Trial Outcomes & Findings for A 10-week Efficacy Study of NOE-105 in Childhood Onset Fluency Disorder (Orpheus) (NCT NCT05583955)

NCT ID: NCT05583955

Last Updated: 2026-02-20

Results Overview

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

75 participants

Primary outcome timeframe

43 days

Results posted on

2026-02-20

Participant Flow

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.

Participant milestones

Participant milestones
Measure	Active Escalating doses of NOE-105 capsules NOE-105: Escalating dose levels of NOE-105 will be given and maximum tolerated dose will be maintained	Placebo Escalating doses of matching placebo Placebo: Escalating dose levels of matching Placebo will be given
Overall Study STARTED	34	31
Overall Study Full analysis set	33	29
Overall Study COMPLETED	19	26
Overall Study NOT COMPLETED	15	5

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A 10-week Efficacy Study of NOE-105 in Childhood Onset Fluency Disorder (Orpheus)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Active n=34 Participants Escalating doses of NOE-105 capsules NOE-105: Escalating dose levels of NOE-105 will be given and maximum tolerated dose will be maintained	Placebo n=31 Participants Escalating doses of matching placebo Placebo: Escalating dose levels of matching Placebo will be given	Total n=65 Participants Total of all reporting groups
Age, Continuous	31.1 years STANDARD_DEVIATION 10.09 • n=14 Participants	34.3 years STANDARD_DEVIATION 10.25 • n=14 Participants	32.6 years STANDARD_DEVIATION 10.22 • n=29 Participants
Sex: Female, Male Female	0 Participants n=14 Participants	0 Participants n=14 Participants	0 Participants n=29 Participants
Sex: Female, Male Male	34 Participants n=14 Participants	31 Participants n=14 Participants	65 Participants n=29 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=14 Participants	0 Participants n=14 Participants	0 Participants n=29 Participants
Race (NIH/OMB) Asian	5 Participants n=14 Participants	1 Participants n=14 Participants	6 Participants n=29 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=14 Participants	1 Participants n=14 Participants	1 Participants n=29 Participants
Race (NIH/OMB) Black or African American	10 Participants n=14 Participants	9 Participants n=14 Participants	19 Participants n=29 Participants
Race (NIH/OMB) White	17 Participants n=14 Participants	15 Participants n=14 Participants	32 Participants n=29 Participants
Race (NIH/OMB) More than one race	0 Participants n=14 Participants	0 Participants n=14 Participants	0 Participants n=29 Participants
Race (NIH/OMB) Unknown or Not Reported	2 Participants n=14 Participants	5 Participants n=14 Participants	7 Participants n=29 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	3 Participants n=14 Participants	3 Participants n=14 Participants	6 Participants n=29 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	31 Participants n=14 Participants	27 Participants n=14 Participants	58 Participants n=29 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=14 Participants	1 Participants n=14 Participants	1 Participants n=29 Participants
Maguire-Leal-Garibaldi Self-rated Stuttering Scale	32.1 score on a scale STANDARD_DEVIATION 14.23 • n=14 Participants	33.3 score on a scale STANDARD_DEVIATION 11.16 • n=14 Participants	32.7 score on a scale STANDARD_DEVIATION 12.86 • n=29 Participants

PRIMARY outcome

Timeframe: 43 days

Population: The full analysis set was used to assess the outcome. The full analysis set includes patients randomized who had at least one dose of study treatment and post randomization MLGSSS assessed.

Outcome measures

Outcome measures
Measure	Active n=33 Participants Escalating doses of NOE-105 capsules NOE-105: Escalating dose levels of NOE-105 will be given and maximum tolerated dose will be maintained	Placebo n=29 Participants Escalating doses of matching placebo Placebo: Escalating dose levels of matching Placebo will be given
Change From Baseline to End of 6 Weeks in Total MLGSSS	-7.0 Score on a scale Standard Deviation 13.32	-5.5 Score on a scale Standard Deviation 6.72

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Active n=33 Participants Escalating doses of NOE-105 capsules NOE-105: Escalating dose levels of NOE-105 will be given and maximum tolerated dose will be maintained	Placebo n=29 Participants Escalating doses of matching placebo Placebo: Escalating dose levels of matching Placebo will be given
Change From Baseline in Sheehan Disability Scale	-0.6 score on a scale Standard Deviation 8.23	-1.3 score on a scale Standard Deviation 5.82

