Hyperphosphataemia
Conditions
Keywords
Dialysis Dependent Chronic Kidney Disease
Brief summary
The main purpose of this study is to see whether PT20 can help people with a high level of phosphate in their blood (called Hyperphosphatemia) that are being treated with dialysis for kidney disease.
Detailed description
PT20 represents a mechanism to address many of the limitations associated with current phosphate binding agents. The available clinical and non clinical evidence suggests that PT20 binds phosphate and prevents its uptake more efficiently than other phosphate binding drugs and may therefore either reduce the pill burden associated with controlling phosphate levels, or result in lower phosphate levels with the same pill burden. The this study is the first to investigate the efficacy and safety of PT20 in subjects with dialysis-dependent chronic kidney disease (CKD).
Interventions
DRUGPT20
Modified ferric oxide adipate tablets
DRUGPlacebo
Placebo tablets matched to each PT20 dose arm
Sponsors
Clinipace Worldwide
Phosphate Therapeutics
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 90 Years
Healthy volunteers
No
Inclusion criteria
* Men or women aged 18 90 years * Subject must have a stable dialysis prescription for at least 28 days prior to start of Screening. * Subject must have the most recent serum phosphate measurement, taken during the 28 days prior to the start of Screening, of ≥ 4.0 mg/dL and ≤ 8 mg/dL.
Exclusion criteria
* Subject's most recent historical pre-dialysis serum bicarbonate value within 14 days prior to the start of Screening (Visit 1) is \< 18 mg/dL. * Subject has, in the opinion of the investigator, severe chronic lung disease and/or carbon dioxide retention.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in Serum Phosphate Concentration From Baseline (Visit 7, Day 1) to Visit 11 (Day 29) | Day 1 to Day 29 | The primary efficacy endpoint was the change in serum phosphate concentration from Baseline (Visit 7, Day 1) to Visit 11 (Day 29). All study specific blood samples were collected, processed and analysed using a central laboratory. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Haemoglobin Concentration From Baseline (Visit 7, Day 1) to Visit 11 (Day 29) | Day 1 to Day 29 | Descriptive statistics were to be used to summarise values and change from Baseline (Visit 7, Day 1) to Day 29 (Visit 11) in haemoglobin concentration. An analysis of covariance (ANCOVA) was to be used to analyse the changes from Baseline (Visit 7, Day 1) to Visit 11 (Day 29), which were to include the fixed, categorical effects of treatment as well as the continuous, fixed covariates of Baseline concentrations for haemoglobin. Only those subjects with available concentrations for both Baseline (Visit 7, Day 1) and Visit 11 (Day 29) were to be included in this analysis. |
| Change in Serum Ferritin Concentration From Baseline (Visit 7, Day 1) to Visit 11 (Day 29) | Day 1 to Day 29 | Descriptive statistics were to be used to summarise values and change from Baseline (Visit 7, Day 1) to Day 29 (Visit 11) in serum ferritin concentration. An analysis of covariance (ANCOVA) was to be used to analyse the changes from Baseline (Visit 7, Day 1) to Visit 11 (Day 29), which were to include the fixed, categorical effects of treatment and week (Visit), as well as the continuous, fixed covariates of Baseline concentrations for serum ferritin. Only those subjects with available concentrations for both Baseline (Visit 7, Day 1) and Visit 11 (Day 29) were to be included in this analysis. |
| Change in Transferrin Saturation From Baseline (Visit 7, Day 1) to Visit 11 (Day 29) | Day 1 to Day 29 | Descriptive statistics were to be used to summarise values and change from Baseline (Visit 7, Day 1) to Day 29 (Visit 11) in transferrin saturation. An analysis of covariance (ANCOVA) was to be used to analyse the changes from Baseline (Visit 7, Day 1) to Day 29 (Visit 11), which were to include the fixed, categorical effects of treatment and week, as well as the continuous, fixed covariates of Baseline concentrations for transferrin saturation. Only those subjects with available concentrations for both Baseline (Visit 7, Day 1) and Visit 11 (Day 29) were to be included in this analysis. |
| Change in Calcium x Phosphate Product From Baseline (Visit 7, Day 1) to Visit 11 (Day 29) | Day 1 to Day 29 | Descriptive statistics were to be used to summarise values and change from Baseline (Visit 7, Day 1) to Day 29 (Visit 11) in Calcium x Phosphate Product . An analysis of covariance (ANCOVA) was to be used to analyse the changes from Baseline (Visit 7, Day 1) to Day 29 (Visit 11), which were to include the fixed, categorical effects of treatment, as well as the continuous, fixed covariates of Baseline concentrations for Calcium x Phosphate Product. Only those subjects with available concentrations for both Baseline (Visit 7, Day 1) and Visit 11 (Day 29) were to be included in this analysis. In CKD subjects (Stages 3-5), the clinical recommendation (KDOQI) is that serum calcium x phosphate product should be maintained at \< 55 mg\^2/dL\^2 (4.4 mmol\^2 /L\^2). |
| Change in Gastrointestinal Symptom Rating System (GSRS) Overall Score From Baseline (Visit 7, Day 1) to Visit 11 (Day 29) | Day 1 to Day 29 | The GSRS assesses the impact of treatment-related GI complications. It includes 15 items divided into 5 subscales (diarrhoea, indigestion, abdominal pain, constipation, and reflux), and uses a seven-grade Likert scale to assess symptoms (1 = no discomfort at all, 7 = very severe discomfort). The total score was calculated as the average score of all 15 items. If data were missing from one or more subscales, the mean of the completed items within the subscale was to be used for the subscale score, provided that more than half of the subscale items were complete. If more than half of the items within a subscale were missing, the subscale score and overall score were also to be defined as missing. The change in overall GSRS score from Baseline (Visit 7, Day 1) to Visit 11 (Day 29) was summarised by treatment and a two-sample t-test was to be used to identify differences between the placebo and PT20 dose groups. The higher the score, the more severe the gastrointestinal symptoms. |
Participant flow
Recruitment details
Male/female subjects with dialysis dependent CKD on prescribed maintenance haemodialysis 3 times/week for 90d before Screening were recruited and randomised to receive PT20 or placebo. Prior to Screening, all subjects were taking at least 1 OPB for a minimum of 28d mean + serum phosphate level ≥ 4.0 mg/dL and ≤ 8.0 mg/dL for the preceding 28 days.
Pre-assignment details
Subjects underwent stratified randomisation into 1 of the 4 PT20 dose groups or equivalent placebo group based pre-randomisation serum phosphate level (ratio of 8:8:8:13:13) - serum phosphate \< 7.5 mg/dL were deemed to have a lower level of serum phosphate; serum phosphate ≥ 7.5 mg/dL were deemed to have a higher level of serum phosphate.
Participants by arm
| Arm | Count |
|---|---|
| Group 1 - PT20 400 mg Tid PT20 400 mg tid (1.2 g/day) administered orally.
Dosing was initiated with the subject's first meal/snack following receipt of study medication at Visit 7 (Day 1). There was to be no change in dose administration level with respect to each cohort in this study | 24 |
| Group 2 - PT20 800 mg Tid PT20 800 mg tid (2.4 g/day) administered orally.
Dosing was initiated with the subject's first meal/snack following receipt of study medication at Visit 7 (Day 1). There was to be no change in dose administration level with respect to each cohort in this study | 23 |
| Group 3 - PT20 1600 mg Tid PT20 1600 mg tid (4.8 g/day) administered orally.
Dosing was initiated with the subject's first meal/snack following receipt of study medication at Visit 7 (Day 1). There was to be no change in dose administration level with respect to each cohort in this study | 22 |
| Group 4 - PT20 3200 mg Tid PT20 3200 mg tid (9.6 g/day) administered orally.
Dosing was initiated with the subject's first meal/snack following receipt of study medication at Visit 7 (Day 1). There was to be no change in dose administration level with respect to each cohort in this study | 36 |
| Group 5 - Placebo Tid Matched Placebo (for PT20) tid administered orally.
