A Phase 2A Trial of FMX-8 Treatment for Anemia in Patients With ESRD on Hemodialysis HD

NCT01873534 | Terminated Phase 2

• 1 other identifier: FX-C-402
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 2

Brief Summary

The trial is an uncontrolled, open-label, parallel group clinical trial. Approximately 10 subjects per dose group in 3 groups will be treated twice weekly for a total of 9 doses, followed by a 4-week observation period. Eligible subjects who have Hgb ≥10.5 g/dL and have stable Hgb levels will start the washout period of one to eight weeks. During the washout period, 30 subjects whose Hgb are \< 10.0 will complete the baseline assessment to confirm their eligibility. Eligible subjects will be randomly assigned to one of the 3 cohorts in a 1:1:1 ratio. Subjects will be admitted on the day of the first dose and stay in the clinic overnight for pharmacokinetic (PK) sampling after the first (day 1) and the last dose (day 29). FMX-8 will be administered as 30 min i.v. infusion. After the 29-day treatment period, the trial subjects will be observed for an additional 28 days to allow safety and immunogenicity assessments.

Conditions

Anemia of Chronic Disease

Keywords

Anemia

Outcome Measures

Primary Outcomes (4)

• The proportion of subjects who achieve an increase in Hgb ≥ 1g/dL from the lowest Hgb concentration post erythropoietin-washout or continuing rise in Hgb concentration for two consecutive weeks

  Weekly for 8 weeks

• Number and Severity of Adverse Events

  8 weeks

• Serum FMX-8 levels

  Serum drug levels (pre-dose, and 25 minutes, 35 minutes, 1, 2, 4, 6, 10, 16 and 24 hrs post-dose) will be used to determine, for each dose, standard pK profiles

  Dosing Days 1 and 29

• Number of Subjects with Positive Serum for Anti-Drug Antibodies

  At 36 and 57 days after first dose of FMX-8

Secondary Outcomes (8)

• Changes in Hgb in each dose group during the treatment and follow-up periods

  Weekly for 8 weeks

• Proportion of subjects that achieve/maintain an absolute Hgb concentration of ≥ 10.0 g/dL for two consecutive weeks

  Weekly for 8 weeks

• Time to beginning of steady increase of Hgb (for two consecutive weeks)

  Weekly for 8 weeks

• Time to Hgb increase ≥1 g/dL

  Weekly for 8 weeks

• Time to full recovery of Hgb to pre- erythropoietin-washout level

  Weekly for 8 weeks

Study Arms (3)

FMX-8 (0.5 mg/kg)

EXPERIMENTAL

0.5 mg/kg FMX-8 IV twice per week for 29 days (9 doses)

Drug: FMX-8

FMX-8 (5 mg/kg)

EXPERIMENTAL

5 mg/kg FMX-8 IV twice per week for 29 days (9 doses)

Drug: FMX-8

FMX-8 (15 mg/kg)

EXPERIMENTAL

15 mg/kg FMX-8 IV twice per week for 29 days (9 doses)

Drug: FMX-8

Interventions

FMX-8 DRUG

FMX-8 is a fusion protein of the human hemojuvelin (HJV) protein.

FMX-8 (0.5 mg/kg); FMX-8 (15 mg/kg); FMX-8 (5 mg/kg)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female patients who are ≥18 years old
• Diagnosed with ESRD and are stable on hemodialysis for more than 3 months
• Maintained stable Hgb for ≥4 weeks prior to screening
• Two consecutive Hgb values ≥10.5 g/dL within 5 weeks of screening
• Body mass index (BMI) between 18 kg/m2 and 42 kg/m2, inclusive, based upon the latest height and weight
• Ferritin levels ≥100 mg/L or Tsat ≥20% or reticulocyte hemoglobin content (CHr) \>25 at screening
• Reasonable clearances on dialysis (KT/V ≥1.0) on two prior determinations within 2.5 months
• Able to provide written informed consent
• Able to understand and follow all trial procedures
• Willing to use contraception as detailed in the protocol

You may not qualify if:

• Hgb remains unchanged without erythropoietin (\<0.5 g/dL decrease during the 8 week maximum erythropoietin-washout period)
• Receipt of iron infusion after the initiation of erythropoietin washout
• Receipt of red blood cell transfusion within four weeks before screening
• Overt gastrointestinal bleeding or other bleeding episode that required transfusion within 2 months prior to screening
• Infection necessitating antibiotic or anti-viral treatment within a month prior to screening
• Requirement for Coumadin (warfarin), Pradaxa or Xarelto
• Hemoglobinopathies such as homozygous sickle-cell disease or thalassemias of all types
• Active hemolysis or chronic hypoxia
• Active malignant diseases (except non-melanoma skin cancer) or life expectancy less than 6 months
• Chronic, uncontrolled or symptomatic inflammatory disease or non-renal cause of anemia such as rheumatoid arthritis, systemic lupus erythematosus, HIV, or systemic acute infection
• On immunosuppressive therapeutics
• Chronic congestive heart failure (New York Heart Association Class III, IV)
• Significant hypertension (≥90 diastolic) based on a sitting diastolic blood pressure at screening
• Kidney transplant within the past year: patients who are off immunosuppressive agents following a failed transplant are eligible for the trial
• End-stage liver disease

Sponsors & Collaborators

• FerruMax Pharmaceuticals, Inc.: lead
• Davita Clinical Research: collaborator

Study Sites (2)

DaVita Arvada Dialysis Center

Arvada, Colorado, 80005, United States

Location

DaVita Minneapolis Dialysis Unit

Minneapolis, Minnesota, 55404, United States

Location

MeSH Terms

Conditions

Anemia

Condition Hierarchy (Ancestors)

Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Leslie Fang, MD, PhD

  FerruMax Pharmaceuticals, Inc.

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 5, 2013
• First Posted: June 10, 2013
• Study Start: June 1, 2013
• Primary Completion: March 1, 2014
• Study Completion: March 1, 2014
• Last Updated: August 11, 2015

Record last verified: 2015-07

Locations

US (2)