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Study of ORL-1B in Patients With Biotinidase Deficiency

NCT ID: NCT03269045

Last Updated: 2018-01-17

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

20 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-08-31

Study Completion Date

2018-01-01

Brief Summary

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

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Detailed Description

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Conditions

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Biotinidase Deficiency

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Treatment with ORL-1B.

Pediatric patients with biotinidase deficiency.

Group Type OTHER

ORL-1B

Intervention Type DRUG

Oral ORL-1B

Interventions

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ORL-1B

Oral ORL-1B

Intervention Type DRUG

Eligibility Criteria

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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.
Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Orpha Labs

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Other Identifiers

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Biot-1

Identifier Type: -

Identifier Source: org_study_id

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