Nervous System Diseases

All Conditions

Symptoms and General Pathology

Neoplasms

Diseases and Abnormalities at or Before Birth

Skin and Connective Tissue Diseases

Rare Diseases

Epilepsy

Sclerosis

Tuberous Sclerosis

Brain Diseases

Central Nervous System Diseases

Hamartoma

Neoplasms, Multiple Primary

Syndrome

Neoplastic Syndromes, Hereditary

Congenital Abnormalities

Malformations of Cortical Development

Nervous System Malformations

Neurocutaneous Syndromes

Heredodegenerative Disorders, Nervous System

Neurodegenerative Diseases

Genetic Diseases, Inborn

Tuberous Sclerosis Complex

All Drugs and Chemicals

Ganaxolone

Ganaxalone

Placebo

This is a Phase 3, global, double-blind, randomized, placebo-controlled study of adjunctive GNX treatment in children and adults with TSC-related epilepsy. The study consists of a 4-week prospective Baseline phase, defined as the first 28 days following screening, followed by a double-blind phase consisting of a 4-week titration period (Day 1 to Day 28) and a 12-week maintenance period (Day 29 to Week 16).

Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy

A Phase 3, Double-blind, Randomized, Placebo-controlled Trial of Adjunctive Ganaxolone (GNX) Treatment in Children and Adults With Tuberous Sclerosis Complex (TSC)-Related Epilepsy (TrustTSC)

Seizure frequency will be calculated as the total number of seizures divided by the number of days with seizure data, multiplied by 28.

Treatment responders are defined as those participants with ≥ 50% reduction from Baseline in seizure frequency

The CGI-I is a 7-point Likert scale that the parent(s)/caregiver(s)/legally authorized representative (LAR)(s) and clinician uses to rate the change in overall seizure control, behavior, safety, and tolerability after initiation of the Investigational product (IP) relative to Baseline (prior to treatment with the IP). It was rated as: 1- "very much improved", 2- "much improved', 3- "minimally improved", 4- "no change", 5- "minimally worse", 6- "much worse", and 7- "very much worse". Higher scores indicated worse condition.

The ADAMS is a rating scale designed to screen for anxiety and depression in individuals with intellectual disability. The 28-question scale is filled out by the parent(s)/caregiver(s)/LAR(s) and is based on the participant's behavior. Each question will be rated on 4-point Likert scale as follows: 0- "behavior has not occurred or is not a problem"; 1- "behavior occurs occasionally or is a mild problem"; 2- "behavior occurs quite often or is a moderate problem"; and 3- "behavior occurs a lot or is a severe problem". Higher scores indicated worse condition.

The SF-36 is a multi-purpose survey designed to capture participant or parent(s)/caregiver(s)/LAR(s) perceptions of own health and well-being. The SF-36 has 36 items grouped in 8 dimensions: physical functioning, physical and emotional limitations, social functioning, bodily pain, general, and mental health, which are the weighted sums of the questions in each section. Each scale is directly transformed into a 0-100 scale on the assumption that each question carries equal weight. Higher scores represent better health-related quality-of-life.

Peds-QL-FIM is designed to measure the impact of pediatric chronic health conditions on parents and the family. It composes of 6 scales measuring parent self-reported functioning: 1) Physical functioning (6 items), 2) Emotional functioning (5 items), 3) Social functioning (4 items), 4) Cognitive functioning (5 items), 5) Communication (3 items), 6) Worry (5 items), and in addition two scales measuring parent-reported family functioning: 7) Daily activities (3 items) and 8) Family relationships (5 items). Each of the items have five Likert response options: 0 = never a problem, 1 = almost never, 2 = sometimes, 3 = often, 4= almost always. Items are reverse-scored and linearly transformed to a 0-100 scale (0 = 100, 1 = 75, 2 = 50, 3 = 25, 4 = 0); higher scores indicate better functioning (less negative impact).

The ELDQOL is a parent/caregiver/LAR-reported measure that examines seizure severity, seizure related injury, anti-seizure medication (ASM) side effects, behavior, mood, physical status, cognitive and social functioning, communication, overall health and quality of life, and family concerns. There are a total of 70 items, some of which are responded as 1=yes/2=no, some of which range 1-4 and others 1-5. For those items, higher is more severe. For mood-related items, however, the scoring is always/often/sometimes/never which are coded as 1-4 but may be good or bad, depending on the mood. Finally: there are qualitative questions with open-ended answers related to "were any questions difficult to answer" and "are there any other comments you would like to make". Each of the 70 items is characterized as its own measurement, and they are not aggregated or tabulated into an overall score.

The CGI-CSID is a 7-point Likert scale in which the parent(s)/caregiver(s)/LAR(s) assesses change in seizure intensity and/or duration after initiation of investigational product relative to Baseline (prior to treatment with investigational product). The scale ranges from 1- very much improved, 2- much improved, 3- minimally improved, 4- no change, 5- minimally worse, 6- much worse, and 7- very much worse. Higher scores indicate worse condition

Marinus Pharmaceuticals

Neurosteroids

Neurotransmitter Agents

GABA Modulators

This is a Phase 3, global, open-label extension (OLE) study of adjunctive GNX treatment in children and adults with TSC who previously participated in either Study 1042-TSC-3001 or Study 1042-TSC-2001

This is an open-label, single arm study with no blinding as all participants will receive adjunctive GNX

Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy

A Phase 3, Open-label Study of Adjunctive Ganaxolone (GNX) Treatment in Children and Adults With Tuberous Sclerosis Complex (TSC)-Related Epilepsy (TrustTSC OLE)

The C-SSRS is a clinician administered assessment tool that evaluates suicidal ideation and behavior. Number of participants that have an affirmative response to the 5 items for suicidal ideation (1. Wish to be dead, 2. Non-specific active suicidal thoughts, 3. Active suicidal ideation with any methods (not plan) without intent to act, 4. Active suicidal ideation with some intent to act, without specific plan, 5. Active suicidal ideation with specific plan and intent) and/or to the 5 items for suicidal behavior (1. Preparatory acts or behavior, 2. Aborted attempt, 3. Interrupted attempt, 4. Actual attempt, 5. Completed suicide) will be reported. Higher scores indicate worse symptoms.

Treatment responders are defined as those participants with ≥ 50% reduction from Baseline in seizure frequency during open-label treatment.

The CGI-CSID is a 7-point Likert scale in which the parent(s)/caregiver(s)/LAR(s) assesses change in seizure intensity and/or duration after initiation of investigational product relative to Baseline (prior to treatment with investigational product). The scale ranges from 1- very much improved, 2- much improved, 3- minimally improved, 4- no change, 5- minimally worse, 6- much worse, and 7- very much worse. Higher scores indicate worse condition.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy	NCT05323734	Tuberous Sclero...	Ganaxalone Placebo	1 Year - 65 Years	Marinus Pharmaceuticals
Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy	NCT05604170	Tuberous Sclero...	Ganaxolone	1 Year - 65 Years	Marinus Pharmaceuticals

Spots Global Cancer Trial Database for adjunctive

Every month we try and update this database with for adjunctive cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Spots Global Cancer Trial Database for adjunctive

Every month we try and update this database with for adjunctive cancer trials from around the world to help patients and their families find trials that might be right for them. We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Every month we try and update this database with for adjunctive cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.