Tourette SyndromePhase 2NCT05434546
A Study to Explore the Effect of Sepranolone in Tourette Syndrome
A Phase 2 study testing Sepranolone in people with Tourette Syndrome and Tourette Syndrome in Adolescence, run by Asarina Pharma at 2 sites in Denmark.
Trial summary
The study was an open-label, randomized, multicenter, parallel, Phase 2a study in adolescents and adult patients with Tourette syndrome that aimed to explore the efficacy of Sepranolone as a treatment for Tourette syndrome, by reducing the severity and frequency of tics. Read more about this study
- Who is it for?
- Ages 12 to 55 years
- Conditions the study is for
- Tourette Syndrome, Tourette Syndrome in Adolescence
- What is being tested?
- Sepranolone
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 26 participants
- Where
- 2 sitesDenmark
- Study dates
- 16 Feb 2022to 1 Feb 2023
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 12 to 55 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Tic severity score ≥ 20 Yale Global Tic Severity Scale (YGTSS) Total Score at baseline
- The patient may have Obsessive-Compulsive Disease (OCD) as a comorbidity
Who cannot join
- Patient has participated in a clinical study over the past 30 days
- Evidence or history of neurological disease that may interfere with the study
- Malignant disease
- Unstable or clinical significant medical condition that could pose a risk
- HIV/ongoing hepatitis
- Clinical significant findings in vital signs
- History of anaphylactic reactions
Trial locations and contacts
2 sites in 1 country.
Denmark2 sites
Herlev Hospital
Copenhagen, Herlev, 2730
Bispebjerg Hospital
Copenhagen, 2400
About this study
Official title: A Randomized, Open-label, Multicenter, Phase 2 Clinical Trial to Explore the Safety and Efficacy of Sepranolone in Pediatric and Adult Patients With Tourette Syndrome
The study was an open-label, randomized, multicenter, parallel, Phase 2a study in adolescents and adult patients with Tourette syndrome that aimed to explore the efficacy of Sepranolone as a treatment for Tourette syndrome, by reducing the severity and frequency of tics. The total study duration from the Screening Visit to the final follow-up visit was approximately 26 weeks and included the following periods:
- A baseline period of 4 weeks between the screening visit (Visit 1) and randomization (4 weeks of baseline period were not needed in well-known adult subjects with stable Tourette syndrome history over the past at least 4 weeks). A school holiday/annual leave period of 2-6 weeks where no study-related activities were done.
- A randomized treatment period of 12 weeks
- A safety follow-up period of 4 weeks
- Being tested
- Sepranolone
- Keywords
- Tourette Syndrome in Adults
- Medical subject headings
- Tourette Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SepranoloneDrug
Also called Isoallopregnanolone
Treatment with 10 mg Sepranolone s.c. twice weekly for 12 weeks alongside patient's standard of care Tourette treatment.
| Study group | Role | What they receive |
|---|---|---|
| Sepranolone | Experimental | Sepranolone 10 mg sc twice weekly for 12 weeks alongside the patient's standard of care Tourette treatment. Receives: Drug: Sepranolone |
| No Intervention | No intervention | Continuation of the patient's standard of care Tourette treatment for 12 weeks. |
What is being measured
The primary outcome is the main question the study is designed to answer.
To investigate the efficacy of Sepranolone to reduce tic severity in patients with Tourette syndrome at 12 weeks, as measured by change in baseline in the The Yale Global Tic Severity Scale (YGTSS) Total Score.
The Yale Global Tic Severity Scale (YGTSS) is a validated scale for assessing the severity of motor and vocal tics in both children and adults with Tourette syndrome. Tics are scored based on a semi-structured interview, including scoring of the number, frequency, intensity, complexity, and interference of tics. The primary endpoint is the total tic score assessment. The score ranges from 0-50, where a higher score indicates a worse outcome.
Measured: Change from baseline at week 4, 8 and 12.
Secondary outcomes (5)
Safety and tolerability of Sepranolone in adolescent and adult patients with Tourette syndrome
Collection of adverse events (AEs) including spontaneous reporting, number of subjects with clinically significant changes in clinical safety laboratory blood and urine test values, vital signs, weight, and injection related events.
Measured: From randomization (day 1) until the end of study visit (week 16).
To evaluate the efficacy of Sepranolone to reduce tic related impairment in patients with Tourette syndrome at 12 weeks, as measured by change in baseline in the Yale Global Tic Severity Scale (YGTSS) Impairment Score.
