E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Friedreich Ataxia (FA) |
Ataxia de Friedreich |
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E.1.1.1 | Medical condition in easily understood language |
Friedreich ataxia is an autosomal recessive, neurodegenerative disease that primarily affects the nervous system and heart. |
La ataxia de Friedreich es una enfermedad neurodegenerativa autosómica recesiva que afecta principalmente al sistema nervioso y al corazón. |
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E.1.1.2 | Therapeutic area | Diseases [C] - Nervous System Diseases [C10] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10017374 |
E.1.2 | Term | Friedreich's ataxia |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To assess the long-term safety of vatiquinone in subjects with FA previously exposed to vatiquinone in Study PTC743-NEU-003-FA or Study PTC743-NEU-005-FA |
Evaluar la seguridad a largo plazo de la vatiquinona en personas con AF a las que previamente se ha administrado vatiquinona en el estudio PTC743-NEU-003-FA o PTC743-NEU-005-FA |
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E.2.2 | Secondary objectives of the trial |
To assess the effects of vatiquinone on the Modified Friedreich Ataxia Rating Scale (mFARS) in subjects with FA previously exposed to vatiquinone in Study PTC743-NEU-003-FA or Study PTC743-NEU-005-FA |
Evaluar los efectos de la vatiquinona según la escala modificada de clasificación de ataxia de Friedreich (EMCAF) en personas con AF a las que previamente se ha administrado vatiquinona en el estudio PTC743-NEU-003-FA o PTC743-NEU-005-FA |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Subject provides consent to participate.
2. Subjects with FA who completed and directly rolled over from a previous vatiquinone clinical study (PTC743-NEU-003-FA [ie, MOVE-FA] or PTC743-NEU-005-FA).
3. Males and females of childbearing potential must be willing to use an effective method of contraception (eg, implants, injectables, transdermal patch, combined oral contraceptives, barrier methods, and intrauterine devices) from the time consent is signed until 30 days after last dose of study drug or Early Termination Visit. Male subjects must agree not to donate sperm during the study and for at least 30 days after the last dose of study drug or Early Termination Visit. Note: Double-barrier method (ie, condom with spermicide) is required if no other methods of contraception are in use. |
1. La persona otorga su consentimiento para participar.
2. Personas con AF que han completado un estudio clínico anterior (PTC743-NEU-003-FA [p. ej., MOVE-FA] o PTC743-NEU-005-FA) y han pasado directamente al estudio actual.
3. Las mujeres y hombres fértiles deberán estar dispuestos a usar un método anticonceptivo efectivo (p. ej. implantes, inyectables, parche transdérmico, anticonceptivos orales combinados, métodos de barrera y dispositivos intrauterinos) a partir del momento en el que se firma el consentimiento hasta 30 días después de la última dosis del medicamento en estudio o de la visita de finalización anticipada. Los hombres deben comprometerse a no donar semen durante el estudio y durante al menos 30 días después de la última dosis del medicamento o de la visita de finalización anticipada. Nota: Si no se utiliza ningún otro método anticonceptivo, será obligatorio el uso de un método de doble barrera (p. ej., preservativo con espermicida). |
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E.4 | Principal exclusion criteria |
1. Current participation in any other interventional study
2. Pregnancy or breastfeeding |
1. Participación actual en cualquier otro estudio intervencional
2. Embarazo o lactancia |
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E.5 End points |
E.5.1 | Primary end point(s) |
Adverse events/serious adverse events (SAEs), laboratory abnormalities, vital signs, and electrocardiograms (ECGs) |
Acontecimientos adversos (AA)/acontecimientos adversos graves (AAG), anomalías de laboratorio, constantes vitales y electrocardiogramas (ECG) |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Frequencies of AEs will be tabulated by MedDRA System Organ Class, Preferred Term, severity, relationship to study drug, and seriousness. Vital signs, laboratory data, and ECG will be summarized by visit. Changes from baseline will be summarized by visit, where appropriate. |
La frecuencia de los AA se presentarán en tablas por la clasificación por órganos y sistemas del diccionario médico para actividades reguladoras (MedDRA), término de preferencia, severidad, relación con el medicamento del estudio y gravedad. Las constantes vitales, los datos de laboratorio y ECG se resumirán en la visita. Los cambios respecto a los valores iniciales se resumirán, en su caso, en la visita. |
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E.5.2 | Secondary end point(s) |
Change from baseline in the mFARS |
Cambio respecto al valor inicial en la EMCAF |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Modified Friedreich Ataxia Rating Scale scores and the changes from baseline will be summarized by visit based on the Safety Analysis Set. |
Los resultados de la escala modificada de ataxia de Friedreich y los cambios con respecto a los valores iniciales se resumirán en la visita en función del conjunto de análisis de seguridad. |
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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 | No |
E.8.1.1 | Randomised | No |
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 | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
Estudio de extensión a largo plazo en sujetos con AF que completaron el estudio PTC743-NEU-003-FA |
Long-Term extension study in subject with FA who completed prior PTC743-NEU-003-FA study |
|
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 1 |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 4 |
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 |
Australia |
Brazil |
Canada |
New Zealand |
United States |
France |
Spain |
Germany |
Italy |
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E.8.7 | Trial has a data monitoring committee | No |
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
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 3 |
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 | 3 |