Trial Outcomes & Findings for Study for Transfusionally Iron Overloaded Children, Adolescents and Adults Using FBS0701 (SSP-004184) (NCT NCT01671111)
NCT ID: NCT01671111
Last Updated: 2021-06-29
Results Overview
Efficacy of SSP-004184 was assessed by determining LIC. Abdominal magnetic resonance imaging (MRI) data were collected by using FerriScan R2 standard procedures and used to determine LIC. A negative change from baseline indicates that LIC decreased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
30 participants
Primary outcome timeframe
Baseline, Week 24 (Cycle 1)
Results posted on
2021-06-29
Participant Flow
A total of 30 participants were enrolled to this open-label extension study (24 participants transferred directly from feeder studies SPD602-201 \[NCT01186419\], SPD602-202 \[NCT01363908\], and SPD602-203 \[NCT01604941\] and 6 participants did not transfer directly and received a chelator other than SSP-004184 after discontinuation from a feeder study).
Participant milestones
| Measure |
SSP-004184AQ
Participants received SSP-004184AQ (magnesium salt of free acid or active form, that is, SSP-004184 \[SPD602, FBS0701\]) capsules orally at a total daily dose of 8-75 milligram per kilogram per day (mg/kg/day) (equivalent to SSP-004184 7-68 mg/kg/day) either once daily or twice daily at the discretion of investigator for up to a maximum of 3 years or until the sponsor decided to stop the study.
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|---|---|
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Overall Study
STARTED
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30
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Overall Study
COMPLETED
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0
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Overall Study
NOT COMPLETED
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30
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Reasons for withdrawal
| Measure |
SSP-004184AQ
Participants received SSP-004184AQ (magnesium salt of free acid or active form, that is, SSP-004184 \[SPD602, FBS0701\]) capsules orally at a total daily dose of 8-75 milligram per kilogram per day (mg/kg/day) (equivalent to SSP-004184 7-68 mg/kg/day) either once daily or twice daily at the discretion of investigator for up to a maximum of 3 years or until the sponsor decided to stop the study.
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Overall Study
Adverse Event
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2
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Overall Study
Lack of Efficacy
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1
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Overall Study
Withdrawal by Subject
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2
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Overall Study
Physician Decision
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1
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Overall Study
Other
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24
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Baseline Characteristics
Study for Transfusionally Iron Overloaded Children, Adolescents and Adults Using FBS0701 (SSP-004184)
Baseline characteristics by cohort
| Measure |
SSP-004184AQ
n=30 Participants
Participants received SSP-004184AQ (magnesium salt of free acid or active form, that is, SSP-004184 \[SPD602, FBS0701\]) capsules orally at a total daily dose of 8-75 mg/kg/day (equivalent to SSP-004184 7-68 mg/kg/day) either once daily or twice daily at the discretion of investigator for up to a maximum of 3 years or until the sponsor decided to stop the study.
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Age, Continuous
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25.5 years
STANDARD_DEVIATION 11.38 • n=93 Participants
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Sex: Female, Male
Female
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17 Participants
n=93 Participants
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Sex: Female, Male
Male
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13 Participants
n=93 Participants
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PRIMARY outcome
Timeframe: Baseline, Week 24 (Cycle 1)Population: Full analysis set (FAS) included all participants in the Safety set who had at least 1 post-baseline primary efficacy assessment. Here, 'n' signifies the number of FAS participants evaluable for the respective time points.
Efficacy of SSP-004184 was assessed by determining LIC. Abdominal magnetic resonance imaging (MRI) data were collected by using FerriScan R2 standard procedures and used to determine LIC. A negative change from baseline indicates that LIC decreased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.
Outcome measures
| Measure |
SSP-004184AQ
n=23 Participants
Participants received SSP-004184AQ (magnesium salt of free acid or active form, that is, SSP-004184 \[SPD602, FBS0701\]) capsules orally at a total daily dose of 8-75 mg/kg/day (equivalent to SSP-004184 7-68 mg/kg/day) either once daily or twice daily at the discretion of investigator for up to a maximum of 3 years or until the sponsor decided to stop the study.
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Change From Baseline in Ferriscan® R2 Liver Iron Concentrations (LIC) at Week 24 (Cycle 1)
Baseline (n=23)
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9.9 milligram per gram (mg/g) dry tissue
Standard Deviation 8.10
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Change From Baseline in Ferriscan® R2 Liver Iron Concentrations (LIC) at Week 24 (Cycle 1)
Change at Week 24 (Cycle 1) (n=13)
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-1.1 milligram per gram (mg/g) dry tissue
Standard Deviation 1.86
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PRIMARY outcome
Timeframe: Baseline, Week 48 (Cycle 1)Population: FAS participants evaluable for this outcome.
