Hemotrial Un proyecto de SEHH

Ensayo clínico

A multi-centre, single intravenous dose, exploratory dose-finding, open label trial on the safety and efficacy of Sym001 in the treatment of Immune Thrombocytopenic Purpura (ITP) in RhD positive, non-splenectomized adult subjects."Estudio abierto, multicéntrico, única dosis intravenosa, de búsqueda de dosis para determinar la seguridad y eficacia de Sym001 en el tratamiento de la púrpura trombocitopénica inmune (PTI) en pacientes no esplenectomizados y RhD positivos"

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Resumen

2017-03-15 04:06:49
2007-006081-15
Sym001-03
A multi-centre, single intravenous dose, exploratory dose-finding, open label trial on the safety and efficacy of Sym001 in the treatment of Immune Thrombocytopenic Purpura (ITP) in RhD positive, non-splenectomized adult subjects."Estudio abierto, multicéntrico, única dosis intravenosa, de búsqueda de dosis para determinar la seguridad y eficacia de Sym001 en el tratamiento de la púrpura trombocitopénica inmune (PTI) en pacientes no esplenectomizados y RhD positivos"
N/A
Sym001-03

PROMOTORES DEL ENSAYO
Nombre del promotor Organización Persona de contacto País
Symphogen A/S Denmark

Fármacos

  INFORMACIÓN DE FÁRMACOS USADOS:
  FÁRMACO 1:
Test
sym001 Solution for injection
Intravenous use

Detalles del Fármaco (Principio Activo):
Sym001

Concentración del fármaco:

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

Trombopenia

Immune Thrombocytopenic Purpura (ITP) in RhD positive, non-splenectomized adult subjects.Púrpura trombocitopénica inmune (PTI) en pacientes adultos no esplenectomizados y RhD positivos

To evaluate the safety of Sym001 following a single intravenous dose, at multiple dose levels in the treatment of ITP in RhD positive, non-splenectomized adult subjects.

To evaluate the efficacy of Sym001 following a single intravenous dose in the treatment of ITP in RhD positive, non-splenectomized adult subjects.To evaluate the binding of Sym001 to RBCs following a single intravenous dose of Sym001 in the treatment of ITP in RhD positive, non-splenectomized adult subjects.To evaluate an appropriate dose range of Sym001 for Phase 3.

No


1. Informed consent obtained before any trial-related procedures.2. Age 18-75 inclusive.3. Confirmed presence of thrombocytopenia with platelet count < 30,000/mm3 at the pre-dose visit. In each cohort a maximum of 3 subjects will be included with platelet counts < 10,000/mm3, in order to limit variability. The average of 2 individual pre-dose platelet counts taken on the dosing day and within > 1 hour interval between each other and both being < 30,000/mm3 will be used to confirm thrombocytopenia.4. History of isolated ITP (thrombocytopenia with no known aetiology, blood smear showing normal appearing platelets or, if performed, a bone marrow showing adequate megacariocytosis and normal erythroid and myeloid morphology).5. RhD-positive serology.6. Previous treatment and response to first line therapy for ITP (corticosteroids, anti-D or IVIg), with response being defined as an increase in platelet count to ? 50,000/mm3.7. female and of child-bearing potential, subject has a negative pregnancy test at screening.8. If subject is a female of child-bearing potential, subject agrees to use a medically accepted form of contraception from the time of enrolment to completion of all follow-up trial visits.

1. Known clinical picture suggestive of other causes of thrombocytopenia, especially systemic lupus erythematosus, antiphospholipid syndrome, Evans syndrome, immunodeficiency states, lymphoproliferative disorders, liver disease, ingestion of drugs such as quinidine/quinine, heparin and sulfonamides and hereditary thrombocytopenia confirmed by relevant laboratory findings.2. Suspected infection with HIV, hepatitis C, H. pylori unless corresponding laboratory tests are negative.3. Clinical splenomegaly (the spleen should not be palpable at more than 1 finger breadth below the costal margin).4. History of abnormal bone marrow examination. 5. At pre-dose visit: bleeding risk requiring emergency treatment for ITP or an ongoing haemorrhage requiring medical intervention.6. History of anaphylaxis or hypersensitivity reactions following IVIg or anti-D treatment.7. History of immune haemolytic anaemia.8. Underlying haemolytic condition (e.g. reticulocyte count > 3%).9. Planned surgery during the trial period.10. Haemoglobin pre-dose value lower than 2g/dL below the lower limit of the laboratory normal range for gender and age.11. History of splenectomy.12. Known malignancy (except basocellular carcinoma).13. Received investigational agent within 90 days prior to enrolment.14. Positive DAT (direct Coombs-test) at screening unless subject has received treatment with anti-D products within 3 months prior to screening with prior negative DAT.15. Known non-responders to most recent anti-D treatment (despite any initial response to treatment). 16. Any other current treatment for ITP except corticosteroids at doses up to 20 mg/day. (Acceptable if the dose has been constant for 2 weeks or more before trial drug administration).17. Therapy with IVIg, anti-D or any other treatment of ITP within 4 weeks prior to enrolment. 18. Therapy with alkylating agents or rituximab within 8 weeks prior to enrolment 19. Any antithrombotic treatment (except acetyl salicylic acid at doses up to 150 mg daily) within 7 days prior to pre-dosing visit or planned during the trial.20. PT/INR and aPTT out of normal range at the pre-dose visit21. History of thrombosis or known thrombophilia.22. Diseases other than ITP that may influence the result of the trial as judged by the Investigator.23. Creatinine, alanine aminotransferase (ALT), alkaline phosphatase (ALP), and Albumin 25% or more outside normal range value (according to the local laboratory) at pre-dose visit.24. Current or planned treatment with erythropoietin.25. Subject is pregnant, breast feeding or intends to become pregnant.

Incidence and severity of Adverse Events (AEs), including Serious Adverse Events (SAEs), Serious Drug Reactions (SDRs) and Adverse Events of Special Interest (AESIs) during the 6-week trial period.

Fase II
  DISEÑO DEL ENSAYO:

No
No

No
No

No
No

No
No
  COMPARADOR DEL ENSAYO CONTROLADO:

No
No

No
No

Si

Centros participantes:

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


14
  TIEMPO ESTIMADO DURACIÓN DEL ENSAYO EN ESPAÑA:


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

Rango de edad:

0
1

1

Sexo:

1
1

Número planeado de pacientes a incluir:

15

Para estudios internacionales:

55
55

Investigadores

  INVESTIGADORES QUE PARTICIPAN EN EL ESTUDIO

Estado actual


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