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| ID | Type | Description | Link |
|---|---|---|---|
| BIVV009-201 | Other Identifier | Bioverativ Therapeutics Inc. |
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The purpose of this study is to explore the safety, preliminary clinical benefit, and activity of BIVV009 in patients with chronic immune thrombocytopenia.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| BIVV009 | Experimental | Participants who weigh less than 75 kilogram (kg) will receive fixed doses of 6.5 grams of BIVV009 intravenous (IV) infusion and participants who weigh 75 kg or more will receive fixed doses of 7.5 grams of BIVV009 every 2 weeks for approximately 21 weeks in Part A (based on time to complete 11 doses of study drug). There will be a 9-week safety follow-up/washout period after administration of the last dose of study drug in Part A. Participants who have been shown to benefit from BIVV009 treatment during Part A, will receive BIVV009 (based on weight) biweekly for up to 52 weeks of BIVV009 after Last Patient In (LPI) in part B. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| BIVV009 6.5 grams | Drug | Participants who weigh less than 75 kilogram (kg) will receive fixed doses of 6.5 grams of BIVV009. |
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| Measure | Description | Time Frame |
|---|---|---|
| Incidence of Treatment-Emergent Adverse Events | An AE is any untoward medical occurrence in a participant participating in a clinical study that does not necessarily have a causal relationship with the pharmaceutical/biological agent under study. A serious adverse event (SAE) is any AE that results in: death, persistent or significant disability/incapacity, requires inpatient hospitalization or prolongation of existing hospitalization, is life-threatening experience, is a congenital anomaly/birth defect and may jeopardize participant and/or may require medical or surgical intervention to prevent one of the outcomes listed above. | Up to 97 weeks |
| Number of Participants With Premature Study Terminations | Number of participants with premature study terminations will be assessed. | Approximately 97 weeks |
| Number of Participants With Clinical Laboratory Abnormalities | Clinical laboratory abnormalities including one or more specific target-organs for toxicity of BIVV009, abnormalities in D-dimer, thrombin-anti-thrombin assay, and Systemic Lupus Erythematosus (SLE) panel. | Approximately 97 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Part A: Change From Baseline in Peripheral Blood Platelet Count at Part A End of Treatment (A-EOT) | Change from baseline in peripheral blood platelet count at A-EOT will be assessed. | Baseline and A-EOT (Day 147) |
| Part A: Change From Baseline in Peripheral Blood Platelet Count during BIVV009 Treatment |
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Inclusion Criteria
Part A:
Part B:
Exclusion Criteria:
Part A:
Part B:
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| Name | Affiliation | Role |
|---|---|---|
| Clinical Sciences & Operations | Sanofi | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Georgetown Lombardi Comprehensive Cancer Center | Georgetown | District of Columbia | 20057 | United States | ||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 35973190 | Result | Broome CM, Roth A, Kuter DJ, Scully M, Smith R, Wang J, Reuter C, Hobbs W, Daak A. Safety and efficacy of classical complement pathway inhibition with sutimlimab in chronic immune thrombocytopenia. Blood Adv. 2023 Mar 28;7(6):987-996. doi: 10.1182/bloodadvances.2021006864. |
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Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
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| BIVV009 7.5 grams | Drug | Participants who weigh 75 kg or more will receive fixed doses of 7.5 grams of BIVV009. |
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Change from baseline in peripheral blood platelet count during BIVV009 treatment will be assessed. |
| Baseline up to Day 147 |
| Part A: Number of Participants who are independent from using combination Immune Thrombocytopenia (ITP) therapy during A-EOT but receive combination ITP therapy after A-EOT | Number of participants who are independent from using combination ITP therapy during A-EOT but receive combination ITP therapy after A-EOT will be assessed. | Day 147 (A-EOT) up to Day 196 (EOS) |
