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NCT03538041

A Study of INCB050465 in Participants With Autoimmune Hemolytic Anemia

Completed Phase 2 Results posted Last updated 11 July 2025
What this trial tests

Phase 2 trial testing Parsaclisib in Autoimmune Hemolytic Anemia in 25 participants. Completed in 2 April 2024.

Timeline
21 November 2018
Primary endpoint
5 August 2021
2 April 2024

Quick facts

Lead sponsorIncyte Corporation
PhasePhase 2
StatusCompleted
Study typeINTERVENTIONAL
Allocationnon randomized
Designparallel
Maskingnone
Primary purposetreatment
Enrollment25
Start date21 November 2018
Primary completion5 August 2021
Estimated completion2 April 2024
Sites12 locations across Austria, France, United States, Italy

Drugs / interventions tested

Conditions studied

Sponsor

Incyte Corporation — full company profile →

Who can join

18 and older, any sex, with Autoimmune Hemolytic Anemia. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Percentage of Participants Attaining a Complete Response at Any Visit From Week 6 to Week 12 Primary · Week 6 to Week 12

A complete response was defined as hemoglobin \>12 grams per deciliter (g/dL) not attributed to a transfusion effect and the normalization of hemolytic markers. No transfusion effect was defined as \> 1 week since the last transfusion.

GroupValue95% CI
Parsaclisib 1 mg QD20.0
Parsaclisib 2.5 mg QD40.0
Percentage of Participants Attaining a Partial Response at Any Visit From Week 6 to Week 12 Primary · Week 6 to Week 12

A partial response was defined as hemoglobin 10-12 g/dL or at least a 2 g/dL increase from Baseline not attributed to a transfusion effect and the normalization of hemolytic markers. No transfusion effect was defined as \> 1 week since the last transfusion.

GroupValue95% CI
Parsaclisib 1 mg QD60.0
Parsaclisib 2.5 mg QD66.7
Number of Participants With Any Treatment-emergent Adverse Event (TEAE) Primary · up to 1638 days

An adverse event (AE) was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related, that occurred after a participant provided informed consent. Abnormal laboratory values or test results occurring after informed consent constituted AEs only if they induced clinical signs or symptoms, were considered clinically meaningful, required therapy, or required changes in the study drug. Anemia and transfusions should not have been reported as AEs unless they represented a clinically meaningful decrease from Baseline in hemoglobin. A

GroupValue95% CI
Parsaclisib 1 mg QD10
Parsaclisib 2.5 mg QD15
Percentage of Participants Attaining a Complete Response During Post-Baseline Visits Secondary · up to 1638 days

A complete response was defined as hemoglobin \>12 g/dL) not attributed to a transfusion effect and the normalization of hemolytic markers. No transfusion effect was defined as \> 1 week since the last transfusion.

Week 1
GroupValue95% CI
Parsaclisib 1 mg QD0.0
Parsaclisib 2.5 mg QD6.7
Week 2
GroupValue95% CI
Parsaclisib 1 mg QD10.0
Parsaclisib 2.5 mg QD6.7
Week 4
GroupValue95% CI
Parsaclisib 1 mg QD22.2
Parsaclisib 2.5 mg QD21.4
Week 6
GroupValue95% CI
Parsaclisib 1 mg QD22.2
Parsaclisib 2.5 mg QD28.6
Week 8
GroupValue95% CI
Parsaclisib 1 mg QD22.2
Parsaclisib 2.5 mg QD23.1
Week 10
GroupValue95% CI
Parsaclisib 1 mg QD22.2
Parsaclisib 2.5 mg QD23.1
Week 12
GroupValue95% CI
Parsaclisib 1 mg QD22.2
Parsaclisib 2.5 mg QD42.9
Follow-up Month 1
GroupValue95% CI
Parsaclisib 1 mg QD0.0
Parsaclisib 2.5 mg QD0.0
Percentage of Participants Attaining a Partial Response During Post-Baseline Visits Secondary · up to 1638 days

A partial response was defined as hemoglobin 10-12 g/dL or at least a 2 g/dL increase from Baseline not attributed to a transfusion effect and the normalization of hemolytic markers. No transfusion effect was defined as \> 1 week since the last transfusion.

