A Phase 2a Study To Evaluate The Pharmacokinetics, Safety, Efficacy, Tolerability, And Pharmacodynamics of Sotatercept (ACE-011) for the Correction of Anemia in Subjects With End-stage Renal Disease on Hemodialysis.

Celgene50 enrolled

Overview

This is the first study in hemodialysis subjects with anemia to evaluate the pharmacokinetics, safety, efficacy, tolerability, and pharmacodynamics of sotatercept (ACE-011)

Part 1: Approximately 8 subjects will be randomized to receive either a single 0.1 mg/kg subcutaneous dose of sotatercept or matching placebo in a 3:1 ratio Part 2: Approximately 8 subjects will be randomized to each of the 3 sequential dose groups (0.3mg/kg or 0.5mg/kg or 0.7 mg/kg) with a 3:1 ratio of sotatercept or placebo (6 subjects in the sotatercept arm and 2 in the placebo arm). A total of 24-36 subjects may be randomized in the 3 dose groups.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

SINGLE

Enrollment

Conditions

Anemia

Interventions

SotaterceptBIOLOGICAL

Part 1: Sotatercept single dose 0.1mg/kg subcutaneous Part 2: Sotatercept starting dose groups of 0.3mg/kg, 0.5mg/kg or 0.7 mg/kg in a sequential design, dosed subcutaneously every 28 days for up to 8 doses

PlaceboBIOLOGICAL

Placebo

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Males or females ≥18 years of age. * Subjects on hemodialysis for at least 12 weeks before screening * Subjects on a stable dose of Erythrocyte Stimulating Agents product to maintain Hemoglobin (Hb) for at least 6 weeks prior to screening. * 3 consecutive pre-dialysis Hb concentrations with a mean ≥10 to ≤ 12 g/dL (≥100 to ≤120 g/L) at screening and one pre-dialysis Hb concentration ≥8 to \< 10 g/dL (≥ 80 to \< 100 g/L) before randomization. * Adequate iron status defined as serum transferrin saturation ≥ 20% before randomization. Exclusion Criteria: * Non renal causes of anemia. * Subjects on peritoneal dialysis. * Systemic hematological disease * High sensitivity C-reactive protein \>50mg/L at screening. * Alanine transaminase (ALT) or aspartate transaminase (AST) laboratory values \> 2 times the upper limit of normal (ULN) at screening. * Uncontrolled diabetes mellitus (HbA1c \> 9) at screening. * Uncontrolled hypertension. * Red Blood Count (RBC) transfusions within 8 weeks prior to screening. * Active serious infection or history of recurrent serious infection likely to recur during the study * History of severe allergic or anaphylactic reactions or hypersensitivity to recombinant proteins or excipients in the investigational product or to the iron products needed to normalize iron levels for subjects. * Subjects that received treatment with another investigational drug or device within 28 days prior to Day 1 * Pregnant or lactating females.

Locations (24)

North American Research Institute

Azusa, California, United States

Outcomes

Primary Outcomes

Observed Maximum Concentration (Cmax)

Cmax is a pharmacokinetic parameter defined as the observed maximum concentration of the study drug in the serum and/or blood. Cmax will be estimated from the sotatercept concentration versus time data using noncompartmental method.