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

Primary Purpose

Transfusional Iron Overload, Beta-thalassemia

Status

Completed

Phase

Phase 1

Locations

International

Study Type

Interventional

Intervention

FBS0701

About this trial

This is an interventional treatment trial for Transfusional Iron Overload focused on measuring Beta-thalassemia, Sickle cell anemia, Transfusional iron overload, Iron overload, Iron chelation

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Children's Hospital of Oakland
Children's Hospital of Boston
Children's Hospital of Philadelphia
Royal Adelaide Hospital
Siriraj Hospital, Mahidol University

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

FBS0701 - 5 escalating doses

Arm Description

5 escalating doses of FBS0701 in 5 cohorts of 4 patients each.

Outcomes

Primary Outcome Measures

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

Steady state plasma level and half-life of FBS0701

Secondary Outcome Measures

Urinary excretion of iron

Full Information

NCT ID

NCT01039636

First Posted

December 23, 2009

Last Updated

February 8, 2012

Sponsor

FerroKin BioSciences, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT01039636

Brief Title

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

Official 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

Study Type

Interventional

2. Study Status

Record Verification Date

January 2011

Overall Recruitment Status

Completed

Study Start Date

November 2009 (undefined)

Primary Completion Date

December 2010 (Actual)

Study Completion Date

undefined (undefined)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

FerroKin BioSciences, Inc.

4. Oversight

Data Monitoring Committee

5. Study Description

Brief Summary

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Transfusional Iron Overload, Beta-thalassemia

Keywords

Beta-thalassemia, Sickle cell anemia, Transfusional iron overload, Iron overload, Iron chelation

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

20 (Actual)

8. Arms, Groups, and Interventions

Arm Title

FBS0701 - 5 escalating doses

Arm Type

Experimental

Arm Description

5 escalating doses of FBS0701 in 5 cohorts of 4 patients each.

Intervention Type

Drug

Intervention Name(s)

FBS0701

Intervention Description

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 Outcome Measure Information:

Title

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

Time Frame

35 days (7 days treatment and 28 days follow up)

Title

Steady state plasma level and half-life of FBS0701

Time Frame

7 days of dosing

Secondary Outcome Measure Information:

Title

Urinary excretion of iron

Time Frame

7 days of dosing

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Ellis Neufeld, MD

Organizational Affiliation

Boston Children's Hospital

Official's Role

Principal Investigator

Facility Information:

Facility Name

Children's Hospital of Oakland

City

Oakland

State/Province

California

ZIP/Postal Code

94609

Country

United States

Facility Name

Children's Hospital of Boston

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02115

Country

United States

Facility Name

Children's Hospital of Philadelphia

City

Philadelphia

State/Province

Pennsylvania

ZIP/Postal Code

19104

Country

United States

Facility Name

Royal Adelaide Hospital

City

Adelaide

Country

Australia

Facility Name

Siriraj Hospital, Mahidol University

City

Bangkok

ZIP/Postal Code

10700

Country

Thailand

12. IPD Sharing Statement

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

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