Trial Outcomes & Findings for A Study to Investigate the Pharmacokinetics, Safety, and Tolerability of Balovaptan in Children With Autism Spectrum Disorder (NCT NCT04049578)
NCT ID: NCT04049578
Last Updated: 2020-10-14
Results Overview
Recruitment status
TERMINATED
Study phase
PHASE1
Target enrollment
2 participants
Primary outcome timeframe
Predose, 2, 4, 6 hours postdose at Week 2; predose at Week 6; predose at Week 12; predose at Week 24, predose at Week 54
Results posted on
2020-10-14
Participant Flow
Participant milestones
| Measure |
Balovaptan
Participants were to receive an oral dose of balovaptan once a day (QD) for a 6-week treatment period, followed by an optional extension period of 48 weeks.
|
|---|---|
|
Overall Study
STARTED
|
2
|
|
Overall Study
COMPLETED
|
1
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
Balovaptan
Participants were to receive an oral dose of balovaptan once a day (QD) for a 6-week treatment period, followed by an optional extension period of 48 weeks.
|
|---|---|
|
Overall Study
Physician Decision
|
1
|
Baseline Characteristics
A Study to Investigate the Pharmacokinetics, Safety, and Tolerability of Balovaptan in Children With Autism Spectrum Disorder
Baseline characteristics by cohort
| Measure |
Balovaptan
n=2 Participants
Participants were to receive an oral dose of balovaptan once a day (QD) for a 6-week treatment period, followed by an optional extension period of 48 weeks.
|
|---|---|
|
Age, Continuous
|
3.45 Years
STANDARD_DEVIATION 0.78 • n=99 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
1 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
White
|
2 Participants
n=99 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
PRIMARY outcome
Timeframe: Predose, 2, 4, 6 hours postdose at Week 2; predose at Week 6; predose at Week 12; predose at Week 24, predose at Week 54Population: Due to low enrollment number patient analysis are not provided to protect participant confidentiality.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Predose, 2, 4, 6 hours postdose at Week 2; predose at Week 6; predose at Week 12; predose at Week 24, predose at Week 54Population: Due to low enrollment number patient analysis are not provided to protect participant confidentiality.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Predose, 2, 4, 6 hours postdose at Week 2; predose at Week 6; predose at Week 12; predose at Week 24, predose at Week 54Population: Due to low enrollment number patient analysis are not provided to protect participant confidentiality.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Predose, 2, 4, 6 hours postdose at Week 2; predose at Week 6; predose at Week 12; predose at Week 24, predose at Week 54Population: Due to low enrollment number patient analysis are not provided to protect participant confidentiality.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Predose, 2, 4, 6 hours postdose at Week 2; predose at Week 6; predose at Week 12; predose at Week 24, predose at Week 54Population: Due to low enrollment number patient analysis are not provided to protect participant confidentiality.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Predose, 2, 4, 6 hours postdose at Week 2; predose at Week 6; predose at Week 12; predose at Week 24, predose at Week 54Population: Due to low enrollment number patient analysis are not provided to protect participant confidentiality.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Up to approximately week 20Outcome measures
| Measure |
Balovaptan
n=2 Participants
Participants were to receive an oral dose of balovaptan once a day (QD) for a 6-week treatment period, followed by an optional extension period of 48 weeks.
|
|---|---|
|
Number of Participants With Adverse Events
|
2 Number of Participants
|
Adverse Events
Balovaptan
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Balovaptan
n=2 participants at risk
Participants were to receive an oral dose of balovaptan once a day (QD) for a 6-week treatment period, followed by an optional extension period of 48 weeks.
|
|---|---|
|
Investigations
Blood thyroid stimulating hormone increased
|
50.0%
1/2 • Number of events 1 • From the first study drug to the data cutoff date: 6 May 2020 (up to approximately 20 weeks)
The safety population is defined as patients who received any amount of study treatment.
|
|
Metabolism and nutrition disorders
Abnormal weight gain
|
50.0%
1/2 • Number of events 1 • From the first study drug to the data cutoff date: 6 May 2020 (up to approximately 20 weeks)
The safety population is defined as patients who received any amount of study treatment.
|
|
Nervous system disorders
Psychomotor hyperactivity
|
50.0%
1/2 • Number of events 1 • From the first study drug to the data cutoff date: 6 May 2020 (up to approximately 20 weeks)
The safety population is defined as patients who received any amount of study treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
50.0%
1/2 • Number of events 1 • From the first study drug to the data cutoff date: 6 May 2020 (up to approximately 20 weeks)
The safety population is defined as patients who received any amount of study treatment.
|
|
Vascular disorders
Haematoma
|
50.0%
1/2 • Number of events 1 • From the first study drug to the data cutoff date: 6 May 2020 (up to approximately 20 weeks)
The safety population is defined as patients who received any amount of study treatment.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The Study being conducted under this Agreement is part of the Overall Study. Investigator is free to publish in reputable journals or to present at professional conferences the results of the Study, but only after the first publication or presentation that involves the Overall Study. The Sponsor may request that Confidential Information be deleted and/or the publication be postponed in order to protect the Sponsor's intellectual property rights.
- Publication restrictions are in place
Restriction type: OTHER