Dosing was initiated with the subject's first meal/snack following receipt of study medication at Visit 7 (Day 1). There was to be no change in dose administration level with respect to each cohort in this study | 36 |
| Total | 141 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 | FG003 | FG004 |
|---|---|---|---|---|---|---|
| Overall Study | Adverse Event | 2 | 0 | 1 | 4 | 0 |
| Overall Study | High Ferritin levels | 3 | 3 | 2 | 2 | 1 |
| Overall Study | Hyperphosphataemia | 1 | 0 | 0 | 0 | 1 |
| Overall Study | Withdrawal by Subject | 0 | 1 | 1 | 1 | 1 |
Baseline characteristics
| Characteristic | Group 2 - PT20 800 mg Tid | Group 3 - PT20 1600 mg Tid | Group 4 - PT20 3200 mg Tid | Group 1 - PT20 400 mg Tid | Group 5 - Placebo Tid | Total |
|---|---|---|---|---|---|---|
| Age, Continuous | 58.0 years | 60.3 years | 55.9 years | 60.4 years | 55.0 years | 57.5 years |
| Baseline Serum Phosphate Concentrations < 7.5 mg/dL | 15 Participants | 11 Participants | 20 Participants | 13 Participants | 20 Participants | 79 Participants |
| Baseline Serum Phosphate Concentrations ≥ 7.5 mg/dL | 8 Participants | 11 Participants | 16 Participants | 11 Participants | 16 Participants | 62 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 9 Participants | 8 Participants | 14 Participants | 9 Participants | 13 Participants | 53 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 14 Participants | 14 Participants | 22 Participants | 15 Participants | 23 Participants | 88 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants | 2 Participants | 0 Participants | 2 Participants |
| Race (NIH/OMB) Asian | 1 Participants | 0 Participants | 2 Participants | 1 Participants | 1 Participants | 5 Participants |
| Race (NIH/OMB) Black or African American | 8 Participants | 9 Participants | 12 Participants | 7 Participants | 16 Participants | 52 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 2 Participants | 0 Participants | 0 Participants | 1 Participants | 0 Participants | 3 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 1 Participants | 0 Participants | 2 Participants | 1 Participants | 3 Participants | 7 Participants |
| Race (NIH/OMB) White | 11 Participants | 13 Participants | 20 Participants | 12 Participants | 16 Participants | 72 Participants |
| Region of Enrollment United States | 23 Participants | 22 Participants | 36 Participants | 24 Participants | 36 Participants | 141 Participants |
| Sex: Female, Male Female | 9 Participants | 11 Participants | 16 Participants | 8 Participants | 14 Participants | 58 Participants |
| Sex: Female, Male Male | 14 Participants | 11 Participants | 20 Participants | 16 Participants | 22 Participants | 83 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk | EG004 affected / at risk |
|---|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 24 | 0 / 23 | 0 / 22 | 0 / 36 | 0 / 36 |
| other Total, other adverse events | 10 / 24 | 11 / 23 | 13 / 22 | 16 / 36 | 16 / 36 |
| serious Total, serious adverse events | 0 / 24 | 1 / 23 | 1 / 22 | 5 / 36 | 5 / 36 |
Outcome results
Primary
Change in Serum Phosphate Concentration From Baseline (Visit 7, Day 1) to Visit 11 (Day 29)
The primary efficacy endpoint was the change in serum phosphate concentration from Baseline (Visit 7, Day 1) to Visit 11 (Day 29). All study specific blood samples were collected, processed and analysed using a central laboratory.
Time frame: Day 1 to Day 29
Population: Intent-to-Treat (ITT) population.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Group 1 - PT20 400 mg Tid | Change in Serum Phosphate Concentration From Baseline (Visit 7, Day 1) to Visit 11 (Day 29) | -0.4 mg/dL |
| Group 2 - PT20 800 mg Tid | Change in Serum Phosphate Concentration From Baseline (Visit 7, Day 1) to Visit 11 (Day 29) | -0.59 mg/dL |
| Group 3 - PT20 1600 mg Tid | Change in Serum Phosphate Concentration From Baseline (Visit 7, Day 1) to Visit 11 (Day 29) | -1.29 mg/dL |
| Group 4 - PT20 3200 mg Tid | Change in Serum Phosphate Concentration From Baseline (Visit 7, Day 1) to Visit 11 (Day 29) | -1.363 mg/dL |
| Group 5 - Placebo Tid | Change in Serum Phosphate Concentration From Baseline (Visit 7, Day 1) to Visit 11 (Day 29) | -0.165 mg/dL |
Comparison: ITT population - statistical analysis of linear model fit using an F-test at the 0.05 level.~It was determined that 150 subjects randomised at a ratio of 8:8:8:13:13 (low dose to high dose, placebo) would have \>90% power to detect either a statistically significant slope for the dose-response relationship, or, if the relationship was not linear, a statistically significant difference between the highest-dose group and the placebo group, using a two-sided 0.05 significance level.p-value: 0.784F-test
Comparison: ITT population sensitivity analysis using baseline observation carried forward (BOCF) to assess statistical analysis of linear model fit using an F-test at the 0.05 level.p-value: 0.905F-test
