Tuberous Sclerosis ComplexPhase 2NCT05059327
Basimglurant (NOE-101) in Children, Adolescents, and Young Adults With TSC
A Phase 2 study of Tuberous Sclerosis Complex, run by Noema Pharma AG at 27 sites in 8 countries.
Trial summary
The study intends to show that basimglurant (NOE-101) provides effective seizure control in children, adolescents and young adults with Tuberous Sclerosis Complex (TSC). Read more about this study
- Who is it for?
- Ages 5 to 30 years
- Conditions the study is for
- Tuberous Sclerosis Complex
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 61 participants
- Where
- 27 sitesin 8 countries
- Study dates
- 3 Mar 2022to 28 Apr 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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)
Show all 11Show fewer
- Stable medications or interventions for epilepsy
- Willingness to complete Patient Reported Outcome assessments
- For female patients of childbearing potential:
- Willingness to undergo serum or urinary pregnancy testing at screening and during the trial period.
- Willingness to use contraception.
Who cannot join
- Neurologic disease other than TSC
- Recent anoxic episode
- Patient weight below 15kg
- Clinically significant unstable medical condition(s)
- Pregnancy or lactation
Trial locations and contacts
27 sites in 8 countries.
27 sites in 8 countries
United States9 sites
Kennedy Krieger Institute (Site #: 110)
Baltimore, Maryland, 21205
Boston Children's Hospital (Site #: 102)
Boston, Massachusetts, 02115-5724
University Hospitals Cleveland Medical Center, Rainbow Babies and Childrens Hospital (Site #: 107)
Cleveland, Ohio, 44106-1716
Duke Children's Hospital and Health Center (Site #: 106)
Durham, North Carolina, 27705-4699
Boston Children's Health Physicians (BCHP) (Site #: 111)
Hawthorne, New York, 10532
The University of Texas Medical School at Houston (Site #: 103)
Houston, Texas, 77030-3000
David Geffen School of Medicine at UCLA (Site #: 101)
Los Angeles, California, 90095
Minnesota Epilepsy Group PA (Site #: 105)
Roseville, Minnesota, 55113-1306
William Beaumont Hospital - Royal Oak (Site #: 104)
Royal Oak, Michigan, 48073-6712
India6 sites
Citi Neuro Centre (Site # 806)
Hyderabad, Andhra Pradesh, 500034
Rainbow Childrens Hospital (Site # 803)
Hyderabad, Andhra Pradesh, 500034
Jaslok Hospital and Research Centre (Site # 801)
Mumbai, Maharashtra, 400026
All India Institute of Medical Sciences (Site # 804)
New Delhi, National Capital Territory of Delhi, 110029
Deenanath Mangeshkar Hospital and Research Centre (Site # 805)
Pune, Maharashtra, 411006
Christian Medical College (Site # 807)
Vellore, Tamil Nadu, 632004
Spain4 sites
Hospital Universitario Germans Trias i Pujol (Site # 194)
Badalona, Barcelona, 08916
Hospital Sant Joan de Deu - PIN (Site # 192)
Esplugues de Llobregat, Barcelona, 08950
Hospital Regional Universitario de Malaga - Hospital General (Site # 193)
Málaga, 29010
Centro de Neurología Avanzada (Site # 191)
Seville, 41013
Turkey (Türkiye)3 sites
Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi (Site #: 905)
Fatih, Istanbul, 34093
Yeditepe University Kosuyolu Hospital (Site #: 904)
Kadıköy, Istanbul, 34718
Istanbul Egitim ve Arastirma Hastanesi (Site #: 901)
Sultangazi, Istanbul, 34096
United Kingdom2 sites
Noahs Ark Children's Hospital (Site # 306)
Cardiff, South Glamorgan, CF14 4XW
Salford Royal Hospital - PPDS (Site # 304)
Salford, Lancashire, M6 8HD
Italy1 site
ASST Grande Ospedale Metropolitano Niguarda - Presidio Ospedaliero Ospedale Niguarda (Site # 183)
Milan, Lombardy, 20162
Poland1 site
Instytut Pomnik Centrum Zdrowia Dziecka (Site # 262)
Warsaw, Masovian Voivodeship, 04-730
About this study
Official 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
The study intends to show that basimglurant (NOE-101) provides effective seizure control in children, adolescents and young adults with Tuberous Sclerosis Complex (TSC).
Read the detailed description
The study drug (NOE-101, basimglurant) is a potent inhibitor of metabotropic glutamate receptor 5 (mGluR5) which controls a wide range of processes in the brain, spinal cord, retina, and peripheral nervous system. In animal studies, the inhibition of this receptor has shown therapeutic potential for the treatment of Tuberous Sclerosis Complex (TSC). This receptor's inhibition decreases the frequency of seizures. In previous clinical trials, the study drug has shown an advantageous safety profile in children and adolescents.
The objective of this study is to find an optimal dose at which the study drug will lead to a decrease in the duration, frequency and intensity of seizures in children, adolescents and young adults with TSC, while being well tolerated. All patients who positively respond and tolerate the medicine will be offered the possibility to continue in an open label extension.
