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| ID | Type | Description | Link |
|---|---|---|---|
| FBS0701-CTP-16 | Other Identifier | Ferrokin | |
| 2011-005675-16 | EudraCT Number |
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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.
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The purpose of this study is to evaluate SSP-004184AQ in patients with transfusional iron overload whose primary diagnosis is hereditary or congenital anemia.
SSP-004184AQ is an iron chelator under development for chronic daily oral administration to patients with transfusional iron overload.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| SPD602 | Experimental | 50 mg/kg/day orally twice daily for 24 weeks |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| SPD602 | Drug | 50 mg/kg/day orally twice daily for 24 weeks |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Liver Iron Concentration (LIC) as Assessed by FerriScan R2 Magnetic Resonance Imaging (MRI) | 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. | Baseline, 12 and 24 weeks |
| Change From Baseline in LIC Adjusted by Transfusional Iron Intake And Assessed by FerriScan R2 MRI | 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. | Baseline, 12 and 24 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in LIC as Assessed by R2* MRI | 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. |
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Inclusion Criteria:
Willing and able to sign the approved informed consent.
Age: 18-60 years old, inclusive, at Screening.
Subjects who have received more than 20 transfusions in their lifetime and who have transfusional iron overload requiring chronic treatment with an iron chelator. N.B.: Sickle Cell Disease subjects receiving regular exchange transfusions and iron overloaded subjects with thalassemia intermedia who are receiving regular transfusions (transfusion dependent thalassemia intermedia) are eligible.
Willing to discontinue all existing iron chelation therapies for a minimum period of one to five days prior to first dose of SSP-004184AQ, the 24 week duration of the study and 1 week after last dose for a total of approximately 26 weeks.
Willing to fast two hours prior to and one hour after each dose.
Serum ferritin >500ng/mL at Screening.
Baseline liver iron concentration is greater than or equal to 5mg iron per g (equivalent dry weight, liver)determined by FerriScan® R2 MRI.
Mean of the previous three pre-transfusion hemoglobin concentrations is greater than or equal to 7.5g/dL.
Adult female subjects should be:
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.
Exclusion Criteria:
As a result of medical review, physical examination, or Screening investigations, the Principal Investigator (PI) considers the subject unfit for the study.
Non-elective hospitalization within the 30 days prior to Baseline testing.
Evidence of clinically relevant oral, cardiovascular, gastrointestinal, hepatic, biliary, renal, endocrine, pulmonary, neurologic, psychiatric, immunologic, bone marrow, or skin disorder that contraindicates dosing with SSP-004184AQ.
Iron overload from causes other than transfusional siderosis.
Evidence of severe renal insufficiency, eg, serum creatinine 1.5X above the upper limit of normal or proteinuria greater than 1 gm per day or a calculated glomerular filtration rate <60mL/min.
Severe iron overload including:
Known sensitivity to magnesium stearate, croscarmellose sodium or SSP-004184AQ.
Platelet count below 100,000/μL or absolute neutrophil count less than 1500/mm3 at Screening.
Insufficient venous access that precludes prescribed blood draws for safety laboratory assessments.
ALT at Screening >200 IU/L.
Use of any investigational agent within the 30 days prior to the Baseline testing.
Pregnant or lactating females.
Cardiac left ventricular ejection fraction
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| Name | Affiliation | Role |
|---|---|---|
| Study Director | Takeda | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Center for Cancer and Blood Diseases | Los Angeles | California | 90027 | United States | ||
| Ann & Robert H. Lurie Children's Hospital of Chicago |
The study started as a double-arm study with once daily (QD) dosing but was amended first to a double-arm study with twice daily (BID) dosing and then to a single-arm study after removing the higher dose. Some participants were enrolled directly to BID dosing, some to QD dosing and then re-enrolled to BID dosing, some completed with QD dosing.
