Trial Outcomes & Findings for Isavuconazole in Preventing Invasive Fungal Infections in Adult Patients With Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome and Neutropenia (NCT NCT03019939)

NCT ID: NCT03019939

Last Updated: 2021-10-26

Results Overview

Participants with proven or possible invasive fungal infections.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 65 participants
• Primary outcome timeframe: Up to 100 days from prophylaxis initiation
• Results posted on: 2021-10-26

Participant Flow

Recruitment Period: April 28, 2017 to July 26, 2019

Participant milestones

Measure	Prevention (Isavuconazole) Patients receive isavuconazole PO every 8 hours for 6 doses and then QD or IV over 1 hour every 8 hours for 6 doses and then QD for up to 4 days for 12 weeks in the absence of disease progression or unacceptable toxicity. Isavuconazole: Given PO or IV
Overall Study STARTED	65
Overall Study COMPLETED	65
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Isavuconazole in Preventing Invasive Fungal Infections in Adult Patients With Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome and Neutropenia

Baseline characteristics by cohort

Measure	Prevention (Isavuconazole) n=65 Participants Patients receive isavuconazole PO every 8 hours for 6 doses and then QD or IV over 1 hour every 8 hours for 6 doses and then QD for up to 4 days for 12 weeks in the absence of disease progression or unacceptable toxicity. Isavuconazole: Given PO or IV
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	31 Participants n=5 Participants
Age, Categorical >=65 years	34 Participants n=5 Participants
Age, Continuous	67 years n=5 Participants
Sex: Female, Male Female	29 Participants n=5 Participants
Sex: Female, Male Male	36 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	3 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	3 Participants n=5 Participants
Race (NIH/OMB) White	57 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	2 Participants n=5 Participants
Region of Enrollment United States	65 participants n=5 Participants

PRIMARY outcome

Timeframe: Up to 100 days from prophylaxis initiation

Participants with proven or possible invasive fungal infections.

Outcome measures

Measure	Prevention (Isavuconazole) n=65 Participants Patients receive isavuconazole PO every 8 hours for 6 doses and then QD or IV over 1 hour every 8 hours for 6 doses and then QD for up to 4 days for 12 weeks in the absence of disease progression or unacceptable toxicity. Isavuconazole: Given PO or IV
Number of Participants With Proven or Probable Invasive Fungal Infections (IFIs)	4 Participants

SECONDARY outcome

Timeframe: Up to 100 days from prophylaxis initiation

Participants with invasive aspergillosissured.

Outcome measures

Measure	Prevention (Isavuconazole) n=65 Participants Patients receive isavuconazole PO every 8 hours for 6 doses and then QD or IV over 1 hour every 8 hours for 6 doses and then QD for up to 4 days for 12 weeks in the absence of disease progression or unacceptable toxicity. Isavuconazole: Given PO or IV
Number of Participants With Invasive Aspergillosis	2 Participants

SECONDARY outcome

Timeframe: Up to 100 days from prophylaxis initiation

Participants with other IFIs will be measured.

Outcome measures

Measure	Prevention (Isavuconazole) n=65 Participants Patients receive isavuconazole PO every 8 hours for 6 doses and then QD or IV over 1 hour every 8 hours for 6 doses and then QD for up to 4 days for 12 weeks in the absence of disease progression or unacceptable toxicity. Isavuconazole: Given PO or IV
Number of Participants With Other Invasive Fungal Infections (IFIs)	2 Participants

SECONDARY outcome

Timeframe: Up to 3 years

Will evaluate versus (vs.) failure (defined as Participants with proven or probable IFI, receipt of any other systemic antifungal agent for +/- 4 days for suspected IFI, occurrence of an adverse events possibly or probably related to the study drug resulting in discontinuation of treatment, or withdrawal from the study with no additional follow-up).

