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SurVival of Lysosomal Acid Lipase Deficiency (LAL-D) Infants Treated With SebelipAse aLfa (VITAL)

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01371825
First Posted: June 13, 2011
Last Update Posted: November 14, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Alexion Pharmaceuticals
June 9, 2011
June 13, 2011
January 14, 2016
April 18, 2016
November 14, 2017
May 2011
August 2014   (Final data collection date for primary outcome measure)
Percentage of Subjects in the PES Surviving to 12 Months of Age [ Time Frame: From week 0 to data cut-off (27 to 164 weeks of treatment) ]
The primary efficacy endpoint was the percentage of subjects (%) in the Primary Efficacy Analysis Set (PES) who survived to at least 12 months of age.
To evaluate treatment emergent Adverse Events (AEs). [ Time Frame: 135 Days ]
The safety and tolerability of weekly infusions of SBC-102 will be assessed by routine monitoring of patients for adverse events (AEs) and monitoring changes from baseline in physical examination findings, vital signs, clinical laboratory evaluations, immunogenicity tests and concomitant therapies.
Complete list of historical versions of study NCT01371825 on ClinicalTrials.gov Archive Site
  • Percentage of Subjects in the PES Surviving at 18 Months of Age [ Time Frame: from week 0 to data cut-off (27 to 164 weeks of treatment) ]
    The percentage of subjects in the Primary Efficacy Analysis Set (PES) who survived to at least 18 months of age.
  • Percentage of Subjects in the PES Surviving at 24 Months of Age [ Time Frame: from week 0 to data cut-off (27 to 164 weeks of treatment) ]
    The percentage of subjects in the Primary Efficacy Analysis Set (PES) who survived to at least 24 months of age.
  • Median Age at Death [ Time Frame: from week 0 to data cut-off (27 to 164 weeks of treatment) ]
  • Effect on Growth Parameters (Weight-for-age) [ Time Frame: from week 0 to data cut-off (27 to 164 weeks of treatment) ]
    Changes from baseline in percentiles for weight-for-age (WFA)
  • Dichotomous Growth Status Indicators [ Time Frame: Month 12 of treatment ]
    The percentages of subjects meeting criteria for each dichotomous indicator of under nutrition, i.e., underweight (at least 2 SD below median for weight-for-age [WFA]), wasting (at least 2 SD below median for weight-for-length or -height [WFL/WFH]), and stunting (at least 2 SD below median for length- or height-for-age [LFA/HFA])
  • Changes in Serum Transaminases [ Time Frame: from week 0 to weeks 1 and 4 ]
    Change from baseline for alanine aminotransferase (ALT) and aspartate aminotransferase (AST)
  • Change in Serum Ferritin [ Time Frame: from week 0 to week 1 ]
    Change from baseline in serum ferritin
  • Percentage of Subjects Achieving Transfusion-free Hemoglobin Normalization [ Time Frame: from week 0 to data cut-off (27 to 164 weeks of treatment) ]
    The percentage of subjects achieving transfusion-free hemoglobin normalization (TFHN) of ≥ 4 weeks at any time during the study (also referred to as short-term TFHN), and the percentage of subjects who maintained TFHN for ≥ 13 weeks beginning at Week 6 (also referred to as sustained early TFHN). A subject was considered to have achieved short-term TFHN if the/she had two post-baseline measurements of hemoglobin, obtained at least 4 weeks apart, that were above the age-adjusted lower limit of normal (LLN), and had no additional hemoglobin measurements below LLN during this minimum 4-week period and no transfusions administered during the minimum 4-week period or for 2 weeks prior to the start of this period.
To determine the effect of SBC-102 on growth. [ Time Frame: 135 Days ]
Changes in weight and length over the course of the trial will be evaluated and compared to standard growth curves.
Not Provided
Not Provided
 
SurVival of Lysosomal Acid Lipase Deficiency (LAL-D) Infants Treated With SebelipAse aLfa
An Open Label, Multicenter, Dose Escalation Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of SBC-102 (Sebelipase Alfa) in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
This is an open-label, repeat-dose, intra-subject dose escalation study of SBC-102 (USAN: sebelipase alfa) in children with growth failure due to LAL Deficiency. Eligible subjects will receive once-weekly (qw) infusions of sebelipase alfa for up to 5 years

Lysosomal Acid Lipase (LAL) Deficiency is a rare autosomal recessive lipid storage disorder that is caused by a marked decrease or almost complete absence of LAL, leading to the accumulation of lipids, predominately cholesteryl esters and triglycerides, in various tissues and cell types. In the liver, accumulation of lipids leads to hepatomegaly, liver dysfunction, and hepatic failure. Although a single disease, LAL Deficiency presents as a clinical continuum with two major phenotypes, Cholesteryl Ester Storage Disease (CESD) and Wolman Disease.

Early Onset LAL Deficiency (Wolman Disease) is extremely rare, with an estimated incidence of less than 2 lives per million. It is characterized by profound malabsorption, growth failure, and hepatic failure, and is usually fatal in the first year of life. There is currently no approved therapy for the treatment of LAL Deficiency.

Interventional
Phase 2
Phase 3
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
  • Lysosomal Acid Lipase Deficiency
  • Wolman Disease
Drug: Sebelipase alfa (SBC-102)
Sebelipase alfa is a recombinant human lysosomal acid lipase (rhLAL). The investigational medicinal product is an enzyme replacement therapy intended for treatment of patients with LAL Deficiency. Dosing will occur once weekly for up to three years.
Experimental: Open-Label sebelipase alfa
All subjects received IV infusions of sebelipase alfa during the open-label treatment period. Subjects received a starting dose of 0.35 mg/kg once weekly (qw) and, after demonstrating acceptable safety and tolerability after at least 2 infusions at this dose, began receiving the per-protocol dose of 1 mg/kg qw. Thereafter, subjects were to continue receiving a dose of 1 mg/kg qw for the duration of the treatment period. However, in the event of disease progression (based on protocol-defined criteria) at any time during treatment with 1 mg/kg qw, an individual subject could receive a dose increase to 3 mg/kg qw and; if necessary, a subsequent dose increase to 5 mg/kg qw (after review and approval by a Safety Committee). Subjects receiving long-term treatment on a stable qw dose could be switched to an every other week (qow) dosing schedule at the same total dose (mg/kg) per infusion.
Intervention: Drug: Sebelipase alfa (SBC-102)

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
9
April 2018
August 2014   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • Subject's parent or legal guardian provides written informed consent/permission prior to any study procedures.
  • Male or female child with documented decreased LAL activity relative to the normal range of the lab performing the assay or documented result of molecular genetic testing (2 mutations) confirming a diagnosis.
  • Growth failure with onset before 6 months of age.

Exclusion Criteria:

  • Clinically important concurrent disease.
  • Has received an investigational product other than sebelipase alfa within 14 days prior to the first dose.
  • Subject is older than 24 months of age.
  • Myeloablative preparation, or other systemic pre-transplant conditioning, for hematopoietic stem cell or liver transplant.
  • Previous hematopoietic stem cell or liver transplant.
  • Known hypersensitivity to eggs.
Sexes Eligible for Study: All
up to 24 Months   (Child)
No
Contact information is only displayed when the study is recruiting subjects
Egypt,   France,   Ireland,   Saudi Arabia,   United Kingdom,   United States
Germany,   India,   Italy,   Taiwan,   Turkey
 
NCT01371825
LAL-CL03
Yes
Not Provided
Plan to Share IPD: Undecided
Alexion Pharmaceuticals
Alexion Pharmaceuticals
Not Provided
Not Provided
Alexion Pharmaceuticals
October 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP