Trial Outcomes & Findings for Use of Sodium Stibogluconate as a Treatment for Leishmaniasis (NCT NCT00657618)

Last Updated: 2020-01-02

Results Overview

The safety of Pentostam (SSG) treatment was evaluated through the daily assessment of AEs during treatment. Additionally, clinical laboratory tests including serum chemistry (glucose, electrolytes, blood urea nitrogen \[BUN\], creatinine, alanine aminotransferase \[ALT\], aspartate aminotransferase \[AST\], total bilirubin, alkaline phosphatase \[ALK\], amylase, and lipase) and hematology (hemoglobin, hematocrit, platelets, and white blood cells with differential); vital signs measurements; electrocardiograms (ECGs); and physical examinations were performed at screening and prior to Pentostam infusion on Days (± 2) 5, 10, 15, 20, 25, and 28 (Days 25 and 28 for patients with visceral or mucocutaneous leishmaniasis only). At the completion of Pentostam treatment, patients were encouraged to arrange follow-up at 2, 6, and 12 months after treatment; however, follow-up was not required.

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

77 participants

Primary outcome timeframe

prior to infusion on days (± 2) 5, 10, 15, 20, 25, and 28

Results posted on

2020-01-02

Participant Flow

77 total patients, 74 treated, 34 completed treatment, 43 withdrawals

Due to low enrollment protocol was amended to remove efficacy objective and only collect safety data. No data entry or statistical analyses of patient data was conducted.

Participant milestones

Participant milestones
Measure	Sodium Stibogluconate (SSG) All consented subjects who meet all inclusion and no exclusion criteria will enter this open label protocol and be treated with Sodium Stibogluconate (SSG). Sodium Stibogluconate (SSG): 100 mg/ml/vial. Treatment for laboratory-confirmed leishmaniasis with SSG 20mg/kg/d intravenously (IV) for 10 days or 20 days; visceral leishmaniasis will be treated with SSG 20mg/kg/d IV for 28 days as a second line of therapy for those failing or intolerant of Ambisome; and mucosal leishmaniasis will be treated with SSG 20mg/kg/d IV for 28 days.
Overall Study STARTED	77
Overall Study COMPLETED	34
Overall Study NOT COMPLETED	43

Reasons for withdrawal

Reasons for withdrawal
Measure	Sodium Stibogluconate (SSG) All consented subjects who meet all inclusion and no exclusion criteria will enter this open label protocol and be treated with Sodium Stibogluconate (SSG). Sodium Stibogluconate (SSG): 100 mg/ml/vial. Treatment for laboratory-confirmed leishmaniasis with SSG 20mg/kg/d intravenously (IV) for 10 days or 20 days; visceral leishmaniasis will be treated with SSG 20mg/kg/d IV for 28 days as a second line of therapy for those failing or intolerant of Ambisome; and mucosal leishmaniasis will be treated with SSG 20mg/kg/d IV for 28 days.
Overall Study Adverse Event	32
Overall Study Withdrawal by Subject	8
Overall Study negative for leishmaniasis	2
Overall Study didn't start due to schedule conflicts	1

Baseline Characteristics

Data was not collected

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Sodium Stibogluconate (SSG) n=77 Participants All consented subjects who meet all inclusion and no exclusion criteria will enter this open label protocol and be treated with Sodium Stibogluconate (SSG). Sodium Stibogluconate (SSG): 100 mg/ml/vial. Treatment for laboratory-confirmed leishmaniasis with SSG 20mg/kg/d intravenously (IV) for 10 days or 20 days; visceral leishmaniasis will be treated with SSG 20mg/kg/d IV for 28 days as a second line of therapy for those failing or intolerant of Ambisome; and mucosal leishmaniasis will be treated with SSG 20mg/kg/d IV for 28 days.
Region of Enrollment United States	77 participants n=77 Participants

PRIMARY outcome

Timeframe: prior to infusion on days (± 2) 5, 10, 15, 20, 25, and 28

Population: 74 patients received at least one dose of IV pentostam

Outcome measures

Outcome measures
Measure	Treatment Only n=74 Participants All consented subjects who meet all inclusion and no exclusion criteria will enter this open label protocol and be treated with Sodium Stibogluconate (SSG). Sodium Stibogluconate (SSG): 100 mg/ml/vial. Treatment for laboratory-confirmed leishmaniasis with SSG 20mg/kg/d intravenously (IV) for 10 days or 20 days; visceral leishmaniasis will be treated with SSG 20mg/kg/d IV for 28 days as a second line of therapy for those failing or intolerant of Ambisome; and mucosal leishmaniasis will be treated with SSG 20mg/kg/d IV for 28 days.
Number of Participants That Discontinued Due to Adverse Experience, by Type of Adverse Experience Myalgia/arthralgia	12 Participants
Number of Participants That Discontinued Due to Adverse Experience, by Type of Adverse Experience Elevated liver-associated enzymes	10 Participants
Number of Participants That Discontinued Due to Adverse Experience, by Type of Adverse Experience Rash	3 Participants
Number of Participants That Discontinued Due to Adverse Experience, by Type of Adverse Experience Subtle elevation of amylase and lipase	1 Participants
Number of Participants That Discontinued Due to Adverse Experience, by Type of Adverse Experience Misdiagnosis	1 Participants
Number of Participants That Discontinued Due to Adverse Experience, by Type of Adverse Experience Decreased white blood cell count	1 Participants
Number of Participants That Discontinued Due to Adverse Experience, by Type of Adverse Experience Nephrotoxicity	1 Participants
Number of Participants That Discontinued Due to Adverse Experience, by Type of Adverse Experience Chemical pancresatitis	1 Participants
Number of Participants That Discontinued Due to Adverse Experience, by Type of Adverse Experience Drug reaction	1 Participants

PRIMARY outcome

Timeframe: prior to infusion on days 2) 5, 10, 15, 20, 25, and 28

Population: 74 patients received at least one dose of IV pentostam

Outcome measures

Outcome measures
Measure	Treatment Only n=74 Participants All consented subjects who meet all inclusion and no exclusion criteria will enter this open label protocol and be treated with Sodium Stibogluconate (SSG). Sodium Stibogluconate (SSG): 100 mg/ml/vial. Treatment for laboratory-confirmed leishmaniasis with SSG 20mg/kg/d intravenously (IV) for 10 days or 20 days; visceral leishmaniasis will be treated with SSG 20mg/kg/d IV for 28 days as a second line of therapy for those failing or intolerant of Ambisome; and mucosal leishmaniasis will be treated with SSG 20mg/kg/d IV for 28 days.
Number of Participants Experiencing Serious or Unexpected Adverse Events, by Type of Serious or Unexpected Adverse Events Elevated lipase level	4 Participants
Number of Participants Experiencing Serious or Unexpected Adverse Events, by Type of Serious or Unexpected Adverse Events Chemical hepatitis	1 Participants
Number of Participants Experiencing Serious or Unexpected Adverse Events, by Type of Serious or Unexpected Adverse Events Allergic reaction with atypical presentation	1 Participants
Number of Participants Experiencing Serious or Unexpected Adverse Events, by Type of Serious or Unexpected Adverse Events Misdiagnosis	1 Participants
Number of Participants Experiencing Serious or Unexpected Adverse Events, by Type of Serious or Unexpected Adverse Events Allergic reaction	1 Participants
Number of Participants Experiencing Serious or Unexpected Adverse Events, by Type of Serious or Unexpected Adverse Events Hospitalized for unhealed cutaneous lesion	1 Participants

