Trial Outcomes & Findings for Safety, Tolerability and Pharmacokinetics Study of STP1 in a Subgroup of Patients With Autism Spectrum Disorder (ASD) (NCT NCT04644003)
NCT ID: NCT04644003
Last Updated: 2024-11-08
Results Overview
Number of Participants with Adverse Events (nature and frequency of non-serious adverse events, serious adverse events and adverse events of special interest).
Recruitment status
COMPLETED
Study phase
PHASE1
Target enrollment
12 participants
Primary outcome timeframe
14 days
Results posted on
2024-11-08
Participant Flow
The patient population is defined as ASD-Phen1 based on STALICLA criteria. The number of patients enrolled were 12 subjects with 6 subjects receiving STP1-Low Dose, 3 subjects receiving STP1-High Dose, and 3 subjects receiving placebo. All 12 subjects (100.0%) completed treatment with 0 screen failures.
Of 12 enrolled participants, 12 met inclusion criteria and were randomized to treatment.
Participant milestones
| Measure |
STP1 Low Dose
1 capsule and 1 tablet per intake
STP1: STP1 is a combination of two drugs, a PDE inhibitor and an NKCC1 inhibitor
|
STP1 High Dose
1 capsule and 1 tablet per intake
STP1: STP1 is a combination of two drugs, a PDE inhibitor and an NKCC1 inhibitor
|
Placebo
1 placebo capsule and 1 placebo tablet per intake
Placebo: Placebo medication (capsule and tablet) identical in appearance to active medication
|
|---|---|---|---|
|
Overall Study
STARTED
|
6
|
3
|
3
|
|
Overall Study
COMPLETED
|
6
|
3
|
3
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Safety, Tolerability and Pharmacokinetics Study of STP1 in a Subgroup of Patients With Autism Spectrum Disorder (ASD)
Baseline characteristics by cohort
| Measure |
STP1 Low Dose
n=6 Participants
1 capsule and 1 tablet per intake
STP1: STP1 is a combination of two drugs, a PDE inhibitor and an NKCC1 inhibitor
|
STP1 High Dose
n=3 Participants
1 capsule and 1 tablet per intake
STP1: STP1 is a combination of two drugs, a PDE inhibitor and an NKCC1 inhibitor
|
Placebo
n=3 Participants
1 placebo capsule and 1 placebo tablet per intake
Placebo: Placebo medication (capsule and tablet) identical in appearance to active medication
|
Total
n=12 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
6 Participants
n=99 Participants
|
3 Participants
n=107 Participants
|
3 Participants
n=206 Participants
|
12 Participants
n=7 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Age, Continuous
|
18.50 years
n=99 Participants
|
20.00 years
n=107 Participants
|
18.00 years
n=206 Participants
|
18.50 years
n=7 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
3 Participants
n=7 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
9 Participants
n=7 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
6 Participants
n=99 Participants
|
3 Participants
n=107 Participants
|
3 Participants
n=206 Participants
|
12 Participants
n=7 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
2 Participants
n=7 Participants
|
|
Race (NIH/OMB)
White
|
5 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
3 Participants
n=206 Participants
|
10 Participants
n=7 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Region of Enrollment
United States
|
6 participants
n=99 Participants
|
3 participants
n=107 Participants
|
3 participants
n=206 Participants
|
12 participants
n=7 Participants
|
|
Body Mass Index
|
36.80 kg/m^2
STANDARD_DEVIATION 5.02 • n=99 Participants
|
37.07 kg/m^2
STANDARD_DEVIATION 6.89 • n=107 Participants
|
25.17 kg/m^2
STANDARD_DEVIATION 5.84 • n=206 Participants
|
33.96 kg/m^2
STANDARD_DEVIATION 7.38 • n=7 Participants
|
PRIMARY outcome
Timeframe: 14 daysPopulation: All patients receiving at least 1 dose of randomised treatment.
Number of Participants with Adverse Events (nature and frequency of non-serious adverse events, serious adverse events and adverse events of special interest).
