Study to Evaluate Safety and Efficacy in Adult Subjects With ITP

Phase 1

Completed

• Conditions: Immune Thrombocytopenic Purpura
• Interventions: Drug: BMS-986004 75 mg IV; Drug: BMS-986004 1500 mg IV; Drug: BMS-986004 225 mg IV; Drug: BMS-986004 675 mg IV

• Registration Number: NCT02273960
• Lead Sponsor: Bristol-Myers Squibb

Brief Summary

The purpose of this study is to assess the safety and tolerability of BMS-986004 when administered in subjects with ITP.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2014-10-03
• Study First Submitted QC: 2014-10-22
• Study First Posted: 2014-10-24
• Study Start: 2014-11-17
• Primary Completion: 2018-01-22
• Study Completion: 2018-01-22
• Results First Submitted: 2019-01-17
• Results First Submitted QC: 2019-03-29
• Results First Posted: 2019-04-01
• Status Verified: 2019-07
• Last Update Submitted: 2019-07-15
• Last Update Posted: 2019-07-16

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 46

Inclusion Criteria

• ≥18 years old, diagnosed with persistent or chronic ITP

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Exclusion Criteria

• Secondary immune thrombocytopenia
• Drug induced thrombocytopenia

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Arm A: BMS-986004	BMS-986004 75 mg IV	BMS-986004 solution intravenously (IV) as specified
Arm D: BMS-986004	BMS-986004 1500 mg IV	BMS-986004 solution intravenously as specified
Arm B: BMS-986004	BMS-986004 225 mg IV	BMS-986004 solution intravenously as specified
Arm C: BMS-986004	BMS-986004 675 mg IV	BMS-986004 solution intravenously as specified

Primary Outcome Measures

Name	Time	Method
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs): Short Term and Long Term	Day 1 to Day 141 (Short term) and Day 1 to Day 398 (Long term)	The primary objective to establish safety was measured by the primary endpoints of AEs and SAEs for both Short term and Long term periods
Number of ECG Abnormalities	Day 1 to Day 141 (Short term) and Day 1 to Day 398 (Long term)	The primary objective to establish safety was measured by investigator identified Electrocardiogram Abnormalities for both Short term and Long term periods. ECG parameters included heart rate, PR interval, QRS interval, and QTcF interval (QT interval corrected for heart rate)
Number of Laboratory Abnormalities of Safety Biomarkers: d-Dimer and Thrombin Anti-Thrombin (TAT)	Day 1 to Day 141 (Short term) and Day 1 to Day 398 (Long Term)	D-dimer and thrombin antithrombin (TAT) in plasma were quantified as measures of thromboembolism risk. D-dimer was evaluated by Enzyme linked immune sorbent assay (ELISA) method (D-dimer reference range 0-0.63 micrograms/milliliters fibrinogen equivalent units \[mcg/ml FEU\]). TAT reference range 0-4.1 ng/ml.

Secondary Outcome Measures

Name	Time	Method
Response Rate (RR) of BMS-986004: Short Term and Long Term	Day 1 to Day 141 (Short term) and Day 1 to Day 398 (Long term)	Overall Response Rate (ORR) was defined as the proportion of participants who achieved a complete response (CR) or response (R). CR was defined as platelet count ≥ 100,000/mm3 and absence of bleeding. R was defined as platelet count ≥ 30,000/mm3 and at least 2-fold increase from the baseline count and absence of bleeding.
Maximum Observed Serum Concentration (Cmax) of BMS-986004	Day 1 (0 hour [h], 2h, 24h, 72h, 168h), Day 15 (0h), Day 29 (0h), Day 43 (0h, 168h), Day 57 (0h, 2h), Day 71 (0h, 2h, 24h, 96h, 168h), Day 85 (0h, 336h, 672h, 1008h, 1344h)	Pharmacokinetic parameter (Cmax) of BMS-986004, derived from serum concentration versus time. who have adequate PK profiles. On study Day 57, non-responders in each treatment group other than the 1500 mg group were dose escalated to the starting dose of the next higher treatment group.
Area Under the Concentration-time Curve in One Dosing Interval [AUC(TAU)] of BMS-986004	Day 1 (0 hour [h], 2h, 24h, 72h, 168h), Day 15 (0h), Day 29 (0h), Day 43 (0h, 168h), Day 57 (0h, 2h), Day 71 (0h, 2h, 24h, 96h, 168h), Day 85 (0h, 336h, 672h, 1008h, 1344h)	Pharmacokinetics of BMS-986004 were derived from serum concentration versus time data. AUC(TAU) = Area under the concentration-time curve in one dosing interval. On study Day 57, non-responders in each treatment group other than the 1500 mg group were dose escalated to the starting dose of the next higher treatment group.
Trough Observed Serum Concentration (Ctrough) of BMS-986004	Day 1 (0 hour [h], 2h, 24h, 72h, 168h), Day 15 (0h), Day 29 (0h), Day 43 (0h, 168h), Day 57 (0h, 2h), Day 71 (0h, 2h, 24h, 96h, 168h), Day 85 (0h, 336h, 672h, 1008h, 1344h)	Pharmacokinetics of BMS-986004 were derived from serum concentration versus time data. Ctrough = Trough observed serum concentration. On study Day 57, non-responders in each treatment group other than the 1500 mg group were dose escalated to the starting dose of the next higher treatment group.
Total Body Clearance (CLT) of BMS-986004	Day 1 (0 hour [h], 2h, 24h, 72h, 168h), Day 15 (0h), Day 29 (0h), Day 43 (0h, 168h), Day 57 (0h, 2h), Day 71 (0h, 2h, 24h, 96h, 168h), Day 85 (0h, 336h, 672h, 1008h, 1344h)	Pharmacokinetics of BMS-986004 were derived from serum concentration versus time data. On study Day 57, non-responders in each treatment group other than the 1500 mg group were dose escalated to the starting dose of the next higher treatment group.
AUC Accumulation Index (AI_AUC) of BMS-986004	Day 1 (0 hour [h], 2h, 24h, 72h, 168h), Day 15 (0h), Day 29 (0h), Day 43 (0h, 168h), Day 57 (0h, 2h), Day 71 (0h, 2h, 24h, 96h, 168h), Day 85 (0h, 336h, 672h, 1008h, 1344h)	AUC accumulation index (AI_AUC) = ratio of AUC(TAU) at steady state to AUC(TAU) after the first dose of BMS-986004. On study Day 57, non-responders in each treatment group other than the 1500 mg group were dose escalated to the starting dose of the next higher treatment group.

Trial Locations

Locations (10)

Oddzial Kliniczny Hematologii i Profilaktyki Chorob Nowotworowych; 🇵🇱 Chorzow, Poland

Emory University; 🇺🇸 Atlanta, Georgia, United States

Rutgers- Robert Wood Johnson Medical School; 🇺🇸 New Brunswick, New Jersey, United States

Georgetown University Medical Center; 🇺🇸 Washington, District of Columbia, United States

Columbus Regional Research Institute; 🇺🇸 Columbus, Georgia, United States

Univ. Of Southern Calif. /Norris Comprehensive Cancer Center; 🇺🇸 Los Angeles, California, United States

Specjalistyczny Gabinet Lekarski Prof. dr hab. Krzysztof Giannopoulos; 🇵🇱 Lublin, Poland

Local Institution; 🇬🇧 London, United Kingdom

Mass General Hospital; 🇺🇸 Boston, Massachusetts, United States

Hamilton Health Sciences/Mc Master Univ Med Ctre; 🇨🇦 Hamilton, Ontario, Canada