Back to resultsBasimglurant in Children, Adolescents, and Young Adults With TSC
Primary Purpose
Tuberous Sclerosis Complex
Status
Recruiting
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Basimglurant with crossover to Placebo
Placebo with crossover to Basimglurant
Sponsored by
About this trial
This is an interventional treatment trial for Tuberous Sclerosis Complex focused on measuring Basimglurant, TSC, Seizures
Eligibility 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:
- Willingness to undergo serum or urinary pregnancy testing at screening and during the trial period.
- 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.
Sites / Locations
- David Geffen School of Medicine at UCLA (Site #: 101)Recruiting
- Nicklaus Children's Hospital (Site # 116)
- Kennedy Krieger Institute (Site #: 110)Recruiting
- Boston Children's Hospital (Site #: 102)Recruiting
- William Beaumont Hospital - Royal Oak (Site #: 104)Recruiting
- Minnesota Epilepsy Group PA (Site #: 105)Recruiting
- Boston Children's Health Physicians (BCHP) (Site #: 111)Recruiting
- Duke Children's Hospital and Health Center (Site #: 106)Recruiting
- Cincinnati Children's Hospital Medical Center - PIN (Site # 115)
- University Hospitals Cleveland Medical Center, Rainbow Babies and Childrens Hospital (Site #: 107)Recruiting
- Texas Scottish Rite Hospital For Children (Site #: 113)Recruiting
- The University of Texas Medical School at Houston (Site #: 103)Recruiting
- Multicare Health System (Site #: 109)Recruiting
- Citi Neuro Centre (Site # 806)Recruiting
- Rainbow Children's Hospital (Site # 803)Recruiting
- All India Institute of Medical Sciences (Site # 804)Recruiting
- Jaslok Hospital and Research Centre (Site # 801)Recruiting
- Deenanath Mangeshkar Hospital and Research Centre (Site # 805)
- Gleneagles Global Health City (Site # 802)Recruiting
- Christian Medical College (Site # 807)Recruiting
- Schneider Children's Medical Center of Israel - Petah Tikvah - PIN (Site #: 502)Recruiting
- The Edmond and Lily Safra Children's Hospital (Site #: 501)Recruiting
- Dana Children's Hospital (Site #: 504)Recruiting
- Hadassah Medical Center - PPDS (Site #: 503)Recruiting
- ASST Santi Paolo e Carlo - Azienda Universitaria-Polo Universitario San Paolo (Site # 181)
- ASST Grande Ospedale Metropolitano Niguarda - Presidio Ospedaliero Ospedale Niguarda (Site # 183)
- Azienda Ospedaliero Universitaria Policlinico Vittorio Emanuele (Site # 182)
- Azienda Ospedaliero Universitaria A Meyer - INCIPIT - PIN (Site # 184)Recruiting
- Instytut Pomnik Centrum Zdrowia Dziecka (Site # 262)Recruiting
- Centrum Medyczne Plejady (Site # 263)Recruiting
- AmiCare Centrum Medyczne (Site # 261)Recruiting
- Hospital Universitario Germans Trias i Pujol (Site # 194)Recruiting
- Hospital Sant Joan de Deu - PIN (Site # 192)Recruiting
- Hospital Regional Universitario de Malaga - Hospital General (Site # 193)Recruiting
- Centro de Neurología Avanzada (Site # 191)Recruiting
- Ankara Etlik Şehir Hastanesi (Site #: 903)Recruiting
- Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi (Site #: 905)Recruiting
- Yeditepe University Kosuyolu Hospital (Site #: 904)Recruiting
- Istanbul Egitim ve Arastirma Hastanesi (Site #: 901)Recruiting
- Acibadem Altunizade Hospital (Site #: 902)
- Salford Royal Hospital - PPDS (Site # 304)Recruiting
- The Royal London Hospital (Site # 302)Recruiting
- St George Hospital (Site # 303)Recruiting
- Cardiff University (Site # 305)Recruiting
- Noahs Ark Children's Hospital (Site # 306)Recruiting
- Queen Elizabeth Hospital Birmingham (Site # 301)Recruiting
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
Arm A (Basimglurant to Placebo)
Arm B (Placebo to Basimglurant)
Arm Description
Basimglurant to Placebo
Placebo to Basimglurant
Outcomes
Primary Outcome Measures
Mean percentage in monthly seizure frequency during the maintenance dosing in Period 2 (Weeks 13 to 16) and Period 4 (Weeks 27-30).