Outcome measures
| Measure |
STP1 Low Dose
n=6 Participants
1 capsule and 1 tablet per intake
STP1: STP1 is a combination of two drugs, a PDE inhibitor and an NKCC1 inhibitor
|
STP1 High Dose
n=3 Participants
1 capsule and 1 tablet per intake
STP1: STP1 is a combination of two drugs, a PDE inhibitor and an NKCC1 inhibitor
|
Placebo
n=3 Participants
1 placebo capsule and 1 placebo tablet per intake
Placebo: Placebo medication (capsule and tablet) identical in appearance to active medication
|
|---|---|---|---|
|
Safety and Tolerability
|
6 Participants
|
3 Participants
|
3 Participants
|
Adverse Events
STP1 Low Dose
Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths
STP1 High Dose
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Placebo
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 |
STP1 Low Dose
n=6 participants at risk
1 capsule and 1 tablet per intake
STP1: STP1 is a combination of two drugs, a PDE inhibitor and an NKCC1 inhibitor
|
STP1 High Dose
n=3 participants at risk
1 capsule and 1 tablet per intake
STP1: STP1 is a combination of two drugs, a PDE inhibitor and an NKCC1 inhibitor
|
Placebo
n=3 participants at risk
1 placebo capsule and 1 placebo tablet per intake
Placebo: Placebo medication (capsule and tablet) identical in appearance to active medication
|
|---|---|---|---|
|
Gastrointestinal disorders
Diarrhoea
|
16.7%
1/6 • Number of events 1 • The total duration of the study for each participant was up to six weeks divided as follows: a screening phase of two weeks (from Day -14 to Day -1); a double-blind treatment phase of two weeks (from Day 1 to Day 14); and a follow-up phase of two weeks after treatment discontinuation (from Day 15 to Day 28).
As per ClinicalTrials.gov definitions.
|
0.00%
0/3 • The total duration of the study for each participant was up to six weeks divided as follows: a screening phase of two weeks (from Day -14 to Day -1); a double-blind treatment phase of two weeks (from Day 1 to Day 14); and a follow-up phase of two weeks after treatment discontinuation (from Day 15 to Day 28).
As per ClinicalTrials.gov definitions.
|
0.00%
0/3 • The total duration of the study for each participant was up to six weeks divided as follows: a screening phase of two weeks (from Day -14 to Day -1); a double-blind treatment phase of two weeks (from Day 1 to Day 14); and a follow-up phase of two weeks after treatment discontinuation (from Day 15 to Day 28).
As per ClinicalTrials.gov definitions.
|
|
Gastrointestinal disorders
Nausea
|
16.7%
1/6 • Number of events 1 • The total duration of the study for each participant was up to six weeks divided as follows: a screening phase of two weeks (from Day -14 to Day -1); a double-blind treatment phase of two weeks (from Day 1 to Day 14); and a follow-up phase of two weeks after treatment discontinuation (from Day 15 to Day 28).
As per ClinicalTrials.gov definitions.
|
0.00%
0/3 • The total duration of the study for each participant was up to six weeks divided as follows: a screening phase of two weeks (from Day -14 to Day -1); a double-blind treatment phase of two weeks (from Day 1 to Day 14); and a follow-up phase of two weeks after treatment discontinuation (from Day 15 to Day 28).
As per ClinicalTrials.gov definitions.
|
66.7%
2/3 • Number of events 2 • The total duration of the study for each participant was up to six weeks divided as follows: a screening phase of two weeks (from Day -14 to Day -1); a double-blind treatment phase of two weeks (from Day 1 to Day 14); and a follow-up phase of two weeks after treatment discontinuation (from Day 15 to Day 28).
As per ClinicalTrials.gov definitions.
|
|
Infections and infestations
Infection
|
0.00%
0/6 • The total duration of the study for each participant was up to six weeks divided as follows: a screening phase of two weeks (from Day -14 to Day -1); a double-blind treatment phase of two weeks (from Day 1 to Day 14); and a follow-up phase of two weeks after treatment discontinuation (from Day 15 to Day 28).
As per ClinicalTrials.gov definitions.
|
33.3%
1/3 • Number of events 1 • The total duration of the study for each participant was up to six weeks divided as follows: a screening phase of two weeks (from Day -14 to Day -1); a double-blind treatment phase of two weeks (from Day 1 to Day 14); and a follow-up phase of two weeks after treatment discontinuation (from Day 15 to Day 28).
As per ClinicalTrials.gov definitions.
|
0.00%
0/3 • The total duration of the study for each participant was up to six weeks divided as follows: a screening phase of two weeks (from Day -14 to Day -1); a double-blind treatment phase of two weeks (from Day 1 to Day 14); and a follow-up phase of two weeks after treatment discontinuation (from Day 15 to Day 28).
As per ClinicalTrials.gov definitions.
|
|
Infections and infestations
Upper respiratory tract infection
|
0.00%
0/6 • The total duration of the study for each participant was up to six weeks divided as follows: a screening phase of two weeks (from Day -14 to Day -1); a double-blind treatment phase of two weeks (from Day 1 to Day 14); and a follow-up phase of two weeks after treatment discontinuation (from Day 15 to Day 28).
