Study of ORL-1B in Patients With Biotinidase Deficiency

January 13, 2018 updated by: Orpha Labs

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

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

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

20

Phase

  • Phase 2
  • Phase 1

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 18 years (ADULT, CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
OTHER: Treatment with ORL-1B.
Pediatric patients with biotinidase deficiency.
Drug: ORL-1B
Oral ORL-1B

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Improvement in seizure frequency
Time Frame: 12 months
Statistically significant improvement in seizure frequency after the ORL-1B treatment.
12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Orpha Labs

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

August 31, 2013

Primary Completion (ACTUAL)

January 1, 2018

Study Completion (ACTUAL)

January 1, 2018

Study Registration Dates

First Submitted

August 28, 2017

First Submitted That Met QC Criteria

August 29, 2017

First Posted (ACTUAL)

August 31, 2017

Study Record Updates

Last Update Posted (ACTUAL)

January 17, 2018

Last Update Submitted That Met QC Criteria

January 13, 2018

Last Verified

January 1, 2018

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • Biot-1

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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