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NCT Number: NCT01039636

Safety and Pharmacokinetic Study of Escalating Multiple Doses of an Iron Chelator in Patients With Iron Overload

The purpose of this research study is to study the safety of increasing doses of FBS0701, and to see how quickly the study medication is absorbed and how quickly it disappears from the bloodstream. FBS0701 is a new, oral iron chelator - a medication taken by mouth that increases the body's elimination of iron. Iron chelators are used in patients who develop iron overload from their transfusions. Four increasing doses of FBS0701 will be tested during this study. The study will start with the lowest dose given to 4 patients (3 mg/kg/day. The next group of 4 patients will receive the next high dose (8mg/kg/day only after the results of the first 4 patients are examined and it is determined safe to continue. Participating patients will take the study medication for 7 days and be followed for 28 days after their last dose to determine if they have any reactions to the study medication - therefore a total of 35 days on study. Patients will need to give up to 17 blood samples over the screening period and first 15 days of the study (a total of about 9 tablespoons). Patients will not need to stay overnight in the clinic but will need to visit the clinic 10 times for screening and on-study visits over the 35 days. Patients currently taking an iron chelator will need to stop that treatment for up to 22 days (up to 5 days before they start the study and for 15 days during the study). The results of this study will be helpful in determining the safety of the drug and the best doses of FBS0701 to be used in the next study which will assess the effectiveness of this new iron chelator.

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Key information

Conditions

Transfusional Iron Overload Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Beta-Thalassemia Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Iron Metabolism Disorders Iron Overload Metabolic Diseases Nutritional and Metabolic Diseases Thalassemia

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Royal Adelaide Hospital, Adelaide, Australia

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • At least 18 years old
  • Transfusion dependent (at least 8 transfusions per year) with transfusional iron overload needing treatment with deperoxamine, deferasirox or deperiprone.
  • Willing to discontinue current iron chelation therapy for 2-5 days prior to enrollment and for a total of up to 3 weeks
  • Willing to fast after midnight prior to each dose
  • Serum ferritin greater than 400ng/ml
  • Liver iron concentration greater than or equal to 1.5 mg iron. (determined by MRI or SQUID within 12 months prior to enrollment)
  • Cardiac iron greater than or equal to 12 milliseconds (determined by MRI t2* within 18 months prior to enrollment)
  • Mean of last 3 pre-transfusion hemoglobin values must be greater than or equal to 7.5 g/dl
  • Agrees to use approved contraception from Screening and until 30 days after last administration of study drug. Abstinence OK.

Exclusion criteria

  • Principal Investigator considers patient unfit for study after conducting a medical review, physical examination and other screening assessments.
  • Non-elective hospitalization with past 30 days
  • Evidence of clinically relevant oral, cardiovascular, gastrointestinal, hepatic, renal, endocrine, pulmonary, neurologic, psychiatric or skin disorder.
  • Evidence of significant renal insufficiency; serum creatine above upper limit of normal or proteinuria greater than 2 gm per day or calculated creatinine clearance of less than or equal to 60ml/min
  • Platelet count below 150,000,000/ml and/or absolute neutrophil count less then 1500/mm3
  • Alkaline phosphatase or AST greater than 5 times the upper limit of normal or ALT greater than 4 times the upper limit of normal
  • Female patients who are pregnant or lactating
  • Use of any investigational agent within the last 30 days

Treatment and study plan

FBS0701

Drug

Oral FBS0701 for 7 days at one of the following doses: 3 mg/kg/day or 8 mg/kg/day or 16 mg/kg/day or 32 mg/kg/day or 40 mg/kg/day

Primary outcomes

  1. Safety and tolerability by AEs, Physical Exam, Clinical Laboratory assessments

    Time frame: 35 days (7 days treatment and 28 days follow up)

  2. Steady state plasma level and half-life of FBS0701

    Time frame: 7 days of dosing

Secondary outcomes

  1. Urinary excretion of iron

    Time frame: 7 days of dosing

Sponsors and collaborators

Lead sponsor

FerroKin BioSciences, Inc.

Industry

Registry information

Official study title

A PHASE 1b OPEN LABEL, MULTI-CENTER, ESCALATING MULTIPLE DOSE STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND PHARMACODYNAMICS OF FBS0701 IN PATIENTS WITH TRANSFUSIONAL IRON OVERLOAD

Important dates

Study start
2009
Primary completion
2010
First posted
Dec 25, 2009
Registry last updated
Feb 10, 2012

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

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This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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