TerminatedPhase 2

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

Stopped: This study was terminated due to treatment stop resulting in an inability to draw conclusions from the data. Evaluation of nonclinical rat findings is ongoing.

United States, Canada, Italy30 participantsStarted 2012-08-14

Plain-language summary

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

Who can participate

Age range

6 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. 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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Change From Baseline in Ferriscan® R2 Liver Iron Concentrations (LIC) at Week 24 (Cycle 1)

Timeframe: Baseline, Week 24 (Cycle 1)

Change From Baseline in Ferriscan® R2 Liver Iron Concentrations (LIC) at Week 48 (Cycle 1)

Timeframe: Baseline, Week 48 (Cycle 1)

Change From Baseline in Ferriscan® R2 Liver Iron Concentrations (LIC) at Week 24 (Cycle 2)

Timeframe: Baseline, Week 24 (Cycle 2)

Change From Baseline in Cardiac T2* Values at Week 24 (Cycle 1)

Timeframe: Baseline, Week 24 (Cycle 1)

Change From Baseline in Cardiac T2* Values at Week 48 (Cycle 1)

Timeframe: Baseline, Week 48 (Cycle 1)

Change From Baseline in Cardiac T2* Values at Week 24 (Cycle 2)

Timeframe: Baseline, Week 24 (Cycle 2)

Trial details

NCT IDNCT01671111

SponsorShire

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2014-04-24

Results submitted2015-07-10

View on ClinicalTrials.gov More Iron Overload Due to Repeated Red Blood Cell Transfusions trials

Who can participate

Age range

6 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. 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.

What they're measuring

Change From Baseline in Ferriscan® R2 Liver Iron Concentrations (LIC) at Week 24 (Cycle 1)

Timeframe: Baseline, Week 24 (Cycle 1)

Change From Baseline in Ferriscan® R2 Liver Iron Concentrations (LIC) at Week 48 (Cycle 1)

Timeframe: Baseline, Week 48 (Cycle 1)

Change From Baseline in Ferriscan® R2 Liver Iron Concentrations (LIC) at Week 24 (Cycle 2)

Timeframe: Baseline, Week 24 (Cycle 2)

Change From Baseline in Cardiac T2* Values at Week 24 (Cycle 1)

Timeframe: Baseline, Week 24 (Cycle 1)

Change From Baseline in Cardiac T2* Values at Week 48 (Cycle 1)

Timeframe: Baseline, Week 48 (Cycle 1)

Change From Baseline in Cardiac T2* Values at Week 24 (Cycle 2)

Timeframe: Baseline, Week 24 (Cycle 2)