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An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase Deficiency

Expanded access is no longer available for this treatment.
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT02376751
First received: February 25, 2015
Last updated: June 6, 2016
Last verified: November 2015
  Purpose

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.


Condition Intervention
Lysosomal Acid Lipase Deficiency
Drug: sebelipase alfa

Study Type: Expanded Access     What is Expanded Access?
Official Title: AN EXPANDED ACCESS PROTOCOL FOR SEBELIPASE ALFA FOR PATIENTS WITH LYSOSOMAL ACID LIPASE DEFICIENCY

Resource links provided by NLM:


Further study details as provided by Alexion Pharmaceuticals:

Intervention Details:
    Drug: sebelipase alfa
    Other Name: SBC-102, recombinant human lysosomal acid lipase, rhLAL
  Eligibility

Ages Eligible for Study:   8 Months and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Criteria

Inclusion Criteria:

  1. Patient is ≥ 8 months of age at commencement of treatment with sebelipase alfa.
  2. Patient has a confirmed diagnosis of LAL Deficiency.
  3. 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.
  4. 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.
  5. Women of childbearing potential must have a negative serum pregnancy test at commencement of treatment with sebelipase alfa.

Exclusion Criteria:

  1. Women who are nursing or pregnant.
  2. 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.
  3. Patients who have received sebelipase alfa as part of a clinical trial that is currently active.
  4. Patients with known hypersensitivity to eggs.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

No Contacts or Locations Provided
  More Information

Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02376751     History of Changes
Other Study ID Numbers: LAL-EA01
Study First Received: February 25, 2015
Last Updated: June 6, 2016

Keywords provided by Alexion Pharmaceuticals:
Cholesteryl Ester Storage Disease
Acid lipase disease
Cholesterol ester hydrolase deficiency
LIPA Deficiency
Wolman disease
Lysosomal Storage Disease

Additional relevant MeSH terms:
Wolman Disease
Cholesterol Ester Storage Disease
Lipidoses
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Infant, Newborn, Diseases
Lipid Metabolism Disorders
Metabolic Diseases

ClinicalTrials.gov processed this record on April 27, 2017