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A multicenter, open-label, phase III study of Abcertin in Gaucher disease.

ABSTRACT: BACKGROUND:Gaucher disease (GD) is caused by a deficiency in the lysosomal enzyme glucocerebrosidase. Enzyme replacement therapy (ERT) is recommended for clinical improvement. METHODS:The efficacy and safety of a new imiglucerase, Abcertin, were assessed in 7 Egyptian patients with treatment-naïve type 1 GD. Each patient was administered a biweekly 60?U/kg dose of Abcertin for 6 months. The primary endpoint was the change in hemoglobin concentration. The secondary endpoints were changes from baseline in platelet counts, spleen and liver volumes, biomarker levels, skeletal parameters, and bone mineral density. RESULTS:The hemoglobin concentration increased by a mean of 1.96?±?0.91?g/dL (range 1.11-2.80?g/dL) or 20.6% (P?=?.001). Statistically significant increases in the platelet count and decreases in the spleen volume and biomarker levels were also observed. There were no severe drug-related adverse events. One patient developed anti-imiglucerase antibodies without neutralizing activity. CONCLUSION:Our study results demonstrate the efficacy and safety of Abcertin in patients with type 1 GD. This suggests that Abcertin can be an alternative ERT option for type 1 GD.

SUBMITTER: Lee BH

PROVIDER: S-EPMC5690733 | biostudies-literature | 2017 Nov

REPOSITORIES: biostudies-literature

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A multicenter, open-label, phase III study of Abcertin in Gaucher disease.

Lee Beom Hee BH Abdalla Ahmed Fathy AF Choi Jin-Ho JH Beshlawy Amal El AE Kim Gu-Hwan GH Heo Sun Hee SH Megahed Ahmed Megahed Hassan AMH Elsayed Mona Abdel Latif MAL Barakat Tarik El-Sayed Mohammad TEM Eid Khaled Mohamed Abd El-Azim KMAE El-Tagui Mona Hassan MH Mahmoud Mona Mohamed Hamdy MMH Fateen Ekram E Park June-Young JY Yoo Han-Wook HW

Medicine 20171101 45

<h4>Background</h4>Gaucher disease (GD) is caused by a deficiency in the lysosomal enzyme glucocerebrosidase. Enzyme replacement therapy (ERT) is recommended for clinical improvement.<h4>Methods</h4>The efficacy and safety of a new imiglucerase, Abcertin, were assessed in 7 Egyptian patients with treatment-naïve type 1 GD. Each patient was administered a biweekly 60 U/kg dose of Abcertin for 6 months. The primary endpoint was the change in hemoglobin concentration. The secondary endpoints were c ...[more]

PMID: 29137040

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A multicenter, open-label, phase III study of Abcertin in Gaucher disease.

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A multicenter, open-label, phase III study of Abcertin in Gaucher disease.

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