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IDES IN ASYMPTOMATIC TTP PATIENTS

Phase 1
Conditions
Asymptomatic antibody-mediated thrombotic thrombocytopenic purpura (TTP) with low ADAMTS13 activity
MedDRA version: 19.0 Level: PT Classification code 10043648 Term: Thrombotic thrombocytopenic purpura System Organ Class: 10005329 - Blood and lymphatic system disorders
Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Registration Number
EUCTR2016-000249-30-GB
Lead Sponsor
Hansa Medical AB
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
ot Recruiting
Sex
Not specified
Target Recruitment
2
Inclusion Criteria

1.Ability to understand and must sign the informed consent form
2.Age 18 years or above
3.Patients diagnosed with acquired TTP with ADAMTS13 levels of = 10 % in clinical remission and with measurable or previously confirmed ADAMTS13 antibodies.
4.Females of childbearing potential and males must use highly effective contraception during the study and at least for 12 weeks after IdeS dosing
Highly effective contraception methods include:
a. Total abstinence (when this is in line with the preferred and usual lifestyle of the subject. Periodic abstinence (e.g. calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception.
b. Female sterilisation (have had surgical bilateral oophorectomy with or without hysterectomy) or tubal ligation at least six weeks before taking study treatment. In case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment.
c. Male sterilisation (at least 6 months prior to screening). For female subjects in the study, the vasectomised male partner should be the sole partner for that subject.
d. Combination of any two of the following methods (a+b or a+c or b+c):
d1) Use of oral, injected or implanted hormonal methods of contraception or other forms of hormonal contraception that have comparable efficacy (failure rate <1%), for example hormone vaginal ring or transdermal hormone contraception.
d2) Placement of an intrauterine device (IUD) or intrauterine system (IUS).
d3) Barrier methods of contraception: condom or occlusive cap (diaphragm or cervical/vault caps) with spermicidal foam/gel/film/cream/vaginal suppository.
In case of use of oral contraception women should have been stable on the same pill for a minimum of 3 months before taking study treatment. Women are considered post-menopausal and not of child bearing potential if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (e.g. age appropriate, history of vasomotor symptoms) or have had surgical bilateral oophorectomy (with or without hysterectomy) or tubal ligation at least six weeks prior to screening. In the case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment is she considered not of child bearing potential

Are the trial subjects under 18? no
Number of subjects for this age range: 0
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 6
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 0

Exclusion Criteria

1.Prior malignancy within 5 years excluding adequately treated basal cell or squamous cell skin cancer, cervical carcinoma in situ
2.Any positive result on screening for serum hepatitis B surface antigen, hepatitis C antibody and human immunodeficiency virus (HIV)
3.Clinical signs of ongoing infectious disease. This includes P-CRP >10.
4.Tested positive for IgE antibodies against IdeS as measured by ImmunoCap.
5.No secondary cause of TTP
6.Rituximab treatment or other antibody-based therapy within 7 days prior to IdeS dosing.
7.Treatment with investigational medicinal product within the last 12 weeks proceeding screening or longer if judged by the investigator to possibly influence the outcome of the current study.
8.Severe other conditions requiring treatment and close monitoring, e.g. cardiac failure > NYHA (New York Heart Association) grade 3, unstable coronary disease or oxygen dependent COPD
9.History of any other clinically significant disease or disorder which, in the opinion of the investigator, may either put the patient at increased risk because of participation in the study, or influence the results or the patient’s ability to participate in the study
10. Hypogammaglobulinemia defined as any values of P-total IgG less than 3 g/L
11.History of severe allergy/hypersensitivity or ongoing allergy/hypersensitivity, as judged by the investigator or history of hypersensitivity to drugs with a similar chemical structure or class to IdeS (e. g. streptokinase and/or staphylokinase)
12.Substance abuse or other concurrent medical condition that, in the investigator’s opinion, could confound study interpretation or affect the patient’s ability to tolerate or complete the study
13.Investigator considers patient unlikely to comply with study procedures, restrictions and requirements
14.Breast feeding women or women with a positive pregnancy test
15.Previously received IdeS treatment

Study & Design

Study Type
Interventional clinical trial of medicinal product
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Secondary Outcome Measures
NameTimeMethod
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