| E.1 Medical condition or disease under investigation |
| E.1.1 | Medical condition(s) being investigated |
Chronic Kidney Disease
Secondary Hyperparathyroidism (SHPT) |
Enfermedad Renal Crónica
Hiperparatiroidismo Secundario (HPTS) |
|
| E.1.1.1 | Medical condition in easily understood language |
Chronic Kidney Disease
Secondary Hyperparathyroidism (SHPT) |
Enfermedad Renal Crónica
Hiperparatiroidismo Secundario (HPTS) |
|
| 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 | 13.1 |
| E.1.2 | Level | LLT |
| E.1.2 | Classification code | 10064848 |
| E.1.2 | Term | Chronic kidney disease |
| E.1.2 | System Organ Class | 10038359 - Renal and urinary disorders |
|
| E.1.2 Medical condition or disease under investigation |
| E.1.2 | Version | 13.1 |
| E.1.2 | Level | PT |
| E.1.2 | Classification code | 10020708 |
| E.1.2 | Term | Hyperparathyroidism secondary |
| E.1.2 | System Organ Class | 10014698 - Endocrine 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% |
| Demostrar la eficacia de cinacalcet para reducir el nivel de hormona paratiroidea intacta (PTHi) en plasma en un porcentaje >= 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 |
- Demostrar la eficacia de cinacalcet para reducir el nivel de PTHi en plasma a < =300 pg/ml (31,8 pmol/l)
- Demostrar el impacto de cinacalcet en el nivel de calcio en suero total corregido
- Demostrar el impacto de cinacalcet en el nivel de fósforo en suero
- Demostrar el impacto de cinacalcet en el producto de calcio x fósforo (Ca x P)
- Evaluar el impacto de cinacalcet en el crecimiento |
|
| E.2.3 | Trial contains a sub-study | Yes |
| E.2.3.1 | Full title, date and version of each sub-study and their related objectives |
| One substudy is described within the 20070208 protocol, Amendment 1, dated 01-Feb-2011. In a subset of subjects who agree to participate, bone densitometry measurements of the forearm will be obtained at Day 1 (baseline), week 30 and week 60. The objective is to assess the impact of cinacalcet on bone mineral density (BMD) measured at the mid shaft radius (among a subset of subjects). |
Hay un sub-estudio descrito en el protocolo del CT 20070208, Enmienda 1 de fecha 1 de febrero de 2011.
Cambio porcentual en la DMO medida en la mitad del radio desde el momento inicial hasta la semana 30 y la semana 60, para los sujetos que participen en los análisis DXA opcionales |
|
| 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. |
- De 6 a menos de 18 años de edad en la selección
- Diagnóstico de ERC y HPTS que reciban HD o DP durante un periodo >=2 meses antes de la aleatorización
-Peso seco >=12,5 kg en la selección
- El PTHi obtenido del laboratorio central debe ser >= 350pg/ml (37,1 pmol/l)
- El calcio en suero (corregido) obtenido del laboratorio central debe ser >=8,8 mg/dl (2,2 mmol/l)
- El fósoforo en suero obtenido del laboratorio central debe ser >= 4,0 mg/dl (1,3 mmol/l) para niños de 6 a menos de 12 años, o >= 3,5 mg/dl (1,1 mmol/l) para niños de 12 a menos de 18 años.
-Para los sujetos que ya estén recibiendo esteroles de la vitamina D (ya sea calcitriol o cualquier otro análogo sintético), una dosis estable definida como ausencia de cambio > 50% dentro de los 2 meses anteriores a la aleatorización.
- Para los sujetos que estén tomando hormonas del crecimiento, una dosis estable definida como ausencia de cambio > 20% dentro de los 2 meses anteriores a la aleatorización.
- Los sujetos que reciban medicación anticonvulsiva deben seguir una dosis estable durante 3 meses, y tener un nivel terapéutico sanguíneo del anticonvulsivo en el momento de la aleatorización
- Los padres o representante legal autorizado del paciente han proporcionado el consentimiento informado y, si lo exige el CEIC de acuerdo con los requisitos locales o nacionales, el niño ha dado su aprobación |
|
| 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. |
- Un estado clínico inestable a criterio del investigador.
