E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
immune thrombocytopenia (ITP) |
trombocitopenia inmunitaria (TPI) |
|
E.1.1.1 | Medical condition in easily understood language |
Blood and lymphatic diseases |
enfermedades hematológicas y limfáticas |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Blood and lymphatic diseases [C15] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 23.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10083842 |
E.1.2 | Term | Immune thrombocytopenia |
E.1.2 | System Organ Class | 10005329 - Blood and lymphatic system disorders |
|
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 evaluate the effect of BIVV020 on the durability of platelet |
• Evaluar el efecto de BIVV020 sobre la persistencia de la respuesta plaquetaria |
|
E.2.2 | Secondary objectives of the trial |
• To assess the safety and tolerability of BIVV020 • To assess the pharmacokinetics of BIVV020 • To assess the response rate of treatment with BIVV020 • To assess the time to response • To assess the effect of treatment with BIVV020 on the requirement for rescue ITP therapy • To assess the immunogenicity of BIVV020 |
• Evaluar la seguridad y tolerabilidad de BIVV020 • Evaluar la farmacocinética de BIVV020 • Evaluar la tasa de respuesta al tratamiento con BIVV020 • Evaluar el tiempo de respuesta • Evaluar el efecto del tratamiento con BIVV020 sobre la necesidad de tratamiento de rescate para la TPI • Evaluar la inmunogenicidad de BIVV020 |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Male and female participants ≥18 years of age at the time of signing the informed consent - Confirmed diagnosis of primary ITP; for participants who previously received sutimlimab in study TDR16218 (NCT03275454), a response to sutimlimab must have been obtained, as defined by platelet count ≥30 × 10^9/L on 2 visits at least 7 days apart - For participants who have not previously received sutimlimab: persistent/chronic ITP (ITP lasting for ≥6 months) and all the following conditions: a) Platelet count ≤30 × 10^9/L on 2 occasions at least 5 days apart during the Screening Period; b) Lack of an adequate platelet count response (as defined by maintenance of sustained platelet count ≥30 × 10^9/L in the absence of bleeding) to at least 2 of the following ITP treatments: IVIg, anti-D immunoglobulin, corticosteroids, splenectomy, rituximab, cyclophosphamide, thrombopoietin agonists, azathioprine, danazol, cyclosporin A, mycophenolate mofetil, or fostamatinib c) If receiving weekly thrombopoietin receptor agonist dosing, the last dose must have been administered ≥7 days before the first dose of BIVV020. If receiving daily thrombopoietin receptor agonist dosing, the last dose must have been administered ≥24 hours before the first dose of BIVV020 d) If applicable, concurrent administration of ITP medications (eg. corticosteroids, IVIg, azathioprine, danazol, cyclosporin A, mycophenolate mofetil, or thrombopoietin receptor agonists) is acceptable provided the patient has been on a stable dose for at least 1 month. e) If previously dosed with rituximab, the last dose of rituximab must have been administered at least 12 weeks before the first dose of BIVV020 - Documented vaccinations against encapsulated bacterial pathogens (Neisseria meningitidis, including serogroup B where available, Haemophilus influenzae, and Streptococcus pneumoniae) within 5 years of enrollment - Contraceptive use for women of childbearing potential and men who are sexually active with a female partner of childbearing potential |
- Los participantes de ambos sexos deben ser ≥18 años de edad en el momento de firmar el consentimiento informado. - Diagnóstico confirmado de TPI primaria; los participantes que hayan recibido previamente sutimlimab en el estudio TDR16218 deben haber mostrado una respuesta a sutimlimab, definida según un recuento plaquetario ≥30 × 109/l en 2 visitas al menos con 7 días de separación - Para los participantes que no hayan recibido previamente sutimlimab: TPI persistente/crónica (TPI durante ≥6 meses) y todas las condiciones siguientes: a. Recuento plaquetario ≤30 × 109/l en 2 ocasiones con al menos 5 días de separación durante la Fase de selección b. Falta de respuesta adecuada del recuento plaquetario (definida como un mantenimiento prolongado del recuento plaquetario ≥30 × 109/l en ausencia de hemorragia) al menos con 2 de los siguientes tratamientos para la TPI: IGIV, inmunoglobulina anti-D |
|
E.4 | Principal exclusion criteria |
Participants are excluded from the study if any of the following criteria apply: - Clinically significant medical history or ongoing chronic illness that would jeopardize the safety of the participant or compromise the quality of the data derived from his/her participation in the study - Clinical diagnosis of SLE - Clinically relevant infection within the month prior to enrollment - History of venous or arterial thrombosis within the year prior to enrollment - Secondary ITP from any cause including lymphoma, chronic lymphocytic leukemia, and drug-induced thrombocytopenia - Positive hepatitis B surface antigen (HBsAg) or active HCV infection - HIV infection - Pregnant or lactating women - Hemoglobin level <10 g/dL |
