A Multicenter, Randomized, Double-blind, Withdrawal Trial Assessing the Efficacy, Safety, and Tolerability of AVTX-801 in Subjects With PGM1-CDG

• Status: Not yet recruiting
• Trial end date: 2024-12-31
• Target enrollment: 0
• Participant gender: All

Summary

This is a multicenter, randomized, double-blind, withdrawal study assessing the efficacy, safety, and tolerability of AVTX-801 in subjects with PGM1-CDG.

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Eva Morava-Kozicz

Criteria

Inclusion Criteria:

1. Subject is aged 6 months to 60 years.

2. Subject has biologically and genetically proven PGM1-CDG.

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

4. Subject has relevant laboratory test values (complete blood count [CBC], ATIII, activated partial thromboplastin time [APTT], CK, comprehensive metabolic panel [CMP], thyroid stimulating hormone [TSH], free thyroxin [T4], insulin like growth factor binding protein 3 [IGBP3], insulin-like growth factor 1 [IgF1], and glycan analysis by mass spectrometry) before initiation of unregulated D-galactose therapy.

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

6. Women of childbearing potential (WOCBP) meeting the criteria below:

1. Non-lactating and has a negative pregnancy test at screening -AND-

2. Uses an acceptable double-barrier method of contraception as determined by the investigator or sub-investigator for the duration of the study and 30 days following the last dose of study drug.

7. Male subjects must agree to use an acceptable double-barrier method of contraception with their partner as determined by the investigator or sub investigator for the duration of the study and 30 days following the last dose of study drug.

Exclusion Criteria:

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

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

10. In the Investigator's opinion, subject has previously experienced any of the following severe AEs from oral galactose:

1. Severe diarrhea

2. Severe, recurrent vomiting

3. Galactosuria

4. Constipation

5. Increased liver glycogen storage.

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

12. Subject is pregnant.

13. Subject has hepatic impairment that would require a dose adjustment, defined by the Investigator.

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