Praziquantel
| Evidence Level: L5 | Predicted Indications: 10 |
Table of Contents
Praziquantel: From Schistosomiasis to Uterine Corpus Epithelioid Leiomyosarcoma
One-Sentence Summary
Praziquantel is an anthelmintic (anti-worm) drug, best known for treating schistosomiasis. The TxGNN model ranks uterine corpus epithelioid leiomyosarcoma as its top predicted new use, but this is a graph-based prediction only, with 0 clinical trials and 0 publications behind it. Among the top 10 predictions, the best-supported is Plasmodium falciparum malaria (5 related trials, 20 publications, but mostly indirect evidence).
Quick Overview
| Item | Content |
|---|---|
| Original Indication | Schistosomiasis and other fluke infections (general pharmacological knowledge; the SAHPRA registry text supplied is blank) |
| Predicted New Indication | Uterine corpus epithelioid leiomyosarcoma |
| TxGNN Prediction Score | 97.28% |
| Evidence Level | L5 (model prediction only) |
| South Africa Market Status | Marketed |
| Number of SAHPRA Registrations | 2 |
| Recommended Decision | Hold |
Why is This Prediction Reasonable?
Currently, detailed mechanism of action data is not available in the Evidence Pack. Praziquantel is known to act on calcium channels and the outer surface (tegument) of schistosomes. Its efficacy in helminth infections is established.
There is no evident link between this action and sarcoma biology. Five of the top 10 predictions are sarcomas: leiomyosarcoma at various sites, retroperitoneal sarcoma, small intestinal sarcoma and dermatofibrosarcoma protuberans. This cluster most likely reflects similarity in the knowledge-graph neighbourhood rather than pharmacology. The high score therefore should not be read as a signal of efficacy.
Clinical Trial Evidence
Currently no related clinical trials registered for uterine corpus epithelioid leiomyosarcoma, or for any of the other sarcoma predictions.
For reference, the best-supported prediction (rank 2, P. falciparum malaria) has these registered trials. None tests praziquantel as an antimalarial.
| Trial Number | Phase | Status | Enrollment | Key Findings |
|---|---|---|---|---|
| NCT02769013 | N/A | Completed | 380 | Effect of neglected tropical diseases on P. falciparum transmission in a co-endemic area |
| NCT00347113 | N/A | Completed | 620 | Effect of schistosome and soil-transmitted helminth infections, and their treatment, on malaria in Tanzanian children |
| NCT03640403 | Phase 3 | Completed | 1555 | Malaria preventive treatment (DHA-piperaquine vs artesunate-amodiaquine) in Tanzanian schoolchildren |
| NCT01722539 | Phase 3 | Completed | 616 | Sulfadoxine-pyrimethamine ± piperaquine preventive treatment in Congolese schoolchildren |
| NCT03893097 | Phase 3 | Completed | 726 | Artesunate-mefloquine vs standard praziquantel for schistosomiasis (the reverse direction: an antimalarial repurposed for schistosomiasis) |
Literature Evidence
Currently no related literature available for uterine corpus epithelioid leiomyosarcoma.
For reference, the most relevant publications for the malaria prediction (rank 2) are listed below.
| PMID | Year | Type | Journal | Key Findings |
|---|---|---|---|---|
| 41159886 | 2026 | Randomised trial (double-blind, placebo-controlled) | J Infect Dis | Praziquantel for P. falciparum infection in asymptomatic Gabonese adults; the supplied abstract is truncated and the results are not available |
| 10531774 | 1998 | Short report | JPMA | 10 patients given praziquantel 30 mg/kg/day; 8 reported cured, 2 with severe disease needed standard antimalarials (uncontrolled) |
| 37957702 | 2023 | RCT | Malar J | Feasibility and safety of combining helminth mass drug administration with seasonal malaria chemoprevention in Senegalese children |
| 25887977 | 2015 | RCT | BMC Infect Dis | Impact of anthelmintic treatment on malaria infection and anaemia in Tanzanian children |
| 21696629 | 2011 | Interventional study | BMC Int Health Hum Rights | Integrated school-based de-worming and malaria treatment in Zimbabwe, 33-month follow-up |
| 37446846 | 2023 | Preclinical | Molecules | Praziquantel analogues and hybrids designed as potential antimalarial and antischistosomal agents |
| 38265982 | 2024 | Scoping review | PLoS Negl Trop Dis | Efforts to integrate malaria and schistosomiasis control programmes |
| 25210876 | 2014 | Cohort | PLoS Negl Trop Dis | S. haematobium co-infection associated with protection from febrile malaria in Mali |
| 17568947 | 2007 | Cohort | Mem Inst Oswaldo Cruz | Anti-malaria antibody responses in children exposed to both P. falciparum and S. haematobium |
| 1545596 | 1992 | Review | Mayo Clin Proc | Overview of antiparasitic agents |
Two other predictions have literature only, with no trials:
- Fascioliasis (rank 8): 18 publications, mostly reviews and case reports. Praziquantel has been tried, but triclabendazole is the established treatment and the older literature does not favour praziquantel.
- Gnathostomiasis (rank 7): 3 publications, all reviews or case reports. One case describes a worm emerging after praziquantel, which is an observation and not evidence of efficacy.
South Africa Market Information
| Registration Number | Product Name | Dosage Form | Approved Indication |
|---|---|---|---|
| Reg. No. P/12/23 | Biltricide | Tablet | Not stated in the registry data supplied |
| Reg. No. 56/12/1162 | Quantimak 600 | Fct (as listed in the registry) | Not stated in the registry data supplied |
Both products are oral solid dosage forms.
Safety Considerations
Please refer to the SAHPRA-approved Professional Information (PI) for safety information. Report adverse drug reactions to SAHPRA.
No drug-interaction records were found in the data supplied.
Conclusion and Next Steps
Decision: Hold
Rationale: The top-ranked prediction (uterine corpus epithelioid leiomyosarcoma) is L5 evidence: a model score with no trials, no literature and no plausible mechanistic link. The same holds for the other sarcoma predictions.
To proceed, the following is needed:
- For malaria (the only prediction with a direct clinical signal), read PMID 41159886 in full. If it confirms a controlled efficacy result, the evidence level could rise from L4 to L3 or L2 and this candidate could be re-evaluated as a research question.
- Obtain the SAHPRA package insert to complete the warnings, contraindications and approved-indication fields, which currently block safety screening.
- Obtain mechanism of action data (for example from DrugBank) to allow proper mechanistic-link analysis.
- Do not advance the sarcoma, fascioliasis or gnathostomiasis predictions without controlled data.
This report is for research reference only and does not constitute medical advice. Repurposing candidates require clinical validation before any clinical use.
Disclaimer
This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.