Childhood-Onset Fluency Disorder
Conditions
Brief summary
This study is designed to evaluate the effectiveness of NOE-105 on speech fluency without the known antipsychotic-induced side effects of commonly used treatments for childhood onset fluency disorder (COFD).
Detailed description
NOE-105 is an investigational selective PDE10A inhibitor with a potential therapeutic effect for the treatment of COFD. In this study adult male patients may be randomized to a double-blind, placebo-controlled, parallel group treatment with NOE-105 or placebo once daily. The study is designed to find the maximum tolerated dose of NOE-105 and thereafter, to maintain the participants at this dose until they have completed a total of 10 weeks treatment period. Following up to 10 weeks of treatment, participants will visit the study site for a follow-up visit within 28 (± 7) days of the date of the last dose of study treatment.
Interventions
DRUGNOE-105
Escalating dose levels of NOE-105 will be given and maximum tolerated dose will be maintained
DRUGPlacebo
Escalating dose levels of matching Placebo will be given
Sponsors
Noema Pharma AG
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
MALE
Age
18 Years to 55 Years
Healthy volunteers
No
Inclusion criteria
1. Patients must be 18 to 55 years of age inclusive, at the time of signing the informed consent. 2. Patients who satisfy DSM-5 criteria for childhood onset fluency disorder and are suitable for pharmacotherapy. 3. Have a history of stuttering for more than or equal to ≥ 2 years with onset consistent to developmental in nature before age 8 years. 4. Patient reported global stuttering experience as "moderate" at screening and baseline. 5. Patients must discontinue all medications used to treat stuttering for at least 14 days prior to receiving study treatment. With the exception of antipsychotic therapies (see exclusion criterion #11), other psychotropic drugs will be allowed provided they have been stable for at least 14 days prior to receiving study treatment and are expected to remain stable for the duration of the study. 6. BMI within the range 19 to 35 kg/m2 (inclusive). 7. Male Contraceptive use should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. Male patients must use a condom during the treatment period and until the end of relevant systemic exposure in the male participant, plus a further 90-day period. In addition, for a non-pregnant WOCBP partner. 8. Capable of giving signed informed consent 9. Able to read and write in English
Exclusion criteria
1. Stuttering is related to a known neurological cause eg, stroke, etc. 2. Low IQ in the opinion of the investigator. 3. Patients with uncontrolled seizure disorders. 4. A history of severe traumatic brain injury or stroke. 5. Patients who are, in the investigator's opinion, at imminent risk of suicide. 6. Known to have tested positive for human immunodeficiency virus. 7. Known DSM-5 diagnosis of substance abuse or dependence. 8. Unstable medical illness or clinically significant abnormalities on screening tests/exams. 9. Any unstable medical conditions or are currently ill (eg, congenital heart disease, arrhythmia or cancer), which, in the investigator's judgment, will put them at a risk of major adverse event during this trial, are expected to progress during the study, or will interfere with safety and efficacy assessments. 10. Initiation of new behavioral therapies for stuttering within 10 weeks prior to baseline. 11. Use of antipsychotic drug therapy within 14 days prior to receiving treatment until the EoT visit. 12. Participation in another clinical study with an IP administered in the last 30 days. 13. Participants with a known hypersensitivity to NOE-105 or any of the excipients of the product. 14. Patient must not intend to use cannabinoids, cocaine, or nonprescribed opiates. 15. Involvement in the planning and/or conduct of the study (applies to both Noema staff and/or staff at the study site). 16. Judgment by the investigator that the patient should not participate in the study if the patient is unlikely to comply with study procedures, restrictions, and requirements. 17. Previous randomization in the present study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline to End of 6 Weeks in Total MLGSSS | 43 days | MLGSSS refers to Maguire-Leal-Garibaldi Self-rated Stuttering Scale. The scale includes ten questions that are assessed on a 0 to 10 scale with 0 being no impact and 10 being highest impact. The total score ranges from 0 to 100 with the higher the score the worse the stuttering. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Sheehan Disability Scale | Up to 71 days | The Sheehan Disability Scale (SDS) is a brief, self-report tool that assesses disability or functional impairment in the domains of (1) Work/School life, (2) Social life, and (3) Family life/home responsibilities. Each is assessed on a 10 point scale with 0 being no impact, and 10 being highest impact. Each item is rated from 0 (not at all) to 10 (extremely) impairment. Each domain score is added to provide a total score from 0-30 total score with 0 (no impairment) and 30 is severe impairment. The change from the Baseline assessment have been provided. |
| Change From Baseline to End Point in Clinician-rated Stuttering Severity Instrument-4 | Up to 71 days | SSI-4 is a validated scale to evaluate speech fluency including frequency, duration, physical concomitants and the naturalness of the speech. The score ranges from 10 (mildest) to 46 (most severe). The results show the change from Baseline in the SSI-4 |
Countries
Australia, United States
Contacts
PRINCIPAL_INVESTIGATORGerald A Maguire, M.D.
Clinical Innovations, Inc. dba CITrials (a CenExel company)
Participant flow
Pre-assignment details
10 participants were not randomized due to withdrawal by patient (n=3), non-compliance with study drug (n=1) or physician decision (n=1), 3 were not eligible for randomization.
Baseline characteristics
| Characteristic | — |
|---|---|
| Age, Continuous | 32.6 years STANDARD_DEVIATION 10.22 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 6 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 31 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 1 Participants |
| Maguire-Leal-Garibaldi Self-rated Stuttering Scale | 32.7 score on a scale STANDARD_DEVIATION 12.86 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 5 Participants |
| Race (NIH/OMB) Black or African American | 19 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 1 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 2 Participants |
| Race (NIH/OMB) White | 17 Participants |
| Sex: Female, Male Female | 0 Participants |
| Sex: Female, Male Male | 34 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 34 | 0 / 31 |
| other Total, other adverse events | 23 / 34 | 13 / 31 |
| serious Total, serious adverse events | 0 / 34 | 0 / 31 |
Outcome results
None listed