Se excluirá del estudio a los participantes que cumplan cualquiera de los siguientes criterios: Antecedentes médicos clínicamente significativos o enfermedad crónica en curso que ponga en peligro la seguridad del participante o la calidad de los datos derivados de su participación en el estudio. - Diagnóstico clínico de LES sistémico. - Infección de importancia clínica en el mes anterior a la inclusión - Antecedentes de trombosis venosa o arterial dentro del año anterior a la inclusión - TPI secundaria por cualquier causa, como linfoma, leucemia linfocítica crónica y trombocitopenia farmacógena. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
-Proportion of participants with a durable platelet Response. Durable platelet response is defined for naïve participants as the proportion of participants with a platelet count ≥50 × 109/L at ≥50% of scheduled visits, or for participants with baseline platelet count <15 × 109/L, a ≥20 × 109/L increase in platelet count from baseline at ≥50% of scheduled visits, without receiving rescue ITP therapy, as assessed from Week 3 to Week 24. Durable platelet response is defined for participants who previously received sutimlimab as maintenance of platelet count ≥30 × 109/L at ≥50% of scheduled visits, without receiving rescue ITP therapy, as assessed from Week 3 to Week 24. |
-Proporción de participantes con una respuesta plaquetaria duradera. La respuesta plaquetaria duradera se define para los participantes sin experiencia como la proporción de los participantes con un recuento plaquetario ≥50 × 109/l al alcanzar ≥50 % de las visitas programadas o, en los participantes con un recuento plaquetario inicial <15 × 109/l, un aumento ≥20 × 109/l del recuento plaquetario desde el inicio hasta ≥50 % de las visitas programadas, sin recibir tratamiento de rescate para la TPI, evaluado desde la Semana 3 hasta la Semana 24. La respuesta plaquetaria duradera se define para los participantes que recibieron previamente sutimlimab como mantenimiento del recuento plaquetario ≥30 × 109/l al alcanzar ≥50 % de las visitas programadas, sin recibir tratamiento de rescate para la TPI, evaluado desde la Semana 3 hasta la Semana 24 |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Week 3 to Week 24 |
De la semana 3 a la semana 24 |
|
E.5.2 | Secondary end point(s) |
1) Number of participants with treatment emergent adverse Events 2) Plasma concentrations of BIVV020 3) Response rate of treatment with BIVV020:Response rate at Weeks 24 and 52, defined as a platelet count ≥50 × 109/L and a greater than 2-fold increase from baseline, measured on 2 occasions at least 7 days apart, with the absence of bleeding (bleeding is defined as bleeding with a score ≥2 on the WHO bleeding scale), and the lack of combination ITP therapy during this period. 4)Time to first platelet Response: Time from baseline to first platelet response, defined as greater than or equal to each of the following values: 50 × 109/L and 100 × 109/L (confirmed by 2 measurements at least 7 days apart) 5) Proportion of patients who did not require rescue therapy for an acute episode of thrombocytopenia after Week 3 6) Number of participants with incidence and titer (if relevant) of anti-BIVV020 antibodies |
1) Número de participantes con acontecimientos adversos emergentes al tratamiento 2) Concentraciones plasmáticas de BIVV020 3) Tasa de respuesta en las Semanas 24 y 52, definida como un recuento plaquetario ≥50 × 109/l y un aumento superior al doble con respecto al inicio, medido en 02 ocasiones dejando al menos 7 días de separación, sin hemorragias (que se define como hemorragia con una puntuación ≥2 en la escala de hemorragias de la OMS) y en ausencia de tratamiento combinado para la TPI durante este periodo 4) Tiempo transcurrido desde el inicio hasta la primera respuesta plaquetaria, definido como un valor mayor o igual que cada uno de los siguientes: 50 × 109/l y 100 × 109/l (confirmado por 2 mediciones al menos con 7 días de separación) 5) Proporción de participantes que no necesiten tratamiento de rescate por episodios agudos de trombocitopenia después de la Semana 3 6) Número de participantes con incidencia y títulos (si es pertinente) de anticuerpos anti-BIVV020 |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
1)Week 52 2)Week 52 3)Weeks 24 and 52 4)Baseline to Week 52 5)From Week 3 to Week 52 6)Week 24, Week 52 |
1) Semana 52 2) Semana 52 3) Semanas 24 y 52 4) De la basal a la semana 52 5) De la semana 3 a la 52 6) Semana 24, Semana 52 |
|
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 | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
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 | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
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 | No |
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
| 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 | 3 |
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 |
United Kingdom |
Austria |
Belgium |
Czechia |
Germany |
Netherlands |
Spain |
|
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
|
LSLV |
Última visita del último paciente |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 8 |
E.8.9.1 | In the Member State concerned days | 9 |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 8 |
E.8.9.2 | In all countries concerned by the trial days | 9 |