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| ID | Title | Description |
|---|---|---|
| FG000 | SPD602 50mg/mg/Day Twice Daily Dosing (BID) | Participants were randomly assigned to 50 mg/kg/day oral BID dosing and continued BID dosing until the end of study (24 weeks) or early discontinuation. |
| FG001 | SPD602 75mg/kg/Day Twice Daily Dosing (BID) |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Baseline, 12 and 24 weeks |
| Change From Baseline in LIC Adjusted by Transfusional Iron Intake And Assessed by R2* MRI | 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. | Baseline, 12 and 24 weeks |
| Change From Baseline in Cardiac T2* Relaxation Rate, an MRI Parameter Used to Estimate Cardiac Iron Load | 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. | Baseline, 12 and 24 weeks |
| Change From Baseline in Serum Ferritin | Serum ferritin levels were determined from serum biochemistry analyses. A negative change from baseline indicates that serum ferritin decreased. | Baseline, 8 and 16 weeks |
| Number of Participants Classified as a Responder by FerriScan R2 MRI Analysis of LIC | 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. | 12 and 24 weeks |
| Number of Participants Classified as a Responder by FerriScan R2 MRI Analysis of LIC Adjusted For Transfusional Iron Intake | 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. | 12 and 24 weeks |
| Number of Participants Classified as a Responder by R2* MRI Analysis of LIC | 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. | 12 and 24 weeks |
| Number of Participants Classified as a Responder by R2* MRI Analysis of LIC Adjusted For Transfusional Iron Intake | 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. | 12 and 24 weeks |
| Number of Participants Classified as a Responder by Serum Ferritin | A responder was defined as a participant whose observed serum ferritin level at the measured time point was less than the baseline value. Serum ferritin levels were determined from serum biochemistry analyses. | 8 and 16 weeks |
| Chicago |
| Illinois |
| 60611 |
| United States |
| Weill Cornell Medical College | New York | New York | 10065 | United States |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Toronto General Hospital | Toronto | Ontario | M5G 2C4 | Canada |
| Ain Shams University Pediatric Hospitals | Cairo | Egypt |
| Cairo University Pediatric Hospitals | Cairo | Egypt |
| Centro della Microcitemia e delle Anemie Congenite | Genova | Genoa | 16128 | Italy |
| San Luigi Hospital Thalassemia Centre | Orbassano | Torino | 10043 | Italy |
| Ospedale Regionale Microcitemie | Cagliari | 09121 | Italy |
| Ospedale Maggiore Policlinico | Milan | 20122 | Italy |
| American University of Beirut Medical Center | Beirut | Lebanon |
Participants were randomly assigned to 75 mg/kg/day oral BID dosing and continued BID dosing until the end of study (24 weeks) or early discontinuation. |
| FG002 | SPD602 50mg/kg/Day Once (QD) Then Twice Daily Dosing (BID) | Participants were randomly assigned to QD dosing then re-enrolled to 50 mg/kg/day oral BID dosing and continued BID dosing until the end of study (24 weeks) or early discontinuation. |
| FG003 | SPD602 75mg/kg/Day Once (QD) Then Twice Daily Dosing (BID) | Participants were randomly assigned to QD dosing then re-enrolled to 75 mg/kg/day oral BID dosing and continued BID dosing until the end of study (24 weeks) or early discontinuation. |
| FG004 | SPD602 50mg/kg/Day Once Daily Dosing (QD) | Participants were randomly assigned to 50 mg/kg/day oral QD dosing and continued QD dosing until the end of study (24 weeks) or early discontinuation. |
| FG005 | SPD602 75mg/kg/Day Once Daily Dosing (QD) | Participants were randomly assigned to 75 mg/kg/day oral QD dosing and continued QD dosing until the end of study (24 weeks) or early discontinuation. |
| COMPLETED |
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| NOT COMPLETED |
|
|
Safety Set, defined as all participants who had taken at least 1 BID dose of investigational product.
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| ID | Title | Description |
|---|---|---|
| BG000 | SPD602 50mg/kg/Day | Participants received SPD602 50mg/kg/day oral dosing either twice daily (BID) or once daily (QD), then BID. |
| BG001 | SPD602 75mg/kg/Day | Participants received SPD602 75mg/kg/day oral dosing either twice daily (BID) or once daily (QD), then BID. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change From Baseline in Liver Iron Concentration (LIC) as Assessed by FerriScan R2 Magnetic Resonance Imaging (MRI) | 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. | 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. | Posted | Mean | Standard Deviation | mg Fe/g*dw | Baseline, 12 and 24 weeks |
|
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| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in LIC as Assessed by R2* MRI | 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. | 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. | Posted | Mean | Standard Deviation | mg Fe/g*dw | Baseline, 12 and 24 weeks |
|
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| Secondary | Change From Baseline in LIC Adjusted by Transfusional Iron Intake And Assessed by R2* MRI | 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. | 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. | Posted | Mean | Standard Deviation | mg Fe/g*dw | Baseline, 12 and 24 weeks |
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| Secondary | Change From Baseline in Cardiac T2* Relaxation Rate, an MRI Parameter Used to Estimate Cardiac Iron Load | 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. | 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. | Posted | Mean | Standard Deviation | milliseconds | Baseline, 12 and 24 weeks |
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| Secondary | Change From Baseline in Serum Ferritin | Serum ferritin levels were determined from serum biochemistry analyses. A negative change from baseline indicates that serum ferritin decreased. | 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. | Posted | Mean | Standard Deviation | ng/mL | Baseline, 8 and 16 weeks |
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| Primary | Change From Baseline in LIC Adjusted by Transfusional Iron Intake And Assessed by FerriScan R2 MRI | 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. | 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. | Posted | Mean | Standard Deviation | mg Fe/g*dw | Baseline, 12 and 24 weeks |
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| Secondary | Number of Participants Classified as a Responder by FerriScan R2 MRI Analysis of LIC | 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. | 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. | Posted | Number | participants | 12 and 24 weeks |
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| Secondary | Number of Participants Classified as a Responder by FerriScan R2 MRI Analysis of LIC Adjusted For Transfusional Iron Intake | 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. | 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. | Posted | Number | participants | 12 and 24 weeks |
