Trial Outcomes & Findings for Evaluation of ALX-0171 in Japanese Children Hospitalized for Respiratory Syncytial Virus Lower Respiratory Tract Infection (NCT NCT03418571)

Last Updated: 2019-07-30

Results Overview

Number of subjects reported with at least 1 serious or non-serious TEAEs in the ALX-0171 1.5 mg/kg treatment group and placebo treatment group.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

15 participants

Primary outcome timeframe

From the subject's first study drug administration until completion of the subject's last visit, an average of 4 weeks

Results posted on

2019-07-30

Participant Flow

Sixteen subjects were screened for Cohort 1. Of these, 15 subjects were randomized. There was 1 screen failure due to consent withdrawal. For Cohort 2, 1 subject was screened but considered a screen failure (consent withdrawal). Consent was obtained from the first subject on 1 March 2018; last subject completed the final visit on 19 October 2018

All randomized subjects received at least 1 administration of study drug and were included in the Safety Population.

Participant milestones

Participant milestones
Measure	ALX-0171 1.5 mg/kg Single inhalation of ALX-0171 1.5 mg/kg once daily for 3 consecutive days.	Placebo Single inhalation of placebo once daily for 3 consecutive days.
Overall Study STARTED	12	3
Overall Study COMPLETED	11	3
Overall Study NOT COMPLETED	1	0

Reasons for withdrawal

Reasons for withdrawal
Measure	ALX-0171 1.5 mg/kg Single inhalation of ALX-0171 1.5 mg/kg once daily for 3 consecutive days.	Placebo Single inhalation of placebo once daily for 3 consecutive days.
Overall Study Consent withdrawn by legal guardian	1	0

Baseline Characteristics

Evaluation of ALX-0171 in Japanese Children Hospitalized for Respiratory Syncytial Virus Lower Respiratory Tract Infection

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	ALX-0171 1.5 mg/kg n=12 Participants Single inhalation of ALX-0171 1.5 mg/kg once daily for 3 consecutive days.	Placebo n=3 Participants Single inhalation of placebo once daily for 3 consecutive days.	Total n=15 Participants Total of all reporting groups
Age, Continuous	7.4 months STANDARD_DEVIATION 4.65 • n=5 Participants	3.5 months STANDARD_DEVIATION 3.04 • n=7 Participants	6.6 months STANDARD_DEVIATION 4.58 • n=5 Participants
Sex: Female, Male Female	5 Participants n=5 Participants	2 Participants n=7 Participants	7 Participants n=5 Participants
Sex: Female, Male Male	7 Participants n=5 Participants	1 Participants n=7 Participants	8 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	12 Participants n=5 Participants	3 Participants n=7 Participants	15 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: From the subject's first study drug administration until completion of the subject's last visit, an average of 4 weeks

Population: Safety Population

Number of subjects reported with at least 1 serious or non-serious TEAEs in the ALX-0171 1.5 mg/kg treatment group and placebo treatment group.

Outcome measures

Outcome measures
Measure	ALX-0171 1.5 mg/kg n=12 Participants Single inhalation of ALX-0171 1.5 mg/kg once daily for 3 consecutive days.	Placebo n=3 Participants Single inhalation of placebo once daily for 3 consecutive days.
Safety and Tolerability of Inhaled ALX-0171 1.5 mg/kg as Measured by the Number of Subjects With at Least 1 Serious or Non-Serious Treatment-emergent Adverse Event (TEAE). At least one serious TEAE	1 participants	0 participants
Safety and Tolerability of Inhaled ALX-0171 1.5 mg/kg as Measured by the Number of Subjects With at Least 1 Serious or Non-Serious Treatment-emergent Adverse Event (TEAE). At least one non-serious TEAE	8 participants	1 participants

PRIMARY outcome

Timeframe: From the subject's first study drug administration until completion of the subject's last visit, an average of 4 weeks

Population: Safety Population

Number of serious and non-serious TEAEs reported in the ALX-0171 1.5 mg/kg treatment group and placebo treatment group.

Outcome measures

Outcome measures
Measure	ALX-0171 1.5 mg/kg n=12 Participants Single inhalation of ALX-0171 1.5 mg/kg once daily for 3 consecutive days.	Placebo n=3 Participants Single inhalation of placebo once daily for 3 consecutive days.
Safety and Tolerability of Inhaled ALX-0171 1.5 mg/kg as Measured by the Number of Serious and Non-serious TEAEs. Serious TEAE	2 TEAE	0 TEAE
Safety and Tolerability of Inhaled ALX-0171 1.5 mg/kg as Measured by the Number of Serious and Non-serious TEAEs. Non-Serious TEAE	18 TEAE	1 TEAE

Adverse Events

ALX-0171 1.5 mg/kg

Serious events: 1 serious events

Other events: 8 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	ALX-0171 1.5 mg/kg n=12 participants at risk Single inhalation of ALX-0171 1.5 mg/kg once daily for 3 consecutive days.	Placebo n=3 participants at risk Single inhalation of placebo once daily for 3 consecutive days.
Infections and infestations Pneumonia hemophilus	8.3% 1/12 • Number of events 1 • From the subject's first study drug administration until the subject's study completion/discontinuation date, an average of 4 weeks.	0.00% 0/3 • From the subject's first study drug administration until the subject's study completion/discontinuation date, an average of 4 weeks.
Infections and infestations Pneumonia bacterial	8.3% 1/12 • Number of events 1 • From the subject's first study drug administration until the subject's study completion/discontinuation date, an average of 4 weeks.	0.00% 0/3 • From the subject's first study drug administration until the subject's study completion/discontinuation date, an average of 4 weeks.