Comparison: ITT population sensitivity analysis using multiple imputation (MI) to assess statistical analysis of linear model fit using an F-test at the 0.05 level.p-value: 0.976F-test
Comparison: Per protocol (PP) population sensitivity analysis to assess statistical analysis of linear model fit using an F-test at the 0.05 level.p-value: 0.914F-test
Comparison: ITT population - statistical analysis of the linear model log(PT20 dose)-response relationship to examine whether the linear trend of the change in phosphate level as a function of the log-transformed dose was statistically significant.p-value: <0.001linear model - log (dose) - response
Comparison: ITT population supportive analysis - Type 3 tests of fixed effects - treatment groupp-value: <0.001Mixed Models Analysis
Comparison: ITT population supportive analysis - Type 3 tests of fixed effects - visitp-value: 0.436Mixed Models Analysis
Comparison: ITT population supportive analysis - Type 3 tests of fixed effects - treatment group versus Visit interactionp-value: 0.959Mixed Models Analysis
Comparison: ITT population supportive analysis - Type 3 tests of fixed effects - baseline serum phosphate concentrationp-value: <0.001Mixed Models Analysis
Comparison: ITT population supportive analysis - Type 3 tests of fixed effects - baseline serum phosphate concentration versus Visit interactionp-value: 0.144Mixed Models Analysis
Comparison: ITT population supportive analysis - mixed model repeated measures for change in serum phosphate concentration
Comparison: ITT population supportive analysis - mixed model repeated measures for change in serum phosphate concentration
Comparison: ITT population supportive analysis - mixed model repeated measures for change in serum phosphate concentration
Comparison: ITT population supportive analysis - mixed model repeated measures for change in serum phosphate concentration
Secondary
Change in Calcium x Phosphate Product From Baseline (Visit 7, Day 1) to Visit 11 (Day 29)
Descriptive statistics were to be used to summarise values and change from Baseline (Visit 7, Day 1) to Day 29 (Visit 11) in Calcium x Phosphate Product . An analysis of covariance (ANCOVA) was to be used to analyse the changes from Baseline (Visit 7, Day 1) to Day 29 (Visit 11), which were to include the fixed, categorical effects of treatment, as well as the continuous, fixed covariates of Baseline concentrations for Calcium x Phosphate Product. Only those subjects with available concentrations for both Baseline (Visit 7, Day 1) and Visit 11 (Day 29) were to be included in this analysis. In CKD subjects (Stages 3-5), the clinical recommendation (KDOQI) is that serum calcium x phosphate product should be maintained at \< 55 mg\^2/dL\^2 (4.4 mmol\^2 /L\^2).
Time frame: Day 1 to Day 29
Population: ITT population.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Group 1 - PT20 400 mg Tid | Change in Calcium x Phosphate Product From Baseline (Visit 7, Day 1) to Visit 11 (Day 29) | -5.515 mg^2/dL^2 | Standard Deviation 13.7966 |
| Group 2 - PT20 800 mg Tid | Change in Calcium x Phosphate Product From Baseline (Visit 7, Day 1) to Visit 11 (Day 29) | -4.035 mg^2/dL^2 | Standard Deviation 13.5196 |
| Group 3 - PT20 1600 mg Tid | Change in Calcium x Phosphate Product From Baseline (Visit 7, Day 1) to Visit 11 (Day 29) | -11.127 mg^2/dL^2 | Standard Deviation 15.1683 |
| Group 4 - PT20 3200 mg Tid | Change in Calcium x Phosphate Product From Baseline (Visit 7, Day 1) to Visit 11 (Day 29) | -10.996 mg^2/dL^2 | Standard Deviation 19.3057 |
| Group 5 - Placebo Tid | Change in Calcium x Phosphate Product From Baseline (Visit 7, Day 1) to Visit 11 (Day 29) | -0.778 mg^2/dL^2 | Standard Deviation 10.286 |
Comparison: Repeated measures ANCOVA for change in Calcium x Phosphate Product from Baseline by PT20 dose group.p-value: 0.004ANCOVA
Comparison: Repeated measures ANCOVA for change in transferrin saturation from Baseline by Baseline Calcium x Phosphate Product.p-value: <0.001ANCOVA
Secondary
Change in Gastrointestinal Symptom Rating System (GSRS) Overall Score From Baseline (Visit 7, Day 1) to Visit 11 (Day 29)
The GSRS assesses the impact of treatment-related GI complications. It includes 15 items divided into 5 subscales (diarrhoea, indigestion, abdominal pain, constipation, and reflux), and uses a seven-grade Likert scale to assess symptoms (1 = no discomfort at all, 7 = very severe discomfort). The total score was calculated as the average score of all 15 items. If data were missing from one or more subscales, the mean of the completed items within the subscale was to be used for the subscale score, provided that more than half of the subscale items were complete. If more than half of the items within a subscale were missing, the subscale score and overall score were also to be defined as missing. The change in overall GSRS score from Baseline (Visit 7, Day 1) to Visit 11 (Day 29) was summarised by treatment and a two-sample t-test was to be used to identify differences between the placebo and PT20 dose groups. The higher the score, the more severe the gastrointestinal symptoms.