- Conditions
- Tuberous Sclerosis Complex
- Being tested
- Basimglurant with crossover to Placebo, Placebo with crossover to Basimglurant
- Keywords
- Basimglurant, TSC, Seizures, NOE-101
- Medical subject headings
- Tuberous Sclerosis, Seizures
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Basimglurant with crossover to PlaceboDrug
Basimglurant with crossover to Placebo
Placebo with crossover to BasimglurantDrug
Placebo with crossover to Basimglurant
| Study group | Role | What they receive |
|---|---|---|
| Arm A (Basimglurant/NOE-101 to Placebo) | Experimental | Basimglurant to Placebo Receives: Drug: Basimglurant with crossover to Placebo |
| Arm B (Placebo to Basimglurant/NOE-101) | Placebo comparator | Placebo to Basimglurant Receives: Drug: Placebo with crossover to Basimglurant |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mean percentage in monthly seizure frequency during the maintenance dosing in Period 2 (Weeks 13 to 16) and Period 4 (Weeks 27-30).
Measured: 30 weeks
Secondary outcomes (5)
Number of patients considered treatment responders.
Measured: 30 weeks
Longest seizure free interval (i.e., seizure free days).
Measured: 30 weeks
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.
Measured: 30 weeks
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.
Measured: 30 weeks
Safety of the study drug in children, adolescents and young adults with seizures associated with TSC.
Measured in terms of incidence, nature, and severity of adverse events, vital signs, physical examination, clinical chemistry, hematology, electrocardiograms, and urinalysis, as well as treatment delays, dose reductions, and dose discontinuations. In addition, suicidal ideation will be assessed using S-STS.
Measured: 82 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Sep 2021
- Study start3 Mar 2022
- Primary completion6 Feb 2025
- Study completion28 Apr 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Tuberous Sclerosis ComplexStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Model description
- This is a multi-center, randomized, double-blind, placebo-controlled, 30-week, cross-over (Part A) followed by a 52-week open-label extension (OLE) (Part B) study.
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 61 participants (actual)
Sponsor and investigators
- Lead sponsor
- Noema Pharma AG
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Director, MD, Study director, Noema Pharma AG
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05059327
- Sponsor's protocol ID
- NOE-TSC-201
- Status last verified
- May 2025
- First submitted
- 20 Aug 2021
- Last updated
- 4 Jun 2025
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT05059327
- Acronym / sponsor ID
- NOE-TSC-201
- Conditions
- Tuberous Sclerosis Complex
- Keywords
- Basimglurant, TSC, Seizures, NOE-101
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 61 participants (actual)
- Lead sponsor
- Noema Pharma AGIndustry (pharma, biotech, devices)
- Study start
- 3 Mar 2022 (actual)
- Primary completion
- 6 Feb 2025 (actual)
- Study completion
- 28 Apr 2025 (actual)
- First posted
- 28 Sep 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study intends to show that basimglurant (NOE-101) provides effective seizure control in children, adolescents and young adults with Tuberous Sclerosis Complex (TSC).
Detailed description
The study drug (NOE-101, basimglurant) is a potent inhibitor of metabotropic glutamate receptor 5 (mGluR5) which controls a wide range of processes in the brain, spinal cord, retina, and peripheral nervous system. In animal studies, the inhibition of this receptor has shown therapeutic potential for the treatment of Tuberous Sclerosis Complex (TSC). This receptor's inhibition decreases the frequency of seizures. In previous clinical trials, the study drug has shown an advantageous safety profile in children and adolescents.
The objective of this study is to find an optimal dose at which the study drug will lead to a decrease in the duration, frequency and intensity of seizures in children, adolescents and young adults with TSC, while being well tolerated. All patients who positively respond and tolerate the medicine will be offered the possibility to continue in an open label extension.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
- Model description
- This is a multi-center, randomized, double-blind, placebo-controlled, 30-week, cross-over (Part A) followed by a 52-week open-label extension (OLE) (Part B) study.
| Arm | Type | Receives |
|---|---|---|
| Arm A (Basimglurant/NOE-101 to Placebo) | Experimental | Drug: Basimglurant with crossover to PlaceboBasimglurant to Placebo |
| Arm B (Placebo to Basimglurant/NOE-101) | Placebo comparator | Drug: Placebo with crossover to BasimglurantPlacebo to Basimglurant |
Who may qualify
- Ages
- 5 to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (11 listed)
- 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
Key exclusion criteria (5 listed)
- Neurologic disease other than TSC
- Recent anoxic episode
- Patient weight below 15kg
- Clinically significant unstable medical condition(s)
- Pregnancy or lactation
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Basimglurant with crossover to PlaceboBasimglurant with crossover to Placebo | Drug | Arm A (Basimglurant/NOE-101 to Placebo) |
| Placebo with crossover to BasimglurantPlacebo with crossover to Basimglurant | Drug | Arm B (Placebo to Basimglurant/NOE-101) |
Primary outcome
Mean percentage in monthly seizure frequency during the maintenance dosing in Period 2 (Weeks 13 to 16) and Period 4 (Weeks 27-30).
Time frame: 30 weeks
5 secondary outcomes
Number of patients considered treatment responders.
Time frame: 30 weeks
Longest seizure free interval (i.e., seizure free days).
Time frame: 30 weeks
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.
Time frame: 30 weeks
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.
Time frame: 30 weeks
Safety of the study drug in children, adolescents and young adults with seizures associated with TSC.
Time frame: 82 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Jun 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Director, MD study director
Noema Pharma AG
27 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study of Tuberous Sclerosis Complex, run by Noema Pharma AG at 27 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 27 sites in United States, India, Spain, Turkey (Türkiye), United Kingdom and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Mean percentage in monthly seizure frequency during the maintenance dosing in Period 2 (Weeks 13 to 16) and Period 4 (Weeks 27-30). (time frame: 30 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Jun 2025.