Secondary Outcome Measures
Number of patients considered treatment responders.
Longest seizure free interval (i.e., seizure free days).
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.
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.
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.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05059327
Brief Title
Basimglurant in Children, Adolescents, and Young Adults With TSC
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
Study Type
Interventional
2. Study Status
Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
March 3, 2021 (Actual)
Primary Completion Date
March 31, 2024 (Anticipated)
Study Completion Date
August 31, 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Noema Pharma AG
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
5. Study Description
Brief Summary
The study intends to show that basimglurant provides effective seizure control in children, adolescents and young adults with Tuberous Sclerosis Complex (TSC).
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Tuberous Sclerosis Complex
Keywords
Basimglurant, TSC, Seizures
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Crossover Assignment
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
ParticipantInvestigator
Allocation
Randomized
Enrollment
54 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Arm A (Basimglurant to Placebo)
Arm Type
Experimental
Arm Description
Basimglurant to Placebo
Arm Title
Arm B (Placebo to Basimglurant)
Arm Type
Placebo Comparator
Arm Description
Placebo to Basimglurant
Intervention Type
Drug
Intervention Name(s)
Basimglurant with crossover to Placebo
Intervention Description
Basimglurant with crossover to Placebo
Intervention Type
Drug
Intervention Name(s)
Placebo with crossover to Basimglurant
Intervention Description
Placebo with crossover to Basimglurant
Primary Outcome Measure Information:
Title
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
Secondary Outcome Measure Information:
Title
Number of patients considered treatment responders.
Time Frame
30 weeks
Title
Longest seizure free interval (i.e., seizure free days).
Time Frame
30 weeks
Title
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
Title
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
Title
Safety of the study drug in children, adolescents and young adults with seizures associated with TSC.
Description
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.
Time Frame
82 weeks
Other Pre-specified Outcome Measures:
Title
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
Title
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
Title
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
10. Eligibility
Sex
All
Minimum Age & Unit of Time
5 Years
Maximum Age & Unit of Time
30 Years
Accepts Healthy Volunteers
No
Eligibility 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:
Willingness to undergo serum or urinary pregnancy testing at screening and during the trial period.
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Noema Pharma
Phone
Please contact via email:
Email
clinicaltrials@noemapharma.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Renata Lazarova, MD
Organizational Affiliation
Noema Pharma AG
Official's Role
Study Director
Facility Information:
Facility Name
David Geffen School of Medicine at UCLA (Site #: 101)
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Individual Site Status
Recruiting
Facility Name
Nicklaus Children's Hospital (Site # 116)
City
Miami
State/Province
Florida
ZIP/Postal Code
33155-3009
Country
United States
Individual Site Status
Not yet recruiting
Facility Name
Kennedy Krieger Institute (Site #: 110)
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21205
Country
United States
Individual Site Status
Recruiting
Facility Name
Boston Children's Hospital (Site #: 102)
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115-5724
