Clinical Trial: Efficacy of Tacrolimus and I.V.-Immunoglobulins in Rasmussen Encephalitis
Study Status: Recruiting
Recruit Status: Unknown status
Study Type: Interventional
Official Title: Efficacy of Tacrolimus and i.v.-Immunoglobulins in Rasmussen Encephalitis With Start of Treatment in the Acute Disease Stage. Prospective, Randomised, Open Parallel Group
Brief Summary: Rasmussen encephalitis (RE) is a rare but severe chronic inflammatory brain disease of unknown origin affecting one brain hemisphere. It is usually accompanied by intractable epilepsy. In addition, it often leads to severe disability due to functional deficits caused by atrophy of one brain hemisphere. Hemispherectomy is an effective means of surgical treatment of the epilepsy. It renders the patient, however, hemiplegic, hemianopic and (if the language dominant hemisphere is affected) aphasic. To slow down or even stop the progressive inflammatory damage to the affected brain hemisphere, immunotherapies may be beneficial. According to a literature survey, tacrolimus (twice daily intake of capsules) and intravenous immunoglobulins (monthly infusions) are the most promising compounds for this. In the investigators' study, these two types of treatment are randomly assigned to patients with disease onset within the last year and not too far advanced disability or hemispheric brain injury. The patients are followed to assess prospectively the functional and brain MRI course of the disease.
Detailed Summary:
1. Trial design/2. Trial interventions Patients are screened in epileptological, neuropediatric, and neurological centers all over Germany. The study design was approved on a meeting in Bonn on April 20, 2002 by several external participants and by the ethical committee of the University of Bonn. Patients with the suspected diagnosis of RE are transferred to the Department of Epileptology of the University of Bonn. If the diagnosis "RE in the acute stage" is confirmed and the patient or (in children) the parents give informed consent, the patients are randomized to one of the two active treatment arms.
The study was started on 1.10.2002. The first patient was included on 20.11.2002.
3. Inclusion/exclusion criteria See appropriate section
4. Duration Recruitment started on 1.10.2002 and will go on until the proposed number of study participants has been included. A patient remains in the study until he or she reaches one of the predefined exit parameters (see below, #4.5). Every patient will be followed under study conditions for at least 12 months (to obtain true long term results).
5. Outcome measures See appropriate section.
6. Methods against bias The patients will be randomized to one of the treatment arms. To avoid unbalanced group sizes and unequal numbers of adolescents and adults, there are two randomization lists, one for patients < 11 years, one for older patients (stratification). Blinding is not possible because the administration of the two drugs is different and would have necessitated the additional use of placebo capsules in the IVIG group and placebo infusions in the tacrolimus group. Because the production and administration of an adequate "
Sponsor: University Hospital, Bonn
Current Primary Outcome: Time to exit, criteria: Deterioration of motor function of the affected side by 15 % (>11 yrs of age: 8%) measured by the "Motricity Index" (scale 0-100) or deterioration of the "Hemispheric ratio" assessed by regular MRI scans by 15% (>11 yrs: 8%). [ Time Frame: until final included subject has been followed for one yer ]
Original Primary Outcome: Time to exit, criteria: Deterioration of motor function of the affected side by 15 % (>11 yrs of age: 8%) measured by the "Motricity Index" (scale 0-100) or deterioration of the "Hemispheric ratio" assessed by regular MRI scans by 15% (>11 yrs: 8%).
Current Secondary Outcome: seizure frequency, "Burden of disease" scale, neuropsychological performance, quality of life, T cell receptor studies (H Wiendl, Würzburg) [ Time Frame: until final included subject has been followed for one yer ]
Original Secondary Outcome: seizure frequency, "Burden of disease" scale, neuropsychological performance, quality of life, T cell receptor studies (H Wiendl, Würzburg)
Information By: University Hospital, Bonn
Dates:
Date Received: October 16, 2007
Date Started: November 2002
Date Completion: April 2010
Last Updated: April 9, 2009
Last Verified: April 2009