- Se sometió a paratiroidectomía en los 6 meses anteriores de la fecha de aleatorización.
- Paratiroidectomía prevista en los 6 meses tras la aleatorización.
- Recibió tratamiento con cinacalcet en un mes antes de la aleatorización.
- Un nuevo brote de crisis convulsivas o el empeoramiento de crisis convulsivas ya existentes en los 6 meses antes de la primera dosis del PI.
- Fecha prevista para un trasplante de riñón de un donante vivo conocido que hace que la finalización del estudio sea poco probable.
Generales:
- Participa actualmente en otro estudio de fármaco o dispositivo en investigación, o han transcurrido menos de 30 días tras finalizar otro estudio con un fármaco o dispositivo, o recibir otro(s) producto(s) en investigación.
- Si el sujeto es sexualmente activo y no está dispuesto/a a usar métodos anticonceptivos eficaces durante el tratamiento y durante al menos un mes (sujetos de sexo femenino) y 3 meses (sujetos de sexo masculino) después del fin del tratamiento.
- Sujeto de sexo femenino que esté embarazada o en periodo de lactancia, o que tenga intención de quedarse embarazada durante el estudio o en un periodo de 1 mes después del fin de tratamiento.
- Sujetos de sexo masculino con pareja embarazada que no estén dispuestos a usar preservativo durante el tratamiento y durante al menos 1 mes después del fin del tratamiento.
- El sujeto tiene una sensibilidad conocida a cualquiera de los productos que se administrarán durante la dosificación.
- El sujeto ha sido incluido previamente en este estudio.
- El sujeto no estará disponible para cumplir con todas las visitas que requiere el protocolo, según el leal saber y entender del sujeto y el investigador.
- Los padres o representante legal del sujeto tienen algún tipo de trastorno que, a criterio del investigador, podría comprometer la capacidad de proporcionar un permiso por escrito del consentimiento informado. |
|
| 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 |
-Consecución de una reducción >=30% desde el momento basal en la PTHi en plasma durante la FEE
|
|
| E.5.1.1 | Timepoint(s) of evaluation of this end point |
| At weeks 25, 27 & 29 |
| En las semanas 25, 27 y 29 |
|
| 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 |
-Consecución de un valor medio de PTHi de <=300 pg/ml (31,8 pmol/l) durante la FEE
-Cambio porcentual en el nivel de calcio sérico total corregido desde el momento basal al valor medio durante la FEE.
-Cambio porcentual en el nivel de fósforo en sérico desde el momento basal al valor medio durante la FEE.
-Cambio porcentual en el producto del calcio y fósforo (Ca x P) desde el momento basal al valor medio durante la FEE.
- Velocidad de crecimiento calculada desde el momento inicial hasta la semana 30, y desde la semana 30 hasta la semana 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. |
- iPTH durante FEE en las semanas 25, 27 y 29
- Cambio porcentual en el serum de calcio y de fósforo y cambio porcentual en el producto del calcio y fósforo (Ca x P) desde el momento basal a las semanas 25, 27 y 29
- Velocidad de crecimiento calculada desde el momento inicial hasta la semana 30, y desde la semana 30 hasta la semana 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 | Yes |
| E.8.4.1 | Number of sites anticipated in Member State concerned | 4 |
| 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 |
| Finland |
| 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. |
| La finalización primaria del estudio se define como la fecha en que el último sujeto completa la última evaluación del estudio, incluidos los análisis de sangre de laboratorio para determinar los parámetros de eficacia o seguridad y las visitas de seguimiento para comprobar la resolución de los acontecimientos adversos |
|
| E.8.9 Initial estimate of the duration of the trial |
| E.8.9.1 | In the Member State concerned years | 4 |
| E.8.9.1 | In the Member State concerned months | 0 |
| E.8.9.1 | In the Member State concerned days | 0 |
| 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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