Biotinidase DeficiencyPhase 1/2NCT03269045
Study of ORL-1B in Patients With Biotinidase Deficiency
A Phase 1/2 study testing ORL-1B in people with Biotinidase Deficiency, run by Orpha Labs.
Trial summary
An Open-label, Single-arm, Phase 2 Study of Biotin in Patients With Biotinidase Deficiency. Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Biotinidase Deficiency
- What is being tested?
- ORL-1B
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- No sites listed yet
- Study dates
- 31 Aug 2013to 1 Jan 2018
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosis of biotinidase deficiency.
- Less than 18 years old.
Who cannot join
- Diagnosis of any other disease that is not a manifestation of biotinidase deficiency.
About this study
Official title: 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.
- Conditions
- Biotinidase Deficiency
- Being tested
- ORL-1B
- Medical subject headings
- Biotinidase Deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ORL-1BDrug
Oral ORL-1B
| Study group | Role | What they receive |
|---|---|---|
| Treatment with ORL-1B. | Other | Pediatric patients with biotinidase deficiency. Receives: Drug: ORL-1B |
What is being measured
The primary outcome is the main question the study is designed to answer.
Improvement in seizure frequency
Statistically significant improvement in seizure frequency after the ORL-1B treatment.
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Aug 2017
- Study start31 Aug 2013
- Primary completion1 Jan 2018
- Study completion1 Jan 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Biotinidase DeficiencyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Orpha Labs
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03269045
- Sponsor's protocol ID
- Biot-1
- Status last verified
- Jan 2018
- First submitted
- 28 Aug 2017
- Last updated
- 17 Jan 2018
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Jan 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Single-arm, Phase 2 Study of ORL-1B in Patients With Biotinidase Deficiency
- ClinicalTrials.gov ID
- NCT03269045
- Acronym / sponsor ID
- Biot-1
- Conditions
- Biotinidase Deficiency
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Orpha LabsIndustry (pharma, biotech, devices)
- Study start
- 31 Aug 2013 (actual)
- Primary completion
- 1 Jan 2018 (actual)
- Study completion
- 1 Jan 2018 (actual)
- First posted
- 31 Aug 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
An Open-label, Single-arm, Phase 2 Study of Biotin in Patients With Biotinidase Deficiency.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment with ORL-1B. | Other | Drug: ORL-1BPediatric patients with biotinidase deficiency. |
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- Diagnosis of biotinidase deficiency.
- Less than 18 years old.
Key exclusion criteria (1 listed)
- Diagnosis of any other disease that is not a manifestation of biotinidase deficiency.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ORL-1BOral ORL-1B | Drug | Treatment with ORL-1B. |
Primary outcome
Improvement in seizure frequency
Time frame: 12 months
Statistically significant improvement in seizure frequency after the ORL-1B treatment.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Jan 2018.
- Individual participant dataSharing planned: No
Related trials for Biotinidase Deficiency
Active, not recruitingObservational study
Early Check: Expanded Screening in Newborns
Spinal Muscular Atrophy, Fragile X Syndrome
RecruitingObservational study
Neonatal Screening of Biotinidase Deficiency: Genotype-phenotype Correlation and Clinical Follow-up
Biotinidase Deficiency
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing ORL-1B in people with Biotinidase Deficiency, run by Orpha Labs.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 17 Jan 2018. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Improvement in seizure frequency (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Jan 2018.