E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Treatment of hyperphosphataemia |
|
E.1.1.1 | Medical condition in easily understood language |
Patients who have kidneys that do not work properly are not able to control the level of serum phosphorus in their blood. The amount of phosphate then rises. |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Nutritional and Metabolic Diseases [C18] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 16.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10064848 |
E.1.2 | Term | Chronic kidney disease |
E.1.2 | System Organ Class | 100000004857 |
|
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
In hyperphosphatemic pediatric patients with chronic kidney disease (CKD) to:
• Evaluate the safety and tolerability of sevelamer carbonate
• Evaluate the efficacy of sevelamer carbonate on the control of serum phosphorus |
|
E.2.2 | Secondary objectives of the trial |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. The patient and/or their parent/legal guardian are willing and able to provide signed informed consent.
2. The patient is 0 to < 19 years old at Visit 1.
3. The patient has CKD requiring dialysis or CKD not on dialysis with an estimated GFR <60 mL/min/1.73m2 based on central laboratory results.
• Patients taking phosphate binder(s) at Screening: At Visit 1a
• Patients not taking phosphate binder(s) at Screening: At Visit 1
4. The patient has a serum phosphorus level greater than the age appropriate upper limit of normal based on central laboratory results (Table 1).
• Patients taking phosphate binder(s) at Screening: At or between Visit 1a and Visit 1b
• Patients not taking phosphate binder(s) at Screening: At Visit 1
5. The patient, if female with reproductive capacity, has a negative pregnancy test based on central laboratory results.
• Patients taking phosphate binder(s) at Screening: At Visit 1a
• Patients not taking phosphate binder(s) at Screening: At Visit 1 |
|
E.4 | Principal exclusion criteria |
1. The patient has active dysphagia, swallowing disorders or a predisposition to or current bowel obstruction, ileus or severe gastrointestinal motility disorder(s) including severe constipation, or major GI tract surgery.
2. The patient has a non-renal cause of hyperphosphatemia.
3. The patient has participated in a study of an investigational drug during the 30 days preceding the start of the Screening Period.
4. The patient has a history of, or active, ethanol or drug dependence or abuse, excluding tobacco use.
5. The patient requires continuous tube feeds. Patients requiring bolus tube feeds are not excluded.
6. The patient has any evidence of active malignancy except for basal cell carcinoma of the skin. A history of malignancy is not an exclusion criterion.
7. The patient is receiving immunosuppressive medication for a functioning organ transplant.
8. The patient uses anti-arrhythmic medications for treatment of arrhythmias. Anti-arrhythmic medications used for other indications are not excluded by this criterion.
9. The patient uses anti-seizure medications for treatment of seizures. Anti-seizure medications used for other indications are not excluded by this criterion.
10. The patient has a known allergy to sevelamer or any of its constituents.
11. The patient is pregnant or breast-feeding, if female.
12. The patient is not willing to use birth control, if sexually active female patient with reproductive capacity.
13. The patient, in the opinion of the Investigator, does not require a phosphate binder.
14. The patient, in the opinion of the Investigator, has any clinically significant unstable medical condition.
15. The patient, in the opinion of the Investigator, is unable to adhere to the requirements of the study. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
The primary efficacy endpoint will be change from Baseline (Visit 2/Week 0) to Visit 3 in serum phosphorus. |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
E.5.2 | Secondary end point(s) |
The secondary efficacy endpoint will be change from Baseline to Visit 11/ET in serum phosphorus. |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
|
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 9 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
France |
Germany |
Lithuania |
Poland |
Turkey |
United States |
|
E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 2 |
E.8.9.1 | In the Member State concerned days | 26 |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 3 |
E.8.9.2 | In all countries concerned by the trial days | 25 |