Overview

A Multicenter, Open-Label, Withdrawal and Treatment Trial Assessing the Efficacy, Safety, and Tolerability of Doses of CERC-801 in Subjects With SLC35A2-CDG

Status:
Not yet recruiting

Trial end date:
2025-03-31

Target enrollment:
0

Participant gender:
All

Summary

This is a multicenter, open-label, withdrawal and treatment study assessing the efficacy, safety, and tolerability of CERC-801 in subjects with SLC35A2-CDG.

Phase:

Phase 2/Phase 3

Accepts Healthy Volunteers?

Details

Lead Sponsor:

Eva Morava-Kozicz

Criteria

Inclusion Criteria:

1. Subject/legally authorized representative (LAR) is able to understand and provide
written informed consent, and assent (as applicable) to participate in this study.

2. Subject has biologically and genetically proven SLC35A2-CDG.

3. Subject has at least one historical measurement of NPCRS before initiation of
unregulated D galactose therapy.

4. Subject has relevant laboratory test values (CBC, ATIII, APTT, CMP, TSH, free T4,
IGBP3, IgF1, and glycan analysis by mass spectrometry) before initiation of
unregulated D galactose therapy.

Exclusion Criteria:

1. Subject has aldolase B deficiency, galactosemia, hemolytic uremic syndrome, or severe
anemia.

2. In the investigator's opinion, subject is not able or willing to comply with the trial
requirements.

3. In the investigator's opinion, subject has a history of galactose intolerance that
precludes the subject from participation in this study.

4. In the investigator's opinion, subject has previously experienced any of the following
severe adverse events from oral galactose:

1. Severe diarrhea

2. Severe, recurrent vomiting

3. Constipation

4. Galactosuria

5. Increased liver glycogen storage

5. Use of investigational compounds or ongoing unregulated D-galactose supplementation or
current enrollment in another trial involving investigational compounds