NCT02640443

Sulfamethoxazole for the Treatment of Primary PREPL Deficiency

Sulfamethoxazole for the Treatment of Primary PREPL Deficiency (In Dutch: Sulfamethoxazole Ter Behandeling Van Primaire PREPL deficiëntie)

• Status: Unknown
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 15 (estimated)
• Sponsor: Universitair Ziekenhuis Brussel · Academic / Other
• Sex: All
• Age: 2 Months
• Healthy volunteers: Not accepted

Summary

The investigators will evaluate whether sulfamethoxazole, a sulfamide antibiotic, improves the symptoms of primary PREPL deficiency (hypotonia-cystinuria syndrome and isolated PREPL deficiency).

Conditions

• Hypotonia Cystinuria Syndrome
• Isolated PREPL Deficiency

Interventions

Type	Name	Description
DRUG	Sulfamethoxazole	60 mg sulfamethoxazole per kg bodyweight (maximal of 3g) divided in 2 doses per day during 3 weeks

Timeline

• Start date: 2015-10-01
• Primary completion: 2016-10-01
• Completion: 2016-10-01
• First posted: 2015-12-29
• Last updated: 2015-12-29