SECONDARY outcome

Timeframe: 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

Outcome measures

Outcome measures
Measure	Active n=33 Participants Escalating doses of NOE-105 capsules NOE-105: Escalating dose levels of NOE-105 will be given and maximum tolerated dose will be maintained	Placebo n=29 Participants Escalating doses of matching placebo Placebo: Escalating dose levels of matching Placebo will be given
Change From Baseline to End Point in Clinician-rated Stuttering Severity Instrument-4	-3.0 units on a scale Standard Deviation 6.5	-2.7 units on a scale Standard Deviation 4.78

POST_HOC outcome

Timeframe: Up to 71 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. Analysis of a subset of participant with a Baseline MLGSSS of \> 24, in other words this analysis excludes those with mild stuttering as measured by MLGSSS. See the description for the first outcome measure for a description of the scale. The results show the change from baseline.

Outcome measures

Outcome measures
Measure	Active n=21 Participants Escalating doses of NOE-105 capsules NOE-105: Escalating dose levels of NOE-105 will be given and maximum tolerated dose will be maintained	Placebo n=24 Participants Escalating doses of matching placebo Placebo: Escalating dose levels of matching Placebo will be given
Change in MLGSSS From Baseline in More Severe Population	-14.8 score on a scale Standard Deviation 12.54	-9.1 score on a scale Standard Deviation 8.81

Adverse Events

Active

Serious events: 0 serious events

Other events: 23 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 13 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Additional Information