The Yale Global Tic Severity Scale (YGTSS) is a validated scale for assessing the severity of motor and vocal tics in both children and adults with Tourette syndrome. Tics are scored based on a semi-structured interview, including scoring of the number, frequency, intensity, complexity, and interference of tics. The secondary endpoint is the impairment score assessment. The score ranges from 0-50, where a higher score indicates a worse outcome.
Measured: Change from baseline at week 4, 8 and 12.
To evaluate the efficacy of Sepranolone to reduce the urge to tic in patients with Tourette syndrome at 12 weeks, as measured by change in baseline in the Premonitory Urge for Tics Scale (PUTS) scale.
The Premonitory Urge for Tics Scale (PUTS) scale is an assessment aiming to quantify the premonitory urge to tic. The scale is reliable and valid instrument for children from above the age of 10 and for adults. This scale is used as a self-report assessment instrument, where the Investigator asks 10 questions, out of which the score for the first 9 questions add up to the total score (i.e., the 9-Item Total). The respondent has 4 alternatives, "not at all true", "a little true", "pretty much true" and "very much true," represented by a score or 1-4, respectively. The total score ranges from 9-36, where a higher score indicates a worse outcome.
Measured: Change from baseline at week 4, 8 and 12.
To evaluate the effect of Sepranolone on activities of daily living in patients with Tourette syndrome at 12 weeks, as measured by change in baseline in the GTS-QoL ADL subscale.
The Gilles de la Tourette Syndrome - Quality of Life (GTS-QoL) physical/activities of daily living (ADL) subscale assesses the impact of the symptoms of Tourette syndrome on the subject's quality of life. The instrument consists of 27 questions, asked by an interviewer, where subjects score the extent of impact on a 5-point verbal scale ranging from "no problems" to "extreme problems." The score ranges from 27-135, where a higher score indicates a worse outcome.
Measured: Change from baseline at week 4, 8 and 12.
To evaluate the effect on Global impression by Sepranolone in patients with Tourette syndrome at 12 weeks, as measured by the Tourette Syndrome-Clinical Global Impression (TS-CGI).
The Tourette Syndrome-Clinical Global Impression (TS-CGI) is a clinician rating of the change in severity of the symptoms of Tourette syndrome. The scale is a 7-step Likert scale, where the following alternatives are represented by a score of 1-7, respectively: "very much worsened," "much worsened," "minimally worsened," "no change," "minimally improved," "much improved," or "very much improved." The score ranges from 1-7, where a higher score indicates a better outcome.
Measured: TS-CGI score at week 4, 8 and 12.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jun 2022
- Study start16 Feb 2022
- Primary completion1 Feb 2023
- Study completion1 Feb 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Tourette SyndromeStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Interventional, open-label, multicenter, randomized, parallel group study
- Masking
- None (open label)
- Enrolment
- 26 participants (actual)
Sponsor and investigators
- Lead sponsor
- Asarina Pharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Nanette Debes, MD, Principal investigator, Copenhagen University Hospital at Herlev
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05434546
- Sponsor's protocol ID
- APH205
- Status last verified
- Jun 2023
- First submitted
- 12 Mar 2022
- Last updated
- 9 Jun 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Jun 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Open-label, Multicenter, Phase 2 Clinical Trial to Explore the Safety and Efficacy of Sepranolone in Pediatric and Adult Patients With Tourette Syndrome
- ClinicalTrials.gov ID
- NCT05434546
- Acronym / sponsor ID
- APH205
- Keywords
- Tourette Syndrome in Adults
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 26 participants (actual)
- Lead sponsor
- Asarina PharmaIndustry (pharma, biotech, devices)
- Study start
- 16 Feb 2022 (actual)
- Primary completion
- 1 Feb 2023 (actual)
- Study completion
- 1 Feb 2023 (actual)
- First posted
- 28 Jun 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study was an open-label, randomized, multicenter, parallel, Phase 2a study in adolescents and adult patients with Tourette syndrome that aimed to explore the efficacy of Sepranolone as a treatment for Tourette syndrome, by reducing the severity and frequency of tics. The total study duration from the Screening Visit to the final follow-up visit was approximately 26 weeks and included the following periods:
- A baseline period of 4 weeks between the screening visit (Visit 1) and randomization (4 weeks of baseline period were not needed in well-known adult subjects with stable Tourette syndrome history over the past at least 4 weeks). A school holiday/annual leave period of 2-6 weeks where no study-related activities were done.