Week 1
GroupValue95% CI
Parsaclisib 1 mg QD50.0
Parsaclisib 2.5 mg QD53.3
Week 2
GroupValue95% CI
Parsaclisib 1 mg QD50.0
Parsaclisib 2.5 mg QD66.7
Week 4
GroupValue95% CI
Parsaclisib 1 mg QD44.4
Parsaclisib 2.5 mg QD64.3
Week 6
GroupValue95% CI
Parsaclisib 1 mg QD44.4
Parsaclisib 2.5 mg QD50.0
Week 8
GroupValue95% CI
Parsaclisib 1 mg QD55.6
Parsaclisib 2.5 mg QD61.5
Week 10
GroupValue95% CI
Parsaclisib 1 mg QD55.6
Parsaclisib 2.5 mg QD61.5
Week 12
GroupValue95% CI
Parsaclisib 1 mg QD55.6
Parsaclisib 2.5 mg QD64.3
Follow-up Month 1
GroupValue95% CI
Parsaclisib 1 mg QD50.0
Parsaclisib 2.5 mg QD0.0
Percentage of Participants Attaining a ≥ 2 g/dL Increase in Hemoglobin From Baseline Secondary · up to 1638 days

Hemoglobin levels were assessed throughout the study.

Week 1
GroupValue95% CI
Parsaclisib 1 mg QD0.0
Parsaclisib 2.5 mg QD26.7
Week 2
GroupValue95% CI
Parsaclisib 1 mg QD10.0
Parsaclisib 2.5 mg QD46.7
Week 4
GroupValue95% CI
Parsaclisib 1 mg QD22.2
Parsaclisib 2.5 mg QD42.9
Week 6
GroupValue95% CI
Parsaclisib 1 mg QD22.2
Parsaclisib 2.5 mg QD35.7
Week 8
GroupValue95% CI
Parsaclisib 1 mg QD33.3
Parsaclisib 2.5 mg QD38.5
Week 10
GroupValue95% CI
Parsaclisib 1 mg QD22.2
Parsaclisib 2.5 mg QD46.2
Week 12
GroupValue95% CI
Parsaclisib 1 mg QD33.3
Parsaclisib 2.5 mg QD50.0
Follow-up Month 1
GroupValue95% CI
Parsaclisib 1 mg QD0.0
Parsaclisib 2.5 mg QD0.0
Change From Baseline in Hemoglobin Secondary · Baseline; up to 1638 days

Change from Baseline was calculated as the post-Baseline value minus the Baseline value.

Baseline
GroupValue95% CI
Parsaclisib 1 mg QD9.1± 0.80
Parsaclisib 2.5 mg QD8.7± 0.85
Change from Baseline at Week 1
GroupValue95% CI
Parsaclisib 1 mg QD0.9± 0.84
Parsaclisib 2.5 mg QD1.2± 1.30
Change from Baseline at Week 2
GroupValue95% CI
Parsaclisib 1 mg QD0.6± 0.96
Parsaclisib 2.5 mg QD1.5± 1.75
Change from Baseline at Week 4
GroupValue95% CI
Parsaclisib 1 mg QD0.9± 1.06
Parsaclisib 2.5 mg QD1.6± 1.79
Change from Baseline at Week 6
GroupValue95% CI
Parsaclisib 1 mg QD1.2± 0.80
Parsaclisib 2.5 mg QD1.7± 1.96
Change from Baseline at Week 8
GroupValue95% CI
Parsaclisib 1 mg QD1.2± 1.14
Parsaclisib 2.5 mg QD2.0± 2.07
Change from Baseline at Week 10
GroupValue95% CI
Parsaclisib 1 mg QD1.3± 1.14
Parsaclisib 2.5 mg QD2.1± 2.22
Change from Baseline at Week 12
GroupValue95% CI
Parsaclisib 1 mg QD1.3± 1.46
Parsaclisib 2.5 mg QD2.5± 2.67
Percentage Change From Baseline in Hemoglobin Secondary · Baseline; up to 1638 days

Percentage change from Baseline was calculated as: (\[post-Baseline value minus the Baseline value\] / Baseline value) x 100.

