Health Condition 1: Q851- Tuberous sclerosis
Conditions
Interventions
Intervention1: Basimglurant (NOE-101): Fixed oral doses of 0.5mg, 1 mg OD orally for 82 weeks therapy of NOE-101 adjunctive to ongoing Anticonvulsive therapy.
Control Intervention1: Matching Placebo:
Sponsors
Noema Pharma AG
CliniRx Research Pvt Ltd
Eligibility
Inclusion criteria
Inclusion criteria: 1. Ability and willingness to provide informed assent or written consent, or consent from their legal representative and willingness to comply with the study procedures. 2. Fluency in the language of the investigator, study staff and the informed assent or consent form when applicable. 3. Age 5 to 30 years. 4. A documented history of TSC, diagnosed according to the International Tuberous Sclerosis Complex diagnostic criteria of 2021 and including a record of either genetic test or MRI/CT scan documenting tumours. 5. Continued seizures associated with TSC (including absences, atonic, tonic, tonic-clonic or myoclonic) despite adequate dosage of at least 2 or more appropriate antiseizure drugs (AEDs) at adequate doses, within approximately the previous 3 years. 6. Refractory seizure treatment status, defined as between 3 and 20 per month over the last year and at least 5 or more seizures within the past 30 days. 7. Currently receiving one or more anti-epileptic drugs (AEDs) with no change in doses in the 30 days prior to enrolment in the study and no planned dose change during the study and through the primary endpoint. 8. All medications or interventions for epilepsy (including ketogenic diet and any neurostimulation devices for epilepsy) must have been stable for 30 days prior to screening and the patient must be willing to maintain a stable regimen throughout the study. The ketogenic diet and neurostimulation treatments are not considered AEDs for the purpose of this study. 9. Patients or their caregiver must be willing to complete daily PRO assessments. 10. For female patients of childbearing potential: a. willingness to undergo serum or urinary pregnancy testing at screening and during the trial period. b. willingness to use contraception.
Exclusion criteria
Exclusion criteria: 1. Etiology of a patientâ??s seizures is a progressive neurologic disease other than TSC. 2. Anoxic episode requiring resuscitation within 6 months of screening. 3. Patient weight below 15kg. 4. Clinically significant unstable medical conditions other than epilepsy including, but not limited to, cardiovascular, gastrointestinal, renal, hepatic, neurological, musculoskeletal, infectious, endocrine, metabolic, haematological, psychiatric, or other major physical impairment that is not stable in the opinion of the investigator and could affect the safety of the patient throughout the study, influence the findings of the study or their interpretation, or might impede the patientâ??s ability to complete the entire duration of the study. 5. Any clinically significant abnormal findings in physical examination, vital signs, haematology, clinical chemistry, or urinalysis during screening which, in the opinion of the investigator, may put the patient at risk because of his/her participation in the study, or might influence the results of the study, or the patientâ??s ability to complete the entire duration of the study. 6. Clinically relevant symptoms or a clinically significant illness in the 4 weeks prior to screening or randomization, other than epilepsy. 7. Current or past use of recreational or medicinal cannabis within the three months prior to study entry and unwillingness to abstain for the duration of the study or a positive result on a urine tetrahydrocannabinol (THC) panel test. Epidiolex is an AED and is therefore allowed for the treatment of TSC with no change in doses in the 30 days prior to enrolment in the study and no planned dose change during the study and through the primary endpoint). 8. Participation in a clinical trial involving another investigational product (IP) in the previous 6 months or at any time in a gene therapy clinical trial. 9. Patient has previously had brain surgery for the treatment of epilepsy. (Surgery for removal of tumours >=6 months prior to entry into the study is acceptable.). 10. Patient has bipolar disorder. 11. Subject is currently taking long-term systemic steroids (excluding inhaled medication for asthma treatment) or any other daily medication known exacerbate epilepsy. An exception will be made for prophylactic medication such as medications for idiopathic nephrotic syndrome or asthma. 12. Pregnancy or lactation.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To evaluate the efficacy of a double-blind, daily basimglurant administration, adjunctive to ongoing anticonvulsive therapy compared with placebo adjunctive to ongoing anticonvulsive therapy in patients with Tuberous Sclerosis Complex (TSC).Timepoint: 86 weeks including 4 week of placebo then 30 weeks of DB study, and 52 weeks of OL study | — |
Secondary
| Measure | Time frame |
|---|---|
| Change from baseline in Sheehan Disability Scale (SDS score at Week 16 in Period 2 and at Week 30 in Period 4. Adverse events, Absolute values and changes from baseline in vital signs, physical examination, electrocardiogram, and clinical laboratory test parameters Treatment delays, dose reductions, and dose discontinuations S-STS score for suicidal ideation Seizure typesTimepoint: 86 weeks | — |
Countries
Australia, India, Israel, Italy, Poland, Spain, Turkey, United Kingdom, United States of America
Contacts
Public ContactShiv Issar
CliniRx Research Pvt Ltd
Outcome results
None listed