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Study for Transfusionally Iron Overloaded Children, Adolescents and Adults Using FBS0701 (SSP-004184)

This study is enrolling participants by invitation only.
Sponsor:
Information provided by (Responsible Party):
Shire Development LLC
ClinicalTrials.gov Identifier:
NCT01671111
First received: August 16, 2012
Last updated: February 14, 2013
Last verified: February 2013
History of Changes
  Purpose

This is a long term extension study for patients with transfusional iron overload whose primary diagnosis is hereditary or congenital anemia who previously participated in other studies with FBS0701.

FBS0701 (SSP-004184) is an iron chelator that will be orally administered daily to patients aged 6 years and older with transfusional iron overload.


Condition Intervention Phase
Iron Overload Due to Repeated Red Blood Cell Transfusions
 Drug: FBS0701 (SSP-004184)
 Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase 2, Open-label, Multicentre, Extension Safety and Tolerability Study for Transfusionally Iron Overloaded Children, Adolescents and Adults Using FBS0701 (SSP-004184)

Resource links provided by NLM:

MedlinePlus related topics: Iron
U.S. FDA Resources

Further study details as provided by Shire Development LLC:

Primary Outcome Measures:
  • Change in liver iron concentration as determined by MRI [ Time Frame: up to 5 years ] [ Designated as safety issue: Yes ]
  • Change in cardiac iron concentration as determined by MRI [ Time Frame: up to 5 years ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Change in serum ferritin levels [ Time Frame: up to 5 years ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 1500
Study Start Date: August 2012
Estimated Study Completion Date: December 2018
Estimated Primary Completion Date: December 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: FBS0701 (SSP-004184) Drug: FBS0701 (SSP-004184)
8-75 mg/kg orally of FBS0701 (SSP-004184) daily
Other Name: SPD602

  Eligibility

Ages Eligible for Study:   6 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patients who have completed one of the FBS0701 (SSP-004184) feeder protocols

Exclusion Criteria:

  • A history of non-compliance in a prior FBS0701 (SSP-004184) study
  • Unwilling to remain off all existing chelation therapies or has restarted prior chelation therapy.
  • Evidence of clinically relevant oral, cardiovascular, gastrointestinal, endocrine, pulmonary, neurologic, psychiatric, immunologic, bone marrow, dermatologic, hepatic or renal dysfunction where treatment with FBS0701 (SSP-004184) is relatively contraindicated.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01671111

Locations
United States, California
Children's Hospital Oakland
Oakland, California, United States, 94609
United States, Massachusetts
Children's Hospital of Boston
Boston, Massachusetts, United States, 02115
Italy
Ospedale Regionale Microcitemie
Cagliari, Italy, 09121
Ospedale Galliera
Genova, Italy, 16128
San Luigi Hospital Thalassemia Centre
Orbassano (Torino), Italy, 10043
United Kingdom
Whittington Hospital
London, United Kingdom, N19 5NF
Sponsors and Collaborators
Shire Development LLC
Investigators
Principal Investigator: Ellis Neufeld, MD Boston Children's Hospital
  More Information

No publications provided

Responsible Party: Shire Development LLC
ClinicalTrials.gov Identifier: NCT01671111     History of Changes
Other Study ID Numbers: SPD602-301
Study First Received: August 16, 2012
Last Updated: February 14, 2013
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Iron Overload
Iron Metabolism Disorders
Metabolic Diseases

ClinicalTrials.gov processed this record on May 16, 2013