Adverse Events

Treatment Only

Serious events: 8 serious events

Other events: 32 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Treatment Only n=74 participants at risk All consented subjects who meet all inclusion and no exclusion criteria will enter this open label protocol and be treated with Sodium Stibogluconate (SSG). Sodium Stibogluconate (SSG): 100 mg/ml/vial. Treatment for laboratory-confirmed leishmaniasis with SSG 20mg/kg/d intravenously (IV) for 10 days or 20 days; visceral leishmaniasis will be treated with SSG 20mg/kg/d IV for 28 days as a second line of therapy for those failing or intolerant of Ambisome; and mucosal leishmaniasis will be treated with SSG 20mg/kg/d IV for 28 days.
Investigations Elevated lipase/aysmpotmatic chemical pancreatitis	5.4% 4/74 • Number of events 4 • prior to infusion on days (± 2) 5, 10, 15, 20, 25, and 28 Safety of Pentostam was evaluated through daily assessment of AEs. Clinical lab tests including serum chemistry (glucose, electrolytes, blood urea nitrogen, creatinine, alanine aminotransferase, aspartate aminotransferase\], total bilirubin, alkaline phosphatase\], amylase, and lipase) and hematology (hemoglobin, hematocrit, platelets, and white blood cells with differential); vital signs measurements; electrocardiograms); and physical examinations were performed at screening and prior to infusion
Investigations Chemical hepatitis	1.4% 1/74 • Number of events 1 • prior to infusion on days (± 2) 5, 10, 15, 20, 25, and 28 Safety of Pentostam was evaluated through daily assessment of AEs. Clinical lab tests including serum chemistry (glucose, electrolytes, blood urea nitrogen, creatinine, alanine aminotransferase, aspartate aminotransferase\], total bilirubin, alkaline phosphatase\], amylase, and lipase) and hematology (hemoglobin, hematocrit, platelets, and white blood cells with differential); vital signs measurements; electrocardiograms); and physical examinations were performed at screening and prior to infusion
Immune system disorders Allergic reaction with atypical presentation	1.4% 1/74 • Number of events 1 • prior to infusion on days (± 2) 5, 10, 15, 20, 25, and 28 Safety of Pentostam was evaluated through daily assessment of AEs. Clinical lab tests including serum chemistry (glucose, electrolytes, blood urea nitrogen, creatinine, alanine aminotransferase, aspartate aminotransferase\], total bilirubin, alkaline phosphatase\], amylase, and lipase) and hematology (hemoglobin, hematocrit, platelets, and white blood cells with differential); vital signs measurements; electrocardiograms); and physical examinations were performed at screening and prior to infusion
Investigations Misdiagnosis	1.4% 1/74 • Number of events 1 • prior to infusion on days (± 2) 5, 10, 15, 20, 25, and 28 Safety of Pentostam was evaluated through daily assessment of AEs. Clinical lab tests including serum chemistry (glucose, electrolytes, blood urea nitrogen, creatinine, alanine aminotransferase, aspartate aminotransferase\], total bilirubin, alkaline phosphatase\], amylase, and lipase) and hematology (hemoglobin, hematocrit, platelets, and white blood cells with differential); vital signs measurements; electrocardiograms); and physical examinations were performed at screening and prior to infusion
Immune system disorders Allergic reaction	1.4% 1/74 • Number of events 1 • prior to infusion on days (± 2) 5, 10, 15, 20, 25, and 28 Safety of Pentostam was evaluated through daily assessment of AEs. Clinical lab tests including serum chemistry (glucose, electrolytes, blood urea nitrogen, creatinine, alanine aminotransferase, aspartate aminotransferase\], total bilirubin, alkaline phosphatase\], amylase, and lipase) and hematology (hemoglobin, hematocrit, platelets, and white blood cells with differential); vital signs measurements; electrocardiograms); and physical examinations were performed at screening and prior to infusion
Product Issues Hospitalization for unhealed lesion	1.4% 1/74 • Number of events 1 • prior to infusion on days (± 2) 5, 10, 15, 20, 25, and 28 Safety of Pentostam was evaluated through daily assessment of AEs. Clinical lab tests including serum chemistry (glucose, electrolytes, blood urea nitrogen, creatinine, alanine aminotransferase, aspartate aminotransferase\], total bilirubin, alkaline phosphatase\], amylase, and lipase) and hematology (hemoglobin, hematocrit, platelets, and white blood cells with differential); vital signs measurements; electrocardiograms); and physical examinations were performed at screening and prior to infusion

