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Open Label Trial on the Safety and Efficacy of Sym001 in the Treatment of Immune Thrombocytopenic Purpura (ITP)

Primary Purpose

Immune Thrombocytopenic Purpura

Status

Completed

Phase

Phase 2

Locations

International

Study Type

Interventional

Intervention

Sym001

About this trial

This is an interventional treatment trial for Immune Thrombocytopenic Purpura focused on measuring Idiopathic, ITP

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Confirmed presence of thrombocytopenia with platelet count < 30,000/mm3 at the pre-dose visit.
History of isolated ITP
RhD-positive serology.
Previous treatment and response to first line therapy for ITP

Exclusion Criteria:

Known clinical picture suggestive of other causes of thrombocytopenia, especially systematic 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.
Suspected infection with HIV, Hepatitis C, H. pylori.
Clinical splenomegaly
History of abnormal bone marrow examination.
Ongoing haemorrhage corresponding to a grade 3 or 4 on the WHO bleeding scale.
Underlying haemolytic condition
History of splenectomy.
Subject is pregnant, breast feeding or intends to become pregnant.

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Arm Description

Patients treated with Sym001

Outcomes

Primary Outcome Measures

Incidence and severity of Adverse Events (AEs), including Serious Adverse Events (SAEs)

Secondary Outcome Measures

Measurements of platelet counts

Full Information

NCT ID

NCT00718692

First Posted

July 18, 2008

Last Updated

January 28, 2019

Sponsor

Symphogen A/S

Collaborators

Swedish Orphan Biovitrum

1. Study Identification

Unique Protocol Identification Number

NCT00718692

Brief Title

Open Label Trial on the Safety and Efficacy of Sym001 in the Treatment of Immune Thrombocytopenic Purpura (ITP)

Official Title

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.

Study Type

Interventional

2. Study Status

Record Verification Date

January 2019

Overall Recruitment Status

Completed

Study Start Date

July 2008 (Actual)

Primary Completion Date

August 2011 (Actual)

Study Completion Date

October 2011 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Symphogen A/S

Collaborators

Swedish Orphan Biovitrum

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

This trial is designed as a multi-centre, single-dose, exploratory dose-finding, open label trial evaluating the safety and efficacy of Sym001 in 4-9 consecutive cohorts. Subjects will receive a single IV dose of Sym001.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Immune Thrombocytopenic Purpura

Keywords

Idiopathic, ITP

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

61 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Arm Type

Experimental

Arm Description

Patients treated with Sym001

Intervention Type

Drug

Intervention Name(s)

Sym001

Intervention Description

Each cohort will receive one single dose of Sym001 according the assigned dose level.

Primary Outcome Measure Information:

Title

Incidence and severity of Adverse Events (AEs), including Serious Adverse Events (SAEs)

Time Frame

6 weeks post dosing

Secondary Outcome Measure Information:

Title

Measurements of platelet counts

Time Frame

From day 1 through week 6

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Confirmed presence of thrombocytopenia with platelet count < 30,000/mm3 at the pre-dose visit. History of isolated ITP RhD-positive serology. Previous treatment and response to first line therapy for ITP Exclusion Criteria: Known clinical picture suggestive of other causes of thrombocytopenia, especially systematic 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. Suspected infection with HIV, Hepatitis C, H. pylori. Clinical splenomegaly History of abnormal bone marrow examination. Ongoing haemorrhage corresponding to a grade 3 or 4 on the WHO bleeding scale. Underlying haemolytic condition History of splenectomy. Subject is pregnant, breast feeding or intends to become pregnant.

Overall Study Officials: