Oxfendazole in Mild Parenchymal Brain Cysticercosis

A Double-blind Multicenter, Randomized Controlled Trial of Single and Multiple Dose Regimens of Oxfendazole for Mild (One or Two Lesions) Parenchymal Brain Cysticercosis, With an Open Comparison Group

The goal of this clinical trial is to compare a single and multiple dose regimens of oxfendazole with the standard treatment in patients with mild (one or two lesions) parenchymal brain cysticercosis. The main question it aims to answer is if OXF will enhance clearance of brain parasites and therefore provide greater cysticidal efficacy, with the potential to provide a single-dose therapy for this type of NCC.

The study cohort will also allow us to identify early imaging markers that predict lesion resolution, as well as factors associated with residual calcification or focal gliosis after lesion resolution. This study will also provide additional information on the safety of the study interventions.

Study Overview

• Status: Not yet recruiting
• Conditions: Brain Cysticercosis
• Intervention / Treatment: Drug: Oxfendazole single dose; Drug: Oxfendazole three doses; Drug: albendazole plus praziquantel regime

Detailed Description

This three-arm randomized controlled phase II/III clinical trial will compare the efficacy and safety of a single-dose regimen with 20 mg/kg oxfendazole and a regimen with three similar doses spread over seven days (day 1, day 4 and day 7), with the most effective antiparasitic regimen available, combined albendazole plus praziquantel for ten days in individuals with mild NCC (with one to two lesions).

Participants will receive treatment with oxfendazole (one or three dose regimens) or the standard treatment (albendazole + praziquantel). At day 15 after treatment onset, an MRI will be performed to evaluate early predictors of lesion resolution. MRI at day 90 will serve to evaluate efficacy (lesion resolution) and a day 180 MRI will evaluate sequelae lesions. CT will be performed at the en of the study to confirm persistence of calcified sequelae lesion.

The study will enroll 544 patients with viable or degenerating parenchymal NCC with no more than two lesions, all in a single brain area. Lesions can be one or two adjacent, viable or degenerating NCC lesions. Patients with only calcified lesions will not be included even if they show perilesional contrast enhancement.

• Study Type: Interventional
• Enrollment (Estimated): 544
• Phase: Phase 2; Phase 3

Contacts and Locations

• Study Contact: Name: Hector H Garcia, MD, PhD; Phone Number: +511 3287360; Email: hgarcia@jhsph.edu
• Study Contact Backup: Name: Javier A Bustos, MD, PhD; Phone Number: +511 3284038; Email: javier.bustos.p@upch.pe

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Male or female individuals between 18 and 75 years of age, with suspected viable or degenerating intraparenchymal brain cysticercosis on neuroimaging (CT or MRI) and fulfill the diagnostic criteria for solitary cysticercus granuloma (Rajshekhar and Chandy, 1997)
2. If female of child-bearing potential and men, willing to use an adequate method of contraception*, including implants, injectables, combined oral contraceptives, effective intrauterine devices, sexual abstinence, or vasectomized partner while participating in the study.
3. Patients with normal laboratory values for hemoglobin, platelet counts, total white blood cells, glucose, creatinine, bilirubin, ALT, and AST.
4. Availability to grant informed consent.

Exclusion Criteria:

1. Multiple lesion sites or more than two adjacent lesions.
2. Suspected neurotuberculosis (Rajshekhar's criteria) [66,67]
3. More than two viable brain cysts.
4. Large brain cysts (> 3cm in diameter)
5. Subarachnoid neurocysticercosis or intraventricular
6. Untreated ocular cysticercosis
7. Previous therapy with ABZ (does not include patients who received single-dose 400 mg ABZ for intestinal parasites), or PZQ in the past twelve months.
8. Active pulmonary tuberculosis evidenced by a positive chest X-ray and positive sputum smears.
9. Systemic disease other than NCC that may affect therapy or short-term prognosis, including but not limited to chronic renal failure, hepatic insufficiency, cardiac failure, and steroid-dependent immune diseases.
10. Patients in unstable condition or with severe intracranial hypertension (ICH). Definition of severe ICH for this study would be the presence of headaches, nausea, and vomiting, and papilledema at fundoscopic examination.
11. Pregnancy
12. History of hypersensitivity to ABZ or PZQ
13. Concurrent treatment with cimetidine, ranitidine, or theophylline.
14. Chronic alcohol or drug abuse.
15. Positive to Strongyloides infection
16. History of reported allergy to contrast substances used in MRI.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Intervention 1; OXF 20 mg/kg/d, single dose, orally	Drug: Oxfendazole single dose; Subjects will receive active oxfendazole, 20 mg/kg/day, orally, as a single dose. Oxfendazole placebo will be used at days 4 and 7 to maintain blinding between the two intervention arms.; Other Names: Intervention 1
Experimental: Intervention 2; OXF 20 mg/kg/d, for three days (1, 4 and 7), orally	Drug: Oxfendazole three doses; Subjects will receive active oxfendazole, 20 mg/kg/day, orally, in three days (1, 4 and 7); Other Names: Intervention 2
Active Comparator: Comparison regimen; Combined treatment with albendazole (15 mg/k/d) plus praziquantel (50 mg/k/d), for 10 days, orally.	Drug: albendazole plus praziquantel regime; Subjects will receive a combination of albendazole plus praziquantel, as a standard treatment for brain cysticercosis (neurocysticercosis), orally, for ten days. Albendazole will be given at 15/kg/d and praziquantel at 50/kg/d.; Other Names: Comparison regimen

