Sulfamethoxazole for the Treatment of Primary PREPL Deficiency
NCT ID: NCT02640443
Last Updated: 2015-12-29
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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UNKNOWN
PHASE2
15 participants
INTERVENTIONAL
2015-10-31
2016-10-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
SINGLE
Study Groups
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Treatment
Treatment with sulfamethoxazole. Subjects will serve as their own control, by using the data from baseline and after treatment stop.
Sulfamethoxazole
60 mg sulfamethoxazole per kg bodyweight (maximal of 3g) divided in 2 doses per day during 3 weeks
Interventions
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Sulfamethoxazole
60 mg sulfamethoxazole per kg bodyweight (maximal of 3g) divided in 2 doses per day during 3 weeks
Eligibility Criteria
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Inclusion Criteria
* who are able to follow the study protocol (for the primary endpoint)
* who have given written informed consent
Exclusion Criteria
* women of child-bearing age unless using a reliable method for contraception and not pregnant at study entrance
* additional diagnosis with influence on muscle force
* not able to follow the study protocol (for the primary endpoint)
* history of sulfonamide hypersensitivity
* diminished renal function based on serum creatinine
* transaminases higher than 3 times the upper limit of normal
* for the pupillometry: eye pathology with the exception of refractive errors, drugs with influence on the pupillary light reflex
2 Months
ALL
No
Sponsors
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KU Leuven
OTHER
Universitair Ziekenhuis Brussel
OTHER
Responsible Party
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Principal Investigators
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Luc Régal, MD
Role: PRINCIPAL_INVESTIGATOR
Universitair Ziekenhuis Brussel
References
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Regal L, Shen XM, Selcen D, Verhille C, Meulemans S, Creemers JW, Engel AG. PREPL deficiency with or without cystinuria causes a novel myasthenic syndrome. Neurology. 2014 Apr 8;82(14):1254-60. doi: 10.1212/WNL.0000000000000295. Epub 2014 Mar 7.
Other Identifiers
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2015/059
Identifier Type: -
Identifier Source: org_study_id