Other adverse events

Other adverse events
Measure	Treatment Only n=74 participants at risk All consented subjects who meet all inclusion and no exclusion criteria will enter this open label protocol and be treated with Sodium Stibogluconate (SSG). Sodium Stibogluconate (SSG): 100 mg/ml/vial. Treatment for laboratory-confirmed leishmaniasis with SSG 20mg/kg/d intravenously (IV) for 10 days or 20 days; visceral leishmaniasis will be treated with SSG 20mg/kg/d IV for 28 days as a second line of therapy for those failing or intolerant of Ambisome; and mucosal leishmaniasis will be treated with SSG 20mg/kg/d IV for 28 days.
Musculoskeletal and connective tissue disorders Myalgia/arthralgia	16.2% 12/74 • Number of events 12 • prior to infusion on days (± 2) 5, 10, 15, 20, 25, and 28 Safety of Pentostam was evaluated through daily assessment of AEs. Clinical lab tests including serum chemistry (glucose, electrolytes, blood urea nitrogen, creatinine, alanine aminotransferase, aspartate aminotransferase\], total bilirubin, alkaline phosphatase\], amylase, and lipase) and hematology (hemoglobin, hematocrit, platelets, and white blood cells with differential); vital signs measurements; electrocardiograms); and physical examinations were performed at screening and prior to infusion
Investigations Elevated liver-assocated enzymes	13.5% 10/74 • Number of events 10 • prior to infusion on days (± 2) 5, 10, 15, 20, 25, and 28 Safety of Pentostam was evaluated through daily assessment of AEs. Clinical lab tests including serum chemistry (glucose, electrolytes, blood urea nitrogen, creatinine, alanine aminotransferase, aspartate aminotransferase\], total bilirubin, alkaline phosphatase\], amylase, and lipase) and hematology (hemoglobin, hematocrit, platelets, and white blood cells with differential); vital signs measurements; electrocardiograms); and physical examinations were performed at screening and prior to infusion
Immune system disorders Rash	4.1% 3/74 • Number of events 3 • prior to infusion on days (± 2) 5, 10, 15, 20, 25, and 28 Safety of Pentostam was evaluated through daily assessment of AEs. Clinical lab tests including serum chemistry (glucose, electrolytes, blood urea nitrogen, creatinine, alanine aminotransferase, aspartate aminotransferase\], total bilirubin, alkaline phosphatase\], amylase, and lipase) and hematology (hemoglobin, hematocrit, platelets, and white blood cells with differential); vital signs measurements; electrocardiograms); and physical examinations were performed at screening and prior to infusion
Investigations Subtle ECG changes	1.4% 1/74 • Number of events 1 • prior to infusion on days (± 2) 5, 10, 15, 20, 25, and 28 Safety of Pentostam was evaluated through daily assessment of AEs. Clinical lab tests including serum chemistry (glucose, electrolytes, blood urea nitrogen, creatinine, alanine aminotransferase, aspartate aminotransferase\], total bilirubin, alkaline phosphatase\], amylase, and lipase) and hematology (hemoglobin, hematocrit, platelets, and white blood cells with differential); vital signs measurements; electrocardiograms); and physical examinations were performed at screening and prior to infusion
Investigations Elevation of amylase and lipase	1.4% 1/74 • Number of events 1 • prior to infusion on days (± 2) 5, 10, 15, 20, 25, and 28 Safety of Pentostam was evaluated through daily assessment of AEs. Clinical lab tests including serum chemistry (glucose, electrolytes, blood urea nitrogen, creatinine, alanine aminotransferase, aspartate aminotransferase\], total bilirubin, alkaline phosphatase\], amylase, and lipase) and hematology (hemoglobin, hematocrit, platelets, and white blood cells with differential); vital signs measurements; electrocardiograms); and physical examinations were performed at screening and prior to infusion
Investigations Misdiagnosis	1.4% 1/74 • Number of events 1 • prior to infusion on days (± 2) 5, 10, 15, 20, 25, and 28 Safety of Pentostam was evaluated through daily assessment of AEs. Clinical lab tests including serum chemistry (glucose, electrolytes, blood urea nitrogen, creatinine, alanine aminotransferase, aspartate aminotransferase\], total bilirubin, alkaline phosphatase\], amylase, and lipase) and hematology (hemoglobin, hematocrit, platelets, and white blood cells with differential); vital signs measurements; electrocardiograms); and physical examinations were performed at screening and prior to infusion
Investigations Decreased white blood cell count	1.4% 1/74 • Number of events 1 • prior to infusion on days (± 2) 5, 10, 15, 20, 25, and 28 Safety of Pentostam was evaluated through daily assessment of AEs. Clinical lab tests including serum chemistry (glucose, electrolytes, blood urea nitrogen, creatinine, alanine aminotransferase, aspartate aminotransferase\], total bilirubin, alkaline phosphatase\], amylase, and lipase) and hematology (hemoglobin, hematocrit, platelets, and white blood cells with differential); vital signs measurements; electrocardiograms); and physical examinations were performed at screening and prior to infusion
Investigations Nephrotoxicity	1.4% 1/74 • Number of events 1 • prior to infusion on days (± 2) 5, 10, 15, 20, 25, and 28 Safety of Pentostam was evaluated through daily assessment of AEs. Clinical lab tests including serum chemistry (glucose, electrolytes, blood urea nitrogen, creatinine, alanine aminotransferase, aspartate aminotransferase\], total bilirubin, alkaline phosphatase\], amylase, and lipase) and hematology (hemoglobin, hematocrit, platelets, and white blood cells with differential); vital signs measurements; electrocardiograms); and physical examinations were performed at screening and prior to infusion
Gastrointestinal disorders Chemical pancreatitis	1.4% 1/74 • Number of events 1 • prior to infusion on days (± 2) 5, 10, 15, 20, 25, and 28 Safety of Pentostam was evaluated through daily assessment of AEs. Clinical lab tests including serum chemistry (glucose, electrolytes, blood urea nitrogen, creatinine, alanine aminotransferase, aspartate aminotransferase\], total bilirubin, alkaline phosphatase\], amylase, and lipase) and hematology (hemoglobin, hematocrit, platelets, and white blood cells with differential); vital signs measurements; electrocardiograms); and physical examinations were performed at screening and prior to infusion
General disorders Drug reaction	1.4% 1/74 • Number of events 1 • prior to infusion on days (± 2) 5, 10, 15, 20, 25, and 28 Safety of Pentostam was evaluated through daily assessment of AEs. Clinical lab tests including serum chemistry (glucose, electrolytes, blood urea nitrogen, creatinine, alanine aminotransferase, aspartate aminotransferase\], total bilirubin, alkaline phosphatase\], amylase, and lipase) and hematology (hemoglobin, hematocrit, platelets, and white blood cells with differential); vital signs measurements; electrocardiograms); and physical examinations were performed at screening and prior to infusion

Additional Information

Roseanne A. Resnner, MD

WRAIR

Phone: 301-295-6400

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place