Primary Visceral LeishmaniasisPhase 2NCT05593666

A Phase II, Multicentre, Randomized, Two-arm Blinded Study to Assess the Efficacy and Safety of Two LXE408 Regimens for Treatment of Patients With Primary Visceral Leishmaniasis

A Phase 2 study testing LXE408 and AmBisome® in people with Primary Visceral Leishmaniasis, run by Drugs for Neglected Diseases at 2 sites in India.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

This is a phase II, multicentre, randomized, two-arm blinded study with an open label calibrator arm in adults and adolescents (≥12 years) with confirmed primary VL. Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Primary Visceral Leishmaniasis
What is being tested?: LXE408 and AmBisome®
Type of treatment: Pill or oral medicine, Injection or infusion
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 101 participants
Where: 2 sitesIndia
Study dates: 27 Dec 2022to 22 Dec 2025
Phase: Phase 2
Are there results?: None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Male and female patients ≥ 18 years (at the time of the screening visit) who are able to comply with the study protocol. Following a favourable interim analysis result, patients ≥12 <18 years will also be enrolled in the trial
Patients for whom written informed consent has been obtained (if aged 18 years and over) or signed by parent(s) or legal guardian for patients under 18 years of age. In the case of minors, assent from the child also needs to be obtained
Primary symptomatic VL (defined as typical parameters including, but not limited to, fever for > 2 weeks, weight loss, and splenomegaly)
Visualization of Leishmania amastigotes by microscopy in tissue samples (spleen or bone marrow)

Who cannot join

Clinical signs of severe VL (jaundice, spontaneous bleeding, edema, ascites, coma, organ failure)
Laboratory abnormalities including ALT/SGPT > 3 times ULN, total bilirubin > 1.5 times ULN, creatinine >1.5 times ULN, amylase or lipase > 1.5 times ULN, haemoglobin < 6 g/dL or other clinically significant abnormal laboratory parameters which, in the opinion of the investigator, may indicate severe VL
Patients with history of previous leishmaniasis and confirmed relapse
Patients with para-kala-azar dermal leishmaniasis
Patients with severe malnutrition (for children ≥12-<18 years: BMI-for-age WHO reference curves by sex, z score < -3; for adults ≥18 years: BMI < 16)
History of congenital or acquired immunodeficiency, including positive HIV (test at screening)

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Known hypersensitivity to amphotericin B deoxycholate or any other constituents of AmBisome®
Concomitant infections such as tuberculosis, severe malaria, or any other serious underlying disease that may interfere with the disease assessment (e.g., cardiac, renal, hepatic, haematologic, and pancreatic)
Infection with hepatitis B (HBV) or hepatitis C virus (HCV). A positive HBV surface antigen (HBsAg) test, or if standard local practice, a positive HBV core antigen test, excludes a subject. Patients with a positive HCV antibody test should have HCV RNA levels measured. Patients with positive (detectable) HCV RNA should be excluded.
Pregnant or nursing (lactating) women
Women of childbearing potential who do not accept to have a pregnancy test done at screening and/or who do not agree to use highly effective contraception while taking the investigational drug and for 5 half-lives or 5 days, whichever is longer, after stopping the investigational drug.
Sexually active males unwilling to use a condom during intercourse while taking the investigational drug and for 5 half-lives or 5 days, whichever is longer, after stopping the investigational drug.

Trial locations and contacts

2 sites in 1 country.

India2 sites

DrugsNeglectedD Investigational Site
Bihār
DrugsNeglectedD Investigational Site
Patna

Other trials in India

About this study

This is a phase II, multicentre, randomized, two-arm blinded study with an open label calibrator arm in adults and adolescents (≥12 years) with confirmed primary VL.

Read the detailed description

This study is run by DNDi with Novartis as co-development partner

Conditions: Primary Visceral Leishmaniasis
Being tested: LXE408, Placebo, AmBisome®

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

LXE408Drug
Film-coated tablets
PlaceboOther
Placebo film-coated tablets
AmBisome®Drug
Sterile lyophilised powder in a 15 mL sterile clear glass vial

Study groups in NCT05593666
Study group	Role	What they receive
LXE408 short regimen	Experimental	LXE408 once daily for seven days (followed by 7 days of placebo). Receives: Drug: LXE408; Other: Placebo
LXE408 long regimen	Experimental	LXE408 once daily for 14 days Receives: Drug: LXE408
Standard of care	Active comparator	AmBisome® 10 mg/kg IV single dose (SDA) Receives: Drug: AmBisome®

What is being measured

The primary outcome is the main question the study is designed to answer.

