We're building a better ClinicalTrials.gov. Check it out and tell us what you think!
Try the New Site
We're building a modernized ClinicalTrials.gov! Visit Beta.ClinicalTrials.gov to try the new functionality.
ClinicalTrials.gov Menu

Safety and Pharmacodynamic Study of an Oral Iron Chelator Given for 6 Months to Patients With Iron Overload

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01186419
Recruitment Status : Completed
First Posted : August 23, 2010
Results First Posted : June 3, 2015
Last Update Posted : June 10, 2021
Information provided by (Responsible Party):
Takeda ( Shire )

Brief Summary:
The purpose of this research study is to evaluate the safety of two doses of FBS0701, a new oral iron chelator, and its effectiveness in clearing iron from the liver. FBS0701 is a medication taken by mouth that causes the body to get rid of iron. Iron chelators are used in patients with β-thalassemia and other forms of anemia who experience iron overload - iron increases in the body as a result of regularly required blood transfusions. Patients who qualify will be randomized to receive one of two doses of FBS0701 for up to 24 weeks (6 months) with a total study duration of up to 33 weeks. These patients will be eligible to participate in a dosing extension for up to 72 weeks. The maximum duration of dosing will be up to 96 weeks. The safety of patients will be monitored frequently during the study by physical exams, ECGs, and blood tests. To assess the amount of iron in the liver and heart, each patient must undergo 6 MRI scans during the study. Patients will not need to stay in the hospital for this study but will need to visit the outpatient clinic up to 28 times over the 96 week period. Patients currently taking an iron chelator will be required to stop for a total of up to 26 weeks. The results of this study will help to determine if FBS0701 may be effective as an iron chelator.

Condition or disease Intervention/treatment Phase
Transfusional Iron Overload Beta-thalassemia Drug: SPD602 (FBS0701, SSP-004184) Phase 2

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 51 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2, 24 Week, Randomized, Open Label, Multi-Center Study to Assess the Safety, Tolerability, and Pharmacodynamics of FBS0701 in the Treatment of Chronic Iron Overload Requiring Chelation Therapy, With a 72 Week Dosing Extension
Actual Study Start Date : August 13, 2010
Actual Primary Completion Date : January 8, 2013
Actual Study Completion Date : January 8, 2013

Arm Intervention/treatment
Experimental: SPD602 (16mg) Drug: SPD602 (FBS0701, SSP-004184)
Oral FBS0701 taken one time daily for up to 96 weeks.

Experimental: SPD602 (32mg) Drug: SPD602 (FBS0701, SSP-004184)
Oral FBS0701 taken one time daily for up to 96 weeks.

Primary Outcome Measures :
  1. Change From Baseline in Liver Iron Concentration (LIC) at 96 Weeks [ Time Frame: Baseline and 96 weeks ]
    LIC was determined by R2 Magnetic Resonance Imaging (MRI).

Secondary Outcome Measures :
  1. Maximum Plasma Concentration (Cmax) of SPD602 [ Time Frame: 92 weeks ]
    Cmax is a term that refers to the maximum (or peak) concentration that a drug achieves in the body after the drug has been administered.

  2. Area Under The Steady-state Plasma Concentration-time Curve (AUC) of SPD602 [ Time Frame: 92 weeks ]
    AUC can be used as a measure of drug exposure. It is derived from drug concentration and time so it gives a measure of how much and how long a drug stays in a body.

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Layout table for eligibility information
Ages Eligible for Study:   18 Years to 60 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Transfusional iron overload due to: hereditary anemias such as sickle cell disease, β-thalassemia and Diamond-Blackfan anemia; acquired anemias such as Myelodysplastic Syndrome and other forms of bone marrow failure. Patients must also be transfusion-dependent and require chronic treatment with deferoxamine, deferasirox, and/or deferiprone.
  • Willing to discontinue all existing iron chelation therapies throughout study period.
  • Serum ferritin greater than 500 ng/mL at Screening.
  • Baseline liver iron concentration and cardiac MRI T2* per protocol requirements.
  • Mean of the previous three pre-transfusion hemoglobin concentrations greater than or equal to 7.5 g/dL.
  • Agrees to use an approved method of contraception throughout study period.

Exclusion Criteria:

  • As a result of medical review, physical examination or Screening investigations, the Principal Investigator considers the patient unfit for the study.
  • Non-elective hospitalization within the 30 days prior to Baseline testing. (Patients with sickle cell anemia who are admitted to the hospital for management of sickle crisis pain whose uncomplicated hospital course was four days or less and who, 14 days prior to Baseline testing, have returned to their previous health status are acceptable.)
  • Evidence of clinically relevant oral, cardiovascular, gastrointestinal, hepatic, renal, endocrine, pulmonary, neurologic, psychiatric, immunologic, bone marrow or skin disorder as determined by the Investigator.
  • Evidence of significant renal insufficiency; possible examples include: serum creatinine above the upper limit of normal, proteinuria greater than 2 gm per day or calculated creatinine clearance of less than 60 mL/minute.
  • Cardiac left ventricular ejection fraction outside of protocol requirements.
  • Known sensitivity to magnesium stearate, croscarmellose sodium or FBS0701.
  • Platelet count below 100,000/µL and/or absolute neutrophil count less than 1500/mm3 at Screening and <50% at Baseline testing by MRI
  • Alkaline phosphatase, AST or ALT outside of protocol requirements.
  • Liver Function Tests: ALT >5 times the local upper limit of normal on two occasions in the previous 12 months or ALT at Screening >200 IU/L
  • Use of any investigational agent within the 30 days prior to the Baseline testing.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01186419

Layout table for location information
United States, California
Children's Hospital and Research Center of Oakland
Oakland, California, United States, 94609
United States, Massachusetts
Children's Hospital of Boston
Boston, Massachusetts, United States, 02115
Ospedale Regionale Microcitemie
Cagliari, Italy
Centro della Microcitemia e delle Anemie Congenite
Genoa, Italy
Thalassemia Center San Luigi Hospital
Orbassano, Italy
Siriraj Hospital, Mahidol University
Bangkok, Thailand
Pediatric Hematology, Ege University Hospital
Izmir, Turkey
United Kingdom
University College London Hospital
London, United Kingdom
Whittington Hospital
London, United Kingdom
Sponsors and Collaborators
Layout table for investigator information
Study Director: Study Director Takeda
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Layout table for additonal information
Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT01186419    
Other Study ID Numbers: SPD602-201
2010-019645-25 ( EudraCT Number )
FBS0701-CTP-04 ( Other Identifier: Ferrokin )
First Posted: August 23, 2010    Key Record Dates
Results First Posted: June 3, 2015
Last Update Posted: June 10, 2021
Last Verified: May 2021
Keywords provided by Takeda ( Shire ):
Sickle cell anemia
Transfusional iron overload
Iron overload
Iron chelation
Additional relevant MeSH terms:
Layout table for MeSH terms
Iron Overload
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hematologic Diseases
Genetic Diseases, Inborn
Iron Metabolism Disorders
Metabolic Diseases