View clinical trials related to Infantile Spasms.
Filter by:Infantile spasms are a type of seizure linked to developmental issues. Unfortunately, they are often misdiagnosed, causing delays in treatment. The purpose of this study is to develop a computer program that can reliably differentiate infantile spasms from similar, yet benign movements in videos. This computer program will learn from videos taken by parents of study participants. Quickly recognizing and treating infantile spasms is crucial for ensuring the best developmental outcomes.
This trial consists of 3 parts: a pilot safety phase, a pivotal randomized controlled phase, and an open-label extension phase. The open-label extension phase only will be described in this record. All participants will receive GWP42003-P.
This trial consists of 3 parts: a pilot safety phase, a pivotal randomized controlled phase, and an open-label extension phase. The pilot phase only will be described in this record. 2 cohorts of 5 participants will be enrolled sequentially. All participants will receive GWP42003-P.
Infantile Spasms syndrome (ISs) is a characterized by epileptic spasms occurring in clusters with an onset in the first year of life. West syndrome represents a subset of ISs that associates spasms in clusters, a hypsarrhythmia EEG pattern and a developmental arrest or regression. Aetiology of ISs is widely heterogeneous including many genetic causes. Many patients, however, remain without etiological diagnosis, which is critical for prognostic purpose and genetic counselling. In the present study, the investigators performed genetic screening of 73 patients with different types of ISs by array-CGH and molecular analysis of 5 genes: CDKL5, STXBP1, KCNQ2, and GRIN2A, whose mutations cause different types of epileptic encephalopathies, including ISs, as well as MAGI2, which was suggested to be related to a subset of ISs.
Infantile Spasms, is an rare age-specific epilepsy of early infancy. A 2012 American Academy Neurology/ Child Neurology Society practice parameter update on the medical treatment of infantile spasms concluded: adrenocorticotrophic hormone or vigabatrin may be offered for short-term treatment of infantile spasms. There was insufficient evidence to recommend the use of prednisolone, dexamethasone, and methylprednisolone. The cost of ACTH and the side effects of vigabatrin have led to the consideration of alternative medications to treat infantile spasms. The United Kingdom Infantile Spasms Study (UKISS) in 2004, comparing the efficacy of intramuscular synthetic ACTH to high dose oral prednisolone, showed a response rate of 74% for ACTH and 70% for prednisolone. Since the UKISS paper was published, many institutions in the United States and Australia have used oral prednisolone instead of ACTH, partly due to the exorbitant cost of intramuscular ACTH but also its ease of use and better adverse event profile compared to ACTH. Prednisolone and vigabatrin are both oral medications, which can be initiated promptly upon diagnosis of infantile spasms, expediting treatment and shortening treatment lag time. Because the UKISS trial is the only Class 3 study providing evidence for oral prednisolone in the first-line treatment of infantile spasms, further prospective studies are needed.
The sponsor is developing a new paediatric formulation of vigabatrin to better adjust the dose to body weight and to limit waste of unused drug. The currently marketed vigabatrin (Sabril™) form only exists as 500 mg film coated tablets (for adults and children above 6 years) and 500 mg granules for oral solution sachets (for infants and children below 6 years). Sabril™ is not adapted for administration to infants when a fraction of the sachet is needed. Manual splitting of the sachet or lengthy and error-prone dilutions are often required. This study is a descriptive, non-randomized, open label multi-centric acceptability study in infants and children affected with infantile spasms. The primary objective is to describe the adherence to the new formulation. Secondary objectives include: - evaluation of the palatability and user-friendliness of the new treatment, - evaluation of the pharmacokinetic parameters of the new formulation, - PK parameters, - evaluation of the tolerance, - measurement of taurine plasma levels. This study will recruit up to 40 patients with infantile spasms and pharmacoresistant partial epilepsy aged 1 month to 6 years in 23 clinical sites in France.
The purpose of this study was to investigate the presence of neuroinflammation in children with infantile spasms using 11C-PK11195 positron emission tomography (PK PET) scan, and its response to ACTH treatment by repeating the PK PET scan after treatment.
Investigators at Boston Children's Hospital are conducting research in order to better understand the genetic factors which may contribute to epilepsy and related disorders. These findings may help explain the broad spectrum of clinical characteristics and outcomes seen in people with epilepsy.
Infantile spasms constitute a unique age specific epilepsy syndrome of infancy, characterized by epileptic spasms often accompanied by neurodevelopmental regression and an EEG finding of hypsarrhythmia. When all 3 components are present, the eponym "West syndrome" is commonly used. West syndrome is a catastrophic epileptic encephalopathy. It does not respond well to standard anti-epileptic drugs. Hormonal therapy is the mainstay in the treatment of infantile spasms. This includes adreno-cortico trophic hormone (ACTH) and oral steroids. Variable dose of prednisolone used in the treatment. Oral prednisolone used in usual dose (2mg/kg) has been shown to be less effective as compared to ACTH. High dose prednisolone (4mg/kg) has been used in the treatment of infantile spasms, which has been shown to be as effective as ACTH. Pyridoxine has been used as first line treatment in Japan, however there is paucity of data on the efficacy of combination of pyridoxine with hormonal therapy. There are no studies comparing add on pyridoxine with high prednisolone versus high dose prednisolone alone in the treatment of infantile spasms. Therefore the study has been planned to see whether the addition of pyridoxine with high dose prednisolone in the treatment of infantile spasms improves the efficacy in terms of spasm cessation.
Infantile spasms (IIS), a characteristic epilepsy syndrome of infancy with often catastrophic developmental consequences, is known in some patients to have many different genetic, metabolic and structural etiologies. However, for most patients IIS is the only presenting clinical feature and the specific cause is unknown. Only two FDA approved pharmacologic treatments for IIS exist, Adrenocorticotropic hormone (ACTH) and vigabatrin. While vigabatrin may be the treatment of choice for Tuberous Sclerosis as a cause for IS, ACTH is the treatment of choice for all others. Unfortunately, a substantial number of patients may still not respond to ACTH and there is no a priori way that suggests which patients may be responders. This has led to the following key questions: Can novel genetic analyses determine known genetic causes of IS with greater efficiency (more timely and cost-effective)? Can novel genetic analyses determine previously unknown disease modifying genes that predispose individuals to develop IS? Can novel genetic analyses elaborate genes and gene polymorphisms that favor ACTH responsiveness? Do these polymorphisms suggest strategies to improve ACTH responsiveness?