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| Secondary | Number of Participants Classified as a Responder by R2* MRI Analysis of LIC | 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. | 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. | Posted | Number | participants | 12 and 24 weeks |
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| Secondary | Number of Participants Classified as a Responder by R2* MRI Analysis of LIC Adjusted For Transfusional Iron Intake | 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. | 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. | Posted | Number | participants | 12 and 24 weeks |
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| Secondary | Number of Participants Classified as a Responder by Serum Ferritin | A responder was defined as a participant whose observed serum ferritin level at the measured time point was less than the baseline value. Serum ferritin levels were determined from serum biochemistry analyses. | 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. | Posted | Number | participants | 8 and 16 weeks |
|
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Not provided
All safety analyses, including analyses of AEs, were based on the Safety Set, which included participants in the 50mg/kg/day BID and 75mg/kg/day BID dosing groups who had taken at least 1 BID dose of investigational product. Participants in the QD only dosing groups were not included in the Safety Set.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | SPD602 50mg/kg/Day | Participants received SPD602 50mg/kg/day oral dosing BID either as originally randomized or as a re-enrolled participant. | 2 | 12 | 9 | 12 | ||
| EG001 | SPD602 75mg/kg/Day | Participants received SPD602 75mg/kg/day oral dosing BID either as originally randomized or as a re-enrolled participant. | 2 | 7 | 7 | 7 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Chest pain | General disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Pain | General disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Sickle cell anaemia with crisis | Congenital, familial and genetic disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Cholelithiasis | Hepatobiliary disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Flank pain | Musculoskeletal and connective tissue disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Neuropathy peripheral | Nervous system disorders | MedDRA (12.1) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Paraesthesia | Nervous system disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Chromaturia | Renal and urinary disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Hypoaesthesia | Nervous system disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Blood urea increased | Investigations | MedDRA (12.1) | Systematic Assessment |
| |
| Faeces discoloured | Gastrointestinal disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (12.1) | Systematic Assessment |
| |
| International normalised ratio increased | Investigations | MedDRA (12.1) | Systematic Assessment |
| |
| Leukocyturia | Renal and urinary disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Limb discomfort | Musculoskeletal and connective tissue disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Sensation of heaviness | Musculoskeletal and connective tissue disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA (12.1) | Systematic Assessment |
| |
| Activated partial thromboplastin time prolonged | Investigations | MedDRA (12.1) | Systematic Assessment |
| |
| Allergic transfusion reaction | Injury, poisoning and procedural complications | MedDRA (12.1) | Systematic Assessment |
| |
| Anion gap increased | Investigations | MedDRA (12.1) | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Blood bicarbonate decreased | Investigations | MedDRA (12.1) | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA (12.1) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Eructation | Gastrointestinal disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Folate deficiency | Metabolism and nutrition disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Glycosuria | Renal and urinary disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Hiatus hernia | Gastrointestinal disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Hypersensitivity | Immune system disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (12.1) | Systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Nitrite urine present | Investigations | MedDRA (12.1) | Systematic Assessment |
| |
| Polyuria | Renal and urinary disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Proteinuria | Renal and urinary disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Red blood cells urine positive | Investigations | MedDRA (12.1) | Systematic Assessment |
| |
| Regurgitation | Gastrointestinal disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA (12.1) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (12.1) | Systematic Assessment |
| |
| Urine protein/creatinine ratio increased | Investigations | MedDRA (12.1) | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Axillary pain | General disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Frequent bowel movements | Gastrointestinal disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Dyspnoea exertional | Respiratory, thoracic and mediastinal disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Throat irritation | Respiratory, thoracic and mediastinal disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Wheezing | Respiratory, thoracic and mediastinal disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Bacteriuria | Infections and infestations | MedDRA (12.1) | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA (12.1) | Systematic Assessment |
| |
| Electrocardiogram T wave abnormal | Investigations | MedDRA (12.1) | Systematic Assessment |
| |
| pH urine increased | Investigations | MedDRA (12.1) | Systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Pruritus allergic | Skin and subcutaneous tissue disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Pallor | Vascular disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Sickle cell anaemia with crisis | Congenital, familial and genetic disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Flank pain | Musculoskeletal and connective tissue disorders | MedDRA (12.1) | Systematic Assessment |
| |
| Neuropathy peripheral | Nervous system disorders | MedDRA (12.1) | Systematic Assessment |
|
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.
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.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Shire | +1 866 842 5335 | ClinicalTransparency@shire.com |
| Male |
|
| paired t-test |
| 0.0400 |
The P-value is from analysis of paired t-test at post treatment timepoint vs. Baseline. |
| 2-Sided |
| Superiority or Other (legacy) |
| Analysis of Week 24 for all participants with BID dosing | paired t-test | 0.6303 | The P-value is from analysis of paired t-test at post treatment timepoint vs. Baseline. | 2-Sided | Superiority or Other (legacy) |
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