Reviewing and Updating the Statistical Analysis Plan and Creating Mock Shells as Part of the Phastar CARES Scheme

Phastar is providing pro-bono support to Medicines Development for Global Health (MDGH) as part of its Phastar CARES scheme. Phastar’s biometrics experts supported the statistical analysis plan (SAP) updates for two related studies on the treatment of onchocerciasis.

Study details

MDGH, a not for profit biopharmaceutical organization developing medicines for neglected diseases primarily affecting people living in poverty, is running two related studies examining the safety and efficacy of moxidectin as a treatment for onchocerciasis (also known as river blindness).

The first is a randomized, double blind, parallel trial in the Democratic Republic of Congo (DRC) to compare safety and efficacy of annual and biannual doses of moxidectin and ivermectin in patients with confirmed onchocerciasis (324 participants enrolled with recruitment now complete).

The second is a randomized, double blind, parallel trial in the Democratic Republic of the Congo and Côte d'Ivoire to assess the safety of single dose moxidectin versus ivermectin in individuals living in onchocerciasis endemic areas, this includes participants from 4 years and older (>12,000 participants).

The overarching goal of the clinical trial program is to achieve the inclusion of moxidectin in the WHO guidelines and essential medicines list for treatment of onchocerciasis, allowing mass community treatment in areas where onchocerciasis is endemic.

Updating the SAP

Phastar initially provided pro bono support to review and update the SAP and create mock shells for study one.

However, study two also required a substantial review of the SAP and associated documents for data management when a second site was added and the study was amended to include children aged down to four years.

Phastar’s biostatistics experts drafted, and reviewed the SAP against the protocol’s primary and secondary objectives and ensured the planned outputs of Tables Figures and Listings met the stated objectives of the protocol and MDGH’s objectives. Phastar’s biostatistics experts are continuing work on the second study.

Why Phastar CARES about MDGH

Phastar CARES is a pro bono scheme allowing charities to access Phastar’s biometrics experts at no cost.

MDGH is a not-for-profit, independent biopharmaceutical organization, developing  and delivering new and improved medicines for diseases that disproportionately affect people in low- and middle-income countries. It is the first not-for-profit organization to independently achieve US FDA approval of a new medicine and moxidectin is the first new medicine registered for onchocerciasis in over 20 years. Developing and delivering new medicines is costly and MDGH is constantly looking for innovative ways to support the work it does, the Phastar CARES scheme is helping MDGH to deliver upon its mission to address health inequity.

Onchocerciasis

More than 100 million people live in areas of Africa endemic for onchocerciasis, also known as river blindness.

Onchocerciasis is a serious, debilitating and stigmatizing neglected infectious disease. It is currently controlled through annual or biannual preventive community directed treatment.

Moxidectin has been shown to be superior to the current treatment in Phase 2 and Phase 3 efficacy clinical trials, with both medicines having similar safety profiles.

The impact of moxidectin has also been extensively studied in mathematical models and the data suggests annual or biannual administration of moxidectin as an alternative to current standard of care could significantly accelerate progress towards onchocerciasis elimination when compared to current treatment.

In 2018, MDGH received United States Food and Drug Administration approval for moxidectin as a single 8 milligram dose for treatment of river blindness in people 12 years of age and older.

The subsequent study is an important step in progressing moxidectin for onchocerciasis and will provide a rich dataset and an opportunity for exploration and supplemental analyses.

The Phase IIIb clinical studies are funded as part of the MoxiMultiDoseMod project.  This project is part of the EDCTP2 Programme supported by the European Union.

Find out more about Phastar’s pro-bono work at: https://phastar.com/cares/pro-bono