Autism Spectrum DisorderPhase 1NCT04644003
Safety, Tolerability and Pharmacokinetics Study of STP1 in a Subgroup of Patients With Autism Spectrum Disorder (ASD)
A Phase 1 study testing STP1 in people with Autism Spectrum Disorder, run by Stalicla SA at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 40 years
- Conditions the study is for
- Autism Spectrum Disorder
- What is being tested?
- STP1
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- 7 Dec 2020to 28 Jan 2022
- Phase
- Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 40 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Male or female individuals, between 18 and 40 years, diagnosed of ASD.
- 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.
- 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.
- Patient and/or parent or legal guardian willing and consenting to participate.
- Patients with ASD and comorbid seizure disorder should be seizure-free for at least 6 months prior to screening.
- Before enrolling in the study, subjects must agree to use double-barrier birth control methods if they engage in intercourse.
Who cannot join
- Patients with an identified genetic cause of ASD in their medical record will be excluded from the study.
- History of traumatic head injury, cerebrovascular disorder, congestive heart failure, hepatic or renal disease.
- Thrombocytopenia.
- Type 1 Diabetes Mellitus or uncontrolled type 2 Diabetes Mellitus, or latent autoimmune diabetes of the adult.
- A significant risk for suicidal behavior.
- Initiation of, or a major change in psychological / behavioral intervention within 4 weeks prior to randomization.
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- Patient with any active infection.
- 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.
- Clinically relevant electrocardiogram (ECG) abnormalities.
- Clinically significant abnormal laboratory test.
- Active clinically significant disease.
- History of malignancy.
- Pregnant (confirmed by laboratory testing) or lactating female patient.
Trial locations and contacts
1 site in 1 country.
United States1 site
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
About this study
Official title: A Phase 1b, Double-Blind, Placebo-Controlled, First-in-Human Study to Evaluate Safety, Tolerability and Pharmacokinetics of a Two-Week Oral Treatment With STP1 in a Subgroup of Patients With Autism Spectrum Disorder
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.
Read the 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.
- Conditions
- Autism Spectrum Disorder
- Being tested
- STP1, Placebo
- Keywords
- ASD, Autism Spectrum Disorder
- Medical subject headings
- Autism Spectrum Disorder
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
STP1Drug
STP1 is a combination of two drugs, a phosphodiesterase (PDE) inhibitor and an NKCC1 inhibitor
PlaceboDrug
Placebo medication (capsule and tablet) identical in appearance to active medication
| Study group | Role | What they receive |
|---|---|---|
| STP1 Low Dose | Experimental | 1 capsule and 1 tablet per intake Receives: Drug: STP1 |
| STP1 High Dose | Experimental | 1 capsule and 1 tablet per intake Receives: Drug: STP1 |
| Placebo | Placebo comparator | 1 placebo capsule and 1 placebo tablet per intake Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 14 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Nov 2020
- Study start7 Dec 2020
- Primary completion28 Jan 2022
- Study completion28 Jan 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Autism Spectrum DisorderStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Stalicla SA
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Craig Erickson, MD, Principal investigator, Children's Hospital Medical Center, Cincinnati
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04644003
- Sponsor's protocol ID
- STP1-C004
- Status last verified
- Aug 2024
- First submitted
- 16 Nov 2020
- Last updated
- 8 Nov 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Erickson CA, Perez-Cano L, Pedapati EV, Painbeni E, Bonfils G, Schmitt LM, Sachs H, Nelson M, De Stefano L, Westerkamp G, de Souza ALS, Pohl O, Laufer O, Issachar G, Blaettler T, Hyvelin JM, Durham LA. Safety, Tolerability, and EEG-Based Target Engagement of STP1 (PDE3,4 Inhibitor and NKCC1 Antagonist) in a Randomized Clinical Trial in a Subgroup of Patients with ASD. Biomedicines. 2024 Jun 27;12(7):1430. doi: 10.3390/biomedicines12071430.
PubMed 39062003
Study at a glance
From the registry record, last updated by the sponsor on 8 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1b, Double-Blind, Placebo-Controlled, First-in-Human Study to Evaluate Safety, Tolerability and Pharmacokinetics of a Two-Week Oral Treatment With STP1 in a Subgroup of Patients With Autism Spectrum Disorder
- ClinicalTrials.gov ID
- NCT04644003
- Acronym / sponsor ID
- STP1-C004
- Conditions
- Autism Spectrum Disorder
- Keywords
- ASD, Autism Spectrum Disorder
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Stalicla SAIndustry (pharma, biotech, devices)
- Study start
- 7 Dec 2020 (actual)
- Primary completion
- 28 Jan 2022 (actual)
- Study completion
- 28 Jan 2022 (actual)
- First posted
- 25 Nov 2020 (actual)
- Results
- Posted 8 Nov 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| STP1 Low Dose | Experimental | Drug: STP11 capsule and 1 tablet per intake |
| STP1 High Dose | Experimental | Drug: STP11 capsule and 1 tablet per intake |
| Placebo | Placebo comparator | Drug: Placebo1 placebo capsule and 1 placebo tablet per intake |
Who may qualify
- Ages
- 18 to 40 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (6 listed)
- Male or female individuals, between 18 and 40 years, diagnosed of ASD.
- 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.
- 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.
- Patient and/or parent or legal guardian willing and consenting to participate.
- Patients with ASD and comorbid seizure disorder should be seizure-free for at least 6 months prior to screening.
Key exclusion criteria (13 listed)
- Patients with an identified genetic cause of ASD in their medical record will be excluded from the study.
- History of traumatic head injury, cerebrovascular disorder, congestive heart failure, hepatic or renal disease.
- Thrombocytopenia.
- Type 1 Diabetes Mellitus or uncontrolled type 2 Diabetes Mellitus, or latent autoimmune diabetes of the adult.
- A significant risk for suicidal behavior.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| STP1STP1 is a combination of two drugs, a phosphodiesterase (PDE) inhibitor and an NKCC1 inhibitor | Drug | STP1 High Dose, STP1 Low Dose |
| PlaceboPlacebo medication (capsule and tablet) identical in appearance to active medication | Drug | Placebo |
Primary outcome
Safety and Tolerability
Time frame: 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).
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Nov 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 39062003Erickson CA, Perez-Cano L, Pedapati EV, Painbeni E, Bonfils G, Schmitt LM, Sachs H, Nelson M, De Stefano L, Westerkamp G, de Souza ALS, Pohl O, Laufer O, Issachar G, Blaettler T, Hyvelin JM, Durham LA. Safety, Tolerability, and EEG-Based Target Engagement of STP1 (PDE3,4 Inhibitor and NKCC1 Antagonist) in a Randomized Clinical Trial in a Subgroup of Patients with ASD. Biomedicines. 2024 Jun 27;12(7):1430. doi: 10.3390/biomedicines12071430.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Craig Erickson, MD principal investigator
Children's Hospital Medical Center, Cincinnati
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing STP1 in people with Autism Spectrum Disorder, run by Stalicla SA at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 8 Nov 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and Tolerability (time frame: 14 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04644003, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Nov 2024.