Hemotrial Un proyecto de SEHH

Ensayo clínico

AN OPEN-LABEL, MULTI-CENTER CONTROLLED CLINICAL TRIAL OF ECULIZUMAB IN ADOLESCENT PATIENTS WITH PLASMA THERAPY-SENSITIVE ATYPICAL HEMOLYTIC UREMIC SYNDROME (AHUS)ESTUDIO CLÍNICO ABIERTO, MULTICÉNTRICO, CONTROLADO DE ADMINISTRACIÓN DE ECULIZUMAB EN PACIENTES ADOLESCENTES CON SÍNDROME HEMOLÍTICO URÉMICO ATÍPICO (SHUA) QUE RESPONDE A PLASMOTERAPIA

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Resumen

2017-03-15 04:10:22
2008-006955-28
C08-003B
AN OPEN-LABEL, MULTI-CENTER CONTROLLED CLINICAL TRIAL OF ECULIZUMAB IN ADOLESCENT PATIENTS WITH PLASMA THERAPY-SENSITIVE ATYPICAL HEMOLYTIC UREMIC SYNDROME (AHUS)ESTUDIO CLÍNICO ABIERTO, MULTICÉNTRICO, CONTROLADO DE ADMINISTRACIÓN DE ECULIZUMAB EN PACIENTES ADOLESCENTES CON SÍNDROME HEMOLÍTICO URÉMICO ATÍPICO (SHUA) QUE RESPONDE A PLASMOTERAPIA
C08-003B

PROMOTORES DEL ENSAYO
Nombre del promotor Organización Persona de contacto País
ALEXION PHARMACEUTICALS, INC. United States

Fármacos

  INFORMACIÓN DE FÁRMACOS USADOS:
  FÁRMACO 1:
Test SOLIRIS 300 mg concentrado para solución para perf
ALEXION EUROPE SAS
Concentrate for solution for infusion
Intravenous use

Detalles del Fármaco (Principio Activo):

h5G1.1-mAb ECULIZUMAB

Concentración del fármaco:

mg/ml milligram equal

10

Contenido del fármaco


No
Si

No
Information no disponible en EudraCT

No
Si

No
No

Information no disponible en EudraCT
No

No
No

No
  INFORMACIÓN DE PLACEBOS USADOS:

No hay placebos asignados al ensayo

Información General



Terapia Celular

Adolescent patients (from 12 and up to 18 years of age) with plasma therapy sensitive Atypical Hemolytic-Uremic Syndrome (aHUS).Pacientes adolescentes (de 12 a 18 años) con síndrome hemolítico urémico atípico (SHUa) que responde a plasmoterapia


Assess the effect of eculizumab on TMA-Event Free status defined as the absence of [1] decrease in platelet count of >25% from the Platelet Count Pre-PT Baseline Set-Point; [2] PT while the patient is receiving eculizumab, and [3] new dialysis for at least 12 weeks in adolescent patients with plasma therapy-sensitive Atypical Hemolytic-Uremic Syndrome (aHUS).

• Evaluate additional efficacy endpoints such as the effect of eculizumab on:- TMA Intervention Rate (# PT and # Dialysis Events/Patient/Day) during the Treatment Period compared with the TMA Intervention Rate prior to the first dose of Investigational Product.- Reduction of thrombotic microangiopathy (TMA) as indicated by thrombocytopenia as measured by platelet count change from baseline through the treatment period.- Key Hemolytic measures.- Quality of Life measures.- Renal function measures.- TMA Remission.• Characterize the overall safety and tolerability of eculizumab.• Describe the pharmacokinetics (PK) and pharmacodynamics (PD) of eculizumab in patients with aHUS.• Perform a series of exploratory efficacy analyses.