Efficacy of SSP-004184 was assessed by determining LIC. Abdominal MRI data were collected by using FerriScan R2 standard procedures and used to determine LIC. A negative change from baseline indicates that LIC decreased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.
Outcome measures
| Measure |
SSP-004184AQ
n=9 Participants
Participants received SSP-004184AQ (magnesium salt of free acid or active form, that is, SSP-004184 \[SPD602, FBS0701\]) capsules orally at a total daily dose of 8-75 mg/kg/day (equivalent to SSP-004184 7-68 mg/kg/day) either once daily or twice daily at the discretion of investigator for up to a maximum of 3 years or until the sponsor decided to stop the study.
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Change From Baseline in Ferriscan® R2 Liver Iron Concentrations (LIC) at Week 48 (Cycle 1)
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-1.0 mg/g dry tissue
Standard Deviation 1.68
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PRIMARY outcome
Timeframe: Baseline, Week 24 (Cycle 2)Population: FAS participants evaluable for this outcome.
Efficacy of SSP-004184 was assessed by determining LIC. Abdominal MRI data were collected by using FerriScan R2 standard procedures and used to determine LIC. A negative change from baseline indicates that LIC decreased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.
Outcome measures
| Measure |
SSP-004184AQ
n=7 Participants
Participants received SSP-004184AQ (magnesium salt of free acid or active form, that is, SSP-004184 \[SPD602, FBS0701\]) capsules orally at a total daily dose of 8-75 mg/kg/day (equivalent to SSP-004184 7-68 mg/kg/day) either once daily or twice daily at the discretion of investigator for up to a maximum of 3 years or until the sponsor decided to stop the study.
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Change From Baseline in Ferriscan® R2 Liver Iron Concentrations (LIC) at Week 24 (Cycle 2)
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-1.5 mg/g dry tissue
Standard Deviation 2.23
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PRIMARY outcome
Timeframe: Baseline, Week 24 (Cycle 1)Population: FAS. Here, 'n' signifies the number of FAS participants evaluable for the respective time points.
The efficacy of SSP-004184 was assessed by determining cardiac iron load. Cardiac MRI data were collected by using T2\* standard procedures and used to determine iron load. A negative change from baseline indicates that iron load increased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.
Outcome measures
| Measure |
SSP-004184AQ
n=23 Participants
Participants received SSP-004184AQ (magnesium salt of free acid or active form, that is, SSP-004184 \[SPD602, FBS0701\]) capsules orally at a total daily dose of 8-75 mg/kg/day (equivalent to SSP-004184 7-68 mg/kg/day) either once daily or twice daily at the discretion of investigator for up to a maximum of 3 years or until the sponsor decided to stop the study.
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Change From Baseline in Cardiac T2* Values at Week 24 (Cycle 1)
Baseline (n=23)
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35.14 milliseconds
Standard Deviation 12.242
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Change From Baseline in Cardiac T2* Values at Week 24 (Cycle 1)
Change at Week 24 (Cycle 1) (n=14)
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-0.41 milliseconds
Standard Deviation 8.069
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PRIMARY outcome
Timeframe: Baseline, Week 48 (Cycle 1)Population: FAS participants evaluable for this outcome.
The efficacy of SSP-004184 was assessed by determining cardiac iron load. Cardiac MRI data were collected by using T2\* standard procedures and used to determine iron load. A negative change from baseline indicates that iron load increased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.
Outcome measures
| Measure |
SSP-004184AQ
n=9 Participants
Participants received SSP-004184AQ (magnesium salt of free acid or active form, that is, SSP-004184 \[SPD602, FBS0701\]) capsules orally at a total daily dose of 8-75 mg/kg/day (equivalent to SSP-004184 7-68 mg/kg/day) either once daily or twice daily at the discretion of investigator for up to a maximum of 3 years or until the sponsor decided to stop the study.
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Change From Baseline in Cardiac T2* Values at Week 48 (Cycle 1)
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-3.47 milliseconds
Standard Deviation 9.619
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PRIMARY outcome
Timeframe: Baseline, Week 24 (Cycle 2)Population: FAS participants evaluable for this outcome.
The efficacy of SSP-004184 was assessed by determining cardiac iron load. Cardiac MRI data were collected by using T2\* standard procedures and used to determine iron load. A negative change from baseline indicates that iron load increased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.
Outcome measures
| Measure |
SSP-004184AQ
n=7 Participants
Participants received SSP-004184AQ (magnesium salt of free acid or active form, that is, SSP-004184 \[SPD602, FBS0701\]) capsules orally at a total daily dose of 8-75 mg/kg/day (equivalent to SSP-004184 7-68 mg/kg/day) either once daily or twice daily at the discretion of investigator for up to a maximum of 3 years or until the sponsor decided to stop the study.
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Change From Baseline in Cardiac T2* Values at Week 24 (Cycle 2)
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-3.91 milliseconds
Standard Deviation 5.551
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SECONDARY outcome
Timeframe: Baseline, Weeks 24 and 48 of Cycle 1, Week 24 of Cycle 2Population: FAS. Here, 'n' signifies the number of FAS participants evaluable for the respective time points.
A negative change from baseline indicates that serum ferritin decreased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.
Outcome measures
| Measure |
SSP-004184AQ
n=23 Participants
Participants received SSP-004184AQ (magnesium salt of free acid or active form, that is, SSP-004184 \[SPD602, FBS0701\]) capsules orally at a total daily dose of 8-75 mg/kg/day (equivalent to SSP-004184 7-68 mg/kg/day) either once daily or twice daily at the discretion of investigator for up to a maximum of 3 years or until the sponsor decided to stop the study.
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Change From Baseline in Serum Ferritin Values at Specified Visits
Baseline (n=23)
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5353.38 nanogram per milliliter
Standard Deviation 4588.423
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Change From Baseline in Serum Ferritin Values at Specified Visits
Change at Week 24 (Cycle 1) (n=12)
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-395.82 nanogram per milliliter
Standard Deviation 569.305
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Change From Baseline in Serum Ferritin Values at Specified Visits
Change at Week 48 (Cycle 1) (n=9)
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-250.87 nanogram per milliliter
Standard Deviation 452.327
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Change From Baseline in Serum Ferritin Values at Specified Visits
Change at Week 24 (Cycle 2) (n=7)
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-384.03 nanogram per milliliter
Standard Deviation 649.990
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Adverse Events
SSP-004184AQ
Serious events: 2 serious events
Other events: 25 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
SSP-004184AQ
n=30 participants at risk
Participants received SSP-004184AQ (magnesium salt of free acid or active form, that is, SSP-004184 \[SPD602, FBS0701\]) capsules orally at a total daily dose of 8-75 mg/kg/day (equivalent to SSP-004184 7-68 mg/kg/day) either once daily or twice daily at the discretion of investigator for up to a maximum of 3 years or until the sponsor decided to stop the study.
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Congenital, familial and genetic disorders
Sickle cell anaemia with crisis
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3.3%
1/30 • Number of events 1 • From start of study treatment up to the end of study (1 week after the end of treatment)
An adverse event (AE) that occurred during the study was considered a treatment-emergent AE (TEAE) if it had a start date and time on or after the study treatment or if it had a start date before the date and time of the study treatment but increased in intensity on or after the date and time of the study treatment.
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Infections and infestations
Bronchitis
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3.3%
1/30 • Number of events 1 • From start of study treatment up to the end of study (1 week after the end of treatment)
An adverse event (AE) that occurred during the study was considered a treatment-emergent AE (TEAE) if it had a start date and time on or after the study treatment or if it had a start date before the date and time of the study treatment but increased in intensity on or after the date and time of the study treatment.
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Other adverse events
| Measure |
SSP-004184AQ
n=30 participants at risk
Participants received SSP-004184AQ (magnesium salt of free acid or active form, that is, SSP-004184 \[SPD602, FBS0701\]) capsules orally at a total daily dose of 8-75 mg/kg/day (equivalent to SSP-004184 7-68 mg/kg/day) either once daily or twice daily at the discretion of investigator for up to a maximum of 3 years or until the sponsor decided to stop the study.
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General disorders
Influenza like illness
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16.7%
5/30 • Number of events 6 • From start of study treatment up to the end of study (1 week after the end of treatment)
An adverse event (AE) that occurred during the study was considered a treatment-emergent AE (TEAE) if it had a start date and time on or after the study treatment or if it had a start date before the date and time of the study treatment but increased in intensity on or after the date and time of the study treatment.
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Infections and infestations
Nasopharyngitis
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16.7%
5/30 • Number of events 6 • From start of study treatment up to the end of study (1 week after the end of treatment)
An adverse event (AE) that occurred during the study was considered a treatment-emergent AE (TEAE) if it had a start date and time on or after the study treatment or if it had a start date before the date and time of the study treatment but increased in intensity on or after the date and time of the study treatment.
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General disorders
Asthenia
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13.3%
4/30 • Number of events 8 • From start of study treatment up to the end of study (1 week after the end of treatment)
An adverse event (AE) that occurred during the study was considered a treatment-emergent AE (TEAE) if it had a start date and time on or after the study treatment or if it had a start date before the date and time of the study treatment but increased in intensity on or after the date and time of the study treatment.
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Gastrointestinal disorders
Nausea
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13.3%
4/30 • Number of events 6 • From start of study treatment up to the end of study (1 week after the end of treatment)
An adverse event (AE) that occurred during the study was considered a treatment-emergent AE (TEAE) if it had a start date and time on or after the study treatment or if it had a start date before the date and time of the study treatment but increased in intensity on or after the date and time of the study treatment.
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Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
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13.3%
4/30 • Number of events 6 • From start of study treatment up to the end of study (1 week after the end of treatment)
An adverse event (AE) that occurred during the study was considered a treatment-emergent AE (TEAE) if it had a start date and time on or after the study treatment or if it had a start date before the date and time of the study treatment but increased in intensity on or after the date and time of the study treatment.
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Nervous system disorders
Paraesthesia
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13.3%
4/30 • Number of events 5 • From start of study treatment up to the end of study (1 week after the end of treatment)
An adverse event (AE) that occurred during the study was considered a treatment-emergent AE (TEAE) if it had a start date and time on or after the study treatment or if it had a start date before the date and time of the study treatment but increased in intensity on or after the date and time of the study treatment.
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Gastrointestinal disorders
Abdominal pain upper
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10.0%
3/30 • Number of events 3 • From start of study treatment up to the end of study (1 week after the end of treatment)
An adverse event (AE) that occurred during the study was considered a treatment-emergent AE (TEAE) if it had a start date and time on or after the study treatment or if it had a start date before the date and time of the study treatment but increased in intensity on or after the date and time of the study treatment.
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Nervous system disorders
Headache
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10.0%
3/30 • Number of events 3 • From start of study treatment up to the end of study (1 week after the end of treatment)
An adverse event (AE) that occurred during the study was considered a treatment-emergent AE (TEAE) if it had a start date and time on or after the study treatment or if it had a start date before the date and time of the study treatment but increased in intensity on or after the date and time of the study treatment.
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Infections and infestations
Pharyngitis
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10.0%
3/30 • Number of events 3 • From start of study treatment up to the end of study (1 week after the end of treatment)
An adverse event (AE) that occurred during the study was considered a treatment-emergent AE (TEAE) if it had a start date and time on or after the study treatment or if it had a start date before the date and time of the study treatment but increased in intensity on or after the date and time of the study treatment.
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Gastrointestinal disorders
Constipation
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6.7%
2/30 • Number of events 2 • From start of study treatment up to the end of study (1 week after the end of treatment)
An adverse event (AE) that occurred during the study was considered a treatment-emergent AE (TEAE) if it had a start date and time on or after the study treatment or if it had a start date before the date and time of the study treatment but increased in intensity on or after the date and time of the study treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
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6.7%
2/30 • Number of events 2 • From start of study treatment up to the end of study (1 week after the end of treatment)
An adverse event (AE) that occurred during the study was considered a treatment-emergent AE (TEAE) if it had a start date and time on or after the study treatment or if it had a start date before the date and time of the study treatment but increased in intensity on or after the date and time of the study treatment.
|
|
Psychiatric disorders
Depression
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6.7%
2/30 • Number of events 2 • From start of study treatment up to the end of study (1 week after the end of treatment)
An adverse event (AE) that occurred during the study was considered a treatment-emergent AE (TEAE) if it had a start date and time on or after the study treatment or if it had a start date before the date and time of the study treatment but increased in intensity on or after the date and time of the study treatment.
|
|
Gastrointestinal disorders
Diarrhoea
|
6.7%
2/30 • Number of events 3 • From start of study treatment up to the end of study (1 week after the end of treatment)
An adverse event (AE) that occurred during the study was considered a treatment-emergent AE (TEAE) if it had a start date and time on or after the study treatment or if it had a start date before the date and time of the study treatment but increased in intensity on or after the date and time of the study treatment.
|
|
Infections and infestations
Gastroenteritis
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6.7%
2/30 • Number of events 4 • From start of study treatment up to the end of study (1 week after the end of treatment)
An adverse event (AE) that occurred during the study was considered a treatment-emergent AE (TEAE) if it had a start date and time on or after the study treatment or if it had a start date before the date and time of the study treatment but increased in intensity on or after the date and time of the study treatment.
|
|
General disorders
Pyrexia
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6.7%
2/30 • Number of events 5 • From start of study treatment up to the end of study (1 week after the end of treatment)
An adverse event (AE) that occurred during the study was considered a treatment-emergent AE (TEAE) if it had a start date and time on or after the study treatment or if it had a start date before the date and time of the study treatment but increased in intensity on or after the date and time of the study treatment.
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Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee If a multicenter publication is not submitted within twelve (12) months after conclusion, abandonment or termination of the Study at all sites, or after Sponsor confirms there shall be no multicenter Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.
- Publication restrictions are in place
Restriction type: OTHER