Open-Label Continuation Treatment Study With Oral MC-1 for the Treatment of Patients With PNPO Deficiency

Medicure

Summary

Continued access to MC-1 is required by subjects who have participated in the MEND-PNPO study, as no other pharmaceutical grade P5P is currently commercially available. The protocol duration is planned for until the finished product is commercially available in the respective countries where subjects are participating in the study.

Eligibility

Age range: 2+ years
Sex: All
Healthy volunteers: No

Detailed criteria

Inclusion Criteria: * The subject should have completed participation in the MEND-PNPO study (Protocol # 16002) and in the opinion of the Principal Investigator would benefit from long-term treatment with MC-1 * Written informed consent (by parent or guardian if under the age of 18) Exclusion Criteria: * The patient has any condition or abnormality which may, in the opinion of the Investigator, compromise the safety of the patient, or influence their ability to comply with study procedures.

Interventions

Drug
Pyridoxal 5'-Phosphate
Oral tablets

Locations (5)

Lucile Packard Children's Hospital
Palo Alto, California
Children's Hospital Colorado
Aurora, Colorado
Boston Children's Hospital
Boston, Massachusetts
Queensland Children's Hospital and Health Service
Brisbane, Queensland
Royal Children's Hospital
Melbourne, Victoria

Open-Label Continuation Treatment Study With Oral MC-1 for the Treatment of Patients With PNPO Deficiency

Summary

Eligibility

Interventions

Locations (5)

Run this study?

Details

Conditions