Week 1
GroupValue95% CI
Parsaclisib 1 mg QD10.1± 9.23
Parsaclisib 2.5 mg QD14.4± 16.31
Week 2
GroupValue95% CI
Parsaclisib 1 mg QD6.0± 10.26
Parsaclisib 2.5 mg QD18.5± 20.71
Week 4
GroupValue95% CI
Parsaclisib 1 mg QD9.9± 10.83
Parsaclisib 2.5 mg QD19.2± 20.65
Week 6
GroupValue95% CI
Parsaclisib 1 mg QD13.3± 8.35
Parsaclisib 2.5 mg QD19.5± 22.35
Week 8
GroupValue95% CI
Parsaclisib 1 mg QD12.7± 12.16
Parsaclisib 2.5 mg QD23.4± 24.57
Week 10
GroupValue95% CI
Parsaclisib 1 mg QD14.1± 11.54
Parsaclisib 2.5 mg QD25.1± 26.30
Week 12
GroupValue95% CI
Parsaclisib 1 mg QD13.9± 15.72
Parsaclisib 2.5 mg QD30.2± 33.95
Follow-up Month 1
GroupValue95% CI
Parsaclisib 1 mg QD0.0± 12.26
Parsaclisib 2.5 mg QD-4.1± 7.92
Percentage of Participants Requiring Transfusions Secondary · Baseline; up to 1638 days

A participant was defined to have required a transfusion if his or her last transfusion was within 7 days of the visit date.

Week 1
GroupValue95% CI
Parsaclisib 1 mg QD0.0
Parsaclisib 2.5 mg QD6.7
Week 2
GroupValue95% CI
Parsaclisib 1 mg QD0.0
Parsaclisib 2.5 mg QD0.0
Week 4
GroupValue95% CI
Parsaclisib 1 mg QD22.2
Parsaclisib 2.5 mg QD0.0
Week 6
GroupValue95% CI
Parsaclisib 1 mg QD0.0
Parsaclisib 2.5 mg QD0.0
Week 8
GroupValue95% CI
Parsaclisib 1 mg QD0.0
Parsaclisib 2.5 mg QD15.4
Week 10
GroupValue95% CI
Parsaclisib 1 mg QD0.0
Parsaclisib 2.5 mg QD7.7
Week 12
GroupValue95% CI
Parsaclisib 1 mg QD0.0
Parsaclisib 2.5 mg QD0.0
Follow-up Month 1
GroupValue95% CI
Parsaclisib 1 mg QD0.0
Parsaclisib 2.5 mg QD0.0
Percentage of Participants Who Achieved Normalization of Hemoglobin, Haptoglobin, Lactate Dehydrogenase (LDH), Reticulocyte Count, Total Bilirubin, Direct Bilirubin, and Indirect Bilirubin Secondary · up to 1638 days

Normalization was determined by the Investigator based on normal ranges for the clinical reference laboratory.

Hemoglobin, Week 1
GroupValue95% CI
Parsaclisib 1 mg QD0.0
Parsaclisib 2.5 mg QD6.7
Hemoglobin, Week 2
GroupValue95% CI
Parsaclisib 1 mg QD0.0
Parsaclisib 2.5 mg QD6.7
Hemoglobin, Week 4
GroupValue95% CI
Parsaclisib 1 mg QD0.0
Parsaclisib 2.5 mg QD14.3
Hemoglobin, Week 6
GroupValue95% CI
Parsaclisib 1 mg QD0.0
Parsaclisib 2.5 mg QD21.4
Hemoglobin, Week 8
GroupValue95% CI
Parsaclisib 1 mg QD0.0
Parsaclisib 2.5 mg QD15.4
Hemoglobin, Week 10
GroupValue95% CI
Parsaclisib 1 mg QD0.0
Parsaclisib 2.5 mg QD15.4
Hemoglobin, Week 12
GroupValue95% CI
Parsaclisib 1 mg QD11.1
Parsaclisib 2.5 mg QD35.7
Hemoglobin, Follow-up Month 1
GroupValue95% CI
Parsaclisib 1 mg QD0.0
Parsaclisib 2.5 mg QD0.0
Percentage of Participants Requiring a Prednisone Dose Change (Increase or Decrease) Secondary · up to 1638 days

Prednisone use was monitored throughout the study.

