Trial Outcomes & Findings for Nebulized Amphotericin B Lipid Complex in Invasive Pulmonary Aspergillosis in Paediatric Patients With Acute Leukaemia (NCT NCT01615809)

Results Overview

is assessed by the proportion of patients who discontinue prophylactic treatment with Abelcet® due to an adverse event that is related or not to the study drug or for intolerability to it. The last week of treatment will have a different calendar for each participant, depending on the number of cicles needed by each patient (it has been anticipated up to 5 cicles of 2-6 weeks each).

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

32 participants

Primary outcome timeframe

at the Baseline visit (week 1) and during the Last week of treatment, up to 6 weeks

Results posted on

2018-03-29

Participant Flow

Participant milestones

Participant milestones
Measure	Amphotericin B (ABELCET®) Patients fulfilling inclusion criteria and those giving the general informed consent for the study will initiate nebulized Amphotericin B prophylaxis treatment, twice a week, during neutropenia periods (coincident with intensive chemotherapy treatment). AMPHOTERICIN B: The study drug will be administered by inhalation, to hospitalised patients or outpatients in the day hospital.The administration regimen for each Abelcet® nebulization will be 10 ml (50 mg) twice a week for the first week, and then from the second week onwards it will be 5 ml (25 mg) with a minimum separation of 72 hours between doses, until the neutrophil count is greater than or equal to 1500 cells/mm3.
Overall Study STARTED	32
Overall Study COMPLETED	22
Overall Study NOT COMPLETED	10

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Nebulized Amphotericin B Lipid Complex in Invasive Pulmonary Aspergillosis in Paediatric Patients With Acute Leukaemia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Amphotericin B (ABELCET®) n=32 Participants Patients fulfilling inclusion criteria and those giving the general informed consent for the study initiate nebulized Amphotericin B prophylaxis treatment, twice a week, during neutropenia periods (coincident with intensive chemotherapy treatment). AMPHOTERICIN B: The study drug will be administered by inhalation, to hospitalised patients or outpatients in the day hospital.The administration regimen for each Abelcet® nebulization was 10 ml (50 mg) twice a week for the first week, and then from the second week onwards was reduced to 5 ml (25 mg) with a minimum separation of 72 hours between doses, until the neutrophil count demonstrated to be greater than or equal to 1500 cells/mm3.
Age, Customized >= 3 years to <=18 years	32 participants n=5 Participants
Sex: Female, Male Female	13 Participants n=5 Participants
Sex: Female, Male Male	19 Participants n=5 Participants
Race/Ethnicity, Customized caucasian	27 participants n=5 Participants
Race/Ethnicity, Customized amerindian	2 participants n=5 Participants
Race/Ethnicity, Customized african	1 participants n=5 Participants
Race/Ethnicity, Customized gypsies	2 participants n=5 Participants
Region of Enrollment Spain	32 center n=5 Participants

PRIMARY outcome

Timeframe: at the Baseline visit (week 1) and during the Last week of treatment, up to 6 weeks

Population: pediatric patients

is assessed by the proportion of patients who discontinue prophylactic treatment with Abelcet® due to an adverse event that is related or not to the study drug or for intolerability to it. The last week of treatment will have a different calendar for each participant, depending on the number of cicles needed by each patient (it has been anticipated up to 5 cicles of 2-6 weeks each).

Outcome measures

Outcome measures
Measure	Amphotericin B (ABELCET®) n=32 Participants Patients fulfilling inclusion criteria and those giving the general informed consent for the study will initiate nebulized Amphotericin B prophylaxis treatment, twice a week, during neutropenia periods (coincident with intensive chemotherapy treatment). AMPHOTERICIN B: The study drug will be administered by inhalation, to hospitalised patients or outpatients in the day hospital.The administration regimen for each Abelcet® nebulization will be 10 ml (50 mg) twice a week for the first week, and then from the second week onwards it will be 5 ml (25 mg) with a minimum separation of 72 hours between doses, until the neutrophil count is greater than or equal to 1500 cells/mm3.
Number of Participants With Adverse Events That Results in the Interruption of Treatment, as a Measure of Safety and Tolerability	0 participants

SECONDARY outcome

Timeframe: at the Baseline visit (week 1) and at the end of the profilaxis treatment phase, up to 6 weeks

Population: At baseline, none of the 32 pediatric patients with acute leukemia included in the clinical trial had API. During the trial period there were 3 patients who developed API

The incidence of invasive pulmonary aspergillosis during the Abelcet® prophylactic treatment period was assessed by the relation between the number of patients with invasive pulmonary aspergillosis and the number of paediatric patients on prophylaxis with Acute Leukaemia (AL) undergoing intensive chemotherapy.