Clinical Study Lead

Noema Pharma

Phone: +1 646-931-0119

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60

Measure	Active n=34 participants at risk Escalating doses of NOE-105 capsules NOE-105: Escalating dose levels of NOE-105 will be given and maximum tolerated dose will be maintained. The adverse event rates could not be allocated per dose because the dose titration occurred over a period of 4 weeks or util maximum tolerated dose reached. Down titration was also allowed	Placebo n=31 participants at risk Escalating doses of matching placebo Placebo: Escalating dose levels of matching Placebo will be given
Nervous system disorders Somnolence	8.8% 3/34 • Adverse events were collected for the duration of the trial (71 days)	0.00% 0/31 • Adverse events were collected for the duration of the trial (71 days)
Nervous system disorders Tremor	5.9% 2/34 • Adverse events were collected for the duration of the trial (71 days)	3.2% 1/31 • Adverse events were collected for the duration of the trial (71 days)
Nervous system disorders Bradykinesia	5.9% 2/34 • Adverse events were collected for the duration of the trial (71 days)	0.00% 0/31 • Adverse events were collected for the duration of the trial (71 days)
Nervous system disorders Dizziness	5.9% 2/34 • Adverse events were collected for the duration of the trial (71 days)	0.00% 0/31 • Adverse events were collected for the duration of the trial (71 days)
Nervous system disorders Akathisia	8.8% 3/34 • Adverse events were collected for the duration of the trial (71 days)	0.00% 0/31 • Adverse events were collected for the duration of the trial (71 days)
Psychiatric disorders Restlessness	8.8% 3/34 • Adverse events were collected for the duration of the trial (71 days)	0.00% 0/31 • Adverse events were collected for the duration of the trial (71 days)
Psychiatric disorders Anxiety	5.9% 2/34 • Adverse events were collected for the duration of the trial (71 days)	0.00% 0/31 • Adverse events were collected for the duration of the trial (71 days)
Psychiatric disorders Apathy	5.9% 2/34 • Adverse events were collected for the duration of the trial (71 days)	0.00% 0/31 • Adverse events were collected for the duration of the trial (71 days)
Psychiatric disorders Insomnia	5.9% 2/34 • Adverse events were collected for the duration of the trial (71 days)	0.00% 0/31 • Adverse events were collected for the duration of the trial (71 days)
Infections and infestations Nasopharyngitis	0.00% 0/34 • Adverse events were collected for the duration of the trial (71 days)	12.9% 4/31 • Adverse events were collected for the duration of the trial (71 days)
Infections and infestations Upper respiratory tract infection	2.9% 1/34 • Adverse events were collected for the duration of the trial (71 days)	6.5% 2/31 • Adverse events were collected for the duration of the trial (71 days)
Musculoskeletal and connective tissue disorders Muscle tightness	5.9% 2/34 • Adverse events were collected for the duration of the trial (71 days)	0.00% 0/31 • Adverse events were collected for the duration of the trial (71 days)
Musculoskeletal and connective tissue disorders Muscle twitching	0.00% 0/34 • Adverse events were collected for the duration of the trial (71 days)	6.5% 2/31 • Adverse events were collected for the duration of the trial (71 days)
Gastrointestinal disorders Vomiting	5.9% 2/34 • Adverse events were collected for the duration of the trial (71 days)	3.2% 1/31 • Adverse events were collected for the duration of the trial (71 days)
Gastrointestinal disorders Nausea	5.9% 2/34 • Adverse events were collected for the duration of the trial (71 days)	0.00% 0/31 • Adverse events were collected for the duration of the trial (71 days)
Cardiac disorders Left ventricular hypertrophy	0.00% 0/34 • Adverse events were collected for the duration of the trial (71 days)	3.2% 1/31 • Adverse events were collected for the duration of the trial (71 days)
General disorders Non-cardiac chest pain	0.00% 0/34 • Adverse events were collected for the duration of the trial (71 days)	3.2% 1/31 • Adverse events were collected for the duration of the trial (71 days)
Infections and infestations Eye infection and bacterial	0.00% 0/34 • Adverse events were collected for the duration of the trial (71 days)	3.2% 1/31 • Adverse events were collected for the duration of the trial (71 days)
Infections and infestations Folliculitis	0.00% 0/34 • Adverse events were collected for the duration of the trial (71 days)	3.2% 1/31 • Adverse events were collected for the duration of the trial (71 days)
Infections and infestations Pertussis	0.00% 0/34 • Adverse events were collected for the duration of the trial (71 days)	3.2% 1/31 • Adverse events were collected for the duration of the trial (71 days)
Infections and infestations Sinusitis	0.00% 0/34 • Adverse events were collected for the duration of the trial (71 days)	3.2% 1/31 • Adverse events were collected for the duration of the trial (71 days)
Investigations Blood cholesterol increased	0.00% 0/34 • Adverse events were collected for the duration of the trial (71 days)	3.2% 1/31 • Adverse events were collected for the duration of the trial (71 days)
Investigations Blood glucose increased	0.00% 0/34 • Adverse events were collected for the duration of the trial (71 days)	3.2% 1/31 • Adverse events were collected for the duration of the trial (71 days)
Nervous system disorders Headache	2.9% 1/34 • Adverse events were collected for the duration of the trial (71 days)	3.2% 1/31 • Adverse events were collected for the duration of the trial (71 days)
Nervous system disorders Migraine	0.00% 0/34 • Adverse events were collected for the duration of the trial (71 days)	3.2% 1/31 • Adverse events were collected for the duration of the trial (71 days)
Psychiatric disorders Dysphemia	0.00% 0/34 • Adverse events were collected for the duration of the trial (71 days)	3.2% 1/31 • Adverse events were collected for the duration of the trial (71 days)
Psychiatric disorders Initial insomnia	0.00% 0/34 • Adverse events were collected for the duration of the trial (71 days)	3.2% 1/31 • Adverse events were collected for the duration of the trial (71 days)
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	0.00% 0/34 • Adverse events were collected for the duration of the trial (71 days)	3.2% 1/31 • Adverse events were collected for the duration of the trial (71 days)
Respiratory, thoracic and mediastinal disorders Throat tightness	0.00% 0/34 • Adverse events were collected for the duration of the trial (71 days)	3.2% 1/31 • Adverse events were collected for the duration of the trial (71 days)
Nervous system disorders Sedation	23.5% 8/34 • Adverse events were collected for the duration of the trial (71 days)	3.2% 1/31 • Adverse events were collected for the duration of the trial (71 days)
Nervous system disorders Dystonia	8.8% 3/34 • Adverse events were collected for the duration of the trial (71 days)	0.00% 0/31 • Adverse events were collected for the duration of the trial (71 days)