Lysosomal Acid Lipase DeficiencyNCT02376751
An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase Deficiency
A study testing sebelipase alfa in people with Lysosomal Acid Lipase Deficiency, run by Alexion Pharmaceuticals, Inc..
Trial summary
This is an open-label, multicenter expanded access protocol to allow patients with a confirmed diagnosis of Lysosomal Acid Lipase (LAL) Deficiency in the United States (US), access to sebelipase alfa (recombinant lysosomal acid lipase [rhLAL]) until commercial product is available. Read more about this study
- Who is it for?
- Ages 8 months and older
- Conditions the study is for
- Lysosomal Acid Lipase Deficiency
- What is being tested?
- sebelipase alfa
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- 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 8 months and older
- All sexes
- Child, Adult, Older adult
Who may be able to join
- Patient is ≥ 8 months of age at commencement of treatment with sebelipase alfa.
- Patient has a confirmed diagnosis of LAL Deficiency.
- Patient or patient's parent or legal guardian (if applicable) consents to participation in the study. If the patient is of minor age, he/she is willing to provide assent where required per local regulations, and if deemed able to do so.
- Male and female patients of childbearing potential must use a highly reliable method of birth control (expected failure rate less than 5% per year) from the time they commence treatment through 4 weeks after the last dose of sebelipase alfa.
- Women of childbearing potential must have a negative serum pregnancy test at commencement of treatment with sebelipase alfa.
Who cannot join
- Women who are nursing or pregnant.
- Patients who received an investigational product within 30 days (for a small molecule) or 60 days (for a biologic) of commencing treatment, and which in the opinion of the investigator or Sponsor, may negatively impact patient safety.
- Patients who have received sebelipase alfa as part of a clinical trial that is currently active.
- Patients with known hypersensitivity to eggs.
About this study
Official title: AN EXPANDED ACCESS PROTOCOL FOR SEBELIPASE ALFA FOR PATIENTS WITH LYSOSOMAL ACID LIPASE DEFICIENCY
This is an open-label, multicenter expanded access protocol to allow patients with a confirmed diagnosis of Lysosomal Acid Lipase (LAL) Deficiency in the United States (US), access to sebelipase alfa (recombinant lysosomal acid lipase [rhLAL]) until commercial product is available.
Patients enrolled in the expanded access protocol will receive 1 mg/kg intravenous infusions of sebelipase alfa every other week.
- Conditions
- Lysosomal Acid Lipase Deficiency
- Being tested
- sebelipase alfa
- Keywords
- Cholesteryl Ester Storage Disease, Acid lipase disease, Cholesterol ester hydrolase deficiency, LIPA Deficiency, Wolman disease, Lysosomal Storage Disease
- Medical subject headings
- Wolman Disease, Cholesterol Ester Storage Disease, Lysosomal Storage Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
sebelipase alfaDrug
Also called SBC-102, recombinant human lysosomal acid lipase, rhLAL
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Mar 2015 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Lysosomal Acid Lipase DeficiencyStudies of the same condition, open and under way.
- More trials run by Alexion Pharmaceuticals, Inc.The sponsor's other registered studies.
- 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
- Expanded access
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02376751
- Sponsor's protocol ID
- LAL-EA01
- Status last verified
- Nov 2015
- First submitted
- 25 Feb 2015
- Last updated
- 8 Jun 2016
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Jun 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- AN EXPANDED ACCESS PROTOCOL FOR SEBELIPASE ALFA FOR PATIENTS WITH LYSOSOMAL ACID LIPASE DEFICIENCY
- ClinicalTrials.gov ID
- NCT02376751
- Acronym / sponsor ID
- LAL-EA01
- Conditions
- Lysosomal Acid Lipase Deficiency
- Keywords
- Cholesteryl Ester Storage Disease, Acid lipase disease, Cholesterol ester hydrolase deficiency, LIPA Deficiency, Wolman disease, Lysosomal Storage Disease
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- First posted
- 3 Mar 2015 (estimated)
- 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.
This is an open-label, multicenter expanded access protocol to allow patients with a confirmed diagnosis of Lysosomal Acid Lipase (LAL) Deficiency in the United States (US), access to sebelipase alfa (recombinant lysosomal acid lipase [rhLAL]) until commercial product is available.
Patients enrolled in the expanded access protocol will receive 1 mg/kg intravenous infusions of sebelipase alfa every other week.
Study design
Who may qualify
- Ages
- 8 months and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Patient is ≥ 8 months of age at commencement of treatment with sebelipase alfa.
- Patient has a confirmed diagnosis of LAL Deficiency.
- Patient or patient's parent or legal guardian (if applicable) consents to participation in the study. If the patient is of minor age, he/she is willing to provide assent where required per local regulations, and if deemed able to do so.
- Male and female patients of childbearing potential must use a highly reliable method of birth control (expected failure rate less than 5% per year) from the time they commence treatment through 4 weeks after the last dose of sebelipase alfa.
- Women of childbearing potential must have a negative serum pregnancy test at commencement of treatment with sebelipase alfa.
Key exclusion criteria (4 listed)
- Women who are nursing or pregnant.
- Patients who received an investigational product within 30 days (for a small molecule) or 60 days (for a biologic) of commencing treatment, and which in the opinion of the investigator or Sponsor, may negatively impact patient safety.
- Patients who have received sebelipase alfa as part of a clinical trial that is currently active.
- Patients with known hypersensitivity to eggs.
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 |
|---|---|---|
| sebelipase alfaAlso: SBC-102, recombinant human lysosomal acid lipase, rhLAL | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jun 2016.
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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 study testing sebelipase alfa in people with Lysosomal Acid Lipase Deficiency, run by Alexion Pharmaceuticals, Inc..
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 5 inclusion and 4 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: status not confirmed, as of the sponsor's last update on 8 Jun 2016. 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.
Are results available for this study?
Results are not available in the source record.
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.
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 8 Jun 2016.