Plasminogen, human-tvmh for the treatment of children and adults with plasminogen deficiency type 1.
NCT02690714
human-tvmh
hypoplasminogenemia
phase 2/3
plasminogen
plasminogen deficiency type 1
replacement therapy
Journal
Haemophilia : the official journal of the World Federation of Hemophilia
ISSN: 1365-2516
Titre abrégé: Haemophilia
Pays: England
ID NLM: 9442916
Informations de publication
Date de publication:
Nov 2023
Nov 2023
Historique:
revised:
11
08
2023
received:
20
03
2023
accepted:
17
08
2023
medline:
14
11
2023
pubmed:
7
9
2023
entrez:
7
9
2023
Statut:
ppublish
Résumé
An open-label phase 2/3 study of plasminogen, human-tvmh administered intravenously in paediatric and adult subjects with type 1 plasminogen deficiency was conducted. Interim data was previously reported. The final data on 15 subjects who completed the study up to a maximum of 124 weeks are reported here. The primary objectives were to evaluate efficacy of plasminogen replacement therapy on clinically evident or visible lesions during 48 weeks of dosing and to achieve an increase in trough plasminogen activity levels by at least an absolute 10% above baseline during 12 weeks of treatment. The primary efficacy endpoint was achieved, as 100% of subjects (n = 11) with visible and assessable non-visible lesions at baseline demonstrated ≥ 50% improvement after 48 weeks of study drug treatment with plasminogen, human-tvmh. All subjects achieved the targeted ≥ 10% increase in trough plasminogen activity above baseline through Week 12. Plasminogen, human-tvmh at a dose of 6.6 mg/kg administered every 2-5 days for 48 weeks and every 1-7 days for up to 124 weeks was well tolerated. This study provides additional evidence regarding the long-term safety and clinical utility of replacement therapy with human plasminogen for the treatment of children and adults with type 1 plasminogen deficiency. Plasminogen, human-tvmh received marketing approval on June 4, 2021. This trial was registered at www. gov as #NCT02690714.
Substances chimiques
Plasminogen
9001-91-6
Banques de données
ClinicalTrials.gov
['NCT02690714']
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
1556-1564Informations de copyright
© 2023 The Authors. Haemophilia published by John Wiley & Sons Ltd.
Références
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