Study of ORL-1B in Patients With Biotinidase Deficiency

Biotinidase Deficiency Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03269045
• Other study ID #: Biot-1
• Status: Completed
• Phase: Phase 1/Phase 2
• First received: August 28, 2017
• Last updated: January 13, 2018
• Start date: August 31, 2013
• Est. completion date: January 1, 2018

Study information

• Verified date: January 2018
• Source: Orpha Labs
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 20
• Est. completion date: January 1, 2018
• Est. primary completion date: January 1, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 18 Years

Eligibility

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.

Related Conditions & MeSH terms

• Biotinidase Deficiency

Intervention

Drug:
• ORL-1B
Oral ORL-1B

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Orpha Labs

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Improvement in seizure frequency	Statistically significant improvement in seizure frequency after the ORL-1B treatment.	12 months