Trial · NCT01604941
Pharmacokinetics of SSP-004184 in the Treatment of Chronic Iron Overload Requiring Chelation Therapy
NCT01604941, Phase 2, Terminated, Shire; results first posted 2015-08-17
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Pharmacokinetics of SSP-004184 in the Treatment of Chronic Iron Overload Requiring Chelation Therapy. Trials, record NCT01604941. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01604941
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Pharmacokinetics of SSP-004184 in the Treatment of Chronic Iron Overload Requiring Chelation Therapy
- NCT number
- NCT01604941
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 32 participants, actual
- Lead sponsor
- Shire
- Conditions
- Iron Overload Due to Repeated Red Blood Cell Transfusions
- Interventions
- SPD602
- First posted
- 2012-05-24
- Last updated
- 2021-06-29
- Other registries' IDs
- eudract:2011-005675-16
- Results first posted
- 2015-08-17
- Limitations and caveats
- 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.
Source ClinicalTrials.gov
Registered outcomes11
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in Liver Iron Concentration (LIC) as Assessed by FerriScan R2 Magnetic Resonance Imaging (MRI) | Baseline, 12 and 24 weeks | The efficacy of SPD602 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. Early Termination was within the protocol defined visit date +/- 14 days window and was mapped to next scheduled MRI visit for 3 participants. |
| Primary | Change From Baseline in LIC Adjusted by Transfusional Iron Intake And Assessed by FerriScan R2 MRI | Baseline, 12 and 24 weeks | The efficacy of SPD602 was assessed by determining LIC and adjusting for transfusional iron intake. 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. For participants who had a blood transfusion on the MRI exam date, the blood transfusion done immediately prior to the MRI exam date was included in the calculation of daily transfusion intake. Early Termination was within the protocol defined visit date +/- 14 days window and was mapped to next scheduled MRI visit for 3 participants. |
| Secondary | Change From Baseline in LIC as Assessed by R2* MRI | Baseline, 12 and 24 weeks | The efficacy of SPD602 was assessed by determining LIC. Abdominal MRI data were collected by using R2\* standard procedures (liver and pancreas) and used to determine LIC. A negative change from baseline indicates that LIC decreased. Early Termination was within the protocol defined visit date +/- 14 days window and was mapped to next scheduled MRI visit for 3 participants. |
| Secondary | Change From Baseline in LIC Adjusted by Transfusional Iron Intake And Assessed by R2* MRI | Baseline, 12 and 24 weeks | The efficacy of SPD602 was assessed by determining LIC and adjusting for transfusional iron intake. Abdominal MRI data were collected by using R2\* standard procedures (liver and pancreas) and used to determine LIC. A negative change from baseline indicates that LIC decreased. For participants who had a blood transfusion on the MRI exam date, the blood transfusion done immediately prior to the MRI exam date was included in the calculation of daily transfusion intake. Early Termination was within the protocol defined visit date +/- 14 days window and was mapped to next scheduled MRI visit for 3 participants. |
| Secondary | Change From Baseline in Cardiac T2* Relaxation Rate, an MRI Parameter Used to Estimate Cardiac Iron Load | Baseline, 12 and 24 weeks | The efficacy of SPD602 was assessed by estimating cardiac iron load. T2\* data from cardiac MRI were collected by using standard procedures and used as an estimate of cardiac iron load. T2\* is an MR relaxation parameter that is reported in milliseconds. Iron within a tissue decreases homogeneity of the magnetic field and shortens the T2\* relaxation rate (Anderson, 2001). Low cardiac T2\* values are associated with increased risk of heart failure (Kirk, 2009). A negative change from baseline in the T2\* relaxation rate indicates that iron load increased. Early Termination was within the protocol defined visit date +/- 14 days window and was mapped to next scheduled MRI visit for 3 participants. |
| Secondary | Change From Baseline in Serum Ferritin | Baseline, 8 and 16 weeks | Serum ferritin levels were determined from serum biochemistry analyses. A negative change from baseline indicates that serum ferritin decreased. |
| Secondary | Number of Participants Classified as a Responder by FerriScan R2 MRI Analysis of LIC | 12 and 24 weeks | A responder was defined as a participant whose observed liver iron concentration (LIC) at the measured time point was less than the baseline value. LIC was assessed by abdominal MRI with the FerriScan R2 according to standard procedures. Early Termination was within the protocol defined visit date +/- 14 days window and was mapped to next scheduled MRI visit for 3 participants. |
| Secondary | Number of Participants Classified as a Responder by FerriScan R2 MRI Analysis of LIC Adjusted For Transfusional Iron Intake | 12 and 24 weeks | A responder was defined as a participant whose observed liver iron concentration (LIC) at the measured time point was less than the baseline value. LIC was assessed by abdominal MRI with the FerriScan R2 according to standard procedures, and the results were adjusted for transfusional iron intake. Early Termination was within the protocol defined visit date +/- 14 days window and was mapped to next scheduled MRI visit for 3 participants. For participants who had a blood transfusion on the MRI exam date, the blood transfusion done immediately prior to the MRI exam date was included in the calculation of daily transfusion intake. |
| Secondary | Number of Participants Classified as a Responder by R2* MRI Analysis of LIC | 12 and 24 weeks | A responder was defined as a participant whose observed liver iron concentration (LIC) at the measured time point was less than the baseline value. LIC was assessed by abdominal MRI with the R2\* according to standard procedures (liver and pancreas). Early Termination was within the protocol defined visit date +/- 14 days window and was mapped to next scheduled MRI visit for 3 participants. |
| Secondary | Number of Participants Classified as a Responder by R2* MRI Analysis of LIC Adjusted For Transfusional Iron Intake | 12 and 24 weeks | A responder was defined as a participant whose observed liver iron concentration (LIC) at the measured time point was less than the baseline value. LIC was assessed by abdominal MRI with the R2\* according to standard procedures (liver and pancreas), and the results were adjusted for transfusional iron intake. Early Termination was within the protocol defined visit date +/- 14 days window and was mapped to next scheduled MRI visit for 3 participants. For participants who had a blood transfusion on the MRI exam date, the blood transfusion done immediately prior to the MRI exam date was included in the calculation of daily transfusion intake. |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 registered outcomesOutcomes11
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline in Liver Iron Concentration (LIC) as Assessed by FerriScan R2 Magnetic Resonance Imaging (MRI) | Baseline, 12 and 24 weeks | The Full Analysis Set, defined as all participants in the Safety Set who had at least 1 post-baseline primary efficacy assessment. The Safety Set was defined as all participants who had taken at least 1 BID dose of investigational product. | mg Fe/g*dw | MEAN |
| SECONDARY | Change From Baseline in LIC as Assessed by R2* MRI | Baseline, 12 and 24 weeks | The Full Analysis Set, defined as all participants in the Safety Set who had at least 1 post-baseline primary efficacy assessment. The Safety Set was defined as all participants who had taken at least 1 BID dose of investigational product. | mg Fe/g*dw | MEAN |
