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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
Results First Submitted: January 14, 2016  
Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions: Lysosomal Acid Lipase Deficiency
Wolman Disease
Intervention: Drug: Sebelipase alfa (SBC-102)

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
A total of 12 Principal Investigators at 12 centers participated in this study, including 9 primary centers in the United Kingdom (UK), United States (US), France, Turkey, Saudia Arabia, Taiwan, Italy, and Egypt.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
To assess eligibility, subjects were screened for a period of up to 3 weeks prior to enrollment in the study. A total of 11 subjects were screened; 2 subjects died during screening. The other 9 subjects, all of whom were ≤ 8 months of age on the date of enrollment, met all eligibility criteria and were enrolled, treated and analysed.

Reporting Groups
  Description
Open-Label Sebelipase Alfa Subjects received IV infusions of sebelipase alfa at a starting dose of 0.35 mg/kg once weekly (qw). Dose was escalated to 1 mg/kg qw once acceptable safety and tolerability had been demonstrated during at least 2 infusions at the dose of 0.35 mg/kg. In the event of disease progression, based on protocol-defined criteria, subjects could be considered for further dose increase to 3 mg/kg qw. Dose escalation to 5 mg/kg qw in subjects who had evidence of continued disease progression was optional. 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.

Participant Flow for 2 periods

Period 1:   Primary Efficacy Analysis Period
    Open-Label Sebelipase Alfa
STARTED   9 
COMPLETED   6 
NOT COMPLETED   3 
Death                3 

Period 2:   Long-term Treatment Period
    Open-Label Sebelipase Alfa
STARTED   6 
COMPLETED   6 [1] 
NOT COMPLETED   0 
[1] This period is currently ongoing.



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Open-Label Sebelipase Alfa Subjects received IV infusions of sebelipase alfa at a starting dose of 0.35 mg/kg once weekly (qw). Dose was escalated to 1 mg/kg qw once acceptable safety and tolerability had been demonstrated during at least 2 infusions at the dose of 0.35 mg/kg. In the event of disease progression, based on protocol-defined criteria, subjects could be considered for further dose increase to 3 mg/kg qw. Dose escalation to 5 mg/kg qw in subjects who had evidence of continued disease progression was optional. 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.

Baseline Measures
   Open-Label Sebelipase Alfa 
Overall Participants Analyzed 
[Units: Participants]
 9 
Age 
[Units: Participants]
Count of Participants
 
<=18 years      9 100.0% 
Between 18 and 65 years      0   0.0% 
>=65 years      0   0.0% 
Age 
[Units: Months]
Median (Full Range)
 3 
 (1.1 to 5.8) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
 
Female      4  44.4% 
Male      5  55.6% 
Ethnicity (NIH/OMB) 
[Units: Participants]
Count of Participants
 
Hispanic or Latino      0   0.0% 
Not Hispanic or Latino      6  66.7% 
Unknown or Not Reported      3  33.3% 
Race (NIH/OMB) 
[Units: Participants]
Count of Participants
 
American Indian or Alaska Native      0   0.0% 
Asian      1  11.1% 
Native Hawaiian or Other Pacific Islander      0   0.0% 
Black or African American      1  11.1% 
White      4  44.4% 
More than one race      0   0.0% 
Unknown or Not Reported      3  33.3% 
Region of Enrollment 
[Units: Participants]
 
Saudi Arabia   0 
Turkey   1 
United States   1 
Ireland   1 
Taiwan   0 
Egypt   1 
United Kingdom   2 
Italy   0 
France   3 
Germany   0 
India   0 


  Outcome Measures
  Show All Outcome Measures

1.  Primary:   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) ]

2.  Secondary:   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) ]

3.  Secondary:   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) ]

4.  Secondary:   Median Age at Death   [ Time Frame: from week 0 to data cut-off (27 to 164 weeks of treatment) ]

5.  Secondary:   Effect on Growth Parameters (Weight-for-age)   [ Time Frame: from week 0 to data cut-off (27 to 164 weeks of treatment) ]

6.  Secondary:   Dichotomous Growth Status Indicators   [ Time Frame: Month 12 of treatment ]

7.  Secondary:   Changes in Serum Transaminases   [ Time Frame: from week 0 to weeks 1 and 4 ]

8.  Secondary:   Change in Serum Ferritin   [ Time Frame: from week 0 to week 1 ]

9.  Secondary:   Percentage of Subjects Achieving Transfusion-free Hemoglobin Normalization   [ Time Frame: from week 0 to data cut-off (27 to 164 weeks of treatment) ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Mark Friedman, Medical Director
Organization: Alexion Pharmaceuticals
phone: 781-357-9953
e-mail: mark.friedman@alxn.com


Publications:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01371825     History of Changes
Other Study ID Numbers: LAL-CL03
First Submitted: June 9, 2011
First Posted: June 13, 2011
Results First Submitted: January 14, 2016
Results First Posted: April 18, 2016
Last Update Posted: November 14, 2017