CompletadoNCT03531255Registro de ClinicalTrials.gov

Pegcetacoplan Long Term Safety and Efficacy Extension Study

PNH

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LO ESENCIAL

De qué trata este estudio

El registro no identifica actualmente este estudio como reclutando. Consulta su estado antes de planificar una evaluación.

Tipo de estudio: De intervención
Fase: Fase 3
Rango de edad: 18 años - No especificado
Sexo admitido: Todos los sexos
Voluntarios sanos: No aceptados
Participantes: 137 Real

Estos son puntos de partida, no una decisión de elegibilidad. El equipo debe revisar todos los requisitos.

Las explicaciones y la navegación están en español. Los textos proporcionados por el registro, incluidos títulos, resúmenes y criterios, se conservan en su idioma original. Puedes pedir orientación en español a WCR.

Acerca de la investigación

This is an Open-label, Non-Randomized, Multi-Center Extension Study. Eligible subjects will have previously completed a pegcetacoplan study.

¿Quién podría participar?

La lista siguiente contiene el texto del patrocinador. Puede incluir condiciones, tratamientos previos, resultados de pruebas y otros requisitos. Aparecer en tu búsqueda no significa que califiques.

Inclusion criteria:

1. Subjects at least 18 years of age with PNH who have participated in a pegcetacoplan clinical trial. Subjects who received treatment with pegcetacoplan must have experienced clinical benefit and adequate tolerability in the opinion of the investigator.

Note: Subjects with PNH who completed a pegcetacoplan clinical trial without receiving pegcetacoplan (or without receiving pegcetacoplan for long enough to demonstrate clinical benefit) may be enrolled in this study if, in the opinion of the Investigator, the subject is expected to demonstrate clinical benefit upon the initiation or continuation of pegcetacoplan therapy.
2. Vaccination against Neisseria meningitidis types A, C, W, Y and B, Streptococcus pneumoniae and Haemophilus influenzae Type B (Hib) either within 2 years prior to Day 1 dosing of this study, or within 14 days after starting treatment with pegcetacoplan. Vaccination is mandatory unless documented evidence exists that subjects are nonresponders to vaccination as evidenced by titers or display titer levels within acceptable local limits. Immunization status checks will be performed to determine whether subjects require primary or booster vaccinations.
3. Willing and able to give written informed consent.
4. Willing and able to self-administer pegcetacoplan (administration by caregiver will be allowed)
5. Women of childbearing potential (WOCBP) defined as any females who have experienced menarche and who are NOT permanently sterile or postmenopausal must have a negative pregnancy test and must agree to continue to use an approved method of contraception for the duration of the study and 90 days after their last dose of study drug. Note: Postmenopausal is defined as 12 consecutive months with no menses without an alternative medical cause.
6. Males must agree to continue to use an approved method of contraception and must agree to refrain from donating sperm for the duration of the study and 90 days after their last dose of study drug.

Exclusion Criteria:

1. Subjects who have withdrawn from a pegcetacoplan clinical study.
2. Any condition that could increase the subject's risk by participating in the study.
3. Any comorbidity or condition (such as malignancy) that, in the opinion of the investigator, could put the subject at increased risk or potentially confound study data.
4. History or presence of hypersensitivity or idiosyncratic reaction to compounds related to the investigational product or SC administration.
5. Known infection with hepatitis B, C, or HIV.
6. Hereditary complement deficiency.
7. History of bone marrow transplant.
8. Concurrent severe aplastic anemia (SAA), defined as currently receiving immunosuppressive therapy for SAA including but not limited to cyclosporin A, tacrolimus, mycophenolate mofetil or anti-thymocyte globulin.
9. History of meningococcal disease.
10. Concomitant treatment with any complement inhibitor (eg, eculizumab, ravulizumab).
11. Pregnancy, breastfeeding, or positive pregnancy test.

Entiende la evaluación inicial

Tratamiento, grupos y participación

El registro puede describir tratamientos y duración sin incluir un calendario completo. Pide al equipo las visitas, procedimientos, riesgos, costos y alternativas. Mantén tu atención habitual salvo que tu médico te indique un cambio.

Asignación: No aplica
Enmascaramiento: Sin enmascaramiento

1,080 mg pegcetacoplan administered subcutaneously

1,080mg pegcetacoplan administered subcutaneously twice weekly or every three days.

Drug: Pegcetacoplan

Pegcetacoplan DRUG

Complement (C3) Inhibitor

Prepara tus preguntas

Qué evalúan los investigadores

Estas medidas indican qué se estudia. No prueban que un tratamiento funcione.

Medidas principales

Number of Participants With Treatment Emergent Adverse Events (TEAEs)

Plazo: From first dose of study drug up to 8 weeks from last dose (up to approximately 4 years or until pegcetacoplan development in participants with PNH was terminated)

An adverse event (AE) is any untoward medical occurrence associated with use of a drug in humans, whether or not considered drug related. An AE can therefore be any unfavorable and unintended sign, including a clinically significant abnormal laboratory finding, symptom, or disease temporally associated with the use of a study drug, whether or not considered related to the study drug. TEAEs were defined as those AEs that occurred or worsened in severity after the first dose of pegcetacoplan.

Number of Participants With Thromboembolic Events

Plazo: From first dose of study drug up to 8 weeks from last dose (up to approximately 4 years or until pegcetacoplan development in participants with PNH was terminated)

No informado en este registro.

Change From Baseline in Hematology Parameters (Erythrocytes)

Plazo: Baseline, Weeks 168 and 264

No informado en este registro.

Change From Baseline in Hematology Parameters (Leukocytes, Platelets and Reticulocytes)

Plazo: Baseline, Weeks 168 and 264

No informado en este registro.

Change From Baseline in Hematology Parameters (Hematocrit)

Plazo: Baseline, Weeks 168 and 264

No informado en este registro.

Change From Baseline in Hematology Parameters (Hemoglobin)

Plazo: Baseline, Weeks 168 and 264

No informado en este registro.

Change From Baseline in Serum Chemistry Parameters [Alanine Aminotransferase, Alkaline Phosphatase, Aspartate Aminotransferase, Lactate Dehydrogenase (LDH)]

Plazo: Baseline, Weeks 168 and 264

No informado en este registro.

Change From Baseline in Serum Chemistry Parameters (Bilirubin)

Plazo: Baseline, Weeks 168 and 264

No informado en este registro.

Number of Participants With Post-Baseline Potentially Clinically Significant Abnormal Coagulation Parameters

Plazo: Baseline, Week 264

Coagulation parameters included Activated Partial Thromboplastin Time (APTT), D-Dimer, Fibrinogen, Prothrombin Intl. Normalized Ratio and Prothrombin Time.

Number of Participants With Post-Baseline Potentially Clinically Significant Abnormal Urinalysis Parameters

Plazo: Baseline, Week 264

Urinalysis parameters included ketones, leukocyte esterase, nitrite, occult blood, pH, protein, specific gravity and urobilinogen.

Number of Participants With Shift From Baseline in Clinically Significant Abnormalities in Electrocardiograms (ECG)

Plazo: Baseline, Week 324

ECG measurements included heart rate, PR, QT, QRS, QTcB and QTcF.

Change From Baseline in Hemoglobin Concentration

Plazo: Baseline, Week 324

No informado en este registro.

Change From Baseline in Lactate Dehydrogenase (LDH) Concentration

Plazo: Baseline, Week 324

No informado en este registro.

Change From Baseline in Indirect Bilirubin Level

Plazo: Baseline, Week 324

No informado en este registro.

Change From Baseline in Absolute Reticulocyte Count (ARC)

Plazo: Baseline, Week 324

No informado en este registro.

Number of Participants Who Received Packed Red Blood Cell Transfusions (PRBCs)

Plazo: From first dose of study drug up to 8 weeks from last dose (up to approximately 4 years or until pegcetacoplan development in participants with PNH was terminated)

Transfusion included whole blood, PRBCs, leukocyte-depleted red blood cells (LDPRCs), and all types of red blood cell (RBC) transfusions.

Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score

Plazo: Baseline, Week 324

The FACIT-Fatigue scale consists of 13 questions that assess fatigue and its impact on daily activities over the past 7 days. Each question is scored on a 5-point scale from 0 ("not at all") to 4 ("very much"). For items where a higher response indicates greater fatigue, the score is reversed so that all items are scored in the same direction. The scores from the 13 items are then added together to calculate the FACIT-Fatigue total score. The total score ranges from 0 to 52, with higher scores indicating less fatigue and better functioning.

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Centros de investigación (62)

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Buscar un centro en este estudio

62 centros

University of Southern CaliforniaLos Angeles, California, United StatesEstado desconocido

De qué trata este estudio

Acerca de la investigación

¿Quién podría participar?

Tratamiento, grupos y participación

Qué evalúan los investigadores

Medidas principales

Centros de investigación (62)

Equipo y fechas

Contactos oficiales del estudio

Consulta a Wellness Clinical Research