Safety and tolerability of intravenous liposomal GM1 in patients with Parkinson disease: A single-center open-label clinical phase I trial (NEON trial)
Abstract
Background: Parkinson disease (PD) is a chronic progressive neurodegenerative disorder leading to motor and non-motor impairment often resulting in severe loss of quality of life. There are symptomatic treatments without effect on the progression of PD. A disease-modifying treatment that could ideally stop the neurodegenerative process is direly needed. Monosialotetrahexosylganglioside (GM1) is a promising molecule with neuroprotective effects in preclinical models of PD and has yielded encouraging results in patients with PD in a randomized placebo-controlled trial. Talineuren (TLN) is a liposomal formulation of GM1 that has been shown to cross the blood-brain barrier in animals. We assessed the safety and pharmacokinetics of TLN in patients with PD. Methods and Findings We prospectively enrolled 12 patients with PD into a single-center, open-label phase I trial to assess the safety and tolerability of weekly infusions with Talineuren. The maximum suitable dose of Talineuren was determined by dose escalation in three patients. Subsequently, these and nine further patients received weekly infusions at the maximum suitable dose of Talineuren over two months (1 patient stopped prematurely). All adverse events were continuously assessed as the primary objective and coded according to the Medical Dictionary for Regulatory Activities (MedDRA(R)). Clinical manifestations of PD were assessed as secondary outcomes using the Movement Disorders Society Unified Parkinsons Disease…
How strong is the evidence?
Evidence Hub reads this as a randomized trial, done in people. Its evidence level is 3 of 5 (Moderate · trial / cohort / secondary analysis). Trials without a large randomized design, cohorts and secondary analyses give moderate evidence. Reported size: 12 participants or records.
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