Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12621000319875
Ethics application status
Approved
Date submitted
16/02/2021
Date registered
22/03/2021
Date last updated
5/07/2024
Date data sharing statement initially provided
22/03/2021
Type of registration
Prospectively registered

Titles & IDs
Public title
An open-label, Phase IIa, multi-center, 12-week prospective study to evaluate the safety and efficacy of NOE-105 at a daily dose range of 2.5mg to 15mg in adult and adolescent male patients with Tourette Syndrome (TS).
Scientific title
An open-label, Phase IIa, multi-center, 12-week prospective study to evaluate the safety and efficacy of NOE-105 at a daily dose range of 2.5mg to 15mg in adult male patients with Tourette Syndrome (TS).
Secondary ID [1] 302832 0
Nil known
Universal Trial Number (UTN)
U1111-1261-4753
Trial acronym
NOE-TTS-211
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Tourette syndrome 319801 0
Condition category
Condition code
Neurological 317728 317728 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
NOE-105, formulated in oral hard gelatine capsules, administered daily, dose-ranging from 2.5mg to 15mg, during 12 weeks. The starting dose of NOE-105 is 2.5mg QD. Based on a clinical assessment and absence of tolerability issues, the investigator may increase the dose weekly by increments of 2.5mg up to a maximum of 15mg QD.
Intervention code [1] 319108 0
Treatment: Drugs
Comparator / control treatment
No control group, open-label study
Control group
Uncontrolled

Outcomes
Primary outcome [1] 325768 0
Response to the investigational drug NOE-105 rated by the Tourette Syndrome Clinical Global Impression of Change (TS-CGI-C). Response is defined as a rating of “Minimally improved” “Much improved” and “Very much improved”
Timepoint [1] 325768 0
Change from Baseline to Week 12 or at patient’s discontinuation of the study if it occurs before Week12 (also called Post Treatment)
Secondary outcome [1] 389081 0
The safety and Tolerability of NOE-105 will be assessed by the review of the patient’s data of :
AEs and SAEs
ECG and Vital signs
Blood samples analysis
Timepoint [1] 389081 0
Change from Baseline to Week 12 or at patient’s discontinuation of the study if it occurs before Week12 (also called Post Treatment)
Secondary outcome [2] 389082 0
Change in tic symptom severity by evaluating change YGTSS of the total tic scores (TTS) of the Yale Global Tic Severity Scale (YGTSS)
Timepoint [2] 389082 0
Change from Baseline to Week 12 or at patient’s discontinuation of the study if it occurs before Week12 (also called Post Treatment)
Secondary outcome [3] 389083 0
Change in severity of patient’s illness rom Baseline. Assessed by TS-CGI Severity (TS-CGI-S) scale
Timepoint [3] 389083 0
Change from Baseline to Week 12 or at patient’s discontinuation of the study if it occurs before Week12 (also called Post Treatment)
Secondary outcome [4] 389084 0
The patient reported Clinical Global Impression of Change (PGI-C) vs baseline
Timepoint [4] 389084 0
Change from Baseline to Week 12 or at patient’s discontinuation of the study if it occurs before Week12 (also called Post Treatment)
Secondary outcome [5] 389085 0
Patient reported rating of the Medication Satisfaction Questionnaire (MSQ)
Timepoint [5] 389085 0
Week 12 or at patient’s discontinuation of the study if it occurs before Week12 (also called Post Treatment)

Eligibility
Key inclusion criteria
1. Ability and willingness to provide written informed consent and to comply with the study procedures.

2. Fluency in the language of the investigator, study staff and the informed consent.

3. Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.

4. Have been under the care of the Investigator for at least 1 year. If not, then the Investigator should liaise closely with the patient’s clinician for the full assessment of the patient.

5. Male patients aged 12 to 50 years.

6. Meeting DSM-5 diagnostic criteria for Tourette Syndrome and requiring drug therapy.

7. Experiencing lack of benefit from their current therapy as evidenced by a CGI severity at least moderately ill or intolerance that impacts patient adherence to treatment at screening visit.

8. Agreement to the following during the study treatment period and for at least 90 days after the last dose of study drug:
• Refrain from donating sperm
o Must agree to use contraception as detailed below:
Agree to use a male condom (with female partner use of an additional highly effective contraceptive method with a failure rate of <1% per year) when having sexual intercourse with a woman of childbearing potential who is not currently pregnant
Agree to use a male condom when engaging in any activity that allows for passage of ejaculate to another person.
Minimum age
12 Years
Maximum age
50 Years
Sex
Males
Can healthy volunteers participate?
No
Key exclusion criteria
Patients who meet any of the following criteria will be excluded from study participation:

1. Secondary tic symptoms accompanied by late-onset tics, Huntington's chorea, neuroacanthocytosis, mental retardation, or autism, if, in the opinion of the investigator, they will interfere with study procedures.

2. IQ<70.

Note: a previously conducted IQ level assessment can be used by the investigator and does not have to be repeated at screening for study purposes.
Additionally, in the absence of a formal IQ test result, the assessment that the targeted IQ level is reached can be based on the investigator’s judgment. The investigator can use proxies to IQ score such as the participant’s education level (i.e. completion of secondary school education) and/or their employment status.

3. Current diagnosis of bipolar disorder, schizophrenia, or Major Depressive Disorder (MDD). Patients with MDD on a stable antidepressant treatment for > 1 month can participate in the study.

