Currently Enrolling
Interventional

VALIANT

Phase 3 Study Assessing the Efficacy and Safety of Pegcetacoplan in Patients with C3 Glomerulapathy (C3G) or Immune-Complex Membranoproliferative Glomerulonephritis (IC-MPGN)

Breve descripción

The purpose of this study is to assess the efficacy and safety of twice weekly pegcetacoplan compared to placebo in patients with C3G or IC-MPGN, on the basis of a reduction in urinary protein levels.

Médico del ensayo/Coordinador del estudio

Elizabeth Carfoli

Correo electrónico
Nombre del sitio

MedResearch Inc.
El Paso, TX

Matrícula estimada

Approximately 90

Fecha estimada de finalización

March 2025

Si no hay un lugar cercano para un ensayo clínico, puede preguntarle al equipo del estudio sobre la posibilidad de reembolsos de viaje (es decir, pagarle de regreso los costos de viaje). Alternativamente, puede preguntar sobre la posibilidad de participar desde casa.
Inscribiéndose actualmente
Intervencionista

VALIANT

Phase 3 Study Assessing the Efficacy and Safety of Pegcetacoplan in Patients with C3 Glomerulapathy (C3G) or Immune-Complex Membranoproliferative Glomerulonephritis (IC-MPGN)

Breve descripción

The purpose of this study is to assess the efficacy and safety of twice weekly pegcetacoplan compared to placebo in patients with C3G or IC-MPGN, on the basis of a reduction in urinary protein levels.

El ensayo es para personas con

Confirmed C3G or IC-MPGN, with or without prior renal transplantation. In addition, participants must:
• Be at least 12 years old
• Be diagnosed with C3G or IC-MPGN.
• Have significant proteinuria (protein in the urine) confirmed by urine collections during the screening process
• Have normal to moderately impaired kidney function, confirmed during the screening process
• Weigh 30kg to 100kg (66 – 220 pounds)
• Be willing to use contraception during and for a period after the study

Objetivo del estudio

The VALIANT study will assess whether pegcetacoplan, an investigational targeted C3 inhibitor, has an effect on the damage to kidney function that is caused by build-up of the complement protein C3 in the kidney.

Qué implica para el paciente?

After completing the screening period to confirm that the patient is able to participate in the study, individuals will be randomly assigned (randomized) to one of two treatment groups.
• Group 1: Will receive pegcetacoplan for the 12-month treatment period.
• Group 2: Will receive placebo for the first 6 months of the treatment period, and then receive pegcetacoplan for the remaining 6 months of the treatment period.
The placebo medication looks like the investigational medication but has no medically active ingredients. There is an equal chance (50%) of being assigned to Group 1 or Group 2 at the start of the study treatment period.
Study participants will not know their study treatment group assignment and they will not have the option of choosing their group.
Study participants who complete the 1-year study treatment period may enter a long-term extension study where they will continue receiving the investigational medication. Participants will enter an 8-week follow-up period after their last dose of the study medication.
Study participants will have approximately 14 in-clinic visits during the 1-year study treatment period and 2 visits during the 8-week follow-up period.
Main study assessments and procedures include, but are not limited to:
• Urine samples, including both samples collected at study visits and samples collected at home
• Adults only: Kidney biopsies (where possible, these will be combined with standard care biopsies)
• Blood samples
• Questionnaires
• Measurements of heart function (ECG)
• Chest X-rays
• Physical examinations
• Completion of an electronic diary to record dosing with study medication and home urine collections

Sobre el medicamento o la intervención

Pegcetacoplan is an investigational, targeted C3 inhibitor. It is administered via a needle, under the skin (subcutaneous), twice weekly at home. Investigational means that pegcetacoplan is not approved by any health authority for the treatment of C3G or IC-MPGN.

El Paso, TX
Preguntas frecuentes

El síndrome nefrótico no es una enfermedad en sí misma, sino un grupo de signos y síntomas que resultan del daño en la parte del riñón que filtra la sangre (glomérulos).

Los síntomas comunes incluyen:

  • Orina espumosa (llamada proteinuria) causada por el «derrame» de proteínas en la orina
  • Hinchazón severa en partes del cuerpo, más notable alrededor de los ojos, manos, pies y abdomen (llamado edema)
  • Aumento de peso debido a la acumulación de líquido adicional
  • Fatiga
  • Pérdida de apetito
  • Bajos niveles de proteínas en sangre (hipoalbuminemia)
  • Niveles de grasa y colesterol en sangre más altos de lo normal (hiperlipidemia)

El síndrome nefrótico generalmente se puede diagnosticar con un análisis de orina.

El síndrome nefrótico puede ser de naturaleza «primaria» o «secundaria».

Las enfermedades que afectan solo a los riñones se denominan causas primarias del síndrome nefrótico. Los médicos a menudo llaman a estas enfermedades «idiopáticas», lo que significa que surgen de una causa desconocida. Algunas de estas enfermedades incluyen:

  • Enfermedad de cambios mínimos (ECM/MCD): más común en los niños
  • Glomeruloesclerosis focal y segmentaria (GSFS/FSGS)
  • Nefropatía membranosa (NM/MN): más común en adultos
  • Nefropatía por IgA (IgAN)

El síndrome nefrótico secundario es causado por una condición sistémica subyacente como diabetes, lupus, VIH y otras.

Kidney Health Gateway es un sitio web que pertenece y es operado por NephCure Kidney International. El objetivo de este sitio web es ayudar a los pacientes con formas raras de síndrome nefrótico primario a conectarse con la atención de expertos y las opciones de tratamiento de vanguardia. Al responder algunas preguntas sobre usted o la condición de su ser querido, podemos brindarle una lista de ensayos clínicos o médicos expertos en su área.

Si tiene preguntas adicionales, visite NephCure.org o envíe un correo electrónico a Info@NephCure.org.

Ver otras preguntas frecuentes
Did you know that some forms of kidney disease can be genetic?Researchers are continually discovering genetic causes of Nephrotic Syndrome.

Learn more about genetic causes of kidney disease and find out if you may be affected.