Outcome measures

Outcome measures
Measure	Miltefosine n=4 Participants Miltefosine: 2.5 mg/kg/day for 28 days
Number of Participants With Clinical Cure of Lesions	3 Participants

PRIMARY outcome

Timeframe: Up to 7 months for CL; Up to 13 months for ML

Population: The safety population included all subjects who received any administration of investigational product.

The number of participants with adverse events (AEs) by occurrence and severity. The Treating Physician monitored participants for the occurrence of AEs from the time the first investigational product was taken on Day 1 through the end of follow up at Month 7 for CL or Month 13 for ML. For the period between Study Day 1 and Study Week 6 (2 weeks after the end of therapy), all AEs regardless of seriousness or relationship to the investigational product were to be recorded on the case report form (CRF). For the period Week 6 to Month 7 for CL, or Month 13 for ML, only AEs requiring medical attention were recorded on the CRF.

Outcome measures

Outcome measures
Measure	Miltefosine n=4 Participants Miltefosine: 2.5 mg/kg/day for 28 days
Number of Participants With Adverse Events	4 Participants

Adverse Events

Miltefosine

Serious events: 0 serious events

Other events: 4 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Miltefosine n=4 participants at risk Miltefosine: 2.5 mg/kg/day for 28 days
Reproductive system and breast disorders Epididymitis	25.0% 1/4 • Number of events 1 • Up to 7 months for CL; Up to 13 months for ML
Gastrointestinal disorders Abdominal Pain Lower	25.0% 1/4 • Number of events 1 • Up to 7 months for CL; Up to 13 months for ML
Gastrointestinal disorders Nausea	50.0% 2/4 • Number of events 7 • Up to 7 months for CL; Up to 13 months for ML
Gastrointestinal disorders Vomiting	50.0% 2/4 • Number of events 8 • Up to 7 months for CL; Up to 13 months for ML
Skin and subcutaneous tissue disorders Rash Pruritic	25.0% 1/4 • Number of events 1 • Up to 7 months for CL; Up to 13 months for ML
Respiratory, thoracic and mediastinal disorders Epistaxis	25.0% 1/4 • Number of events 1 • Up to 7 months for CL; Up to 13 months for ML

Additional Information

Jonathan D. Berman

Fast-Track Drugs and Biologics, LLC

Phone: 301-922-2097

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place