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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: EUCTR
Last refreshed on: 5 November 2012
Main ID:  EUCTR2011-001354-29-ES
Date of registration: 19/07/2012
Prospective Registration: Yes
Primary sponsor: LFB BIOTECHNOLOGIES
Public title: An open-label, multicentre efficacy and safety study of a human immunoglobulin (project code I10E) in patients with primary Immune ThrombocytoPenia (ITP)
Scientific title: An open-label, multicentre efficacy and safety study of I10E in patients with primary Immune ThrombocytoPenia (ITP)
Date of first enrolment: 24/09/2012
Target sample size: 40
Recruitment status: Authorised-recruitment may be ongoing or finished
URL:  https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2011-001354-29
Study type:  Interventional clinical trial of medicinal product
Study design:  Controlled: no Randomised: no Open: no Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no  
Phase: 
Countries of recruitment
Belarus France Germany Hungary Italy Poland Russian Federation Spain
Ukraine
Contacts
Name: Clinical trial information desk   
Address:  ZA de Courtaboeuf, 3, avenue des Tropiques 91940 Les Ulis Cedex France
Telephone: +33169 82 70 10
Email:
Affiliation:  LFB BIOTECHNOLOGIES
Name: Clinical trial information desk   
Address:  ZA de Courtaboeuf, 3, avenue des Tropiques 91940 Les Ulis Cedex France
Telephone: +33169 82 70 10
Email:
Affiliation:  LFB BIOTECHNOLOGIES
Key inclusion & exclusion criteria
Inclusion criteria:
1. Both genders

2. Age between 18 to 65 year old

3. Chronic Primary ITP
- ITP diagnosis being defined by ASH-2011 and BCSH 2010 criteria adopting the new consensus terminology proposed by an international working group (Rodeghiero et al, 2009):
? Isolated thrombocytopenia diagnosed with platelet count
<100 x 10exp9/l and no abnormality of cells of other haematological
lineage and,
? Normal bone marrow (if available), or history of response to an
ITP treatment (corticosteroids, IVIg, anti-D) and,
? Failure to find any other causes of thrombocytopenia.

- Chronic ITP with bleeding(s) or an increased risk of bleeding:
? More than 12 months from diagnosis of ITP and,
? Platelet counts < 30 x 10exp9/l at the time of inclusion.
Note: Refractory ITP may be included and is defined by the failure to achieve a response or by the loss of response after splenectomy and the need of treatment (s) to minimize the risk of bleeding considered as clinically
significant by the investigator.

4. Written informed consent.
5. Patient is covered by health care insurance system.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 40
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
1. Known hypersensitivity to the active substance or to any of the excipients.

2. Patient with IgA deficiency except if the absence of anti IgA antibodies is documented.

3. History of cardiac insufficiency (NYHA III/IV), cardiomyopathy, congestive heart failure, or severe hypertension (systolic blood pressure > or = to160 mmHg and diastolic blood pressure > or = to 100 mmHg).

4. History of thrombotic episodes (including deep vein thrombosis, myocardial infarction, cerebrovascular accident, pulmonary embolism) within the last 12 months.

5. Patient known to be infected with hepatitis B virus, hepatitis C virus, or human immunodeficiency virus.

6. Treatment that could induce thrombocytopenia within 15 days prior to inclusion.

7. Recent previous treatment, the action of which may interfere with the assessment of the investigational medicinal product:
- Initiation of corticosteroids or regular increase in the steroid dose within the last 4 weeks (EMA/CHMP/BPWP/94033/2007 rev.2),
- IVIg within the last 4 weeks,
- Anti-D within the last 4 weeks,
- Cyclosporin A within 4 weeks,
- Immunomodulator (as vincristin, vinblastin) within the last month,
- Immunosuppressor (azathioprine, cyclophosphamide) within the last 4 weeks,
- Anti-CD20 (rituximab) within the last 4 months,
- Antigonadotropin Hormone (danazol) within the last 6 months,
- Thrombopoietin receptors agonist (eltrombopag, romiplostim) within the last 4 weeks,

8. Splenectomy required during the study period or within the two previous months.

9. Severe haemorrhagic syndrome whether life-threatening or not, such as intracranial haemorrhage, gastrointestinal haemorrhage, gynaecological
haemorrhage with deglobulisation of more than 2g/dL or major cutaneous-mucosal haemorrhagic syndrome.

10. Severe bleeding or planned surgery requiring platelets transfusion at time of inclusion, or whole blood transfusion.

11. Plasma exchange 4 weeks before inclusion.

12. Concomitant disease that may induce a secondary immune thrombocytopenia, as:
- Clinical active systemic lupus erythematous with more than 4 American College of Rheumatology criteria,
- Lymphoproliferative disease or active malignant condition requiring antineoplastic or cytotoxic treatment.

13. Known hepatic disorder including total bilirubin > 2 x upper limit of normal range, alanine aminotransferase (ALT) or aspartate amino transferase (AST) > 3 x upper limit of normal range.