| SECONDARY | Change From Baseline in LIC Adjusted by Transfusional Iron Intake And Assessed by R2* MRI | Baseline, 12 and 24 weeks | The Full Analysis Set, defined as all participants in the Safety Set who had at least 1 post-baseline primary efficacy assessment. The Safety Set was defined as all participants who had taken at least 1 BID dose of investigational product. | mg Fe/g*dw | MEAN |
| SECONDARY | Change From Baseline in Cardiac T2* Relaxation Rate, an MRI Parameter Used to Estimate Cardiac Iron Load | Baseline, 12 and 24 weeks | The Full Analysis Set, defined as all participants in the Safety Set who had at least 1 post-baseline primary efficacy assessment. The Safety Set was defined as all participants who had taken at least 1 BID dose of investigational product. | milliseconds | MEAN |
| SECONDARY | Change From Baseline in Serum Ferritin | Baseline, 8 and 16 weeks | The Full Analysis Set, defined as all participants in the Safety Set who had at least 1 post-baseline primary efficacy assessment. The Safety Set was defined as all participants who had taken at least 1 BID dose of investigational product. | ng/mL | MEAN |
| PRIMARY | Change From Baseline in LIC Adjusted by Transfusional Iron Intake And Assessed by FerriScan R2 MRI | Baseline, 12 and 24 weeks | The Full Analysis Set, defined as all participants in the Safety Set who had at least 1 post-baseline primary efficacy assessment. The Safety Set was defined as all participants who had taken at least 1 BID dose of investigational product. | mg Fe/g*dw | MEAN |
| SECONDARY | Number of Participants Classified as a Responder by FerriScan R2 MRI Analysis of LIC | 12 and 24 weeks | The Full Analysis Set, defined as all participants in the Safety Set who had at least 1 post-baseline primary efficacy assessment. The Safety Set was defined as all participants who had taken at least 1 BID dose of investigational product. | participants | NUMBER |
| SECONDARY | Number of Participants Classified as a Responder by FerriScan R2 MRI Analysis of LIC Adjusted For Transfusional Iron Intake | 12 and 24 weeks | The Full Analysis Set, defined as all participants in the Safety Set who had at least 1 post-baseline primary efficacy assessment. The Safety Set was defined as all participants who had taken at least 1 BID dose of investigational product. | participants | NUMBER |
| SECONDARY | Number of Participants Classified as a Responder by R2* MRI Analysis of LIC | 12 and 24 weeks | The Full Analysis Set, defined as all participants in the Safety Set who had at least 1 post-baseline primary efficacy assessment. The Safety Set was defined as all participants who had taken at least 1 BID dose of investigational product. | participants | NUMBER |
| SECONDARY | Number of Participants Classified as a Responder by R2* MRI Analysis of LIC Adjusted For Transfusional Iron Intake | 12 and 24 weeks | The Full Analysis Set, defined as all participants in the Safety Set who had at least 1 post-baseline primary efficacy assessment. The Safety Set was defined as all participants who had taken at least 1 BID dose of investigational product. | participants | NUMBER |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 outcomesResults by group44
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline in Liver Iron Concentration (LIC) as Assessed by FerriScan R2 Magnetic Resonance Imaging (MRI) | SPD602 50 mg/kg/d | Week 12, n=5,10 | -3.4 | Standard Deviation 6.4 | mg Fe/g*dw |
| Change From Baseline in Liver Iron Concentration (LIC) as Assessed by FerriScan R2 Magnetic Resonance Imaging (MRI) | All Participants With BID Dosing | Week 12, n=5,10 | -3.8 | Standard Deviation 5.0 | mg Fe/g*dw |
| Change From Baseline in Liver Iron Concentration (LIC) as Assessed by FerriScan R2 Magnetic Resonance Imaging (MRI) | SPD602 50 mg/kg/d | Week 24, n=1,2 | -5.3 | Standard Deviation NA | mg Fe/g*dw |