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Efficacy: completely resolution or persistence only as small lesion remnants.	Proportion of participants whose lesions completely resolved or persist only as small lesion remnants (<20% of the original lesion size).	Three months after treatment onset

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Clinical effectiveness: Seizure relapses.	The frequency of seizure relapses in individuals whose lesions resolved compared to those who had remaining viable or degenerating lesions on month 3 MRI	In the initial 12 months after treatment
Safety: Serious adverse events	Proportion of participants in each group who develop serious or severe adverse events, and the numbers of adverse events per group	In the initial 12 months after treatment

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Early imaging markers predicting lesion resolution.	Imaging characteristics on MRI at day 15 after treatment onset that are associated with successful lesion resolution on MRI.	Month 3
Quality of life in Epilepsy (QOLIE-31) score around antiparasitic treatment in NCC, to assess emotional and psychological effects, and medical and social effects.	Factors associated with reduced quality of life before and after anti-parasitic treatment for mild parenchymal NCC	Before treatment and 12 months after antiparasitic treatment.
Number of baseline seizures associated with seizure relapses.	Number of pre-treatment seizures associated with seizure relapses after initiation of antiparasitic treatment.	During the initial 12 months after antiparasitic treatment.
Antiparasitic treatment type associated with gliosis or residual calcification.	Antiparasitic treatment type (oxfendazole single dose, or oxfendazole three doses, or combined albendazole plus praziquantel treatment) associated with focal gliosis or residual calcification.	Month 6 and month 12
Antiparasitic treatment type associated with seizure relapses.	Antiparasitic treatment type (oxfendazole single dose, or oxfendazole three doses, or combined albendazole plus praziquantel treatment) associated with seizure relapses.	During the initial 12 months after antiparasitic treatment.
Cysticercus size associated with seizure relapses.	Measurement of cysticercus size, in millimeters, to evaluate its association with seizure relapses.	During the initial 12 months after antiparasitic treatment.
Cysticercus size associated with gliosis or residual calcification.	Measurement of cysticercus size, in millimeters, to evaluate its association with gliosis or residual calcification.	Month 6 and month 12

Collaborators and Investigators

• Sponsor: Universidad Peruana Cayetano Heredia
• Collaborators: Oxfendazole Development Group
• Investigators: Principal Investigator: Hector H Garcia, MD, PhD, Universidad Peruana Cayetano Heredia

Study record dates

Study Major Dates

• Study Start (Estimated): December 1, 2026
• Primary Completion (Estimated): March 15, 2030
• Study Completion (Estimated): December 15, 2030

Study Registration Dates

• First Submitted: August 12, 2024
• First Submitted That Met QC Criteria: August 19, 2024
• First Posted (Actual): August 22, 2024

Study Record Updates

• Last Update Posted (Actual): May 1, 2026
• Last Update Submitted That Met QC Criteria: April 27, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Keywords: Cysticercosis; Cysticercosis treatment
• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Infections; Parasitic Diseases; Central Nervous System Infections; Central Nervous System Helminthiasis; Central Nervous System Parasitic Infections; Cestode Infections; Helminthiasis; Taeniasis; Neurocysticercosis; Cysticercosis; Organic Chemicals; Heterocyclic Compounds; Benzimidazoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Acids, Acyclic; Carboxylic Acids; Carbamates; Albendazole; oxfendazole
• Other Study ID Numbers: 211661

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No