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Trial of lactose supplementation in CDG

Phase 2
Conditions
Congenital disorder of glycosylation
Registration Number
JPRN-jRCTs061200059
Lead Sponsor
Maegaki Yoshihiro
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
ot Recruiting
Sex
All
Target Recruitment
5
Inclusion Criteria

Patients with
1) CDG having the mutation in one of the genes of SLC35A2, PGM1, SLC39A8, or TMEM165.
2) At least one epileptic seizure 28 days prior to the drug administration.

Exclusion Criteria

Patients with
1) Dairy allergies.
2) Lactose intolerance.
3) Diabetes mellitus.
4) Severe liver, renal, or cardiac dysfunction.
5) Pregnancy or nursing.
6) Participating in other clinical trials or stidies within three months before enrollment.
7) Judgement to be ineligible for trial.

Study & Design

Study Type
Interventional
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Differences of frequencies of epileptic seizures between baseline and 52 weeks later.
Secondary Outcome Measures
NameTimeMethod
Variabilities of<br>1) Epileptic seizures<br>2) Scores of Nijmegen Pediatric CDG Rating Scale<br>3) Interictal epileptiform discarges in EEG<br>4) Serum galactosylglycan
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