Outcome measures

Measure	Prevention (Isavuconazole) n=65 Participants Patients receive isavuconazole PO every 8 hours for 6 doses and then QD or IV over 1 hour every 8 hours for 6 doses and then QD for up to 4 days for 12 weeks in the absence of disease progression or unacceptable toxicity. Isavuconazole: Given PO or IV
Number of Participants With Treatment Success	46 Participants

SECONDARY outcome

Timeframe: Up to 3 years

Will evaluate versus success. Success is defined as Participants with proven or probable IFI, receipt of any other systemic antifungal agent for +/- 4 days for suspected IFI, occurrence of an adverse events possibly or probably related to the study drug resulting in discontinuation of treatment, or withdrawal from the study with no additional follow-up).

Outcome measures

Measure	Prevention (Isavuconazole) n=65 Participants Patients receive isavuconazole PO every 8 hours for 6 doses and then QD or IV over 1 hour every 8 hours for 6 doses and then QD for up to 4 days for 12 weeks in the absence of disease progression or unacceptable toxicity. Isavuconazole: Given PO or IV
Number of Participants Who Failed Treatment	19 Participants

SECONDARY outcome

Timeframe: Up to 3 years

Time from date of treatment start until date of death due to any cause or last Follow-up.

Outcome measures

Measure	Prevention (Isavuconazole) n=65 Participants Patients receive isavuconazole PO every 8 hours for 6 doses and then QD or IV over 1 hour every 8 hours for 6 doses and then QD for up to 4 days for 12 weeks in the absence of disease progression or unacceptable toxicity. Isavuconazole: Given PO or IV
Overall Survival (OS)	19.9 Months Interval 0.9 to 19.9

SECONDARY outcome

Timeframe: Up to 3 years

Time measured in days from start of treatment to IFI or off study date

Outcome measures

Measure	Prevention (Isavuconazole) n=65 Participants Patients receive isavuconazole PO every 8 hours for 6 doses and then QD or IV over 1 hour every 8 hours for 6 doses and then QD for up to 4 days for 12 weeks in the absence of disease progression or unacceptable toxicity. Isavuconazole: Given PO or IV
Invasive Fungal Infections (IFIs)-Free Survival	86 Days Interval 7.0 to 86.0

SECONDARY outcome

Timeframe: Up to 3 years

Time to death from any cause will be measured.

Outcome measures

Measure	Prevention (Isavuconazole) n=65 Participants Patients receive isavuconazole PO every 8 hours for 6 doses and then QD or IV over 1 hour every 8 hours for 6 doses and then QD for up to 4 days for 12 weeks in the absence of disease progression or unacceptable toxicity. Isavuconazole: Given PO or IV
Time to Death From Any Cause	4 Months Interval 1.1 to 16.4

SECONDARY outcome

Timeframe: Up to 3 years

Death's from invasive fungal infections

Outcome measures

Measure	Prevention (Isavuconazole) n=65 Participants Patients receive isavuconazole PO every 8 hours for 6 doses and then QD or IV over 1 hour every 8 hours for 6 doses and then QD for up to 4 days for 12 weeks in the absence of disease progression or unacceptable toxicity. Isavuconazole: Given PO or IV
Number of Participants With Death Related to Invasive Fungal Infections (IFIs)	0 Participants

SECONDARY outcome

Timeframe: Up to 3 years

Time measured in days from start of treatment to invasive fungal infections

Outcome measures

Measure	Prevention (Isavuconazole) n=65 Participants Patients receive isavuconazole PO every 8 hours for 6 doses and then QD or IV over 1 hour every 8 hours for 6 doses and then QD for up to 4 days for 12 weeks in the absence of disease progression or unacceptable toxicity. Isavuconazole: Given PO or IV
Time to Diagnosis of Proven or Probable Invasive Fungal Infections (IFIs)	24 Days Interval 7.0 to 28.0

SECONDARY outcome

Timeframe: Up to 3 years

Time days from start of empiric anti-fungal therapy.

Outcome measures

Measure	Prevention (Isavuconazole) n=65 Participants Patients receive isavuconazole PO every 8 hours for 6 doses and then QD or IV over 1 hour every 8 hours for 6 doses and then QD for up to 4 days for 12 weeks in the absence of disease progression or unacceptable toxicity. Isavuconazole: Given PO or IV
Time to Initiation of Empiric Anti-fungal Therapy	22 Days Interval 7.0 to 80.0

Adverse Events

Prevention (Isavuconazole)

Serious events: 30 serious events

Other events: 14 other events

Deaths: 4 deaths

Serious adverse events

Measure	Prevention (Isavuconazole) n=65 participants at risk Patients receive isavuconazole PO every 8 hours for 6 doses and then QD or IV over 1 hour every 8 hours for 6 doses and then QD for up to 4 days for 12 weeks in the absence of disease progression or unacceptable toxicity. Isavuconazole: Given PO or IV
General disorders Abdominal Pain	1.5% 1/65 • Number of events 1 • Up to 3 years
Renal and urinary disorders Acute Kidney Injury	1.5% 1/65 • Number of events 1 • Up to 3 years
Musculoskeletal and connective tissue disorders Arthralgia	1.5% 1/65 • Number of events 2 • Up to 3 years
General disorders Death	1.5% 1/65 • Number of events 1 • Up to 3 years
Respiratory, thoracic and mediastinal disorders Dyspnea	1.5% 1/65 • Number of events 1 • Up to 3 years
General disorders Fever	6.2% 4/65 • Number of events 5 • Up to 3 years
Infections and infestations Infection	3.1% 2/65 • Number of events 2 • Up to 3 years
Infections and infestations Lung Infection	1.5% 1/65 • Number of events 1 • Up to 3 years
Gastrointestinal disorders Nausea	1.5% 1/65 • Number of events 1 • Up to 3 years
Blood and lymphatic system disorders Neutropenic Fever	26.2% 17/65 • Number of events 19 • Up to 3 years
General disorders Pain Extremity	1.5% 1/65 • Number of events 1 • Up to 3 years
Infections and infestations Sepsis	6.2% 4/65 • Number of events 4 • Up to 3 years
Infections and infestations Skin Infection	1.5% 1/65 • Number of events 1 • Up to 3 years
Cardiac disorders Supraventricular Tachycardia	1.5% 1/65 • Number of events 1 • Up to 3 years
Infections and infestations Upper Respiratory Infection	1.5% 1/65 • Number of events 1 • Up to 3 years

Other adverse events

Measure	Prevention (Isavuconazole) n=65 participants at risk Patients receive isavuconazole PO every 8 hours for 6 doses and then QD or IV over 1 hour every 8 hours for 6 doses and then QD for up to 4 days for 12 weeks in the absence of disease progression or unacceptable toxicity. Isavuconazole: Given PO or IV
Investigations Alanine Aminotransferase Increased	4.6% 3/65 • Number of events 3 • Up to 3 years
Investigations Aspartate Aminotransferase Increased	3.1% 2/65 • Number of events 2 • Up to 3 years
Gastrointestinal disorders Colitis	1.5% 1/65 • Number of events 1 • Up to 3 years
Reproductive system and breast disorders Enlarged Prostate	1.5% 1/65 • Number of events 1 • Up to 3 years
General disorders Fever	3.1% 2/65 • Number of events 2 • Up to 3 years
Vascular disorders Flushing	1.5% 1/65 • Number of events 1 • Up to 3 years
Gastrointestinal disorders Gastrointestinal Other	1.5% 1/65 • Number of events 1 • Up to 3 years
Nervous system disorders Headache	1.5% 1/65 • Number of events 1 • Up to 3 years
Infections and infestations Infection	1.5% 1/65 • Number of events 1 • Up to 3 years
Blood and lymphatic system disorders Neutropenic Fever	10.8% 7/65 • Number of events 8 • Up to 3 years
Gastrointestinal disorders Oral Pain	3.1% 2/65 • Number of events 2 • Up to 3 years
Infections and infestations Sinusitis	1.5% 1/65 • Number of events 1 • Up to 3 years
Metabolism and nutrition disorders Tumor Lysis Syndrome	1.5% 1/65 • Number of events 1 • Up to 3 years

Additional Information

Prithviraj Bose MD/Associate Porfessor

The University of Texas MD Anderson Cancer Center

Phone: 713-792-7747

Email: PBose@mdanderson.org

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place