Study of ORL-1B in Patients With Biotinidase Deficiency

An Open-label, Single-arm, Phase 2 Study of Biotin in Patients With Biotinidase Deficiency.

Completed

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Key information

Conditions

Biotinidase Deficiency Amino Acid Metabolism, Inborn Errors Carbohydrate Metabolism, Inborn Errors Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Metabolic Diseases Metabolism, Inborn Errors Multiple Carboxylase Deficiency Nutritional and Metabolic Diseases

Age range

Up to 18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

Diagnosis of biotinidase deficiency.
Less than 18 years old.

Exclusion criteria

Diagnosis of any other disease that is not a manifestation of biotinidase deficiency.

Treatment and study plan

ORL-1B

Drug

Oral ORL-1B

Primary outcomes

Improvement in seizure frequency

Time frame: 12 months

Statistically significant improvement in seizure frequency after the ORL-1B treatment.

Sponsors and collaborators

Lead sponsor

Orpha Labs

Industry

Registry information

Official study title

An Open-label, Single-arm, Phase 2 Study of ORL-1B in Patients With Biotinidase Deficiency

Important dates

Study start: 2013
Primary completion: 2018
Study completion: 2018
First posted: Aug 31, 2017
Registry last updated: Jan 17, 2018

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed