Autism Spectrum Disorder
Conditions
Keywords
ASD, Autism Spectrum Disorder
Brief summary
The main purpose of this study is to evaluate safety and tolerability in a subgroup of patients with Autism Spectrum Disorder (ASD). In addition, Pharmacokinetics and Pharmacodynamics, as well as efficacy of STP1 are being explored.
Detailed description
After obtaining written informed consent, those patients who are deemed eligible for the study, will be randomized on Day 1, in a double-blinded manner, in a 3:1ratio to receive either oral STP1 (twice daily) or placebo (twice daily). The total study duration is 6 weeks, including a screening phase of up to 2 weeks, a treatment phase of 2 weeks and a post-treatment follow-up phase of 2 weeks.
Interventions
DRUGSTP1
STP1 is a combination of two drugs, a phosphodiesterase (PDE) inhibitor and an NKCC1 inhibitor
DRUGPlacebo
Placebo medication (capsule and tablet) identical in appearance to active medication
Sponsors
Stalicla SA
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 40 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: 1. Male or female individuals, between 18 and 40 years, diagnosed of ASD. 2. Patients will be assessed for specific developmental anthropometric & anatomical criteria as well as personal and family medical history as assessed by the ASD-Phen1 semi structured interview form. 3. Patients must have a parent or reliable caregiver who can provide information about the pre-natal period and early developmental period, as required by the protocol. 4. Patient and/or parent or legal guardian willing and consenting to participate. 5. Patients with ASD and comorbid seizure disorder should be seizure-free for at least 6 months prior to screening. 6. Before enrolling in the study, subjects must agree to use double-barrier birth control methods if they engage in intercourse. Key
Exclusion criteria
1. Patients with an identified genetic cause of ASD in their medical record will be excluded from the study. 2. History of traumatic head injury, cerebrovascular disorder, congestive heart failure, hepatic or renal disease. 3. Thrombocytopenia. 4. Type 1 Diabetes Mellitus or uncontrolled type 2 Diabetes Mellitus, or latent autoimmune diabetes of the adult. 5. A significant risk for suicidal behavior. 6. Initiation of, or a major change in psychological / behavioral intervention within 4 weeks prior to randomization. 7. Patient with any active infection. 8. Systolic blood pressure (SBP) \<80 mmHg or diastolic blood pressure (DBP) \<40 mmHg or a drop in SBP of ≥20 mm Hg, or in DBP of ≥10 mm Hg, during the orthostatic recordings. 9. Clinically relevant electrocardiogram (ECG) abnormalities. 10. Clinically significant abnormal laboratory test. 11. Active clinically significant disease. 12. History of malignancy. 13. Pregnant (confirmed by laboratory testing) or lactating female patient.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety and Tolerability | 14 days | Number of Participants with Adverse Events (nature and frequency of non-serious adverse events, serious adverse events and adverse events of special interest). |
Countries
United States
Participant flow
Recruitment details
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.
Pre-assignment details
Of 12 enrolled participants, 12 met inclusion criteria and were randomized to treatment.
Participants by arm
| Arm | Count |
|---|---|
| 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 | 6 |
| 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 | 3 |
| Placebo 1 placebo capsule and 1 placebo tablet per intake
Placebo: Placebo medication (capsule and tablet) identical in appearance to active medication | 3 |
| Total | 12 |
Baseline characteristics
| Characteristic | STP1 Low Dose | STP1 High Dose | Placebo | Total |
|---|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 6 Participants | 3 Participants | 3 Participants | 12 Participants |
| Age, Continuous | 18.50 years | 20.00 years | 18.00 years | 18.50 years |
| Body Mass Index | 36.80 kg/m^2 STANDARD_DEVIATION 5.02 | 37.07 kg/m^2 STANDARD_DEVIATION 6.89 | 25.17 kg/m^2 STANDARD_DEVIATION 5.84 | 33.96 kg/m^2 STANDARD_DEVIATION 7.38 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 6 Participants | 3 Participants | 3 Participants | 12 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants | 1 Participants | 0 Participants | 2 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 5 Participants | 2 Participants | 3 Participants | 10 Participants |
| Region of Enrollment United States | 6 participants | 3 participants | 3 participants | 12 participants |
| Sex: Female, Male Female | 1 Participants | 1 Participants | 1 Participants | 3 Participants |
| Sex: Female, Male Male | 5 Participants | 2 Participants | 2 Participants | 9 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 6 | 0 / 3 | 0 / 3 |
| other Total, other adverse events | 5 / 6 | 2 / 3 | 2 / 3 |
| serious Total, serious adverse events | 0 / 6 | 0 / 3 | 0 / 3 |
Outcome results
Primary
Safety and Tolerability
Number of Participants with Adverse Events (nature and frequency of non-serious adverse events, serious adverse events and adverse events of special interest).
Time frame: 14 days
Population: All patients receiving at least 1 dose of randomised treatment.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| STP1 Low Dose | Safety and Tolerability | 6 Participants |
| STP1 High Dose | Safety and Tolerability | 3 Participants |
| Placebo | Safety and Tolerability | 3 Participants |