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Sulfamethoxazole for the Treatment of Primary PREPL Deficiency

NCT02640443 · Status: UNKNOWN · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 15

Last updated 2015-12-29

No results posted yet for this study

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

DRUG

Sulfamethoxazole

60 mg sulfamethoxazole per kg bodyweight (maximal of 3g) divided in 2 doses per day during 3 weeks

Sponsors & Collaborators

  • KU Leuven

    collaborator OTHER

  • Universitair Ziekenhuis Brussel

    lead OTHER

Principal Investigators

  • Luc Régal, MD · Universitair Ziekenhuis Brussel

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
SINGLE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-10-31
Primary Completion
2016-10-31
Completion
2016-10-31

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02640443 on ClinicalTrials.gov