Other adverse events

Additional Information

Medical Monitor

Ablynx NV

Phone: +32 9 262 00 00

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Publication of any results from this study will be according to the principles of the Declaration of Helsinki, in particular point 30, and will require prior review and written agreement of the Sponsor.
Publication restrictions are in place

Restriction type: OTHER

Measure	ALX-0171 1.5 mg/kg n=12 participants at risk Single inhalation of ALX-0171 1.5 mg/kg once daily for 3 consecutive days.	Placebo n=3 participants at risk Single inhalation of placebo once daily for 3 consecutive days.
Skin and subcutaneous tissue disorders Miliaria	25.0% 3/12 • Number of events 3 • From the subject's first study drug administration until the subject's study completion/discontinuation date, an average of 4 weeks.	0.00% 0/3 • From the subject's first study drug administration until the subject's study completion/discontinuation date, an average of 4 weeks.
Skin and subcutaneous tissue disorders Dermatitis contact	8.3% 1/12 • Number of events 1 • From the subject's first study drug administration until the subject's study completion/discontinuation date, an average of 4 weeks.	0.00% 0/3 • From the subject's first study drug administration until the subject's study completion/discontinuation date, an average of 4 weeks.
Skin and subcutaneous tissue disorders Dermatitis diaper	8.3% 1/12 • Number of events 1 • From the subject's first study drug administration until the subject's study completion/discontinuation date, an average of 4 weeks.	0.00% 0/3 • From the subject's first study drug administration until the subject's study completion/discontinuation date, an average of 4 weeks.
Skin and subcutaneous tissue disorders Erythema	8.3% 1/12 • Number of events 1 • From the subject's first study drug administration until the subject's study completion/discontinuation date, an average of 4 weeks.	0.00% 0/3 • From the subject's first study drug administration until the subject's study completion/discontinuation date, an average of 4 weeks.
Infections and infestations Upper respiratory tract infection	16.7% 2/12 • Number of events 2 • From the subject's first study drug administration until the subject's study completion/discontinuation date, an average of 4 weeks.	0.00% 0/3 • From the subject's first study drug administration until the subject's study completion/discontinuation date, an average of 4 weeks.
Infections and infestations Otitis media	8.3% 1/12 • Number of events 1 • From the subject's first study drug administration until the subject's study completion/discontinuation date, an average of 4 weeks.	0.00% 0/3 • From the subject's first study drug administration until the subject's study completion/discontinuation date, an average of 4 weeks.
Infections and infestations Otitis media acute	8.3% 1/12 • Number of events 1 • From the subject's first study drug administration until the subject's study completion/discontinuation date, an average of 4 weeks.	0.00% 0/3 • From the subject's first study drug administration until the subject's study completion/discontinuation date, an average of 4 weeks.
Infections and infestations Pneumonia bacterial	8.3% 1/12 • Number of events 1 • From the subject's first study drug administration until the subject's study completion/discontinuation date, an average of 4 weeks.	0.00% 0/3 • From the subject's first study drug administration until the subject's study completion/discontinuation date, an average of 4 weeks.
Gastrointestinal disorders Diarrhoea	8.3% 1/12 • Number of events 1 • From the subject's first study drug administration until the subject's study completion/discontinuation date, an average of 4 weeks.	0.00% 0/3 • From the subject's first study drug administration until the subject's study completion/discontinuation date, an average of 4 weeks.
Gastrointestinal disorders Gastric ulcer	8.3% 1/12 • Number of events 1 • From the subject's first study drug administration until the subject's study completion/discontinuation date, an average of 4 weeks.	0.00% 0/3 • From the subject's first study drug administration until the subject's study completion/discontinuation date, an average of 4 weeks.
Blood and lymphatic system disorders Iron deficiency anemia	8.3% 1/12 • Number of events 1 • From the subject's first study drug administration until the subject's study completion/discontinuation date, an average of 4 weeks.	0.00% 0/3 • From the subject's first study drug administration until the subject's study completion/discontinuation date, an average of 4 weeks.
Ear and labyrinth disorders Middle ear effusion	8.3% 1/12 • Number of events 1 • From the subject's first study drug administration until the subject's study completion/discontinuation date, an average of 4 weeks.	0.00% 0/3 • From the subject's first study drug administration until the subject's study completion/discontinuation date, an average of 4 weeks.
General disorders Edema	8.3% 1/12 • Number of events 1 • From the subject's first study drug administration until the subject's study completion/discontinuation date, an average of 4 weeks.	0.00% 0/3 • From the subject's first study drug administration until the subject's study completion/discontinuation date, an average of 4 weeks.
Hepatobiliary disorders Hepatic function abnormal	0.00% 0/12 • From the subject's first study drug administration until the subject's study completion/discontinuation date, an average of 4 weeks.	33.3% 1/3 • Number of events 1 • From the subject's first study drug administration until the subject's study completion/discontinuation date, an average of 4 weeks.
Nervous system disorders Myoclonus	8.3% 1/12 • Number of events 1 • From the subject's first study drug administration until the subject's study completion/discontinuation date, an average of 4 weeks.	0.00% 0/3 • From the subject's first study drug administration until the subject's study completion/discontinuation date, an average of 4 weeks.
Respiratory, thoracic and mediastinal disorders Upper respiratory tract inflammation	8.3% 1/12 • Number of events 1 • From the subject's first study drug administration until the subject's study completion/discontinuation date, an average of 4 weeks.	0.00% 0/3 • From the subject's first study drug administration until the subject's study completion/discontinuation date, an average of 4 weeks.