Time frame: Day 1 to Day 29
Population: Safety Set
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Group 1 - PT20 400 mg Tid | Change in Gastrointestinal Symptom Rating System (GSRS) Overall Score From Baseline (Visit 7, Day 1) to Visit 11 (Day 29) | -0.9 score on a scale | Standard Deviation 3.75 |
| Group 2 - PT20 800 mg Tid | Change in Gastrointestinal Symptom Rating System (GSRS) Overall Score From Baseline (Visit 7, Day 1) to Visit 11 (Day 29) | 2.8 score on a scale | Standard Deviation 5.97 |
| Group 3 - PT20 1600 mg Tid | Change in Gastrointestinal Symptom Rating System (GSRS) Overall Score From Baseline (Visit 7, Day 1) to Visit 11 (Day 29) | 0.4 score on a scale | Standard Deviation 9.8 |
| Group 4 - PT20 3200 mg Tid | Change in Gastrointestinal Symptom Rating System (GSRS) Overall Score From Baseline (Visit 7, Day 1) to Visit 11 (Day 29) | 1.6 score on a scale | Standard Deviation 8.48 |
| Group 5 - Placebo Tid | Change in Gastrointestinal Symptom Rating System (GSRS) Overall Score From Baseline (Visit 7, Day 1) to Visit 11 (Day 29) | 0.1 score on a scale | Standard Deviation 5.16 |
p-value: 0.292paired z-test
p-value: 0.047paired z-test
p-value: 0.872paired z-test
p-value: 0.288paired z-test
Secondary
Change in Haemoglobin Concentration From Baseline (Visit 7, Day 1) to Visit 11 (Day 29)
Descriptive statistics were to be used to summarise values and change from Baseline (Visit 7, Day 1) to Day 29 (Visit 11) in haemoglobin concentration. An analysis of covariance (ANCOVA) was to be used to analyse the changes from Baseline (Visit 7, Day 1) to Visit 11 (Day 29), which were to include the fixed, categorical effects of treatment as well as the continuous, fixed covariates of Baseline concentrations for haemoglobin. Only those subjects with available concentrations for both Baseline (Visit 7, Day 1) and Visit 11 (Day 29) were to be included in this analysis.
Time frame: Day 1 to Day 29
Population: ITT population.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Group 1 - PT20 400 mg Tid | Change in Haemoglobin Concentration From Baseline (Visit 7, Day 1) to Visit 11 (Day 29) | 0.02 g/dL | Standard Deviation 0.953 |
| Group 2 - PT20 800 mg Tid | Change in Haemoglobin Concentration From Baseline (Visit 7, Day 1) to Visit 11 (Day 29) | -0.25 g/dL | Standard Deviation 1.085 |
| Group 3 - PT20 1600 mg Tid | Change in Haemoglobin Concentration From Baseline (Visit 7, Day 1) to Visit 11 (Day 29) | -0.22 g/dL | Standard Deviation 0.958 |
| Group 4 - PT20 3200 mg Tid | Change in Haemoglobin Concentration From Baseline (Visit 7, Day 1) to Visit 11 (Day 29) | 0.18 g/dL | Standard Deviation 0.913 |
| Group 5 - Placebo Tid | Change in Haemoglobin Concentration From Baseline (Visit 7, Day 1) to Visit 11 (Day 29) | -0.13 g/dL | Standard Deviation 0.892 |
Comparison: Repeated measures ANCOVA for change in haemoglobin concentration from Baseline by PT20 dose group.p-value: 0.497ANCOVA
Comparison: Repeated measures ANCOVA for change in haemoglobin concentration from Baseline by baseline hemoglobin.p-value: <0.001ANCOVA
Secondary
Change in Serum Ferritin Concentration From Baseline (Visit 7, Day 1) to Visit 11 (Day 29)
Descriptive statistics were to be used to summarise values and change from Baseline (Visit 7, Day 1) to Day 29 (Visit 11) in serum ferritin concentration. An analysis of covariance (ANCOVA) was to be used to analyse the changes from Baseline (Visit 7, Day 1) to Visit 11 (Day 29), which were to include the fixed, categorical effects of treatment and week (Visit), as well as the continuous, fixed covariates of Baseline concentrations for serum ferritin. Only those subjects with available concentrations for both Baseline (Visit 7, Day 1) and Visit 11 (Day 29) were to be included in this analysis.
