Avatrombopag for Thrombocytopenia in People With Cancer

NCT04437953 | Withdrawn Phase 2 FDA Drug

• 1 other identifier: 19-483
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

This study will test whether avatrombopag is an effective treatment for thrombocytopenia in people who have both cancer and a liver disease (such as cirrhosis, cholangitis, or hepatitis). Researchers will look at whether giving participants avatrombopag for 3 weeks can raise their platelet levels enough for them to begin chemotherapy. The study will also test whether avatrombopag can continue to be effective against thrombocytopenia while participants are on chemotherapy for 12 weeks or longer. In addition, researchers will determine how safe the study drug is in participants.

Conditions

Thrombocytopenia; Cancer; Liver Diseases

Keywords

Avatrombopag; 19-483

Outcome Measures

Primary Outcomes (1)

• Number of patients who achieve platelet counts of ≥ 100,000/mcL

  the platelet count is ≥ 100,000/mcL, this will be considered successful achievement of the primary endpoint

  within 3 weeks of treatment

Secondary Outcomes (1)

• Number of patients who can undergo systemic cancer therapy without more than one dose delay or dose reduction

  12 weeks

Study Arms (1)

Avatrombopag

EXPERIMENTAL

Patients will receive an initial dose of Avatrombopag 60 mg on Day 1.Starting on Day 2, the dose will be Avatrombopag 20 mg daily.

Drug: Avatrombopag

Interventions

Avatrombopag DRUG

Patients will receive an initial dose of Avatrombopag 60 mg on Day 1. Starting on Day 2, the dose will be Avatrombopag 20 mg daily. Once a patient has reached a platelet target of \> 100,000/mcL patients can initiate systemic cancer therapy. Avatrombopag will be continued as maintenance (subscript "m") and titrated weekly with a goal to maintain a platelet count of ≥ 100,000/mcL during administration of systemic cancer therapy. Duration of maintenance therapy will be at least 12 weeks (indicated by Week 1m, Week 2m, … 12m) starting from time of platelet correction and resumption of systemic cancer therapy. End of Treatment (EOT) is up to 15 weeks on study Week 12m.

Avatrombopag

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients with history of locally advanced or metastatic solid tumor, with a plan to initiate cancer directed systemic therapy for adjuvant or palliative purposes.
• years of age or greater
• Known liver disease attributed to one of the following:
• Chronic HBV and/or chronic HCV with Cirrhosis
• Alcoholic Cirrhosis
• Nonalcoholic Fatty Liver Disease with Cirrhosis
• Primary Biliary Cholangitis
• Primary Sclerosing Cholangitis
• Autoimmune Hepatitis
• Hereditary Hemochromatosis
• Wilsons Disease
• Alpha-1 Antitrypsin Deficiency
• Congestive Hepatopathy
• Cirrhosis or known pre-existing liver dysfunction of unknown cause, or not otherwise specified
• No specific Child-Pugh Score will be required for eligibility.

You may not qualify if:

• History of immune causes of thrombocytopenia (ITP).
• Presence of leukemia or myelodysplastic syndrome.
• Known bone metastases sufficient to result in at least one site of cortical bone destruction, osteolytic lesions, or osteoblastic lesions, on radiologic imaging.
• Patients who have previously received thrombopoietin mimetics such as romiplostim, eltrombopag, etc.
• Patients who require emergent systemic cancer therapy will be excluded.
• Patients who require emergent radiation therapy will be excluded.
• a. Note: Concomitant radiation therapy with systemic cancer therapy is permitted.
• Pancytopenia at enrollment (Hemoglobin \<9 g/dL and/or Absolute Neutrophil Count \< 1500/mcL).
• a. G-CSF and Red Blood Cell transfusions to treat anemia and neutropenia and achieve adequate hemoglobin and ANC are permitted.
• Patients with serum sodium ≤130 mEq/L will be excluded.
• Patients with a known bleeding disorder or platelet dysfunction will be excluded
• Baseline Prothrombin Time (PT) that is greater than 2" above the upper limit of normal.
• Activated Partial Thromboplastin Time (aPTT) that is greater than 3" above the upper limit of normal.
• Patients with known genetic prothrombotic conditions (Factor V Leiden, Prothrombin 20210A, Antithrombin deficiency or Protein C or S deficiency) or history of antiphospholipid syndrome.
• Patients on anticoagulation or NSAIDs within 7 days of enrollment will be excluded, with the exception of celecoxib.

Sponsors & Collaborators

• Memorial Sloan Kettering Cancer Center: lead
• Sobi, Inc.: collaborator

Related Links

• Memorial Sloan Kettering Cancer Center

MeSH Terms

Conditions

Thrombocytopenia; Neoplasms; Liver Diseases

Interventions

avatrombopag

Condition Hierarchy (Ancestors)

Blood Platelet Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Cytopenia; Digestive System Diseases

Study Officials

• Gerald Soff, MD

  Memorial Sloan Kettering Cancer Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: A Phase II single arm study will be an open label study examining the use of Avatrombopag.

Study Record Dates

• First Submitted: June 17, 2020
• First Posted: June 18, 2020
• Study Start: May 27, 2020
• Primary Completion: December 3, 2020
• Study Completion: December 3, 2020
• Last Updated: December 8, 2020

Record last verified: 2020-12

Data Sharing

• IPD Sharing: Will share