| Part A: Number of Participants who Achieve Complete Response Through A-EOT | Complete response (CR) is defined as a platelet count greater than or equal to (>=) 100*10^9/liter (L) measured on 2 occasions at least 7 days apart and the absence of bleeding on and through these two visits and the lack of combination ITP therapy on and through these two visits. | Up to Day 147 |
| Part A: Number of Participants who Achieve Response Through A-EOT | Response or Better: Response (R) is defined as a platelet count >= 30*10^9/L and a greater than 2-fold increase from baseline measured on 2 occasions at least 7 days apart and the absence of bleeding on and through these two visits and the lack of combination ITP therapy on and through these two visits; and CR: A platelet count >=100*10^9/L measured on 2 occasions at least 7 days apart and the absence of bleeding on and through these two visits and the lack of combination ITP therapy on and through these two visits. | Up to Day 147 |
| Part A: Duration of Complete Response per Each CR | Duration of CR is defined as the number of consecutive days in which a patient's peripheral blood platelet count is >= 100*10^9/L and the absence of bleeding, and the lack of platelet transfusions or other ITP therapy. | Up to Day 196 |
| Part A: Duration of Response per Each Response | Duration of response is defined as the number of consecutive days in which a patient's peripheral blood platelet count is >= 30*10^9/L and the absence of bleeding, and the lack of platelet transfusions or other ITP therapy. | Up to Day 196 |
| Part A: Time to First Platelet Response | Time to first platelet response is defined as greater than or equal to 30*10^9/L, 50*10^9/L, 100*10^9/L (confirmed by a consecutive platelet response at least 7 days apart). | Up to Day 196 |
| Part A: Number of Participants who Report Loss of Response Among Those who Achieve Response | Number of participants who report loss of response among those who achieve response will be reported. For a participant with a response (R), the loss of the response is defined as a platelet count < 30*10^9/L measured on 2 consecutive occasions at least 1 day apart, or a less than 2-fold increase in platelet count from baseline measured on 2 consecutive occasions at least 1 day apart, or the presence of bleeding, or use of the combination ITP therapy. | Up to Day 196 |
| Part A: Number of Participants who Report Loss of Complete Response Among Those who Achieve Complete Response | Number of participants who report loss of complete response among those who achieve complete response will be reported. For a participant with a complete response (CR), loss of complete response is defined as a platelet count less than (<) 100*10^9/L measured on 2 consecutive occasions more than 1 day apart and/or the presence of bleeding or use of the combination ITP therapy. | Up to Day 196 |
| Part B: Change From Baseline in Peripheral Blood Platelet Count to B-EOT | Change from baseline (Part B) in peripheral blood platelet count to B-EOT will be assessed. | Baseline up to 52 weeks |
| Part B: Number of Participants who Achieve CR and the Lack of Platelet Transfusions or Other ITP Therapy During Treatment Period | Number of participants who achieve CR and the lack of platelet transfusions or other ITP therapy during Part B treatment period will be reported. Complete response (CR): A platelet count >= 100*10^9/L measured on 2 occasions at least 7 days apart and the absence of bleeding on and through these two visits and the lack of combination ITP therapy on and through these two visits. | Up to 52 weeks |
| Part B: Number of Participants who Achieve Response Through Part B End of Treatment (B-EOT) | Number of participants who achieve response through B-EOT will be reported. | Up to 52 weeks |
| Part B: Duration of Complete Response per Each CR | Duration of CR is defined as the number of consecutive days in which a participant's peripheral blood platelet count is >= 100*10^9/L and the absence of bleeding, and the lack of platelet transfusions or other ITP therapy. | Up to 52 weeks |
| Part B: Duration of Response per each Response | Duration of response is defined as the number of consecutive days in which a participant's peripheral blood platelet count is >= 30*10^9/L and the absence of bleeding, and the lack of platelet transfusions or other ITP therapy. | Up to 52 weeks |
| Part B: Number of Participants who achieve a platelet count >= 100*10^9/L on 2 consecutive occasions at least 7 days apart and have the absence of bleeding on and through these two visits and use any combination ITP therapy through B-EOT | Number of participants who achieve a platelet count >= 100*10^9/L on 2 consecutive occasions at least 7 days apart and have the absence of bleeding on and through these two visits and use any combination ITP therapy through B-EOT will be assessed. | Up to 52 weeks |
| Part B: Number of Participants who achieve a platelet count >=30*10^9/L, a >2-fold increase from baseline measured on 2 consecutive occasions at least 7 days apart, have absence of bleeding on and through these two visits, use any combination ITP therapy | Number of participants who achieve a platelet count >= 30*10^9/L and a greater than (>) 2-fold increase from baseline measured on 2 consecutive occasions at least 7 days apart and have the absence of bleeding on and through these two visits and use any combination ITP therapy through B-EOT will be assessed. | Up to 52 weeks |
| Part B: Number of Participants who do not Require Other Immune Thrombocytopenia (ITP) Therapy (non-transfusion) During the Part B Treatment Period | Number of Participants who do not require other ITP therapy (non-transfusion) following the last BIVV009 dose will be assessed. | Up to 52 weeks |
| Part B: Number of Participants who do not Require Platelet Transfusions During the Part B Treatment Period | Number of Participants who do not require platelet transfusions during the Part B treatment period will be reported. | Up to 52 weeks |
| Part B: Number of Participants who Experience any Bleeding Episode, Bleeding by Grade or Serious Bleeding | Number of participants who experience any bleeding episode, bleeding by grade or serious bleeding according to the International Working Group (IWG) Bleeding Assessment Tool (BAT) will be reported. | Up to 52 weeks |
| Plasma Concentrations of BIVV009 | Plasma concentrations of BIVV009 will be assessed. | Approximately 97 weeks |
| Maximum Observed Plasma Concentration (Cmax) of BIVV009 | Maximum observed concentration of BIVV009 in plasma will be assessed. | Approximately 97 weeks |
| Time to Reach Maximum Observed Plasma Concentration (Tmax) of BIVV009 | Time to Reach Maximum Observed Plasma Concentration (Tmax) of BIVV009 will be assessed. | Approximately 97 weeks |
| Area Under the Concentration-time Curve (AUC) From Hour 0 to the last quantifiable time point (AUC [0-t]) of BIVV009 | AUC (0-t) is the area under the Concentration-time curve (AUC) from hour 0 to the last quantifiable time point of BIVV009. | Approximately 97 weeks |
| Number of Participants With Anti-drug antibodies (ADAs) Against BIVV009 | Blood samples will be collected to determine number of participants with anti-drug antibodies (ADAs) against BIVV009. | Up to 97 weeks |
| Complement System Classical Pathway Levels as Measured by WIESLAB Assay | Inhibition by BIVV009 of the complement system classical pathway measured by the WIESLAB assay. | Up to 97 weeks |
| Total Complement (CH50) Levels | Complement CH50 is a blood test that helps us determine whether protein abnormalities and deficiencies in the complement system are responsible for any increase in autoimmune activity. It will be assessed using complement assays. | Up to 97 weeks |
| Total Complement Factor C4 Levels | Total C4 Levels will be assessed in plasma using complement assays. | Up to 97 weeks |
| C1 Complex Components: C1q | C1q Levels will be assessed in plasma using complement assays. | Up to 97 weeks |
| Thrombopoietin Level | Thrombopoietin level will be assessed in plasma using complement assays. | Up to 97 weeks |
| Massachusetts General Hospital - Cancer Center |
| Boston |
| Massachusetts |
| 02144 |
| United States |
| University of Pittsburgh Medical Center (UPMC) Hilman Cancer Center | Pittsburgh | Pennsylvania | 15232 | United States |
| Essen University Hospital Department of Hematology | Essen | 45147 | Germany |
| University College Hospital | London | WC1E 6HX | United Kingdom |
| ID | Term |
|---|---|
| D016553 | Purpura, Thrombocytopenic, Idiopathic |
| D001327 | Autoimmune Diseases |
| D006402 | Hematologic Diseases |
| D007154 | Immune System Diseases |
| D001791 | Blood Platelet Disorders |
| ID | Term |
|---|---|
| D011696 | Purpura, Thrombocytopenic |
| D011693 | Purpura |
| D001778 | Blood Coagulation Disorders |
| D006425 | Hemic and Lymphatic Diseases |
| D057049 | Thrombotic Microangiopathies |
| D013921 | Thrombocytopenia |
| D000095542 | Cytopenia |
| D006474 | Hemorrhagic Disorders |
| D006470 | Hemorrhage |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D012877 | Skin Manifestations |
| D012816 | Signs and Symptoms |
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