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

Primary Purpose

Childhood-Onset Fluency Disorder

Status
Active
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
NOE-105
Placebo
Sponsored by
Noema Pharma AG
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Childhood-Onset Fluency Disorder

Eligibility Criteria

18 Years - 55 Years (Adult)MaleDoes not accept healthy volunteers

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.

Sites / Locations

  • Noema Investigator site
  • Noema Investigator site
  • Noema Investigator site
  • Noema Investigator site
  • Noema Investigator site
  • Noema Investigator site
  • Noema Investigator site
  • Noema Investigator site
  • Noema Investigator site

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Active

Placebo

Arm Description

Escalating doses of NOE-105 capsules

Escalating doses of matching placebo

Outcomes

Primary Outcome Measures

Change from baseline to end point in severity subset of the MLGSSS
MLGSSS refers to Maguire-Leal-Garibaldi Self-rated Stuttering Scale
Number of participants with adverse events
An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment
Severity of the adverse events
Adverse events will be categorized as mild, moderate or severe by the investigator
Change in the hematological parameters
Platelet count, RBC count, Hemoglobin, hematocrit and differential WBC count will be assessed
Change in clinical chemistry
Urea, creatinine, potassium, SGOT, SGPT, glucose, sodium chloride, magnesium, phosphates, calcium, total and conjugated bilirubin, GGT, total protein albumin, phosphokinase, and plasma prolactin will be assessed
Change in the vital signs
Temperature, weight, height, pulse rate and blood pressure will be assessed.

Secondary Outcome Measures

Change from baseline to end point in SDS
SDS refers to Sheehan disability scale
PGI-S rating at end point
PGI-S refers to patient global impression of severity
CGI-C rating at end point
Clinician global impression of change
Rating of the medication satisfaction questionnaire at end point
To evaluate the patient's satisfaction in treatment with NOE-105
Change from baseline to end point in clinician-rated stuttering severity instrument-4
To evaluate the effect of NOE-105 on the change in stuttering severity
PGI-C rating at end point
PGI-C refers to patient global impression of change
Change in mood as rated by the patient through change from baseline to end point in Quick inventory of depressive symptomology (QIDS-16)

Full Information

First Posted
June 16, 2022
Last Updated
August 10, 2023
Sponsor
Noema Pharma AG
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1. Study Identification

Unique Protocol Identification Number
NCT05583955
Brief Title
A 10-week Efficacy Study of NOE-105 in Childhood Onset Fluency Disorder (Orpheus)
Official Title
A Double-blind, Placebo-controlled, Phase IIb, Multi-center, Ten-week Prospective Study to Evaluate the Efficacy and Safety of NOE-105 in Adult Male Patients With Childhood Onset Fluency Disorder (Orpheus)
Study Type
Interventional

2. Study Status

Record Verification Date
August 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
July 25, 2022 (Actual)
Primary Completion Date
October 31, 2023 (Anticipated)
Study Completion Date
December 31, 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Noema Pharma AG

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Childhood-Onset Fluency Disorder

