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| ID | Type | Description | Link |
|---|---|---|---|
| 2021-000292-37 | EudraCT Number |
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ITP patients with low platelet count and active bleeding symptoms are at risk of life-threatening bleeding and therefore require a treatment with a rapid effect, reliable, and sustained. The combination of intravenous immunoglobulin (IVIg) and prednisone (1 mg/kg per day), is more rapidly and more frequently effective than high dose methylprednisolone to increase the platelet count. This combination is therefore usually given in patients with platelets count < 20 x 109/L and moderate to severe bleeding manifestations. Based on common practice in France and on French ITP guidelines, on average 50 % of patients with ITP and profound thrombocytopenia do actually receive IVIg (mostly during the initial phase of the disease) corresponding to approximately 1,500 ITP patients per year in France.
Whereas IVIg is usually well tolerated, renal insufficiency and congestive heart failure may occur, moreover IVIg are costly and non-easily available with supply difficulties in many countries including France.
High dose dexamethasone (DXM) (ie: 40 mg/d for 4 days) has recently emerged as a promising treatment for ITP. One recent meta-analysis as well as a controlled prospective trial suggest that the initial overall response was higher (> 80 %) and the time to response was shorter with dexamethasone (DXM) 40 mg/d given for 4 days compared to standard prednisone.
The investigators hypothesize that DXM could be a reasonable non-inferior alternative to IVIg, more convenient for patients with less adverse events and economically cost-effective for patients with moderate and severe bleeding manifestations.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Experimental group | Experimental | Oral dexamethasone (Neofordex®) 40 mg (Day1 to Day 4), ± an additional 4-days cycle of dexamethasone between days 10 and 21 |
|
| Control | Active Comparator | IVIg (1g/kg D1-D2) plus prednisone (1 mg/kg/day x 21 days (3 weeks)) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Neofordex® | Drug | Oral dexamethasone (Neofordex®) 40 mg (Day1 to Day 4), ± an additional 4-days cycle of dexamethasone between days 10 and 21. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Time to achieve an initial response (R) within 5 days. | 5 days |
| Measure | Description | Time Frame |
|---|---|---|
| Time to achieve an initial complete response (CR) in the two arms | complete response (CR): defined by a platelet count > 100 x 109/L in the absence use of any other ITP directed therapies between Day 1 and Day 5 | between Day 1 and Day 5 |
| Duration of overall response from Day 1 to the end of the study in the two arms. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Matthieu MAHEVAS, MD,PhD | Contact | +33 (1) 49 81 20 76 | matthieu.mahevas@aphp.fr |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Henri Mondor Hospital | Recruiting | Créteil | 94010 | France |
DATAS ARE OWN BY ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS, PLEASE CONTACT SPONSOR FOR FURTHER INFORMATION
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| ID | Term |
|---|---|
| D016553 | Purpura, Thrombocytopenic, Idiopathic |
| ID | Term |
|---|---|
| D011696 | Purpura, Thrombocytopenic |
| D011693 | Purpura |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
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| ID | Term |
|---|---|
| D003907 | Dexamethasone |
| D016756 | Immunoglobulins, Intravenous |
| C495669 | Octagam |
| ID | Term |
|---|---|
| D011246 | Pregnadienetriols |
| D011245 | Pregnadienes |
| D011278 | Pregnanes |
| D013256 | Steroids |
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| Intravenous immunoglobulins | Drug | IVIg (1g/kg D1-D2) plus oral prednisone (1 mg/kg/day x 21 days (3 weeks)) |
|
|
| Day 1 to 6 months |
| Proportion of early treatment switches across arms | before day 5 |
| Number of new bleeding manifestations between Day 1 and Day 5 in the two arms. | between Day 1 and Day 5 |
| Rates of response (R) and complete response (CR) in the two arms. | at Day 28 and at 6 months |
| Number of new bleeding manifestations in the two arms. | Between Day 5 and Day 28 |
| Number of adverse events in the two arms. | up to 6 months |
| Number of responders in patients with positive and negative anti-platelets antibodies in the two arms. | At 6 months |
| Number of outcome in patients with positive and negative anti-platelets antibodies in the two arms. | At 6 months |
| D006425 |
| Hemic and Lymphatic Diseases |
| D057049 | Thrombotic Microangiopathies |
| D013921 | Thrombocytopenia |
| D001791 | Blood Platelet Disorders |
| D000095542 | Cytopenia |
| D006474 | Hemorrhagic Disorders |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
| D006470 | Hemorrhage |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D012877 | Skin Manifestations |
| D012816 | Signs and Symptoms |
| D000072473 |
| Fused-Ring Compounds |
| D011083 | Polycyclic Compounds |
| D013259 | Steroids, Fluorinated |
| D007074 | Immunoglobulin G |
| D007132 | Immunoglobulin Isotypes |
| D000906 | Antibodies |
| D007136 | Immunoglobulins |
| D007162 | Immunoproteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D012712 | Serum Globulins |
| D005916 | Globulins |