Phase 4 trial of miltefosine for the treatment of Indian visceral leishmaniasis

J Infect Dis. 2007 Aug 15;196(4):591-8. doi: 10.1086/519690. Epub 2007 Jun 29.


Background: Visceral leishmaniasis (VL) is a major public health problem in Bihar, accounting for 90% of all cases in India. Development of high levels of resistance to various existing drugs necessitated the search for alternative orally administered drugs. Hospital-based studies have shown that oral miltefosine is a highly effective treatment for VL both in adults and in children.

Methods: An open, single-arm trial was designed to investigate the feasibility of treatment of VL patients with miltefosine in field conditions in 13 centers in Bihar.

Results: The phase 4 study was conducted among 1132 adult and pediatric VL patients. Compliance was good, with 1084 (95.5%) patients completing the full 28-day treatment course. Nine hundred and seventy-one (85.8%) patients returned for the final cure assessment at 6 months after treatment. The final cure rate was 82% by intention to treat analysis and 95% by per protocol analysis (similar to the 94% cure rate in hospitalized patients). Treatment-related adverse events of common toxicity criteria grade 3 occurred in ~3% of patients, including gastrointestinal toxicity and rise in aspertate amino transferase, alanine amino transferase, or serum creatinine levels, similar to previous clinical experience.

Conclusion: This study supports the use of miltefosine in an outpatient setting in an area where VL is endemic.

Publication types

  • Clinical Trial
  • Clinical Trial, Phase IV
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Administration, Oral
  • Adolescent
  • Adult
  • Aged
  • Alanine Transaminase / blood
  • Antiprotozoal Agents / administration & dosage
  • Antiprotozoal Agents / adverse effects
  • Antiprotozoal Agents / therapeutic use*
  • Aspartate Aminotransferases / blood
  • Capsules / administration & dosage
  • Chemical and Drug Induced Liver Injury
  • Child
  • Child, Preschool
  • Creatinine / blood
  • Drug Administration Schedule
  • Female
  • Gastrointestinal Diseases / chemically induced
  • Humans
  • India
  • Leishmaniasis, Visceral / drug therapy*
  • Liver Diseases / blood
  • Male
  • Middle Aged
  • Patient Compliance
  • Phosphorylcholine / administration & dosage
  • Phosphorylcholine / adverse effects
  • Phosphorylcholine / analogs & derivatives*
  • Phosphorylcholine / therapeutic use
  • Treatment Outcome


  • Antiprotozoal Agents
  • Capsules
  • Phosphorylcholine
  • miltefosine
  • Creatinine
  • Aspartate Aminotransferases
  • Alanine Transaminase