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
67 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Active
Arm Type
Experimental
Arm Description
Escalating doses of NOE-105 capsules
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Escalating doses of matching placebo
Intervention Type
Drug
Intervention Name(s)
NOE-105
Other Intervention Name(s)
Escalating dose levels of NOE-105
Intervention Description
Escalating dose levels of NOE-105 will be given and maximum tolerated dose will be maintained
Intervention Type
Drug
Intervention Name(s)
Placebo
Other Intervention Name(s)
Placebo to match
Intervention Description
Escalating dose levels of matching Placebo will be given
Primary Outcome Measure Information:
Title
Change from baseline to end point in severity subset of the MLGSSS
Description
MLGSSS refers to Maguire-Leal-Garibaldi Self-rated Stuttering Scale
Time Frame
Up to 71 days
Title
Number of participants with adverse events
Description
An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment
Time Frame
Up to 71 days
Title
Severity of the adverse events
Description
Adverse events will be categorized as mild, moderate or severe by the investigator
Time Frame
Up to 71 days
Title
Change in the hematological parameters
Description
Platelet count, RBC count, Hemoglobin, hematocrit and differential WBC count will be assessed
Time Frame
Up to 71 days
Title
Change in clinical chemistry
Description
Urea, creatinine, potassium, SGOT, SGPT, glucose, sodium chloride, magnesium, phosphates, calcium, total and conjugated bilirubin, GGT, total protein albumin, phosphokinase, and plasma prolactin will be assessed
Time Frame
Up to 71 days
Title
Change in the vital signs
Description
Temperature, weight, height, pulse rate and blood pressure will be assessed.
Time Frame
Up to 71 days
Secondary Outcome Measure Information:
Title
Change from baseline to end point in SDS
Description
SDS refers to Sheehan disability scale
Time Frame
Up to 71 days
Title
PGI-S rating at end point
Description
PGI-S refers to patient global impression of severity
Time Frame
Up to 71 days
Title
CGI-C rating at end point
Description
Clinician global impression of change
Time Frame
Up to 71 days
Title
Rating of the medication satisfaction questionnaire at end point
Description
To evaluate the patient's satisfaction in treatment with NOE-105
Time Frame
Up to 71 days
Title
Change from baseline to end point in clinician-rated stuttering severity instrument-4
Description
To evaluate the effect of NOE-105 on the change in stuttering severity
Time Frame
Up to 71 days
Title
PGI-C rating at end point
Description
PGI-C refers to patient global impression of change
Time Frame
Up to 71 days
Title
Change in mood as rated by the patient through change from baseline to end point in Quick inventory of depressive symptomology (QIDS-16)
Time Frame
Up to 71 days

10. Eligibility

Sex
Male
Gender Based
Yes
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
55 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Patients must be 18 to 55 years of age inclusive, at the time of signing the informed consent. Patients who satisfy DSM-5 criteria for childhood onset fluency disorder and are suitable for pharmacotherapy. Have a history of stuttering for more than or equal to ≥ 2 years with onset consistent to developmental in nature before age 8 years. Patient reported global stuttering experience as "moderate" at screening and baseline. 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. BMI within the range 19 to 35 kg/m2 (inclusive). 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. Capable of giving signed informed consent Able to read and write in English Exclusion Criteria: Stuttering is related to a known neurological cause eg, stroke, etc. Low IQ in the opinion of the investigator. Patients with uncontrolled seizure disorders. A history of severe traumatic brain injury or stroke. Patients who are, in the investigator's opinion, at imminent risk of suicide. Known to have tested positive for human immunodeficiency virus. Known DSM-5 diagnosis of substance abuse or dependence. Unstable medical illness or clinically significant abnormalities on screening tests/exams. 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. Initiation of new behavioral therapies for stuttering within 10 weeks prior to baseline. Use of antipsychotic drug therapy within 14 days prior to receiving treatment until the EoT visit. Participation in another clinical study with an IP administered in the last 30 days. Participants with a known hypersensitivity to NOE-105 or any of the excipients of the product. Patient must not intend to use cannabinoids, cocaine, or nonprescribed opiates. Involvement in the planning and/or conduct of the study (applies to both Noema staff and/or staff at the study site). 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. Previous randomization in the present study.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Gerald A Maguire, M.D.
Organizational Affiliation
Clinical Innovations, Inc. dba CITrails (a CenExel company)
Official's Role
Principal Investigator
Facility Information:
Facility Name
Noema Investigator site
City
Bellflower
State/Province
California
ZIP/Postal Code
90706
Country
United States
Facility Name
Noema Investigator site
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32256
Country
United States
Facility Name
Noema Investigator site
City
Orlando
State/Province
Florida
ZIP/Postal Code
32801
Country
United States
Facility Name
Noema Investigator site
City
Overland Park
State/Province
Kansas
ZIP/Postal Code
66210
Country
United States
Facility Name
Noema Investigator site
City
Berlin
State/Province
New Jersey
ZIP/Postal Code
08009
Country
United States
Facility Name
Noema Investigator site
City
Memphis
State/Province
Tennessee
ZIP/Postal Code
38119
Country
United States
Facility Name
Noema Investigator site
City
Brookvale
State/Province
New South Wales
ZIP/Postal Code
2100
Country
Australia
Facility Name
Noema Investigator site
City
Miranda
State/Province
New South Wales
ZIP/Postal Code
2228
Country
Australia
Facility Name
Noema Investigator site
City
Sydney
State/Province
New South Wales
ZIP/Postal Code
2109
Country
Australia

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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