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

Stopped early · Phase 2

Conditions studied: Iron Overload Due to Repeated Red Blood Cell Transfusions

In brief

The purpose of this extension study is to evaluate SSP-004184AQ in patients with transfusional iron overload and to provide data on long term safety and efficacy. SSP-004184AQ is an iron chelator under development for chronic daily oral administration to patients with transfusional iron overload

Key facts

Study ID

NCT01671111

Run by

Shire

People needed

30

Starts

2012-08-14

Expected to finish

2014-04-24

Last updated by the study team

2021-06-29

Who can join

Age: 6 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Subjects and/or parents willing and able to sign the approved informed consent/and assent (based on institutional guidelines).
• An understanding, ability, and willingness to fully comply with study procedures and restrictions.
• Has either completed a previous SSP-004184AQ study or prematurely discontinued from (with agreement from the Investigator and Shire physician) from one of the SSP-004184AQ studies.
• If applicable, female subjects should be either:
• Post-menopausal (12 consecutive months of spontaneous amenorrhea), or
• Surgically sterile, or
• Females of child-bearing potential must have a negative urine pregnancy test at the Qualification and Enrollment Visit prior to dosing on Day 1. Females of child-bearing potential must agree to abstain from sexual activity that could result in pregnancy or agree to use acceptable methods of contraception.
• For subjects that did not transition directly from their feeder protocol and have initiated treatment with a chelator other than SSP-004184AQ:
• Willing to discontinue all existing iron chelation therapies for a minimum period of 1 to 5 days prior to first dose of SSP-004184AQ.
• (Note: This inclusion criterion is also applicable for subjects that were receiving chelators other than SSP-004184AQ in their feeder protocol.)
• Serum ferritin greater than 500 ng/mL at the Qualification and Enrollment Visit.
• Liver iron concentration (LIC) greater than or equal to 2.0 mg iron per g (equivalent dry weight, liver) determined by FerriScan® R2 MRI at the Qualification and Enrollment Visit (Day -28 to Day -8).
• (Note: Younger subjects for whom MRI is not feasible will be considered iron overloaded on the basis of serum ferritin only.) - Mean of the previous 3 pre-transfusion hemoglobin concentrations greater than or equal to 7.5 g/dL.
• Exclusion:
• Unwilling to remain off all other existing chelation therapies during SSP-004184AQ dosing and for up to 24 hours from last dose.
• A history of non-compliance in a prior FerroKin/Shire-sponsored SSP-004184AQ study (excluding dose suspensions that were medically warranted).
• Cardiac MRI T2* less than 10.0 milliseconds at the Qualification and Enrollment Visit.
• Cardiac left ventricular ejection fraction less than 50% at the Qualification and Enrollment Visit by MRI or below the locally determined normal range by echocardiography if MRI information is not available.
• Evidence of clinically relevant oral, cardiovascular, gastrointestinal, endocrine, pulmonary, neurologic, psychiatric, immunologic, bone marrow, dermatologic, hepatic, biliary, or renal dysfunction where, in the opinion of the Investigator or the Shire Study Physician, treatment with SSP-004184AQ is relatively contraindicated.
• Platelet count below 100,000/µL or absolute neutrophil count less than 1500/mm3 at the Qualification and Enrollment Visit.
• Known sensitivity to any ingredient in the SSP-004184AQ formulation.
• Pregnant or lactating females.
• For subjects that did not transition directly from their feeder protocol and have initiated treatment with a chelator other than SSP-004184AQ:
• In younger subjects for whom MRI is not feasible, evidence of severe cardiac dysfunction, as assessed by the Investigator.
• Non-elective hospitalization within the 30 days prior to receiving the first dose of SSP 004184AQ.

Where it is running

• Children's Hospital Oakland — Oakland, California, United States
• Children's Hospital of Boston — Boston, Massachusetts, United States
• The Hospital for Sick Children — Toronto, Ontario, Canada
• Toronto General Hospital — Toronto, Ontario, Canada
• San Luigi Hospital Thalassemia Centre — Orbassano, Torino, Italy
• Ospedale Regionale Microcitemie — Cagliari, Italy
• Ospedale Galliera — Genova, Italy
• Siriraj Hospital, Mahidol University — Bangkok, Thailand
• Whittington Hospital — London, United Kingdom

Full record on ClinicalTrials.gov

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