Proportion of patients with initial cure at Day 28 for LXE408
Initial cure defined as clinical improvement of Visceral leishmaniasis (VL), absence of parasites in the spleen or bone marrow (microscopy), and no rescue therapy on or before Day 28.
Measured: Day 28

Secondary outcomes (14)

Proportion of patients with initial cure at Day 28 for AmBisome®
Initial cure defined as clinical improvement of Visceral leishmaniasis (VL), absence of parasites in the spleen or bone marrow (microscopy), and no rescue therapy on or before Day 28.
Measured: Day 28
Proportion of patients with definitive cure at Day 180 for LXE408 and AmBisome®
Definitive cure described as initial cure at Day 28, no requirement for rescue treatment throughout the study, no death associated to VL and absence of any clinical parameters of VL at Day 180.
Measured: Day 180
Mortality
All-cause mortality and mortality not associated with Visceral leishmaniasis (VL)
Measured: Days 28 and 180
Cmax for LXE408
Maximum Observed Blood-drug Concentrations for LXE408
Measured: Days 1 and 7
Tmax for LXE408
Time to Reach Maximum Blood-drug Concentrations for LXE408
Measured: Days 1 and 7
AUCtau for LXE408
Area Under The Plasma Concentration-time Curve Over A Dosing Interval for LXE408
Measured: Days 1 and 7
CLss/F for LXE408
Apparent Clearance for LXE408
Measured: Days 1 and 7
Cmax for Amphotericin B
Maximum Observed Blood-drug Concentrations for Amphotericin B
Measured: Days 1 and 7
AUC0-24h for Amphotericin B
Area under the plasma concentration-time curve from time zero to 24h for Amphotericin B
Measured: Day 1
AUC0-infinity for Amphotericin B
Area under the plasma concentration-time curve from time zero to infinity for Amphotericin B
Measured: Day 1
Blood parasite clearance
Blood parasite clearance over time, as measured by quantitative polymerase chain reaction (qPCR) from blood samples at defined time points and at any suspicion of relapse during the trial.
Measured: Baseline and Days 1, 3, 5, 7, 10, 14, 28 and 56
Proportion of patients with a positive loop-mediated isothermal amplification (LAMP) from blood samples
Proportion of patients with a positive loop-mediated isothermal amplification (LAMP) from blood samples at defined time points and at any suspicion of relapse during the trial.
Measured: Baseline and Days 28 and 56
Tissue parasite loads
Tissue parasite loads, as measured by qPCR from tissue samples (spleen or bone marrow) collected at defined time points and at any suspicion of relapse during the trial.
Measured: Baseline and Day 28
Proportion of patients with a positive loop-mediated isothermal amplification (LAMP) from tissue samples
Proportion of patients with a positive loop-mediated isothermal amplification (LAMP) from tissue samples at defined time points and at any suspicion of relapse during the trial.
Measured: Baseline and Day 28

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

27 Dec 202222 Dec 2025

First posted25 Oct 2022
Study start27 Dec 2022
Primary completion15 Apr 2025
Study completion22 Dec 2025

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Primary Visceral LeishmaniasisStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Triple blind
Who is masked: Participant, Investigator, Outcomes assessor
Enrolment: 101 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT05593666
Sponsor's protocol ID: DNDi-LXE408-01-VL
Other IDs: CLXE408A12201R
Status last verified: Apr 2026
First submitted: 20 Oct 2022
Last updated: 30 Apr 2026
Results posted: No
Data monitoring committee: Yes
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: Undecided

Study at a glance

From the registry record, last updated by the sponsor on 30 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase II, Multicentre, Randomized, Two-arm Blinded Study to Assess the Efficacy and Safety of Two LXE408 Regimens for Treatment of Patients With Primary Visceral Leishmaniasis
ClinicalTrials.gov ID: NCT05593666
Acronym / sponsor ID: DNDi-LXE408-01-VL
Conditions: Primary Visceral Leishmaniasis
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 101 participants (actual)
Lead sponsor: Drugs for Neglected DiseasesUniversity, hospital or other
Collaborators: Novartis Pharmaceuticals
Study start: 27 Dec 2022 (actual)
Primary completion: 15 Apr 2025 (actual)
Study completion: 22 Dec 2025 (actual)
First posted: 25 Oct 2022 (actual)
Results: Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

This is a phase II, multicentre, randomized, two-arm blinded study with an open label calibrator arm in adults and adolescents (≥12 years) with confirmed primary VL.