- A randomized treatment period of 12 weeks
- A safety follow-up period of 4 weeks
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Interventional, open-label, multicenter, randomized, parallel group study
| Arm | Type | Receives |
|---|---|---|
| Sepranolone | Experimental | Drug: SepranoloneSepranolone 10 mg sc twice weekly for 12 weeks alongside the patient's standard of care Tourette treatment. |
| No Intervention | No intervention | –Continuation of the patient's standard of care Tourette treatment for 12 weeks. |
Who may qualify
- Ages
- 12 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- Tic severity score ≥ 20 Yale Global Tic Severity Scale (YGTSS) Total Score at baseline
- The patient may have Obsessive-Compulsive Disease (OCD) as a comorbidity
Key exclusion criteria (7 listed)
- Patient has participated in a clinical study over the past 30 days
- Evidence or history of neurological disease that may interfere with the study
- Malignant disease
- Unstable or clinical significant medical condition that could pose a risk
- HIV/ongoing hepatitis
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 |
|---|---|---|
| SepranoloneAlso: IsoallopregnanoloneTreatment with 10 mg Sepranolone s.c. twice weekly for 12 weeks alongside patient's standard of care Tourette treatment. | Drug | Sepranolone |
Primary outcome
To investigate the efficacy of Sepranolone to reduce tic severity in patients with Tourette syndrome at 12 weeks, as measured by change in baseline in the The Yale Global Tic Severity Scale (YGTSS) Total Score.
Time frame: Change from baseline at week 4, 8 and 12.
The Yale Global Tic Severity Scale (YGTSS) is a validated scale for assessing the severity of motor and vocal tics in both children and adults with Tourette syndrome. Tics are scored based on a semi-structured interview, including scoring of the number, frequency, intensity, complexity, and interference of tics. The primary endpoint is the total tic score assessment. The score ranges from 0-50, where a higher score indicates a worse outcome.
5 secondary outcomes
Safety and tolerability of Sepranolone in adolescent and adult patients with Tourette syndrome
Time frame: From randomization (day 1) until the end of study visit (week 16).
To evaluate the efficacy of Sepranolone to reduce tic related impairment in patients with Tourette syndrome at 12 weeks, as measured by change in baseline in the Yale Global Tic Severity Scale (YGTSS) Impairment Score.
Time frame: Change from baseline at week 4, 8 and 12.
To evaluate the efficacy of Sepranolone to reduce the urge to tic in patients with Tourette syndrome at 12 weeks, as measured by change in baseline in the Premonitory Urge for Tics Scale (PUTS) scale.
Time frame: Change from baseline at week 4, 8 and 12.
To evaluate the effect of Sepranolone on activities of daily living in patients with Tourette syndrome at 12 weeks, as measured by change in baseline in the GTS-QoL ADL subscale.
Time frame: Change from baseline at week 4, 8 and 12.
To evaluate the effect on Global impression by Sepranolone in patients with Tourette syndrome at 12 weeks, as measured by the Tourette Syndrome-Clinical Global Impression (TS-CGI).
Time frame: TS-CGI score at week 4, 8 and 12.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Jun 2023.
- Individual participant dataSharing planned: No
Related trials for Tourette Syndrome
Not yet recruiting
Evaluating and Promoting Resilience in Adolescents With Tourette Syndrome
Tourette Syndrome, Tic Disorders
Not yet recruiting
Transcutaneous Auricular Vagus Nerve Stimulation for Chronic Tic Disorders/Tourette's Syndrome in Children and Adolescents
Tourette Syndrome, Chronic Tic Disorder
Recruiting
Deep Brain Stimulation Therapy in Movement Disorders
Parkinson's Disease
Recruiting
Exploring the Role of Neuroactive Steroids in Tourette Syndrome
Tourette Syndrome in Children
Active, not recruiting
Tourette Deep Brain Stimulation (DBS) Target Detection & Suppression
Tourette Syndrome
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.
Nanette Debes, MD principal investigator
Copenhagen University Hospital at Herlev
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Sepranolone in people with Tourette Syndrome and Tourette Syndrome in Adolescence, run by Asarina Pharma at 2 sites in Denmark.
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 2 inclusion and 7 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 9 Jun 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Denmark. 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. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
To investigate the efficacy of Sepranolone to reduce tic severity in patients with Tourette syndrome at 12 weeks, as measured by change in baseline in the The Yale Global Tic Severity Scale (YGTSS) Total Score. (time frame: Change from baseline at week 4, 8 and 12.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 9 Jun 2023.