Niemann-Pick Disease type C1 is a rare, genetic disease causing cholesterol accumulation in cells, leading to dysfunction of liver, lung, spleen and brain and premature death
The multi-center, randomized, double-blind, parallel group trial without a placebo arm randomized 12 patients ranging in age from 2 to 39 years across clinical sites in the UK, Israel and Sweden.
Patients were treated intravenously with Trappsol Cyclo, the company's proprietary formulation of hydroxypropyl beta cyclodextrin, over 8-9 hours for a total of 24 doses in a 48-week treatment period. Three doses of Trappsol Cyclo were evaluated: 1500 mg/kg, 2000 mg/kg, and 2500 mg/kg.
All patients received study drug, and the top line data summarizes the results from all dose levels combined.
All dose groups showed a favorable safety profile, consistent with previously reported data from a companion Phase 1 clinical trial. 15 SAEs were reported in five patients, the company said.
Of these, three were considered by the investigator to be at least possibly related to the study drug: one hearing related event resolved within a month (Grade 2); the two other events were in a pediatric patient related to infiltrated peripheral cannula which resolved quickly, and which did not interfere with study completion.
Nine patients completed the trial and three were withdrawn by treating physicians. One patient was withdrawn at Week 24 due to intercurrent illness, another who had completed the 36 Week assessment was withdrawn due to COVID-19 travel restrictions which prohibited further participation.
The third patient's parents refused to consent for efficacy assessments prior to Week 12, and the patient was withdrawn from the study and not replaced in keeping with the protocol. No patient was withdrawn due to concerns over safety of the drug.
Of the nine patients who completed the study, eight met the first efficacy endpoint. Patients improved in disease-related domains that included the ability to walk, speak, swallow, behavioral features, incontinence, fine motor skills, saccadic eye movements, and cognition.
The 17-domain NPC Severity Scores for all patients between baseline and last available assessment showed overall improvement in 6 patients and worsening in 5.
One patient had only baseline data and was excluded from the analysis. Based on natural history data with no intervention, patients would be expected to decline on average by 1.5 points over this time period.
The three patients who were withdrawn from the study by treating physicians worsened by six and eight points at their last assessment or had a single baseline score.
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