Efficacy of anti-sclerostin monoclonal antibody BPS804 in adult patients with hypophosphatasia.

ABSTRACT:

Background

Hypophosphatasia (HPP) is a rare genetic disorder resulting in variable alterations of bone formation and mineralization that are caused by mutations in the ALPL gene, encoding the tissue-nonspecific alkaline phosphatase (ALP) enzyme.

Methods

In this phase IIA open-label, single-center, intra-patient, dose-escalating study, adult patients with HPP received 3 ascending intravenous doses of 5, 10, and 20 mg/kg BPS804, a fully human anti-sclerostin monoclonal antibody, on days 1, 15, and 29, respectively. Patients were followed for 16 weeks after the last dose. We assessed the pharmacodynamics, pharmacokinetics, preliminary efficacy, and safety of BPS804 administrations at specified intervals during treatment and follow-up.

Results

Eight patients (mean age 47.

SUBMITTER: Seefried L 

PROVIDER: S-EPMC5451251 | biostudies-literature | 2017 Jun

REPOSITORIES: biostudies-literature