Clinical trial NCT02228655: An Exploratory Study to Evaluate FMX-8 to Treat Anemia in CKD
Official title: An Exploratory, Uncontrolled, Open-labeled Trial to Evaluate the Effect of FMX-8 Treatment for Anemia in Patients With Chronic Kidney Disease (CKD), Stage 4 or 5
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: EARLY_PHASE1
Conditions: Anemia in Chronic Kidney Disease
Keywords: Chronic Kidney Disease
CKD
Anemia of Chronic Illness
Lead sponsor: FerruMax Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2014-08-29
Enrollment: 2
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: FMX-8 is a new type of drug being tested for the treatment of anemia in chronic illnesses.


Eligibility criteria: Inclusion Criteria:

* a documented hemoglobin level to be less than 10 g/dL at screening
* diagnoses of CKD 4 or 5
* body mass index (BMI) between 18 kg/m2 and 42 kg/m2, inclusive, based upon the height and weight at screening
* ferritin levels ≥100 ng/ml or Tsat ≥20% at screening
* erythropoietin (EPO) level greater than 8 ng/mL
* able to provide written informed consent
* able to understand and follow all trial procedures
* willing to use contraception as detailed in the protocol

Exclusion Criteria:

* receipt of red blood cell (RBC) 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
* requiring Coumadin (warfarin), Pradaxa®, Eliquis®, 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 except topical corticosteroids or nasal sprays
* 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
* known hypersensitivity to recombinant protein therapies
* female patients who are pregnant or breast feeding
* previous exposure to FMX-8
* previous exposure to Epogen®, Procrit® (erythropoietin) Aranesp® (darbepoietin alpha), Omontys® or Hematide® (peginesatide) anemia treatment
* uncontrolled hyperparathyroidism (PTH \>750) based upon latest PTH determination within the past 4 months
* inability to comply with the trial scheduled visits

Primary outcomes:
measure: Change in hemoglobin concentration
timeFrame: 57 day evaluation period
Secondary outcomes:
Other outcomes:
Locations:
city: Boston
country: United States
facility: Beth Israel Deaconess Medical Center
state: Massachusetts
Study officials:
affiliation: Beth Israel Deaconess Medical Center
name: Stewart Lecker, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Babitt JL, Huang FW, Wrighting DM, Xia Y, Sidis Y, Samad TA, Campagna JA, Chung RT, Schneyer AL, Woolf CJ, Andrews NC, Lin HY. Bone morphogenetic protein signaling by hemojuvelin regulates hepcidin expression. Nat Genet. 2006 May;38(5):531-9. doi: 10.1038/ng1777. Epub 2006 Apr 9.
citation: Babitt JL, Huang FW, Xia Y, Sidis Y, Andrews NC, Lin HY. Modulation of bone morphogenetic protein signaling in vivo regulates systemic iron balance. J Clin Invest. 2007 Jul;117(7):1933-9. doi: 10.1172/JCI31342.
citation: Huang FW, Pinkus JL, Pinkus GS, Fleming MD, Andrews NC. A mouse model of juvenile hemochromatosis. J Clin Invest. 2005 Aug;115(8):2187-91. doi: 10.1172/JCI25049.
citation: KDOQI. KDOQI Clinical Practice Guideline and Clinical Practice Recommendations for anemia in chronic kidney disease: 2007 update of hemoglobin target. Am J Kidney Dis. 2007 Sep;50(3):471-530. doi: 10.1053/j.ajkd.2007.06.008. No abstract available.
citation: Theurl I, Schroll A, Sonnweber T, Nairz M, Theurl M, Willenbacher W, Eller K, Wolf D, Seifert M, Sun CC, Babitt JL, Hong CC, Menhall T, Gearing P, Lin HY, Weiss G. Pharmacologic inhibition of hepcidin expression reverses anemia of chronic inflammation in rats. Blood. 2011 Nov 3;118(18):4977-84. doi: 10.1182/blood-2011-03-345066. Epub 2011 Jul 5.
citation: Weiss G, Goodnough LT. Anemia of chronic disease. N Engl J Med. 2005 Mar 10;352(10):1011-23. doi: 10.1056/NEJMra041809. No abstract available.
citation: Weiner DE. Causes and consequences of chronic kidney disease: implications for managed health care. J Manag Care Pharm. 2007 Apr;13(3 Suppl):S1-9. doi: 10.18553/jmcp.2007.13.s3.1.