4. Patients with uncontrolled seizure disorders.

5. A history of severe traumatic brain injury or stroke.

6. Any unstable medical conditions or are currently ill (e.g., 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, or will interfere with safety and efficacy assessments.

7. Require cognitive-behavioral therapy (CBT, including habitual inversion therapy, cognitive therapy, relaxation training, etc.) during the trial period UNLESS started at least 8 weeks prior to study start.

8. Positive urine drug screen for cannabinoids, cocaine, or nonprescribed opiates.

9. Participated in any clinical trial of any investigational treatments within the past 30 days.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Not applicable
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Not applicable
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
All patients start with a dose of 2.5mg QD and see their dose increase weekly by increments of 2.5mg up to a maximum of 15.0mg QD based on investigator clinical assessment of the effect of NOE-105 on tics associated with Tourette Syndrome and tolerability.
Phase
Phase 2
Type of endpoint/s
Safety/efficacy
Statistical methods / analysis
Descriptive statistics will be used to summarize baseline patient characteristics, treatment with NOE-105, and safety and efficacy variables. Categorical and nominal variables will be summarized by frequency and percentage. Continuous variables will be summarized by descriptive statistics such as n, mean, median, standard deviation, minimum, and maximum.

Recruitment
Recruitment status
Completed
Date of first participant enrolment
Anticipated
29/03/2021
Actual
23/06/2021
Date of last participant enrolment
Anticipated
29/04/2022
Actual
20/06/2023
Date of last data collection
Anticipated
29/07/2022
Actual
3/11/2023
Sample size
Target
18
Accrual to date
Final
15
Recruitment in Australia
Recruitment state(s)
QLD,WA,VIC
Recruitment hospital [1] 18399 0
The Wesley Hospital - Auchenflower
Recruitment hospital [2] 18400 0
Royal Melbourne Hospital - City campus - Parkville
Recruitment hospital [3] 26757 0
Perth Children's Hospital - Nedlands
Recruitment postcode(s) [1] 32493 0
4066 - Auchenflower
Recruitment postcode(s) [2] 32494 0
3050 - Parkville
Recruitment postcode(s) [3] 42806 0
6009 - Nedlands
Recruitment outside Australia
Country [1] 24712 0
Germany
State/province [1] 24712 0

Funding & Sponsors
Funding source category [1] 307252 0
Commercial sector/Industry
Name [1] 307252 0
Noema Pharma Australia PTY LTD
Country [1] 307252 0
Australia
Primary sponsor type
Commercial sector/Industry
Name
Noema Pharma Australia Pty Ltd
Address
Suite 2003, 109 Pitt Street, SYDNEY NSW 2000
PO Box Q175, QVB NSW 1230
Country
Australia
Secondary sponsor category [1] 307875 0
None
Name [1] 307875 0
Address [1] 307875 0
Country [1] 307875 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 307347 0
Royal Melbourne Hospital HREC
Ethics committee address [1] 307347 0
Ethics committee country [1] 307347 0
Australia
Date submitted for ethics approval [1] 307347 0
26/01/2021
Approval date [1] 307347 0
17/03/2021
Ethics approval number [1] 307347 0
HREC/72847/MH-2021
Ethics committee name [2] 307620 0
Belberry Limited
Ethics committee address [2] 307620 0
Ethics committee country [2] 307620 0
Australia
Date submitted for ethics approval [2] 307620 0
23/12/2020
Approval date [2] 307620 0
17/02/2021
Ethics approval number [2] 307620 0
2020-12-1330
Ethics committee name [3] 310715 0
MHH Ethikkomission
Ethics committee address [3] 310715 0
Ethics committee country [3] 310715 0
Germany
Date submitted for ethics approval [3] 310715 0
27/09/2021
Approval date [3] 310715 0
15/02/2022
Ethics approval number [3] 310715 0
10034_AMG_mono_2021
Ethics committee name [4] 315646 0
Child and Adolescent Health Service Human Research Ethics Committee
Ethics committee address [4] 315646 0
Ethics committee country [4] 315646 0
Australia
Date submitted for ethics approval [4] 315646 0
22/11/2022
Approval date [4] 315646 0
14/03/2023
Ethics approval number [4] 315646 0
RGS0000005794

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 106922 0
Dr Snehal Shah
Address 106922 0
Perth children’s Hospital, 15 Hospital Avenue, Nedlands WA 6009
Country 106922 0
Australia
Phone 106922 0
+61 8 6456 4320
Fax 106922 0
Email 106922 0
[email protected]
Contact person for public queries
Name 106923 0
Snehal Shah
Address 106923 0
Perth children’s Hospital, 15 Hospital Avenue, Nedlands WA 6009
Country 106923 0
Australia
Phone 106923 0
+61 8 6456 4320
Fax 106923 0
Email 106923 0
[email protected]
Contact person for scientific queries
Name 106924 0
Snehal Shah
Address 106924 0
Perth children’s Hospital, 15 Hospital Avenue, Nedlands WA 6009
Country 106924 0
Australia
Phone 106924 0
+61 8 6456 4320
Fax 106924 0
Email 106924 0
[email protected]

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

Doc. No.TypeCitationLinkEmailOther DetailsAttachment
11112Study protocol  [email protected]



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.