Country
United States
Individual Site Status
Recruiting
Facility Name
William Beaumont Hospital - Royal Oak (Site #: 104)
City
Royal Oak
State/Province
Michigan
ZIP/Postal Code
48073-6712
Country
United States
Individual Site Status
Recruiting
Facility Name
Minnesota Epilepsy Group PA (Site #: 105)
City
Roseville
State/Province
Minnesota
ZIP/Postal Code
55113-1306
Country
United States
Individual Site Status
Recruiting
Facility Name
Boston Children's Health Physicians (BCHP) (Site #: 111)
City
Hawthorne
State/Province
New York
ZIP/Postal Code
10532
Country
United States
Individual Site Status
Recruiting
Facility Name
Duke Children's Hospital and Health Center (Site #: 106)
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27705-4699
Country
United States
Individual Site Status
Recruiting
Facility Name
Cincinnati Children's Hospital Medical Center - PIN (Site # 115)
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229-3026
Country
United States
Individual Site Status
Not yet recruiting
Facility Name
University Hospitals Cleveland Medical Center, Rainbow Babies and Childrens Hospital (Site #: 107)
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44106-1716
Country
United States
Individual Site Status
Recruiting
Facility Name
Texas Scottish Rite Hospital For Children (Site #: 113)
City
Dallas
State/Province
Texas
ZIP/Postal Code
75219-3924
Country
United States
Individual Site Status
Recruiting
Facility Name
The University of Texas Medical School at Houston (Site #: 103)
City
Houston
State/Province
Texas
ZIP/Postal Code
77030-3000
Country
United States
Individual Site Status
Recruiting
Facility Name
Multicare Health System (Site #: 109)
City
Tacoma
State/Province
Washington
ZIP/Postal Code
98405-4048
Country
United States
Individual Site Status
Recruiting
Facility Name
Citi Neuro Centre (Site # 806)
City
Hyderabad
State/Province
Andhra Pradesh
ZIP/Postal Code
500034
Country
India
Individual Site Status
Recruiting
Facility Name
Rainbow Children's Hospital (Site # 803)
City
Hyderabad
State/Province
Andhra Pradesh
ZIP/Postal Code
500034
Country
India
Individual Site Status
Recruiting
Facility Name
All India Institute of Medical Sciences (Site # 804)
City
New Delhi
State/Province
Delhi
ZIP/Postal Code
110029
Country
India
Individual Site Status
Recruiting
Facility Name
Jaslok Hospital and Research Centre (Site # 801)
City
Mumbai
State/Province
Maharashtra
ZIP/Postal Code
400026
Country
India
Individual Site Status
Recruiting
Facility Name
Deenanath Mangeshkar Hospital and Research Centre (Site # 805)
City
Pune
State/Province
Maharashtra
ZIP/Postal Code
411006
Country
India
Individual Site Status
Not yet recruiting
Facility Name
Gleneagles Global Health City (Site # 802)
City
Chennai
State/Province
Tamil Nadu
ZIP/Postal Code
600100
Country
India
Individual Site Status
Recruiting
Facility Name
Christian Medical College (Site # 807)
City
Vellore
State/Province
Tamil Nadu
ZIP/Postal Code
632004
Country
India
Individual Site Status
Recruiting
Facility Name
Schneider Children's Medical Center of Israel - Petah Tikvah - PIN (Site #: 502)
City
Petah Tikva
State/Province
HaMerkaz
ZIP/Postal Code
49202
Country
Israel
Individual Site Status
Recruiting
Facility Name
The Edmond and Lily Safra Children's Hospital (Site #: 501)
City
Ramat Gan
State/Province
Tel-Aviv
ZIP/Postal Code
52621
Country
Israel
Individual Site Status
Recruiting
Facility Name
Dana Children's Hospital (Site #: 504)
City
Tel Aviv-Yafo
State/Province
Tel-Aviv
ZIP/Postal Code
64239
Country
Israel
Individual Site Status
Recruiting
Facility Name
Hadassah Medical Center - PPDS (Site #: 503)
City
Jerusalem
ZIP/Postal Code
91120
Country
Israel
Individual Site Status
Recruiting
Facility Name
ASST Santi Paolo e Carlo - Azienda Universitaria-Polo Universitario San Paolo (Site # 181)
City
Milano
State/Province
Lombardia
ZIP/Postal Code
20142
Country
