Trial Profile
An open-label single-centre pilot study to evaluate the safety and tolerability of pentosan polysulfate sodium in subjects with mucopolysaccharidosis type I (MPS I)
Status:
Recruiting
Phase of Trial:
Phase I/II
Latest Information Update: 19 Aug 2022
Price :
$35
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At a glance
- Drugs Pentosan polysulfate (Primary)
- Indications Mucopolysaccharidosis I; Pain
- Focus Adverse reactions
- Sponsors Paradigm Biopharma
- 12 Aug 2022 According to a Paradigm Biopharma media release, four patients with this rare disease have been enrolled, and three have completed the 48-week treatment, with a 6-month treatment extension available.
- 12 Aug 2022 According to a Paradigm Biopharma media release, the data from the clinical trial participants treated to date will be presented at ICLD 2023.
- 12 Aug 2022 According to a Paradigm Biopharma media release, interim results were presented at the 14th International Congress of Inborn Errors of Metabolism in Sydney (2021)