A Study of INCB050465 in Participants With Autoimmune Hemolytic Anemia

A Phase 2, Open-Label Study of INCB050465 in Participants With Autoimmune Hemolytic Anemia

Sponsors

Lead Sponsor: Incyte Corporation

Source Incyte Corporation
Brief Summary

The purpose of this study is to evaluate the safety and efficacy of parsaclisib administered orally to participants with autoimmune hemolytic anemia (AIHA) who have decreased hemoglobin and evidence of ongoing hemolysis that requires treatment intervention.

Overall Status Recruiting
Start Date July 2, 2018
Completion Date December 2021
Primary Completion Date September 2020
Phase Phase 2
Study Type Interventional
Primary Outcome
Measure Time Frame
Proportion of participants attaining a complete response (CR) Week 6 to Week 12
Proportion of participants attaining a partial response (PR) From Baseline to Week 6 to Week 12
Number of participants with a treatment-emergent adverse event (TEAE) Up to 28 weeks
Secondary Outcome
Measure Time Frame
Proportion of participants attaining a CR during post baseline visits Up to 28 weeks
Proportion of participants attaining a PR during post baseline visits From Baseline up to 28 weeks
Proportion of participants attaining an increase in hemoglobin from baseline From Baseline up to 28 weeks
Change from baseline of hemoglobin From Baseline up to 28 weeks
Proportion of participants requiring transfusions From Baseline up to 28 weeks
Proportion of participants who achieve normalization of hemolytic markers Up to 28 weeks
Change of daily usage of prednisone From Baseline up to 28 weeks
Change from baseline in Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-F) sub-scale questionnaire From Baseline up to 28 weeks
Enrollment 25
Condition
Intervention

Intervention Type: Drug

Intervention Name: Parsaclisib

Description: Parsaclisib administered orally.

Other Name: INCB050465

Eligibility

Criteria:

Inclusion Criteria:

- Diagnosis of AIHA based on the presence of hemolytic anemia and serological evidence of anti-erythrocyte antibodies, detectable by the direct antiglobulin test.

- Participants who have disease progression after treatment with standard therapies that are known to confer clinical benefit, or who are intolerant to treatment, or who refuse standard treatment. There is no limit to the number of prior treatment regimens.

- Hemoglobin 7 to 10 g/dL.

- No evidence of a lymphoproliferative malignancy or other autoimmune-related underlying conditions.

- Eastern Cooperative Oncology Group performance status of 0 to 2.

- Willingness to avoid pregnancy or fathering children.

Exclusion Criteria:

- Pregnant or breastfeeding women.

- Concurrent conditions and history of other protocol-specified diseases.

- ANC < 1.5 × 10^9/L.

- Platelet count < 100 × 10^9/L.

- Severely impaired liver function.

- Impaired renal function with estimated creatinine clearance less than 45 mL/min.

- Anti-phospholipid antibodies positive or elevated anti-streptolysin antibodies.

- Positive serology test results for hepatitis B surface antigen or core antibody, or hepatitis C virus antibody with detectable RNA at screening, consistent with active or chronic infection.

- Known HIV infection or positivity on immunoassay.

- History or presence of an abnormal ECG that, in the investigator's opinion, is clinically meaningful.

- Known hypersensitivity or severe reaction to parsaclisib or its excipients.

Gender: All

Minimum Age: 18 Years

Maximum Age: N/A

Healthy Volunteers: No

Overall Official
Last Name Role Affiliation
Kathleen Butler, MD Study Director Incyte Corporation
Overall Contact

Last Name: Incyte Corporation Call Center (US)

Phone: 1.855.463.3463

Email: [email protected]

Location
Facility: Status:
Georgetown University Georgetown Lombardi Comprehensive Cancer Center Division Hematology/Oncology Podium B | Washington, District of Columbia, 20007, United States Not yet recruiting
University of Minnesota | Minneapolis, Minnesota, 55455, United States Recruiting
Washington University School of Medicine in St. Louis | Saint Louis, Missouri, 63110, United States Recruiting
Montefiore Medical Center Department Of Oncology | Bronx, New York, 10467, United States Recruiting
Tirol Kliniken | Innsbruck, 6020, Austria Withdrawn
Medical University of Vienne Klinische Abteilung für Hämatologie und Hämostaseologie | Vienna, 1090, Austria Recruiting
Hôpital Henri Mondor | Créteil, 94000, France Recruiting
Hopital Claude Huriez CHRU de Lille | Lille, 59037, France Recruiting
Hopital de la Timone | Marseille, 13385, France Withdrawn
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico | Milano, 20122, Italy Recruiting
A.O.U. Federico II | Napoli, 80131, Italy Recruiting
AOU Maggiore della Carità | Novara, 28100, Italy Recruiting
Location Countries

Austria

France

Italy

United States

Verification Date

December 2019

Responsible Party

Type: Sponsor

Keywords
Has Expanded Access No
Condition Browse
Number Of Arms 2
Arm Group

Label: Cohort 1

Type: Experimental

Description: Parsaclisib at the protocol-defined dose for 12 weeks followed by extension period, with a dose-increase option at Week 6 for participants who fulfill dose increase criteria.

Label: Cohort 2

Type: Experimental

Description: Parsaclisib at the protocol-defined dose for 12 weeks followed by extension period.

Patient Data No
Study Design Info

Allocation: Non-Randomized

Intervention Model: Parallel Assignment

Primary Purpose: Treatment

Masking: None (Open Label)

Source: ClinicalTrials.gov