Outcome measures

Outcome measures
Measure	Amphotericin B (ABELCET®) n=32 Participants Patients fulfilling inclusion criteria and those giving the general informed consent for the study will initiate nebulized Amphotericin B prophylaxis treatment, twice a week, during neutropenia periods (coincident with intensive chemotherapy treatment). AMPHOTERICIN B: The study drug will be administered by inhalation, to hospitalised patients or outpatients in the day hospital.The administration regimen for each Abelcet® nebulization will be 10 ml (50 mg) twice a week for the first week, and then from the second week onwards it will be 5 ml (25 mg) with a minimum separation of 72 hours between doses, until the neutrophil count is greater than or equal to 1500 cells/mm3.
Efficacy of Primary Prophylaxis With Nebulized Abelcet® on the Incidence of Invasive Pulmonary Aspergillosis	3 participants

SECONDARY outcome

Timeframe: at the Baseline visit (week 1) and at the end of the profilaxis treatment phase, up to 6 weeks

Percentage of deaths related to Invasive Pulmonary Aspergillosis during the prophylactic treatment period with Abelcet® in paediatric patients with Acute Leukaemia undergoing intensive chemotherapy.

Outcome measures

Outcome measures
Measure	Amphotericin B (ABELCET®) n=32 Participants Patients fulfilling inclusion criteria and those giving the general informed consent for the study will initiate nebulized Amphotericin B prophylaxis treatment, twice a week, during neutropenia periods (coincident with intensive chemotherapy treatment). AMPHOTERICIN B: The study drug will be administered by inhalation, to hospitalised patients or outpatients in the day hospital.The administration regimen for each Abelcet® nebulization will be 10 ml (50 mg) twice a week for the first week, and then from the second week onwards it will be 5 ml (25 mg) with a minimum separation of 72 hours between doses, until the neutrophil count is greater than or equal to 1500 cells/mm3.
Invasive Pulmonary Aspergillosis -Related Mortality During Primary Prophylaxis With Abelcet®.	0 percentage of deaths Interval 0.0 to 0.0

Adverse Events

Amphotericin B (ABELCET®)

Serious events: 22 serious events

Other events: 32 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Amphotericin B (ABELCET®) n=32 participants at risk Patients fulfilling inclusion criteria and those giving the general informed consent for the study will initiate nebulized Amphotericin B prophylaxis treatment, twice a week, during neutropenia periods (coincident with intensive chemotherapy treatment). AMPHOTERICIN B: The study drug will be administered by inhalation, to hospitalised patients or outpatients in the day hospital.The administration regimen for each Abelcet® nebulization will be 10 ml (50 mg) twice a week for the first week, and then from the second week onwards it will be 5 ml (25 mg) with a minimum separation of 72 hours between doses, until the neutrophil count is greater than or equal to 1500 cells/mm3.
Blood and lymphatic system disorders febrile neutropenia	50.0% 16/32 • Number of events 26
General disorders fever	25.0% 8/32 • Number of events 13
Metabolism and nutrition disorders hyperglycemia	6.2% 2/32 • Number of events 3
Blood and lymphatic system disorders Febrile Aplasia	9.4% 3/32 • Number of events 3
Nervous system disorders Methotrexate-induced neurotoxicity	9.4% 3/32 • Number of events 3
Blood and lymphatic system disorders macroscopic hematuria	3.1% 1/32 • Number of events 3

Other adverse events

Other adverse events
Measure	Amphotericin B (ABELCET®) n=32 participants at risk Patients fulfilling inclusion criteria and those giving the general informed consent for the study will initiate nebulized Amphotericin B prophylaxis treatment, twice a week, during neutropenia periods (coincident with intensive chemotherapy treatment). AMPHOTERICIN B: The study drug will be administered by inhalation, to hospitalised patients or outpatients in the day hospital.The administration regimen for each Abelcet® nebulization will be 10 ml (50 mg) twice a week for the first week, and then from the second week onwards it will be 5 ml (25 mg) with a minimum separation of 72 hours between doses, until the neutrophil count is greater than or equal to 1500 cells/mm3.
General disorders Fever	100.0% 32/32 • Number of events 32
Gastrointestinal disorders abdominal pain	90.6% 29/32 • Number of events 32
General disorders paleness	87.5% 28/32 • Number of events 32
General disorders physical pain	75.0% 24/32 • Number of events 32
Gastrointestinal disorders diarrhea	65.6% 21/32 • Number of events 32

Additional Information

Rosa Maria morales Palau

Fundaciò per la Recerca i la Docencia sant Joan de Deu

Phone: 936009751

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: GT60