14. Known chronic renal insufficiency or creatinine clearance values < 80 ml/min in adult patients (Modified Diet in Renal Disease calculation).

15. Medical history of haemolysis or haemolytic anaemia during prior IVIg therapy or any other concomitant disease of clotting system (i.e. haemophilia).

16. Administration of another investigational medicinal product within the last month.

17. Any serious medical condition that would interfere with the clinical assessment of I10E or prevent the patient to comply with the protocol requirements.

18. Pregnant with positive results on a urine pregnancy test or breastfeeding woman, or woman of childbearing potential without effective contraception (effective contraception are: injectable, patch or combined
oral estro-progestative or progestative contraceptives, intra-uterine devices of type 'copper T' and levonorgest releasing IU systems, depot intramuscular medroxyprogesteron, subcutaneous implants of
progestative contraceptive implants, barrier methods of contrac


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes
Health Condition(s) or Problem(s) studied
ITP diagnosis being defined by ASH-2011 and BCSH 2010 criteria adopting the new consensus terminology proposed by an international working group (Rodeghiero et al, 2009)
MedDRA version: 14.1 Level: LLT Classification code 10023095 Term: ITP System Organ Class: 10005329 - Blood and lymphatic system disorders
Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Intervention(s)

Product Name: HUMAN NORMAL IMMUNOGLOBULIN FOR INTRVENOUS USE
Product Code: I10E
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN FOR INTRAVENOUS USE
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 100-

Primary Outcome(s)
Main Objective: To assess the efficacy of I10E in increasing platelet count and controlling bleedings in patients suffering from primary Immune ThrombocytoPenia (ITP).
Primary end point(s): The primary endpoint is the number and percentage of patients with responses (including complete responses) during the investigational period.

Patients with response (R):
- Platelet counts > or = to 30 x 10 exp9/l and at least 2-fold increase of baseline platelet count confirmed on at least 2 separate visits at least 7 days apart.
- And absence of new bleeding.

Patients with complete response (CR):
- Platelet count > or = to 100 x 10 exp9/l, confirmed on at least 2 separate visits at least 7 days apart.
- And absence of new bleeding.

Patients with no response (NR):
- Patients who are neither responder nor complete responder.

Patients with loss of CR or R:
- Platelet count < 100 x 109/l (from CR) or platelet count < 30 x 109/l or
less than 2-fold increase of baseline platelet count (from R). Platelet
counts confirmed on at least 2 separate visits approximately 1 day apart.
- Or occurrence of new bleeding.
Secondary Objective: To assess the biological and clinical safety profile of I10E.
Timepoint(s) of evaluation of this end point: confirmed on at least 2 separate visits at least 7 days apart during the study period.
Secondary Outcome(s)
Secondary end point(s): Efficacy criteria:
- Number and % of patients with complete response (CR) during the investigational period.
- Time to response i.e. time from starting treatment to time of platelet response achievement (platelet count > or = to 30 x 10 exp9/l) in the responder
population.
- Maximum platelet counts and time to reach the maximum platelet count.
- Number and % of non-responders patients (NR) during the investigational period.
- Number and % of patients with a loss of R and CR during the investigational period.
- Duration of response defined as the number of days from the first day of the response (CR or R) to the first day of the loss of response (CR or R).
- Number and % of patients with platelet count > or = to 50 x 10 exp9/l at Day 5, Day 6 and Day 7.
- Evaluation of pre-existing bleedings (Khellaf score and WHO score assessed at baseline, Day 2, Day 14 and Day 30)

Safety criteria:
All adverse events (AEs), non serious or serious adverse events (SAEs), from
all subjects followed throughout the clinical study will be recorded and
reported regardless of whether the AE is determined to be related to the
product or not (pre-medication is not permitted to avoid masking AEs).
Safety evaluation will include the monitoring of the following items:
- Vital signs (blood pressure, heart rate, temperature) will be monitored
by the study nurse during infusions,
- Other adverse events that occurred during and between both infusions
will be noted by the study nurse or the investigator,
- Adverse events that occurred during the follow-up period will be
recorded by the patients in their diaries and reviewed carefully by the
investigator at Day 14 and at the end-of-study visit,
- Renal function, hepatic function and blood cells will be monitored by
biological tests (see study plan table).
All AEs that begin during the infusion or within 72 hours after an infusion
will be classified and analysed as temporally associated AEs (TAAEs) i.e.
infusional AEs. A phone call between the patient and the investigator is
scheduled at Day 5 to ensure the collect of these AEs.
The patient will be asked to complete carefully the diary (nature of event,
severity, date of initiation, hour at initiation of symptoms, duration of
symptoms).
AEs will be evaluated with regard to the infusion rates.
In this study, worsening of thrombocytopenia and bleeding events will not be
regarded as adverse events as they will already be reported as efficacy criteria.
Timepoint(s) of evaluation of this end point: see details provided in section E 5.2.
Secondary ID(s)
2011-001354-29-DE
I10E-0719
Source(s) of Monetary Support
LFB BIOTECHNOLOGIES
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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