Week 1, increased
GroupValue95% CI
Parsaclisib 1 mg QD0.0
Parsaclisib 2.5 mg QD6.7
Week 1, decreased
GroupValue95% CI
Parsaclisib 1 mg QD20.0
Parsaclisib 2.5 mg QD6.7
Week 2, increased
GroupValue95% CI
Parsaclisib 1 mg QD0.0
Parsaclisib 2.5 mg QD0.0
Week 2, decreased
GroupValue95% CI
Parsaclisib 1 mg QD20.0
Parsaclisib 2.5 mg QD13.3
Week 4, increased
GroupValue95% CI
Parsaclisib 1 mg QD0.0
Parsaclisib 2.5 mg QD0.0
Week 4, decreased
GroupValue95% CI
Parsaclisib 1 mg QD10.0
Parsaclisib 2.5 mg QD6.7
Week 6, increased
GroupValue95% CI
Parsaclisib 1 mg QD0.0
Parsaclisib 2.5 mg QD0.0
Week 6, decreased
GroupValue95% CI
Parsaclisib 1 mg QD10.0
Parsaclisib 2.5 mg QD6.7
Change From Baseline in the Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-F) Subscale Scores Secondary · Baseline; up to 1638 days

The FACIT-F subscale is a 13-item instrument designed to assess fatigue/tiredness and its impact on daily activities and functioning in a number of chronic diseases. Participants were asked to respond to 13 statements that people with the illness have said are important on the following scale: 0, not at all; 1, a little bit; 2, somewhat; 3, quite a bit; 4, very much. Participants were asked to indicate the response as it applied to the last 7 days. The total fatigue subscale score ranges from 0 to 52; a higher score indicates more severe impact on daily activities and functioning.

Baseline
GroupValue95% CI
Parsaclisib 1 mg QD32.6± 12.80
Parsaclisib 2.5 mg QD30.1± 13.88
Week 6
GroupValue95% CI
Parsaclisib 1 mg QD6.7± 12.44
Parsaclisib 2.5 mg QD8.2± 9.28
Week 12
GroupValue95% CI
Parsaclisib 1 mg QD6.2± 16.08
Parsaclisib 2.5 mg QD5.4± 15.49
Follow-up Month 1
GroupValue95% CI
Parsaclisib 1 mg QD2.0± 2.83
Parsaclisib 2.5 mg QD2.3± 10.69
Follow-up Month 2
GroupValue95% CI
Parsaclisib 1 mg QD1.5± 7.78
Parsaclisib 2.5 mg QD7.0± 15.56
Follow-up Month 3
GroupValue95% CI
Parsaclisib 1 mg QD-2.0± 21.21
Parsaclisib 2.5 mg QD13.0± 14.14

Adverse events — posted to ClinicalTrials.gov

Time frame: up to a maximum of 1638 days. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Parsaclisib 1 mg QD
Serious: 6/10 (60%)
Deaths: 0/10
Parsaclisib 2.5 mg QD
Serious: 5/15 (33%)
Deaths: 1/15

Serious adverse events (28 terms)

ReactionSystemParsaclisib 1 mg QDParsaclisib 2.5 mg QD
Acute respiratory failureRespiratory, thoracic and mediastinal disorders
COVID-19 pneumoniaInfections and infestations
Amoebic dysenteryInfections and infestations
Aortic thrombosisVascular disorders
Atypical mycobacterial infectionInfections and infestations
Autoimmune haemolytic anaemiaBlood and lymphatic system disorders
Bacterial sepsisInfections and infestations
COVID-19Infections and infestations
Campylobacter colitisInfections and infestations
Cytomegalovirus infection reactivationInfections and infestations
Diabetes insipidusEndocrine disorders
DiarrhoeaGastrointestinal disorders
Enterococcal infectionInfections and infestations
FallInjury, poisoning and procedural complications
Femur fractureInjury, poisoning and procedural complications
Haemolytic anaemiaBlood and lymphatic system disorders
HypernatraemiaMetabolism and nutrition disorders
Interstitial lung diseaseRespiratory, thoracic and mediastinal disorders
Invasive ductal breast carcinomaNeoplasms benign, malignant and unspecified (incl cysts and polyps)
PneumoniaInfections and infestations
Pseudomonas infectionInfections and infestations
PsoriasisSkin and subcutaneous tissue disorders
Pulmonary fibrosisRespiratory, thoracic and mediastinal disorders
Pulmonary haemorrhageRespiratory, thoracic and mediastinal disorders
Respiratory failureRespiratory, thoracic and mediastinal disorders
Other adverse events (123 terms — click to expand)

