NCT03269045

Brief Summary

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

Trial Health

100
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
20

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Aug 2013

Longer than P75 for phase_1

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

August 31, 2013

Completed
4 years until next milestone

First Submitted

Initial submission to the registry

August 28, 2017

Completed
3 days until next milestone

First Posted

Study publicly available on registry

August 31, 2017

Completed
4 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2018

Completed
Last Updated

January 17, 2018

Status Verified

January 1, 2018

Enrollment Period

4.3 years

First QC Date

August 28, 2017

Last Update Submit

January 13, 2018

Conditions

Biotinidase Deficiency

Outcome Measures

Primary Outcomes (1)

  • Improvement in seizure frequency

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

    12 months

Study Arms (1)

Treatment with ORL-1B.

OTHER

Pediatric patients with biotinidase deficiency.

Drug: ORL-1B

Interventions

ORL-1BDRUG

Oral ORL-1B

Treatment with ORL-1B.

Eligibility Criteria

AgeUp to 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Diagnosis of biotinidase deficiency.
  • Less than 18 years old.

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

MeSH Terms

Conditions

Biotinidase Deficiency

Condition Hierarchy (Ancestors)

Multiple Carboxylase DeficiencyAmino Acid Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesCarbohydrate Metabolism, Inborn ErrorsMetabolic DiseasesNutritional and Metabolic Diseases

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 28, 2017

First Posted

August 31, 2017

Study Start

August 31, 2013

Primary Completion

January 1, 2018

Study Completion

January 1, 2018

Last Updated

January 17, 2018

Record last verified: 2018-01

Data Sharing

IPD Sharing
Will not share