Investigational New Drug (IND) Protocol Benznidazole for treatment of Chagas Disease (American trypanosomiasis)

Study ID
STU 022013-066

Cancer Related
No

Healthy Volunteers
No

Study Sites

  • Parkland Health & Hospital System

Contact
Jason Gillman
214-648-8806
jason.gillman@utsouthwestern.edu

Principal Investigator
Jason Gillman

Summary

Benznidazole and nifurtimox are the only drugs with proven efficacy against Trypanosoma cruzi infection (Chagas disease). These drugs have been used clinically for nearly 40 years in Latin america. neither drug is approved in the united States because of the limited market and the lack of profitability. However, the need for available chemotherapy in the united States is increasing, largely because screening of blood donors was implemented early in 2007 and has identified many chronically infected individuals who may benefit from treatment. The Centers for Disease Control and Prevention (CDC) has sponsored an investigational new Drug (inD) application for nifurtimox since 1986. nifurtimox production has been insecure in the past. The drug has gastrointestinal and neurologic adverse effects, interactions with other drugs, a long duration of therapy, and is poorly tolerated by some patients. For these reasons and because most treatment data from randomized clinical trials have been obtained from benznidazole rather than nifurtimox, there is a demand for benznidazole availability in the united States. We propose to provide benznidazole for treatment of Chagas Disease, specifically for patients with acute or congenital infection, reactivated infection, chronic infection in the absence of advanced cardiac disease manifestations, and for prophylaxis when indicated in the setting of proven exposure. The drug will be provided for individual patients on request of the treating physician after consultation with CDC staff. Physicians will be requested to notify CDC of any adverse event following drug administration.

Participant Eligibility

Beznidazole will be made available for treatment of patients 18 year of age and older:
1) having a history consistent with exposure to T. cruzi and having laboratory evidence of T. cruzi infection, including:
a) parasitologic evidence of infection, e.g., microscopic or lolecular detection of T. cruzi in blood or tissues, and/or
b) serologic evidence of infection.
2) not having laboratory evidence of infection but having a documented history of exposure to T. cruzi.

The decision of eligibility and consideration of the risks and benefits of treatment will be made through discussion between the CDC medical officer in the Parasitic Diseases Branch or his/her designee and the treating physician.

Individuals will be required to sign and informed consent form. If unable to sign, the next of kin or legally authorized representative will be required to sign. Persons who are otherwise able to sign who refuse to sign the consent form will be excluded from his program.