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Basimglurant in Children, Adolescents, and Young Adults With TSC

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ClinicalTrials.gov Identifier: NCT05059327
Recruitment Status : Recruiting
First Posted : September 28, 2021
Last Update Posted : October 4, 2022
Sponsor:
Information provided by (Responsible Party):
Noema Pharma AG

Brief Summary:
The study intends to show that basimglurant provides effective seizure control in children, adolescents and young adults with Tuberous Sclerosis Complex (TSC).

Condition or disease Intervention/treatment Phase
Tuberous Sclerosis Complex Drug: Basimglurant with crossover to Placebo Drug: Placebo with crossover to Basimglurant Phase 2

Detailed Description:

The study drug (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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 54 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Intervention 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 (Participant, Investigator)
Primary Purpose: Treatment
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
Actual Study Start Date : March 3, 2021
Estimated Primary Completion Date : June 14, 2024
Estimated Study Completion Date : July 15, 2024


Arm Intervention/treatment
Experimental: Arm A (Basimglurant to Placebo)
Basimglurant to Placebo
Drug: Basimglurant with crossover to Placebo
Basimglurant with crossover to Placebo

Placebo Comparator: Arm B (Placebo to Basimglurant)
Placebo to Basimglurant
Drug: Placebo with crossover to Basimglurant
Placebo with crossover to Basimglurant




Primary Outcome Measures :
  1. Mean percentage change from baseline of seizure frequency during the maintenance dosing in Period 2 (Weeks 13 to 16) and Period 4 (Weeks 27-30). [ Time Frame: 30 weeks ]

Secondary Outcome Measures :
  1. Number of patients considered treatment responders. [ Time Frame: 30 weeks ]
  2. Longest seizure free interval (i.e., seizure free days). [ Time Frame: 30 weeks ]
  3. Change in the severity of symptoms of TSCas 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 ]
  4. Changes 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 ]
  5. Safety of the study drug in children and adolescents with seizures associated with TSC. [ Time Frame: 82 weeks ]
    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.


Other Outcome Measures:
  1. Change in seriousness of disease as assessed by Most Impactful Symptoms Scale in Periods 2 (weeks 13 to 16) and Period 4 (weeks 27 to 30) compared to baseline. [ Time Frame: 30 weeks ]
  2. Frequency of seizures detected by the wearable device evaluated as the change from Baseline compared to study treatment in Period 1 (weeks 13 to 16) and Period 4 (weeks 27 to 30). [ Time Frame: 30 weeks ]
  3. Intensity of seizures detected by the wearable device evaluated as the change from Baseline compared to study treatment Periods 1 and 4. [ Time Frame: 30 weeks ]


Information from the National Library of Medicine

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Ages Eligible for Study:   5 Years to 30 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
  • A documented history of TSC,
  • Refractory seizure treatment status
  • Currently receiving one or more anti-epileptic drugs (AEDs)
  • Stable medications or interventions for epilepsy
  • Willingness to complete Patient Reported Outcome
  • 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.

Exclusion Criteria (summary):

  • Neurologic disease other than TSC.
  • Recent anoxic episode
  • Patient weight below 15kg
  • Clinically significant unstable medical condition(s).
  • Pregnancy or lactation.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05059327


Contacts
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Contact: Noema Pharma Please contact via email: clinicaltrials@noemapharma.com

Locations
Show Show 24 study locations
Sponsors and Collaborators
Noema Pharma AG
Investigators
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Study Director: Renata Lazarova, MD Noema Pharma AG
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Responsible Party: Noema Pharma AG
ClinicalTrials.gov Identifier: NCT05059327    
Other Study ID Numbers: NOE-TSC-201
First Posted: September 28, 2021    Key Record Dates
Last Update Posted: October 4, 2022
Last Verified: September 2022

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Noema Pharma AG:
Basimglurant
TSC
Seizures
Additional relevant MeSH terms:
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Tuberous Sclerosis
Sclerosis
Pathologic Processes
Hamartoma
Neoplasms
Neoplasms, Multiple Primary
Neoplastic Syndromes, Hereditary
Malformations of Cortical Development, Group I
Malformations of Cortical Development
Nervous System Malformations
Nervous System Diseases
Neurocutaneous Syndromes
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Congenital Abnormalities
Genetic Diseases, Inborn