Trial Outcomes & Findings for Posaconazole Prophylaxis During ATG Treatment for hMDS/AA Patients (NCT NCT03318159)

Results Overview

Define Invasive fungal infections according to guidelines of the Invasive Fungal Infections Cooperative Group of the European Organization for Research and Treatment of Cancer and Mycoses Study Group of the National Institute of Allergy and Infectious Diseases (Ascioglu S, Rex JH, de Pauw B, et al. Defining opportunistic invasive fungal infections in immunocompromised patients with cancer and hematopoietic stem cell transplants: an international consensus. Clin Infect Dis. 2008;46:1813-1821.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

20 participants

Primary outcome timeframe

during 4 weeks of posaconazole prophylaxis (i.e. upto 4 weeks)

Results posted on

2024-12-20

Participant Flow

Participant milestones

Participant milestones
Measure	Posaconazole Prophylaxis Group aplastic anemia / hypoplastic myelodysplastic syndrome patients undergoing antithymocyte globulin treatment and receiving posaconazole as prophylaxis antifungal agent Posaconazole: Dosing of posaconazole Posaconazole tablet 300 mg twice daily on day 1; Maintenance dose: 300 mg once daily on day 2 and thereafter. Treatment period: 4 weeks \*if posaconazole tablet intolerance: posaconazole suspension 200mg tid
Overall Study STARTED	20
Overall Study COMPLETED	14
Overall Study NOT COMPLETED	6

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Posaconazole Prophylaxis During ATG Treatment for hMDS/AA Patients

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Posaconazole Prophylaxis Group n=20 Participants aplastic anemia / hypoplastic myelodysplastic syndrome patients undergoing antithymocyte globulin treatment and receiving posaconazole as prophylaxis antifungal agent Posaconazole: Dosing of posaconazole Posaconazole tablet 300 mg twice daily on day 1; Maintenance dose: 300 mg once daily on day 2 and thereafter. Treatment period: 4 weeks \*if posaconazole tablet intolerance: posaconazole suspension 200mg tid
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	14 Participants n=5 Participants
Age, Categorical >=65 years	6 Participants n=5 Participants
Sex: Female, Male Female	10 Participants n=5 Participants
Sex: Female, Male Male	10 Participants n=5 Participants
Race/Ethnicity, Customized Asian/ Not Hispanic or Latino	20 Participants n=5 Participants
Region of Enrollment South Korea	20 Participants n=5 Participants

PRIMARY outcome

Timeframe: during 4 weeks of posaconazole prophylaxis (i.e. upto 4 weeks)

Define Invasive fungal infections according to guidelines of the Invasive Fungal Infections Cooperative Group of the European Organization for Research and Treatment of Cancer and Mycoses Study Group of the National Institute of Allergy and Infectious Diseases (Ascioglu S, Rex JH, de Pauw B, et al. Defining opportunistic invasive fungal infections in immunocompromised patients with cancer and hematopoietic stem cell transplants: an international consensus. Clin Infect Dis. 2008;46:1813-1821.

Outcome measures

Outcome measures
Measure	Posaconazole Prophylaxis Group n=20 Participants aplastic anemia / hypoplastic myelodysplastic syndrome patients undergoing antithymocyte globulin treatment and receiving posaconazole as prophylaxis antifungal agent Posaconazole: Dosing of posaconazole Posaconazole tablet 300 mg twice daily on day 1; Maintenance dose: 300 mg once daily on day 2 and thereafter. Treatment period: 4 weeks \*if posaconazole tablet intolerance: posaconazole suspension 200mg tid
the Incidence of Proven/Probable/Possible Fungal Infection	20 Participants

SECONDARY outcome

Timeframe: through study completion, an average of 18 months

The overall survival (OS) curves will be estimated using the Kaplan-Meier method

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: through study completion, an average of 18 months

Define Invasive fungal infections according to guidelines of the Invasive Fungal Infections Cooperative Group of the European Organization for Research and Treatment of Cancer and Mycoses Study Group of the National Institute of Allergy and Infectious Diseases (Ascioglu S, Rex JH, de Pauw B, et al. Defining opportunistic invasive fungal infections in immunocompromised patients with cancer and hematopoietic stem cell transplants: an international consensus. Clin Infect Dis. 2008;46:1813-1821.

Outcome measures

Outcome data not reported

Adverse Events

Posaconazole Prophylaxis Group

Serious events: 4 serious events

Other events: 4 other events

Deaths: 6 deaths

Serious adverse events

Serious adverse events
Measure	Posaconazole Prophylaxis Group n=20 participants at risk aplastic anemia / hypoplastic myelodysplastic syndrome patients undergoing antithymocyte globulin treatment and receiving posaconazole as prophylaxis antifungal agent Posaconazole: Dosing of posaconazole Posaconazole tablet 300 mg twice daily on day 1; Maintenance dose: 300 mg once daily on day 2 and thereafter. Treatment period: 4 weeks \*if posaconazole tablet intolerance: posaconazole suspension 200mg tid
Infections and infestations Herpes zoster	5.0% 1/20 • 6 months Definition of adverse event and serious adverse event, used to collect adverse event informations are not differs from the clinicaltrials.gov definitions.
Blood and lymphatic system disorders Neutropenia	5.0% 1/20 • 6 months Definition of adverse event and serious adverse event, used to collect adverse event informations are not differs from the clinicaltrials.gov definitions.
Infections and infestations Cellulitis	5.0% 1/20 • 6 months Definition of adverse event and serious adverse event, used to collect adverse event informations are not differs from the clinicaltrials.gov definitions.
Cardiac disorders Cardiac arrest	5.0% 1/20 • 6 months Definition of adverse event and serious adverse event, used to collect adverse event informations are not differs from the clinicaltrials.gov definitions.

Other adverse events

Other adverse events
Measure	Posaconazole Prophylaxis Group n=20 participants at risk aplastic anemia / hypoplastic myelodysplastic syndrome patients undergoing antithymocyte globulin treatment and receiving posaconazole as prophylaxis antifungal agent Posaconazole: Dosing of posaconazole Posaconazole tablet 300 mg twice daily on day 1; Maintenance dose: 300 mg once daily on day 2 and thereafter. Treatment period: 4 weeks \*if posaconazole tablet intolerance: posaconazole suspension 200mg tid
Investigations Liver function abnormality	20.0% 4/20 • 6 months Definition of adverse event and serious adverse event, used to collect adverse event informations are not differs from the clinicaltrials.gov definitions.

Additional Information

Dr. Koh

Seoul National University Hospital

Phone: 82220727217

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place