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CompletedNCT03269045Updated Jan 17, 2018

Study of ORL-1B in Patients With Biotinidase Deficiency

A Phase 1/2 interventional study of ORL-1B in Biotinidase Deficiency, sponsored by Orpha Labs. Completed. Open to participants aged Up to 18 Years. Per ClinicalTrials.gov, last updated 2018-01-17.

Sponsored by Orpha Labs · Phase 1/2, Interventional, and Treatment

From the registry’s dates

  • Registered 3 years 11 months after the study started (first participant enrolled Aug 2013, registered Aug 2017).
Phase
Phase 1/2
Study type
Interventional
Enrollment
20
Allocation
Not applicable
Ages
Up to 18 Years
Sex
All
01

Study summary

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

02

Conditions studied

  • Biotinidase Deficiency
03

In context

Lead sponsor

Orpha Labs is the lead sponsor of 4 studies on the registry; none are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
Up to 18 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

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

Exclusion criteria

Exclusion Criteria:

  • Diagnosis of any other disease that is not a manifestation of biotinidase deficiency.
05

Study design

Phase
Phase 1 / Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
20 participants (actual)

Study arms

  • Other
    Treatment with ORL-1B.

    Pediatric patients with biotinidase deficiency.

    Drug: ORL-1B

Interventions

  • DrugORL-1B

    Oral ORL-1B

06

What researchers measure

Primary outcomes

  1. Improvement in seizure frequency

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

    Time frame: 12 months

07

Study locations

No study locations are listed for this record.

08

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Ran from Aug 2013 to primary completion in Jan 2018, 52 months.

How these figures are worked out, and which studies are left out of them.

Registered
Aug 28, 2017
First posted
Aug 31, 2017
StartActual
Aug 31, 2013
Primary completionActual
Jan 1, 2018
CompletionActual
Jan 1, 2018
Last update
Jan 17, 2018
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Jan 17, 2018, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT03269045
Lead sponsor
Orpha Labs
Responsible party
Sponsor
First posted
Aug 31, 2017
Start date
Aug 31, 2013
Primary completion
Jan 1, 2018
Completion
Jan 1, 2018
Last update
Jan 17, 2018

Oversight

Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Jan 2018. You cannot join it, but the record below documents what was studied.

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Discussion

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