No


1. Male or female patients from 12 and up to 18 years of age who have been diagnosed with Atypical Hemolytic-Uremic Syndrome (aHUS).2. Patients must be receiving PT for aHUS and must be observed to (i) receive ?1 PT treatment every two weeks and no more than 3 PT treatments/week (at an unchanged frequency) for at least 8 weeks immediately prior to first dose of Investigational Product and (ii) receive the same volume of PP or PE and units of FFP for at least 8 weeks immediately prior to first dose of Investigational Product.3. Platelet Count Pre-PT Baseline Set-Point (collected immediately prior to the Qualifying PT Episode) is within 75% of the average of the pre-PT platelet counts collected at Screening and during the Observation Period.4. Known complement regulatory protein genetic abnormality, i.e., a mutation in Complement Protein 3, factor H or associated factor, factor I, or membrane cofactor protein 1 (MCP-1) or known Factor B gain-of- function mutation, or known anti-CFH antibody ("aHUS lesions").• Patients diagnosed with aHUS with any of these aHUS lesions are eligible and will be assigned to one of the following parallel categories during the treatment period of the trial:- (Category 1) Complement Protein 3 or factor H or factor I functional deficiency or abnormal factor interaction (C3/CFH/CFI FFP Group);- (Category 2) Factor B Gain of Function;- (Category 3) Anti-CFH Antibody (Anti-CFH Group);- (Category 4) MCP-1 deficiency (MCP-1 Group);5. Patients diagnosed with HUS of the atypical type without documented complement regulatory protein genetic abnormality or known anti-CFH antibody are eligible if other etiologies of HUS have been ruled out as confirmed in the Exclusion Criteria (i.e., including Shiga-toxin negative, non-infectious, nondrug-exposure-related [e.g., cyclosporine]), no known HIV positivity, and anti-phospholipid antibody negative). Thrombotic thrombocytopenic purpura also must be ruled out (i.e., ADAMTS-13 activity must be > 5%; see Exclusion Criteria). Patients meeting these conditions will be assigned to Category 5. In addition, these patients will undergo genetic testing to determine if a mutation can be identified. If a mutation is identified, the patient will be reassigned to the appropriate category.6. Lactate dehydrogenase (LDH) level ?ULN.7. Creatinine level ? ULN for age.8. Female patients of childbearing potential must be practicing an effective, reliable and medically acceptable contraceptive regimen during the entire duration of the study, including the follow-up period.9. Patient's parents/legal guardian must be willing and able to give written informed consent and patients must be willing to give written informed assent (if applicable as determined by the IRB/IEC).10. Able and willing to comply with study procedures.

1. ADAMTS-13 inhibitor or deficiency (i.e., ADAMTS-13 activity <5%) as measured at the screening visit. 2. Malignancy.3. Typical HUS (Shiga toxin +).4. Known HIV infection.5. Identified drug exposure-related HUS.6. Infection-related HUS.7. Patients with a confirmed diagnosis of sepsis defined as positive blood cultures within 7 days of the screening visit and not treated with antibiotics to which the organism is sensitive.8. Presence or suspicion of active and untreated systemic bacterial infection that, in the opinion of the Investigator confounds an accurate diagnosis of aHUS or impedes the ability to manage the aHUS disease.9. Pregnancy or lactation.10. Unresolved meningococcal disease.11. Known Systemic Lupus Erythematosus (SLE) or antiphospholipid antibody positivity or syndrome.12. Any medical or psychological condition that, in the opinion of the investigator, could increase the patient's risk by participating in the study or confound the outcome of the study.13. Patients receiving IVIg or Rituximab therapy.14. Patients receiving other immunosuppressive therapies such as steroids, mTOR inhibitors or FK506 inhibitors are excluded unless: [1] part of a post-transplant anti-rejection regime, [2] patient has confirmed anti-CFH antibody requiring immunosuppressive therapy and [3] dose of such medications have been unchanged for at least 4 weeks prior to the screening period.15. Patients receiving Erythrocyte Stimulating Agents (ESAs) unless already on a stable dose for at least 4 weeks prior to the screening period.16. Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedures beginning 4 weeks prior to screening and throughout the entire trial.

The primary efficacy endpoint for this protocol is TMA-Event Free status defined as the absence of [1] decrease in platelet count of >25% from the Platelet Count Pre-PT Baseline Set-Point; [2] PT while the patient is receiving eculizumab, and [3] new dialysis for at least 12 weeks in adolescent patients with plasma therapysensitive Atypical Hemolytic-Uremic Syndrome (aHUS). Dialysis events occurring within the 14 days after the first dose of Investigational Product will not be considered as a new Treatment Period dialysis event. In addition, dialysis events that commence within the 14 days before the first dose of Investigational Product and continue up to 14 days after the first dose of Investigational Product will not be considered a new Treatment Period dialysis event;

Fase II
  DISEÑO DEL ENSAYO:

Si
No

Si
No

No
No

No
Si
  COMPARADOR DEL ENSAYO CONTROLADO:

No
No

Si
No

Si

Centros participantes:


Si
Information no disponible en EudraCT
  TIEMPO ESTIMADO DURACIÓN DEL ENSAYO:

1
0
  TIEMPO ESTIMADO DURACIÓN DEL ENSAYO EN ESPAÑA:


  POBLACIÓN DE PACIENTES EN EL ESTUDIO:
  Población de pacientes: 1

Rango de edad:


1
0

0

Sexo:


1
1

Número planeado de pacientes a incluir:


4

Para estudios internacionales:


8
15

Investigadores

  INVESTIGADORES QUE PARTICIPAN EN EL ESTUDIO

Estado actual


EC Finalizado



EC Finalizado