| Change From Baseline in Liver Iron Concentration (LIC) as Assessed by FerriScan R2 Magnetic Resonance Imaging (MRI) | All Participants With BID Dosing | Week 24, n=1,2 | -2.1 | Standard Deviation 4.5 | mg Fe/g*dw |
| Change From Baseline in LIC as Assessed by R2* MRI | SPD602 50 mg/kg/d | Week 12, n=5,9 | -3.2 | Standard Deviation 2.3 | mg Fe/g*dw |
| Change From Baseline in LIC as Assessed by R2* MRI | All Participants With BID Dosing | Week 12, n=5,9 | -3.2 | Standard Deviation 2.1 | mg Fe/g*dw |
| Change From Baseline in LIC as Assessed by R2* MRI | SPD602 50 mg/kg/d | Week 24, n=1,2 | -2.4 | Standard Deviation NA | mg Fe/g*dw |
| Change From Baseline in LIC as Assessed by R2* MRI | All Participants With BID Dosing | Week 24, n=1,2 | -3.3 | Standard Deviation 1.3 | mg Fe/g*dw |
| Change From Baseline in LIC Adjusted by Transfusional Iron Intake And Assessed by R2* MRI | SPD602 50 mg/kg/d | Week 12, n=5,9 | -6.0 | Standard Deviation 4.5 | mg Fe/g*dw |
| Change From Baseline in LIC Adjusted by Transfusional Iron Intake And Assessed by R2* MRI | All Participants With BID Dosing | Week 12, n=5,9 | -6.2 | Standard Deviation 3.2 | mg Fe/g*dw |
1 to 10 of 44Source ClinicalTrials.gov
See all 44 results by groupParticipant flow18
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | SPD602 50mg/mg/Day Twice Daily Dosing (BID) | 11 | |
| Overall Study | STARTED | SPD602 75mg/kg/Day Twice Daily Dosing (BID) | 4 | |
| Overall Study | STARTED | SPD602 50mg/kg/Day Once (QD) Then Twice Daily Dosing (BID) | 3 | |
| Overall Study | STARTED | SPD602 75mg/kg/Day Once (QD) Then Twice Daily Dosing (BID) | 3 | |
| Overall Study | STARTED | SPD602 50mg/kg/Day Once Daily Dosing (QD) | 4 | |
| Overall Study | STARTED | SPD602 75mg/kg/Day Once Daily Dosing (QD) | 7 | |
| Overall Study | COMPLETED | SPD602 50mg/mg/Day Twice Daily Dosing (BID) | 0 | |
| Overall Study | COMPLETED | SPD602 75mg/kg/Day Twice Daily Dosing (BID) | 0 | |
| Overall Study | COMPLETED | SPD602 50mg/kg/Day Once (QD) Then Twice Daily Dosing (BID) | 0 | |
| Overall Study | COMPLETED | SPD602 75mg/kg/Day Once (QD) Then Twice Daily Dosing (BID) | 0 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 participant flowAdverse events152
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Paraesthesia | Nervous system disorders | SPD602 50mg/kg/Day | 2 | 12 | 3 |
| other | Paraesthesia | Nervous system disorders | SPD602 75mg/kg/Day | 4 | 7 | 5 |
| other | Chromaturia | Renal and urinary disorders | SPD602 50mg/kg/Day | 2 | 12 | 2 |
| other | Chromaturia | Renal and urinary disorders | SPD602 75mg/kg/Day | 2 | 7 | 2 |
| other | Constipation | Gastrointestinal disorders | SPD602 50mg/kg/Day | 1 | 12 | 1 |
| other | Constipation | Gastrointestinal disorders | SPD602 75mg/kg/Day | 3 | 7 | 4 |
| other | Headache | Nervous system disorders | SPD602 50mg/kg/Day | 2 | 12 | 4 |
| other | Headache | Nervous system disorders | SPD602 75mg/kg/Day | 2 | 7 | 2 |
| other | Hypoaesthesia | Nervous system disorders | SPD602 50mg/kg/Day | 2 | 12 | 4 |
| other | Hypoaesthesia | Nervous system disorders | SPD602 75mg/kg/Day | 2 | 7 | 3 |
1 to 10 of 152Source ClinicalTrials.gov
See all 152 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | SPD602 50mg/kg/Day | ||
| Total, all-cause mortality | SPD602 75mg/kg/Day | ||
| Total, other adverse events | SPD602 50mg/kg/Day | 9 | 12 |
| Total, other adverse events | SPD602 75mg/kg/Day | 7 | 7 |
| Total, serious adverse events | SPD602 50mg/kg/Day | 2 | 12 |
| Total, serious adverse events | SPD602 75mg/kg/Day | 2 | 7 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| SPD602 50mg/kg/Day vs SPD602 75mg/kg/Day: serious adverse events |
Source Trial comparisons
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
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GET https://conductscience.org/api/trials/v1/NCT01604941/results?page=2