| E.1 Medical condition or disease under investigation |
| E.1.1 | Medical condition(s) being investigated |
Chronic Kidney Disease
Secondary Hyperparathyroidism (SHPT) |
|
| E.1.1.1 | Medical condition in easily understood language |
Chronic Kidney Disease
Secondary Hyperparathyroidism (SHPT) |
|
| E.1.1.2 | Therapeutic area | Diseases [C] - Hormonal diseases [C19] |
| MedDRA Classification |
| E.1.2 Medical condition or disease under investigation |
| E.1.2 | Version | 14.0 |
| E.1.2 | Level | LLT |
| E.1.2 | Classification code | 10064848 |
| E.1.2 | Term | Chronic kidney disease |
| E.1.2 | System Organ Class | 10038359 - Renal and urinary disorders |
|
| 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 |
To demonstrate the efficacy of cinacalcet for reducing the plasma intact parathyroid hormone
level (iPTH) by ≥ 30% |
|
| E.2.2 | Secondary objectives of the trial |
• To demonstrate the efficacy of cinacalcet for lowering the plasma iPTH level to
≤ 300 pg/mL (31.8 pmol/L)
• To demonstrate the impact of cinacalcet on corrected total serum calcium level
• To demonstrate the impact of cinacalcet on serum phosphorus level
• To demonstrate the impact of cinacalcet on the calcium x phosphorus product (Ca x P)
• To assess the impact of cinacalcet on growth
|
|
| E.2.3 | Trial contains a sub-study | No |
| E.3 | Principal inclusion criteria |
- Age 6 to less than 18 years at screening
- Diagnosed with CKD and SHPT receiving HD or PD for ≥ 2 months before
randomization
- Dry weight ≥ 12.5 kg at screening
- iPTH obtained from the central laboratory must be ≥ 350 pg/mL (37.1 pmol/L)
- Serum calcium (corrected) obtained from the central laboratory must be
≥ 8.8 mg/dL (2.2 mmol/L)
- Serum phosphorus obtained from the central laboratory ≥ 4.0 mg/dL
(1.3 mmol/L) for children 6 to less than 12 years old, or ≥ 3.5 mg/dL
(1.1 mmol/L) for children 12 to less than 18 years old
- For subjects already receiving vitamin D sterols (either calcitriol or a synthetic
analog thereof), a stable dose defined as no change > 50% within the
preceding 2 months from randomization
- For subjects taking growth hormone, a stable dose defined as no change
> than 20% within the preceding 2 months prior to randomization
- Subjects on anti-convulsant medication must be on a stable dose for
3 months, and have a therapeutic blood level of the anti-convulsant at the
time of randomization
- The parent or legally acceptable representative of the patient has provided
informed consent and if required by the IRB/IEC according to local or country
requirements, child has provided assent. |
|
| E.4 | Principal exclusion criteria |
- An unstable medical condition in the judgment of the investigator
- Underwent parathyroidectomy in the 6 months before the date of
randomization
- Anticipated parathyroidectomy within 6 months after randomization
- Received therapy with cinacalcet within 1 month prior to randomization
- A new onset of seizure or worsening of a pre-existing seizure disorder within
6 months prior to first dose of IP
- Scheduled date for kidney transplant from a known living donor that makes
completion of the study unlikely.
General:
- Currently enrolled in another investigational device or drug study, or less than
30 days since ending another investigational device or drug study(s), or
receiving other investigational agent(s)
- If sexually active, subject is not willing to use highly effective contraception
during treatment and for at least one month (females) and 3 months (males)
after the end of treatment
- Subject is pregnant or breast feeding, or planning to become pregnant during
study or within 1 month after the end of treatment
- Male subject with a pregnant partner who is not willing to use a condom
during treatment and for at least 1 month after the end of treatment
- Subject has known sensitivity to any of the products to be administered
during dosing
- Subject previously has entered this study
- Subject will not be available for protocol-required study visits, to the best of
the subject and investigator’s knowledge
- Subject’s parent or legal representative has any kind of disorder that, in the
opinion of the investigator, may compromise the ability to give written
parental permission for informed consent.
|
|
| E.5 End points |
| E.5.1 | Primary end point(s) |
• Achievement of a ≥ 30% reduction from baseline in mean plasma iPTH during
the EAP |
|
| E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
| E.5.2 | Secondary end point(s) |
• Achievement of a mean iPTH value ≤ 300 pg/mL (31.8 pmol/L) during the EAP
• Percent change in corrected total serum calcium from baseline to the mean value
during the EAP
• Percent change in serum phosphorus from baseline to the mean value
during the EAP
• Percent change in calcium and phosphorus product (Ca x P) from baseline to the
mean value during the EAP
• Growth velocity calculated from baseline to Week 30, and from Week 30 to
Week 60 |
|
| E.5.2.1 | Timepoint(s) of evaluation of this end point |
- iPTH during EAP at weeks 25, 27 & 29.
- Percent change in serum calcium, in serum phosphorus and in calcium and phosphorus product (Ca x P) from baseline to weeks 25, 27 & 29
- Growth velocity from baseline to week 30, and from week 30 to week 60. |
|
| 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) | No |
| E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
| 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 | No |
| E.8.1.3 | Single blind | No |
| E.8.1.4 | Double blind | Yes |
| 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 | Information not present in EudraCT |
| E.8.5 | The trial involves multiple Member States | Yes |
| E.8.5.1 | Number of sites anticipated in the EEA | 25 |
| 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 |
| Argentina |
| Australia |
| Belgium |
| Brazil |
| Germany |
| Hungary |
| Mexico |
| Peru |
| Poland |
| Russian Federation |
| Slovakia |
| Spain |
| 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
|
Primary completion is defined as the date on which the last subject completes the last study assessment or last visit, including laboratory blood draws for determination of
efficacy and/or safety parameters, and follow-up visits for monitoring resolution of adverse events. |
|
| E.8.9 Initial estimate of the duration of the trial |
| E.8.9.1 | In the Member State concerned years | |
| E.8.9.1 | In the Member State concerned months | |
| E.8.9.1 | In the Member State concerned days | |
| E.8.9.2 | In all countries concerned by the trial years | 4 |
| E.8.9.2 | In all countries concerned by the trial months | 0 |
| E.8.9.2 | In all countries concerned by the trial days | 0 |
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