Detailed description

This study is run by DNDi with Novartis as co-development partner

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Triple blind (participant, investigator, outcomes assessor)
Number of arms: 3

Arm	Type	Receives
LXE408 short regimen	Experimental	Drug: LXE408, Other: PlaceboLXE408 once daily for seven days (followed by 7 days of placebo).
LXE408 long regimen	Experimental	Drug: LXE408LXE408 once daily for 14 days
Standard of care	Active comparator	Drug: AmBisome®AmBisome® 10 mg/kg IV single dose (SDA)

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (4 listed)

Male and female patients ≥ 18 years (at the time of the screening visit) who are able to comply with the study protocol. Following a favourable interim analysis result, patients ≥12 <18 years will also be enrolled in the trial
Patients for whom written informed consent has been obtained (if aged 18 years and over) or signed by parent(s) or legal guardian for patients under 18 years of age. In the case of minors, assent from the child also needs to be obtained
Primary symptomatic VL (defined as typical parameters including, but not limited to, fever for > 2 weeks, weight loss, and splenomegaly)
Visualization of Leishmania amastigotes by microscopy in tissue samples (spleen or bone marrow)

Key exclusion criteria (12 listed)

Clinical signs of severe VL (jaundice, spontaneous bleeding, edema, ascites, coma, organ failure)
Laboratory abnormalities including ALT/SGPT > 3 times ULN, total bilirubin > 1.5 times ULN, creatinine >1.5 times ULN, amylase or lipase > 1.5 times ULN, haemoglobin < 6 g/dL or other clinically significant abnormal laboratory parameters which, in the opinion of the investigator, may indicate severe VL
Patients with history of previous leishmaniasis and confirmed relapse
Patients with para-kala-azar dermal leishmaniasis
Patients with severe malnutrition (for children ≥12-<18 years: BMI-for-age WHO reference curves by sex, z score < -3; for adults ≥18 years: BMI < 16)

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
LXE408Film-coated tablets	Drug	LXE408 long regimen, LXE408 short regimen
PlaceboPlacebo film-coated tablets	Other	LXE408 short regimen
AmBisome®Sterile lyophilised powder in a 15 mL sterile clear glass vial	Drug	Standard of care

Primary outcome

Proportion of patients with initial cure at Day 28 for LXE408
Time frame: Day 28
Initial cure defined as clinical improvement of Visceral leishmaniasis (VL), absence of parasites in the spleen or bone marrow (microscopy), and no rescue therapy on or before Day 28.

14 secondary outcomes

Proportion of patients with initial cure at Day 28 for AmBisome®
Time frame: Day 28
Proportion of patients with definitive cure at Day 180 for LXE408 and AmBisome®
Time frame: Day 180
Mortality
Time frame: Days 28 and 180
Cmax for LXE408
Time frame: Days 1 and 7
Tmax for LXE408
Time frame: Days 1 and 7
AUCtau for LXE408
Time frame: Days 1 and 7
CLss/F for LXE408
Time frame: Days 1 and 7
Cmax for Amphotericin B
Time frame: Days 1 and 7
AUC0-24h for Amphotericin B
Time frame: Day 1
AUC0-infinity for Amphotericin B
Time frame: Day 1
Blood parasite clearance
Time frame: Baseline and Days 1, 3, 5, 7, 10, 14, 28 and 56
Proportion of patients with a positive loop-mediated isothermal amplification (LAMP) from blood samples
Time frame: Baseline and Days 28 and 56
Tissue parasite loads
Time frame: Baseline and Day 28
Proportion of patients with a positive loop-mediated isothermal amplification (LAMP) from tissue samples
Time frame: Baseline and Day 28

Results and safety

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 30 Apr 2026.
Individual participant dataSharing planned: Undecided

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

The record lists no contact. Use the official record to reach the sponsor.

2 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing LXE408 and AmBisome® in people with Primary Visceral Leishmaniasis, run by Drugs for Neglected Diseases at 2 sites in India.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 30 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in India. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Proportion of patients with initial cure at Day 28 for LXE408 (time frame: Day 28). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05593666.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Apr 2026.