Time frame: Day 1 to Day 29
Population: ITT population.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Group 1 - PT20 400 mg Tid | Change in Serum Ferritin Concentration From Baseline (Visit 7, Day 1) to Visit 11 (Day 29) | 50 ng/mL | Standard Deviation 206.23 |
| Group 2 - PT20 800 mg Tid | Change in Serum Ferritin Concentration From Baseline (Visit 7, Day 1) to Visit 11 (Day 29) | 48.8 ng/mL | Standard Deviation 231.41 |
| Group 3 - PT20 1600 mg Tid | Change in Serum Ferritin Concentration From Baseline (Visit 7, Day 1) to Visit 11 (Day 29) | 10.1 ng/mL | Standard Deviation 107.05 |
| Group 4 - PT20 3200 mg Tid | Change in Serum Ferritin Concentration From Baseline (Visit 7, Day 1) to Visit 11 (Day 29) | 69.9 ng/mL | Standard Deviation 172.85 |
| Group 5 - Placebo Tid | Change in Serum Ferritin Concentration From Baseline (Visit 7, Day 1) to Visit 11 (Day 29) | -67.0 ng/mL | Standard Deviation 181.73 |
Comparison: Repeated measures ANCOVA for change in serum ferritin concentration from Baseline by PT20 dose group.p-value: 0.243ANCOVA
Comparison: Repeated measures ANCOVA for change in serum ferritin concentration from Baseline by Visit.p-value: 0.867ANCOVA
Comparison: Repeated measures ANCOVA for change in serum ferritin concentration from Baseline by baseline serum ferritin concentrationp-value: 0.186ANCOVA
Secondary
Change in Transferrin Saturation From Baseline (Visit 7, Day 1) to Visit 11 (Day 29)
Descriptive statistics were to be used to summarise values and change from Baseline (Visit 7, Day 1) to Day 29 (Visit 11) in transferrin saturation. An analysis of covariance (ANCOVA) was to be used to analyse the changes from Baseline (Visit 7, Day 1) to Day 29 (Visit 11), which were to include the fixed, categorical effects of treatment and week, as well as the continuous, fixed covariates of Baseline concentrations for transferrin saturation. Only those subjects with available concentrations for both Baseline (Visit 7, Day 1) and Visit 11 (Day 29) were to be included in this analysis.
Time frame: Day 1 to Day 29
Population: ITT population.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Group 1 - PT20 400 mg Tid | Change in Transferrin Saturation From Baseline (Visit 7, Day 1) to Visit 11 (Day 29) | 2.20 percent | Standard Deviation 8.649 |
| Group 2 - PT20 800 mg Tid | Change in Transferrin Saturation From Baseline (Visit 7, Day 1) to Visit 11 (Day 29) | 0.40 percent | Standard Deviation 11.856 |
| Group 3 - PT20 1600 mg Tid | Change in Transferrin Saturation From Baseline (Visit 7, Day 1) to Visit 11 (Day 29) | 5.84 percent | Standard Deviation 15.178 |
| Group 4 - PT20 3200 mg Tid | Change in Transferrin Saturation From Baseline (Visit 7, Day 1) to Visit 11 (Day 29) | -4.59 percent | Standard Deviation 14.735 |
| Group 5 - Placebo Tid | Change in Transferrin Saturation From Baseline (Visit 7, Day 1) to Visit 11 (Day 29) | -0.45 percent | Standard Deviation 8.754 |
Comparison: Repeated measures ANCOVA for change in transferrin saturation from Baseline by PT20 dose group.p-value: 0.068ANCOVA
Comparison: Repeated measures ANCOVA for change in transferrin saturation from Baseline by Visit.p-value: 0.013ANCOVA
Comparison: Repeated measures ANCOVA for change in transferrin saturation from Baseline by Baseline Transferrin Saturation.p-value: <0.001ANCOVA