As per ClinicalTrials.gov definitions.
|
33.3%
1/3 • Number of events 1 • The total duration of the study for each participant was up to six weeks divided as follows: a screening phase of two weeks (from Day -14 to Day -1); a double-blind treatment phase of two weeks (from Day 1 to Day 14); and a follow-up phase of two weeks after treatment discontinuation (from Day 15 to Day 28).
As per ClinicalTrials.gov definitions.
|
0.00%
0/3 • The total duration of the study for each participant was up to six weeks divided as follows: a screening phase of two weeks (from Day -14 to Day -1); a double-blind treatment phase of two weeks (from Day 1 to Day 14); and a follow-up phase of two weeks after treatment discontinuation (from Day 15 to Day 28).
As per ClinicalTrials.gov definitions.
|
|
Psychiatric disorders
Trichotillomania
|
16.7%
1/6 • Number of events 1 • The total duration of the study for each participant was up to six weeks divided as follows: a screening phase of two weeks (from Day -14 to Day -1); a double-blind treatment phase of two weeks (from Day 1 to Day 14); and a follow-up phase of two weeks after treatment discontinuation (from Day 15 to Day 28).
As per ClinicalTrials.gov definitions.
|
0.00%
0/3 • The total duration of the study for each participant was up to six weeks divided as follows: a screening phase of two weeks (from Day -14 to Day -1); a double-blind treatment phase of two weeks (from Day 1 to Day 14); and a follow-up phase of two weeks after treatment discontinuation (from Day 15 to Day 28).
As per ClinicalTrials.gov definitions.
|
0.00%
0/3 • The total duration of the study for each participant was up to six weeks divided as follows: a screening phase of two weeks (from Day -14 to Day -1); a double-blind treatment phase of two weeks (from Day 1 to Day 14); and a follow-up phase of two weeks after treatment discontinuation (from Day 15 to Day 28).
As per ClinicalTrials.gov definitions.
|
|
Renal and urinary disorders
Pollakiuria
|
16.7%
1/6 • Number of events 1 • The total duration of the study for each participant was up to six weeks divided as follows: a screening phase of two weeks (from Day -14 to Day -1); a double-blind treatment phase of two weeks (from Day 1 to Day 14); and a follow-up phase of two weeks after treatment discontinuation (from Day 15 to Day 28).
As per ClinicalTrials.gov definitions.
|
0.00%
0/3 • The total duration of the study for each participant was up to six weeks divided as follows: a screening phase of two weeks (from Day -14 to Day -1); a double-blind treatment phase of two weeks (from Day 1 to Day 14); and a follow-up phase of two weeks after treatment discontinuation (from Day 15 to Day 28).
As per ClinicalTrials.gov definitions.
|
0.00%
0/3 • The total duration of the study for each participant was up to six weeks divided as follows: a screening phase of two weeks (from Day -14 to Day -1); a double-blind treatment phase of two weeks (from Day 1 to Day 14); and a follow-up phase of two weeks after treatment discontinuation (from Day 15 to Day 28).
As per ClinicalTrials.gov definitions.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
16.7%
1/6 • Number of events 1 • The total duration of the study for each participant was up to six weeks divided as follows: a screening phase of two weeks (from Day -14 to Day -1); a double-blind treatment phase of two weeks (from Day 1 to Day 14); and a follow-up phase of two weeks after treatment discontinuation (from Day 15 to Day 28).
As per ClinicalTrials.gov definitions.
|
0.00%
0/3 • The total duration of the study for each participant was up to six weeks divided as follows: a screening phase of two weeks (from Day -14 to Day -1); a double-blind treatment phase of two weeks (from Day 1 to Day 14); and a follow-up phase of two weeks after treatment discontinuation (from Day 15 to Day 28).
As per ClinicalTrials.gov definitions.
|
0.00%
0/3 • The total duration of the study for each participant was up to six weeks divided as follows: a screening phase of two weeks (from Day -14 to Day -1); a double-blind treatment phase of two weeks (from Day 1 to Day 14); and a follow-up phase of two weeks after treatment discontinuation (from Day 15 to Day 28).
As per ClinicalTrials.gov definitions.
|
Additional Information
Eric Painbeni, PhD - Head of Clinical Operations
STALICLA SA
Phone: +41 (0)79 918 33 16
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place