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The purpose of the study is to evaluate the efficacy, tolerability, and safety of vigabatrin versus rapamycin as a preventive treatment in infants with Tuberous Sclerosis Complex (TSC).
This is a two-arm, randomized, double-blind and double-dummy, placebo controlled study to evaluate the efficacy, tolerability, and safety of vigabatrin versus rapamycin as a preventive treatment in infants with TSC. The study consists of 3 phases for each patient: screening, core blinded phase, and open-label follow-up phase. Patients who meet the eligibility criteria will be randomized to receive vigabatrin or rapamycin. The randomization ratio is 1:1. Randomization will be stratified by the sex and the presence of epileptiform activity on baseline videoEEG (video electroencephalography) recording (yes versus no). Approximately 60 infants are planned to be enrolled in the study.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Vigabatrin arm | Experimental | Vigabatrin in capsules co-administered with placebo in liquid. |
|
| Rapamycin arm | Experimental | Rapamycin in liquid co-administered with placebo in capsules. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Vigabatrin | Drug | Vigabatrin in capsules administered orally, initially (between V1 and V2) once daily in the evening,and starting from V2 administered two times daily. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Occurrence of clinical seizures in the blinded phase of the study, | 730 days | |
| Summarized volume of TSC-associated tumors ≥ 125% of initial value within the blinded phase of the study | 730 days |
| Measure | Description | Time Frame |
|---|---|---|
| Total volume of TSC-associated tumors within the blinded phase and the whole study | 730 days | |
| The risk for high risk of autism assessed with psychological test at 6, 12, 18, 24 months | 6, 12, 18, 24 months |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Katarzyna Kotulska-Jozwiak | Contact | +48 22 8157404 | k.kotulska@ipczd.pl | |
| Monika Szkop | Contact | +48 22 815 74 04 | m.szkop@ipczd.pl |
| Name | Affiliation | Role |
|---|---|---|
| Katarzyna Kotulska-Jozwiak | The Children's Memorial Health Institute | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Medical University of Warsaw, Department of Pediatric Neurology | Not yet recruiting | Warsaw | 02-091 | Poland |
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| ID | Term |
|---|---|
| D014402 | Tuberous Sclerosis |
| D004827 | Epilepsy |
| D009369 | Neoplasms |
| ID | Term |
|---|---|
| D006222 | Hamartoma |
| D009378 | Neoplasms, Multiple Primary |
| D009386 | Neoplastic Syndromes, Hereditary |
| D065703 | Malformations of Cortical Development, Group I |
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| ID | Term |
|---|---|
| D020888 | Vigabatrin |
| D020123 | Sirolimus |
| D005440 | Fluid Therapy |
| ID | Term |
|---|---|
| D005680 | gamma-Aminobutyric Acid |
| D000613 | Aminobutyrates |
| D002087 | Butyrates |
| D000144 | Acids, Acyclic |
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Triple
| Rapamycin | Drug | Rapamycin in liquid administered orally, in the morning, every other day or daily depending on the patient's body weight. The starting dose of rapamycin will be calculated according to the body weight of the patient measured at V1. |
|
| Placebo | Drug | Placebo in liquid administered orally, once daily, in the morning. The starting dose of placebo in liquid will be calculated according to the body weight of the patient measured at V1. |
|
|
| Placebo | Drug | Placebo in granules administered orally, initially once daily in the evening,and after reaching the targeted dose administered two times daily. |
|
|
| The risk for low developmental quotient (< 70 points in Bayley Scales of Infant Development, measured at the end of the blinded phase and at the end of the entire study) at the end of the study | 730 days |
| The risk of drug-resistant epilepsy at any point of the study | 730 days |
| Occurrence of adverse events within the blinded phase of the study | 730 days |
| Number of adverse events across the whole study | 730 days |
| Parameters of physical development (weight gain history) across the whole study | 730 days |
| Parameters of physical development (height gain history) across the whole study | 730 days |
| Children's Memorial Health Institute, Neurology and Epileptology | Recruiting | Warsaw | 04-730 | Poland |
|
| D054220 | Malformations of Cortical Development |
| D009421 | Nervous System Malformations |
| D009422 | Nervous System Diseases |
| D020752 | Neurocutaneous Syndromes |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D030342 | Genetic Diseases, Inborn |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D002264 |
| Carboxylic Acids |
| D009930 | Organic Chemicals |
| D000596 | Amino Acids |
| D000602 | Amino Acids, Peptides, and Proteins |
| D018942 | Macrolides |
| D007783 | Lactones |
| D004358 | Drug Therapy |
| D013812 | Therapeutics |