Evaluate Efficacy and Safety/Tolerability Profiles of Antroquinonol in Acute Myeloid Leukemia (AML) Adult Patients

Open-label, Non-randomized, Phase IIa Study to Evaluate Efficacy and Safety/Tolerability Profiles of Antroquinonol in Adult Patients With Relapsed Acute Myeloid Leukemia (AML) or at Initial Diagnosis When no Intensive Treatment is Possible

This is a IIa phase IIa open-label, non-randomized clinical trial of Antroquinonol, capsule, 100 mg (Golden Biotechnology Corporation, Taiwan) in patients with AML.

Study Overview

• Status: Completed
• Conditions: Leukemia, Myeloid, Acute
• Intervention / Treatment: Drug: Antroquinonol

Detailed Description

The given pilot clinical trial of a medicinal product for medical use, is held to evaluate preliminary efficacy and safety/tolerability profiles of antroquinonol in adult patients with relapsed AML or at initial diagnosis when no intensive treatment is possible.

• Study Type: Interventional
• Enrollment (Actual): 12
• Phase: Phase 2

Contacts and Locations

Study Locations

• Russian Federation
  • Moscow, Russian Federation, 125101
    • City clinical hospital n.a. S.P.Botkin
  • Tula, Russian Federation, 300053
    • Tula Regional Clinical Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 70 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Diagnosis of relapsed AML after at least 1 chemotherapy regimen or at initial diagnosis when no intensive treatment possible (based on standard criteria).
2. Men and women 18 to 70 years of age.
3. ECOG performance status = 0 or 1.
4. Ability to comply with the study requirements and give written informed consent.
5. Expected survival more than 3 months.
6. Patients with preserved reproductive potential agree to use, with their partner, adequate contraception throughout the study and for 30 days thereafter (contraceptive methods with reliability greater than 90%: cervical caps with spermicide, diaphragms with spermicide, condoms with intravaginal spermicide, non-hormonal intrauterine devices), or true sexual abstinence.

Exclusion Criteria:

1. Acute promyelocytic leukemia (APL).
2. Hemoglobin ≤ 7g/dL or platelet ≤ 50,000.

3. Abnormal liver and renal function:

   • Total bilirubin > 2 mg/dL;
   • AST and ALT > 2.5 × ULN;
   • Creatinine > 1.5 × ULN, OR creatinine clearance < 50 mL/min/1.73m2.
4. The subject has not recovered to grade ≤ 1 adverse events due to investigational or chemotherapeutic drugs or stem cell transplantation which were administered > 4 weeks prior to study enrollment.
5. Patient who has had in the past 3 years any previous or concomitant malignancy EXCEPT adequately treated basal or squamous cell carcinoma of the skin, in situ carcinoma of the cervix or bladder, in situ ductal carcinoma of the breast.
6. Subjects with any serious active infection (i.e., requiring an intravenous antibiotic, antifungal, or antiviral agent).
7. Subjects with known human immunodeficiency virus, active hepatitis B or C.
8. Subjects who have any other life-threatening illness or organ system dysfunction, which in the opinion of the Investigator, would either compromise subject safety or interfere with the evaluation of the safety of the study drug.
9. Known or suspected substance abuse or alcohol abuse.
10. Patients with history of seizure disorders or central nervous system leukemia.
11. Patients with uncontrolled, intercurrent illness including, but not limited to, symptomatic neurological illness; active, uncontrolled, systemic infection considered opportunistic, life-threatening, or clinically significant at the time of treatment; symptomatic congestive heart failure; unstable angina pectoris; cardiac arrhythmia; significant pulmonary disease or hypoxia; or psychiatric illness/social situations that would limit compliance with study requirements.
12. Inability to swallow oral medications or a recent acute gastrointestinal disorder with diarrhea e.g., Crohn's disease, malabsorption, or CTCAE Grade > 2 diarrhea of any etiology at baseline.
13. Prior major surgery or trauma within 28 days prior to first dose of study drug.
14. A positive urine pregnancy test (strip) for female patients of childbearing potential.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Antroquinonol; Patients will receive Antroquinonol 200 mg BID on Day 1 for 4 weeks or until transfusion of red blood cell or platelet ≧ 2, unacceptable toxicity, non-compliance or withdrawal of consent by the patient, or the investigator decides to discontinue treatment, whichever comes first.	Drug: Antroquinonol; Antroquinonol: 100 mg and corn oil 100 mg encapsulated in a gelatin capsule administered orally. Dose will be selected(200mg TID or 300mg TID with SOC) after phase I, then follow up the best dose for 40 Patients for the efficacy.; Other Names: Hocena

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Complete blood count measurements according International Working Group (IWG) response criteria	Evaluation of preliminary efficacy profile of antroquinonol in adult patients with relapsed AML or at initial diagnosis when no intensive treatment is possible.	2 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants alive at 4weeks	patients' situation	4 weeks
Number of Participants alive at 24 weeks	patients' situation	24 weeks

Collaborators and Investigators

• Sponsor: Golden Biotechnology Corporation

Study record dates

Study Major Dates

• Study Start (Actual): February 20, 2019
• Primary Completion (Actual): December 31, 2020
• Study Completion (Actual): December 31, 2020

Study Registration Dates

• First Submitted: January 27, 2019
• First Submitted That Met QC Criteria: January 28, 2019
• First Posted (Actual): January 30, 2019

Study Record Updates

• Last Update Posted (Actual): February 3, 2021
• Last Update Submitted That Met QC Criteria: February 1, 2021
• Last Verified: February 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Leukemia; Leukemia, Myeloid; Leukemia, Myeloid, Acute
• Other Study ID Numbers: GHAML-2-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No