Trial reportClinical and translational science2021
Dose selection for intracerebroventricular cerliponase alfa in children with CLN2 disease, translation from animal to human in a rare genetic disease.
Trial report in Clinical and translational science, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
7 citing papers in PubMed, 9 citations in OpenAlex.
- Conversion of a ventricular rickham reservoir to a subcutaneous chest port for repeated intraventricular enzyme replacement therapy: technical note and surgical considerations.Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery · 2026Article
- Patient and Family Perspective on Transition from Ventricular Access Device to Chest-Sited Port for Intracerebroventricular Infusion in CLN2 Disease.Children (Basel, Switzerland) · 2026Article
- Variability in the circulation of cerebrospinal fluid: causes and clinical implications for intraventricular drug delivery.Frontiers in drug delivery · 2026Review
- Comparison ofMolecular therapy. Nucleic acids · 2025Article
- Novel surgical approach for intraventricular cerliponase alfa enzyme replacement therapy via central venous access device (CVAD) port in neuronal ceroid lipofuscinosis type 2 (CLN2) disease.Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery · 2025Article
- Neurological glycogen storage diseases and emerging therapeutics.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2024Review
- Haptoglobin Treatment for Aneurysmal Subarachnoid Hemorrhage: Review and Expert Consensus on Clinical Translation.Stroke · 2023Review
Corrections and comments
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Authors and funding
8 authors at 2 institutions in 1 country.
Funding
Abstract
Neuronal ceroid lipofuscinosis type 2 (CLN2 disease) is an ultra-rare pediatric neurodegenerative disorder characterized by deficiency of the lysosomal enzyme tripeptidyl peptidase-1 (TPP1). In the absence of adequate TPP1, lysosomal storage material accumulation occurs in the central nervous system (CNS) accompanied by neurodegeneration and neurological decline that culminates in childhood death. Cerliponase alfa is a recombinant human TPP1 enzyme replacement therapy administered via intracerebroventricular infusion and approved for the treatment of CLN2 disease. Here, we describe two allometric methods, calculated by scaling brain mass across species, that informed the human dose selection and exposure prediction of cerliponase alfa from preclinical studies in monkeys and a dog model of CLN2 disease: (1) scaling of dose using a human-equivalent dose factor; and (2) scaling of compartmental pharmacokinetic (PK) model parameters. Source PK data were obtained from cerebrospinal fluid (CSF) samples from dogs and monkeys, and the human exposure predictions were confirmed with CSF data from the first-in-human clinical study. Nonclinical and clinical data were analyzed using noncompartmental analysis and nonlinear mixed-effect modeling approaches. Both allometric methods produced CSF exposure predictions within twofold of the observed exposure parameters maximum plasma concentration (C
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.