Italy
Individual Site Status
Not yet recruiting
Facility Name
ASST Grande Ospedale Metropolitano Niguarda - Presidio Ospedaliero Ospedale Niguarda (Site # 183)
City
Milano
State/Province
Lombardia
ZIP/Postal Code
20162
Country
Italy
Individual Site Status
Not yet recruiting
Facility Name
Azienda Ospedaliero Universitaria Policlinico Vittorio Emanuele (Site # 182)
City
Catania
State/Province
Sicilia
ZIP/Postal Code
20142
Country
Italy
Individual Site Status
Not yet recruiting
Facility Name
Azienda Ospedaliero Universitaria A Meyer - INCIPIT - PIN (Site # 184)
City
Firenze
State/Province
Toscana
ZIP/Postal Code
50139
Country
Italy
Individual Site Status
Recruiting
Facility Name
Instytut Pomnik Centrum Zdrowia Dziecka (Site # 262)
City
Warsaw
State/Province
Mazowieckie
ZIP/Postal Code
04-730
Country
Poland
Individual Site Status
Recruiting
Facility Name
Centrum Medyczne Plejady (Site # 263)
City
Kraków
ZIP/Postal Code
30-363
Country
Poland
Individual Site Status
Recruiting
Facility Name
AmiCare Centrum Medyczne (Site # 261)
City
Łódź
ZIP/Postal Code
91-495
Country
Poland
Individual Site Status
Recruiting
Facility Name
Hospital Universitario Germans Trias i Pujol (Site # 194)
City
Badalona
State/Province
Barcelona
ZIP/Postal Code
08916
Country
Spain
Individual Site Status
Recruiting
Facility Name
Hospital Sant Joan de Deu - PIN (Site # 192)
City
Esplugues De Llobregat
State/Province
Barcelona
ZIP/Postal Code
08950
Country
Spain
Individual Site Status
Recruiting
Facility Name
Hospital Regional Universitario de Malaga - Hospital General (Site # 193)
City
Málaga
ZIP/Postal Code
29010
Country
Spain
Individual Site Status
Recruiting
Facility Name
Centro de Neurología Avanzada (Site # 191)
City
Sevilla
ZIP/Postal Code
41013
Country
Spain
Individual Site Status
Recruiting
Facility Name
Ankara Etlik Şehir Hastanesi (Site #: 903)
City
Yenimahalle
State/Province
Ankara
ZIP/Postal Code
06170
Country
Turkey
Individual Site Status
Recruiting
Facility Name
Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi (Site #: 905)
City
Fatih
State/Province
Istanbul
ZIP/Postal Code
34093
Country
Turkey
Individual Site Status
Recruiting
Facility Name
Yeditepe University Kosuyolu Hospital (Site #: 904)
City
Kadıköy
State/Province
Istanbul
ZIP/Postal Code
34718
Country
Turkey
Individual Site Status
Recruiting
Facility Name
Istanbul Egitim ve Arastirma Hastanesi (Site #: 901)
City
Sultangazi
State/Province
Istanbul
ZIP/Postal Code
34096
Country
Turkey
Individual Site Status
Recruiting
Facility Name
Acibadem Altunizade Hospital (Site #: 902)
City
Üsküdar
ZIP/Postal Code
34662
Country
Turkey
Individual Site Status
Not yet recruiting
Facility Name
Salford Royal Hospital - PPDS (Site # 304)
City
Salford
State/Province
Lancashire
ZIP/Postal Code
M6 8HD
Country
United Kingdom
Individual Site Status
Recruiting
Facility Name
The Royal London Hospital (Site # 302)
City
London
State/Province
London, City Of
ZIP/Postal Code
E1 1BB
Country
United Kingdom
Individual Site Status
Recruiting
Facility Name
St George Hospital (Site # 303)
City
London
State/Province
London, City Of
ZIP/Postal Code
SW17 0QT
Country
United Kingdom
Individual Site Status
Recruiting
Facility Name
Cardiff University (Site # 305)
City
Cardiff
State/Province
South Glamorgan
ZIP/Postal Code
CF10 3AT
Country
United Kingdom
Individual Site Status
Recruiting
Facility Name
Noahs Ark Children's Hospital (Site # 306)
City
Cardiff
State/Province
South Glamorgan
ZIP/Postal Code
CF14 4XW
Country
United Kingdom
Individual Site Status
Recruiting
Facility Name
Queen Elizabeth Hospital Birmingham (Site # 301)
City
Birmingham
State/Province
West Midlands
ZIP/Postal Code
B15 2TH
Country
United Kingdom
Individual Site Status
Recruiting
12. IPD Sharing Statement
Learn more about this trial
Basimglurant in Children, Adolescents, and Young Adults With TSC
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