ReactionSystemParsaclisib 1 mg QDParsaclisib 2.5 mg QD
PyrexiaGeneral disorders
DiarrhoeaGastrointestinal disorders
Oedema peripheralGeneral disorders
ArthralgiaMusculoskeletal and connective tissue disorders
COVID-19Infections and infestations
HeadacheNervous system disorders
NauseaGastrointestinal disorders
Pain in extremityMusculoskeletal and connective tissue disorders
PneumoniaInfections and infestations
RashSkin and subcutaneous tissue disorders
Autoimmune haemolytic anaemiaBlood and lymphatic system disorders
CoughRespiratory, thoracic and mediastinal disorders
DizzinessNervous system disorders
DyspnoeaRespiratory, thoracic and mediastinal disorders
HyperuricaemiaMetabolism and nutrition disorders
HypokalaemiaMetabolism and nutrition disorders
Muscle spasmsMusculoskeletal and connective tissue disorders
NeutropeniaBlood and lymphatic system disorders
PsoriasisSkin and subcutaneous tissue disorders
Rash pruriticSkin and subcutaneous tissue disorders
SARS-CoV-2 test positiveInvestigations
VomitingGastrointestinal disorders
Abdominal distensionGastrointestinal disorders
Abdominal painGastrointestinal disorders
AgitationPsychiatric disorders
AnaemiaBlood and lymphatic system disorders
Angina pectorisCardiac disorders
ArrhythmiaCardiac disorders
AstheniaGeneral disorders
BacteraemiaInfections and infestations
BlisterSkin and subcutaneous tissue disorders
Blood bilirubin increasedInvestigations
Blood creatinine increasedInvestigations
Blood potassium decreasedInvestigations
Bone painMusculoskeletal and connective tissue disorders
Breath sounds abnormalInvestigations
Cardiac discomfortCardiac disorders
Chest discomfortGeneral disorders
ChromaturiaRenal and urinary disorders
Chronic obstructive pulmonary diseaseRespiratory, thoracic and mediastinal disorders

Most-reported serious reactions: Acute respiratory failure, COVID-19 pneumonia, Amoebic dysentery, Aortic thrombosis, Atypical mycobacterial infection, Autoimmune haemolytic anaemia, Bacterial sepsis, COVID-19.

Data from ClinicalTrials.gov NCT03538041 adverse events section.

Sponsor's own description

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.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. New Insights in Autoimmune Hemolytic Anemia: From Pathogenesis to Therapy Stage 1.
    Barcellini W, Zaninoni A, Giannotta JA, Fattizzo B. · · 2020 · cited 75× · PMID 33261023 · DOI 10.3390/jcm9123859
  2. Autoimmune hemolytic anemia: current knowledge and perspectives.
    Michalak SS, Olewicz-Gawlik A, Rupa-Matysek J, Wolny-Rokicka E, et al · · 2020 · cited 72× · PMID 33292368 · DOI 10.1186/s12979-020-00208-7
  3. Infectious Complications in Autoimmune Hemolytic Anemia.
    Giannotta JA, Fattizzo B, Cavallaro F, Barcellini W. · · 2021 · cited 23× · PMID 33466516 · DOI 10.3390/jcm10010164
  4. Targeting PI3K Signaling to Overcome Tumor Immunosuppression: Synergistic Strategies to Enhance Cancer Vaccine Efficacy.
    Cui R, Luo Z, Zhang X, Yu X, et al · · 2025 · cited 10× · PMID 40266213 · DOI 10.3390/vaccines13030292
  5. Autoimmune Hemolytic Anemias: Challenges in Diagnosis and Therapy.
    Barcellini W, Fattizzo B. · · 2024 · cited 10× · PMID 39371250 · DOI 10.1159/000540475
  6. Rise of the planet of rare anemias: An update on emerging treatment strategies.
    Fattizzo B, Motta I. · · 2022 · cited 8× · PMID 36698833 · DOI 10.3389/fmed.2022.1097426
  7. Parsaclisib for the treatment of primary autoimmune hemolytic anemia: Results from a phase 2, open-label study.
    Barcellini W, Pane F, Patriarca A, Murakhovskaya I, et al · · 2024 · cited 7× · PMID 39435908 · DOI 10.1002/ajh.27493
  8. The EHA Research Roadmap: Anemias.
    Iolascon A, Rivella S, Anagnou NP, Camaschella C, et al · · 2021 · cited 7× · PMID 34522846 · DOI 10.1097/hs9.0000000000000607

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Other trials of Parsaclisib

Trials testing the same drug.

Other recruiting trials for Autoimmune Hemolytic Anemia

Currently open trials in the same condition.

Other Incyte Corporation trials

Trials by the same sponsor.

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Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03538041.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing