Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT05059327




Registration number
NCT05059327
Ethics application status
Date submitted
20/08/2021
Date registered
28/09/2021
Date last updated
7/11/2023

Titles & IDs
Public title
Basimglurant in Children, Adolescents, and Young Adults With TSC
Scientific title
A Phase 2B, Multicenter, 30-week, Prospective, Cross-over, Double-blind, Randomized, Placebo-controlled Study Followed by a 52-Week Open-label Extension Study to Evaluate the Efficacy and Safety of Basimglurant Adjunctive to Ongoing Anticonvulsive Therapy in Children, Adolescents, and Young Adults With Uncontrolled Seizures Associated With Tuberous Sclerosis Complex
Secondary ID [1] 0 0
NOE-TSC-201
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Tuberous Sclerosis Complex 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Basimglurant with crossover to Placebo
Treatment: Drugs - Placebo with crossover to Basimglurant

Experimental: Arm A (Basimglurant to Placebo) - Basimglurant to Placebo

Placebo Comparator: Arm B (Placebo to Basimglurant) - Placebo to Basimglurant


Treatment: Drugs: Basimglurant with crossover to Placebo
Basimglurant with crossover to Placebo

Treatment: Drugs: Placebo with crossover to Basimglurant
Placebo with crossover to Basimglurant
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Mean percentage in monthly seizure frequency during the maintenance dosing in Period 2 (Weeks 13 to 16) and Period 4 (Weeks 27-30).
Timepoint [1] 0 0
30 weeks
Secondary outcome [1] 0 0
Number of patients considered treatment responders.
Timepoint [1] 0 0
30 weeks
Secondary outcome [2] 0 0
Longest seizure free interval (i.e., seizure free days).
Timepoint [2] 0 0
30 weeks
Secondary outcome [3] 0 0
Change in the severity of symptoms of TSC as measured by Caregiver Global Impression of Change (CGIC) score during maintenance dosing in Period 2 (Weeks 13 to 16) and Period 4 (Weeks 27-30) compared to Baseline.
Timepoint [3] 0 0
30 weeks
Secondary outcome [4] 0 0
Change in the Sheehan Disability Scale during maintenance dosing in Period 2 (Weeks 13 to 16) and Period 4 (Weeks 27-30) compared to baseline.
Timepoint [4] 0 0
30 weeks
Secondary outcome [5] 0 0
Safety of the study drug in children, adolescents and young adults with seizures associated with TSC.
Timepoint [5] 0 0
82 weeks

Eligibility
Key inclusion criteria
Inclusion Criteria (summary):

- Ability and willingness to provide informed assent or written consent or consent from
their legal representative.

- Fluency in the language of the study staff

- Age 5 to 30 years at study entry

- A documented history of TSC

- Refractory seizure history

- Currently receiving one or more anti-epileptic drugs (AEDs)

- Stable medications or interventions for epilepsy

- Willingness to complete Patient Reported Outcome assessments

- For female patients of childbearing potential:

1. Willingness to undergo serum or urinary pregnancy testing at screening and during
the trial period.

2. Willingness to use contraception.
Minimum age
5 Years
Maximum age
30 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Exclusion Criteria (summary):

- Neurologic disease other than TSC

- Recent anoxic episode

- Patient weight below 15kg

- Clinically significant unstable medical condition(s)

- Pregnancy or lactation

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Crossover
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
3/03/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
31/08/2025
Actual
Sample size
Target
54
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Maryland
Country [3] 0 0
United States of America
State/province [3] 0 0
Massachusetts
Country [4] 0 0
United States of America
State/province [4] 0 0
Michigan
Country [5] 0 0
United States of America
State/province [5] 0 0
Minnesota
Country [6] 0 0
United States of America
State/province [6] 0 0
New York
Country [7] 0 0
United States of America
State/province [7] 0 0
North Carolina
Country [8] 0 0
United States of America
State/province [8] 0 0
Ohio
Country [9] 0 0
United States of America
State/province [9] 0 0
Texas
Country [10] 0 0
United States of America
State/province [10] 0 0
Washington
Country [11] 0 0
India
State/province [11] 0 0
Andhra Pradesh
Country [12] 0 0
India
State/province [12] 0 0
Delhi
Country [13] 0 0
India
State/province [13] 0 0
Maharashtra
Country [14] 0 0
India
State/province [14] 0 0
Tamil Nadu
Country [15] 0 0
Israel
State/province [15] 0 0
HaMerkaz
Country [16] 0 0
Israel
State/province [16] 0 0
Tel-Aviv
Country [17] 0 0
Israel
State/province [17] 0 0
Jerusalem
Country [18] 0 0
Italy
State/province [18] 0 0
Lombardia
Country [19] 0 0
Italy
State/province [19] 0 0
Sicilia
Country [20] 0 0
Italy
State/province [20] 0 0
Toscana
Country [21] 0 0
Poland
State/province [21] 0 0
Mazowieckie
Country [22] 0 0
Poland
State/province [22] 0 0
Kraków
Country [23] 0 0
Poland
State/province [23] 0 0
Lódz
Country [24] 0 0
Spain
State/province [24] 0 0
Barcelona
Country [25] 0 0
Spain
State/province [25] 0 0
Málaga
Country [26] 0 0
Spain
State/province [26] 0 0
Sevilla
Country [27] 0 0
Turkey
State/province [27] 0 0
Ankara
Country [28] 0 0
Turkey
State/province [28] 0 0
Istanbul
Country [29] 0 0
Turkey
State/province [29] 0 0
Üsküdar
Country [30] 0 0
United Kingdom
State/province [30] 0 0
Lancashire
Country [31] 0 0
United Kingdom
State/province [31] 0 0
London, City Of
Country [32] 0 0
United Kingdom
State/province [32] 0 0
South Glamorgan
Country [33] 0 0
United Kingdom
State/province [33] 0 0
West Midlands

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Noema Pharma AG
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The study intends to show that basimglurant provides effective seizure control in children,
adolescents and young adults with Tuberous Sclerosis Complex (TSC).
Trial website
https://clinicaltrials.gov/ct2/show/NCT05059327
Trial related presentations / publications
Public notes
This record is viewable in the ANZCTR as it had previously listed Australia and/or New Zealand as a recruitment site, however these sites have since been removed

Contacts
Principal investigator
Name 0 0
Renata Lazarova, MD
Address 0 0
Noema Pharma AG
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Noema Pharma
Address 0 0
Country 0 0
Phone 0 0
Please contact via email:
Fax 0 0
Email 0 0
clinicaltrials@noemapharma.com
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT05059327