Safety, Efficacy and Pharmacokinetics of an Oral Iron Chelator Given for a Year to Pediatric Patients With Iron Overload
This study is currently recruiting participants.
Verified May 2013 by Shire Development LLC
Sponsor:
Shire Development LLC
Information provided by (Responsible Party):
Shire Development LLC
ClinicalTrials.gov Identifier:
NCT01363908
First received: May 30, 2011
Last updated: May 3, 2013
Last verified: May 2013
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Purpose
This is an open-label study to assess the pharmacokinetics, safety, efficacy and tolerability of FBS0701. The study consists of two phases: the pharmacokinetic phase, using a single 16 mg/kg dose of FBS0701; and the chronic dosing phase, during which patients will receive an additional 48 weeks of FBS0701 dosing. Two age groups will be studied: 6-<12, and 12-<18 years old. The study is designed to initially assess the pharmacokinetics and safety of FBS0701 in older children (adolescents, 12-<18 years old) and then if deemed safe, in younger children (6-<12 years old).
| Condition | Intervention | Phase |
|---|---|---|
|
Transfusional Iron Overload Beta-Thalassemia |
Drug: FBS0701 |
Phase 2 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | A Phase 2, Open Label, Multi-Center, Single-Dose Pharmacokinetics, and Multiple Dose Study of the Safety, Efficacy and Tolerability of FBS0701 in a Pediatric Population With Transfusional Iron Overload |
Resource links provided by NLM:
Further study details as provided by Shire Development LLC:
Primary Outcome Measures:
- Single-dose Pharmacokinetics: The concentrations of FBS0701 will be measured in plasma from all patients using a validated assay method. Pharmacokinetic parameters will be tabulated and summarized. [ Time Frame: 8 days ] [ Designated as safety issue: Yes ]Plasma levels, half-life of FBS0701 and other pharmacokinetic parameters following a single capsule dose of FBS0701 at 16 mg/kg with 7 days of follow-up.
- Multiple Dose FBS0701 Study: Safety and Tolerability Based on Clinical Assessments; Efficacy Based on Clinical Response [ Time Frame: 48 weeks ] [ Designated as safety issue: Yes ]Clinical assessments include incidence and severity of adverse events, physical examinations including vital signs, clinical laboratory values, and ECGs); clinical response to be evaluated based on changes in liver and cardiac iron concentration determined by MRI in patients able to be studied using this method; serum ferritin in all patients.
| Estimated Enrollment: | 30 |
| Study Start Date: | August 2011 |
| Estimated Study Completion Date: | December 2014 |
| Estimated Primary Completion Date: | October 2014 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: 25 mg/kg of FBS0701
Oral FBS0701 taken once daily for 48 weeks
|
Drug: FBS0701
Iron chelation
|
|
Experimental: 35 mg/kg dose of FBS0701
Oral FBS0701 taken once daily for 48 weeks
|
Drug: FBS0701
Iron chelation
|
Detailed Description:
Pharmacokinetic Phase: Patients will receive a single 16 mg/kg dose of FBS0701 in capsule form.
Chronic Dosing Phase: Patients will receive FBS0701 capsules daily for 48 weeks. Doses may range from 8-40 mg/kg/d.
Eligibility| Ages Eligible for Study: | 6 Years to 17 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria
- Able to swallow whole capsules.
- Transfusion-dependent patients who have transfusional iron overload requiring chronic treatment with deferoxamine, deferasirox, or deferiprone.
- Willing to discontinue all existing iron chelation therapies throughout study period.
- For patients able to have an MRI, Baseline liver iron concentration and cardiac MRI T2* per protocol requirements.
- Serum ferritin greater than 500 ng/mL at Screening.
- 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 the 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.
- Iron overload from causes other than transfusional hemosiderosis.
- Severe cardiac dysfunction.
- Non-elective hospitalization within the past 30 days prior to Baseline testing.
- Evidence of clinically significant oral, cardiovascular, gastrointestinal, hepatic, renal, endocrine, pulmonary, neurologic, psychiatric, or skin disorder that contra-indicates dosing with FBS0701.
- Evidence of significant renal insufficiency.
- Known sensitivity to any ingredient in the FBS0701 formulation.
- Platelet count below 100,000/µL or absolute neutrophil count less than 1500/mm3 at Screening.
- Screening ALT outside of protocol requirements.
- Use of any investigational agent within the 30 days prior to Baseline testing.
- Pregnant or lactating females.
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01363908
Contacts
| Contact: Shire Call Center | +1 866-842-5335 |
Locations
| United States, California | |
| Children's Hospital Oakland | Withdrawn |
| Oakland, California, United States, 94609 | |
| United States, Massachusetts | |
| Children's Hospital Boston | Recruiting |
| Boston, Massachusetts, United States, 02115 | |
| Principal Investigator: Rachael Grace | |
| Canada, Ontario | |
| Toronto Sick Kids Hospital | Recruiting |
| Toronto, Ontario, Canada | |
| Principal Investigator: Isaac Odame | |
| Italy | |
| Ospedale Regionale Mecrocitemie | Recruiting |
| Cagliari, Italy, 09121 | |
| Principal Investigator: Renzo Galanello | |
| Centro della Microcitemia e delle Anemie Congenite | Recruiting |
| Genoa, Italy | |
| Principal Investigator: Gian Luca Forni | |
| Thalassemia Center San Luigi Hospital | Recruiting |
| Orbassano, Italy | |
| Principal Investigator: Antonio Piga | |
Sponsors and Collaborators
Shire Development LLC
Investigators
| Principal Investigator: | Ellis J Neufeld, M.D. | Children's Hospital Boston |
More Information
No publications provided
| Responsible Party: | Shire Development LLC |
| ClinicalTrials.gov Identifier: | NCT01363908 History of Changes |
| Other Study ID Numbers: | SPD602-202, FBS0701-CTP-07 |
| Study First Received: | May 30, 2011 |
| Last Updated: | May 3, 2013 |
| Health Authority: | United States: Food and Drug Administration |
Keywords provided by Shire Development LLC:
|
Beta-Thalassemia Sickle Cell Anemia Transfusional iron overload Iron Overload Iron Chelation |
Additional relevant MeSH terms:
|
Iron Overload Iron Metabolism Disorders Beta-Thalassemia Thalassemia Anemia, Hemolytic, Congenital Anemia, Hemolytic Anemia Hematologic Diseases Hemoglobinopathies |
Genetic Diseases, Inborn Metabolic Diseases Iron Trace Elements Micronutrients Growth Substances Physiological Effects of Drugs Pharmacologic Actions |
ClinicalTrials.gov processed this record on May 19, 2013