Enrollment Begins for Phase 3 Niemann-Pick Disease Type C1 Trial

According to a story from MarketWatch, the biotechnology company Cyclo Therapeutics announced that is has proceeded with the enrollment for its phase 3 clinical trial. This trial will evaluate an original formulation of hydroxypropyl beta cyclodextrin called Trappsol® Cyclo™ as a treatment for type 1 Niemann-Pick disease type C (NPC1). This therapy is delivered intravenously. Cyclo is focused on developing life-changing, innovative therapies.

About Niemann-Pick Disease Type C

Niemann-Pick disease type C is a form of lysosomal storage disease which is characterized by a deficiency, not in an enzyme, but most typically in a type of transporter protein that prevents water-soluble molecules from moving within a cell. It is caused by mutations of either the NPC1 or NPC2 gene. There is a broad disparity in the severity and presentation of symptoms, making symptoms an unreliable method for diagnosis. They may appear in childhood or as late as a patient’s sixth decade of life. Such symptoms include spleen and/or liver enlargement, jaundice, severe depression, ataxia, epilepsy, difficulty speaking and swallowing, dystonia, poor muscle tone, bipolar disorder, microcephaly, progressive loss of hearing, progressive dementia, and psychosis. Most treatment is supportive, but there are some medicines that can delay disease progression and prolong life. Lifespan is connected to the onset of symptoms, with those with the earliest symptoms usually dying sooner. To learn more about this illness, click here.

About The Trial

With limited options available for people that are living with this illness, the beginning of enrollment for a late stage trial is an exciting moment. This trial will specifically evaluate a 2000 mg/kg dose of Trappsol Cyclo alongside standard of care (SOC) versus a placebo infusion and SOC. This study is hoping to include a total of 93 patients of varying ages (both children and adults). This clinical trial is expected to last a total of 96 weeks, with a 48 week interim analysis.

In the US and nations that are following US Food and Drug Administration (FDA) guidelines, a 4-Domain modified Niemann-Pick Disease Type C Severity Scale will be used to evaluate the efficacy of the treatment. In the EU and nations following EMA guidelines, a 5-Domain Niemann-Pick Disease Type C Severity Scale will be used.

Trappsol Cyclo has earned Orphan Drug designation in the US and EU as well as Rare Pediatric Disease and Fast Track designations in the US.

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