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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02112994




Registration number
NCT02112994
Ethics application status
Date submitted
20/03/2014
Date registered
14/04/2014
Date last updated
4/12/2019

Titles & IDs
Public title
Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency
Scientific title
A Multi-Center, Open-Label Study of Sebelipase Alfa in Patients With Lysosomal Acid Lipase Deficiency
Secondary ID [1] 0 0
2011-004287-30
Secondary ID [2] 0 0
LAL-CL06
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Lysosomal Acid Lipase Deficiency 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Sebelipase Alfa

Experimental: Sebelipase Alfa - Pediatric and adult participants initiated IV treatment with sebelipase alfa at a dose of 1 mg/kg qow. Participants were considered for a dose adjustment at the discretion of the Investigator and in consultation with the Sponsor. Dose escalation to 3 mg/kg qow was considered if pre-defined dose-escalation criteria were met. If these criteria continued to be met, a subsequent dose escalation to 3 mg/kg every week (qw) was considered. Dose decreases as low as 0.35 mg/kg qow were permitted based upon evidence of intolerance to sebelipase alfa treatment. Participants who completed the 96-week treatment period were permitted to continue receiving sebelipase alfa in an expanded treatment period for up to 48 weeks, pending local drug availability and study participation status.


Treatment: Drugs: Sebelipase Alfa
IV infusion of sebelipase alfa
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Participants Experiencing Severe Treatment-emergent Adverse Events (TEAEs)
Timepoint [1] 0 0
Screening, Week 144
Secondary outcome [1] 0 0
Percent Change In Serum Lipids From Baseline To Week 144
Timepoint [1] 0 0
Baseline, Week 144
Secondary outcome [2] 0 0
Participants Testing Positive For Anti-drug Antibodies (ADAs)
Timepoint [2] 0 0
Week 144
Secondary outcome [3] 0 0
Percent Change In Body Mass Index (BMI)-For-Age Percentile From Baseline To Week 144 In Pediatric Participants
Timepoint [3] 0 0
Baseline, Week 144
Secondary outcome [4] 0 0
Shift In Child-Pugh Status From Baseline To Week 144
Timepoint [4] 0 0
Baseline, Week 144

Eligibility
Key inclusion criteria
Key

1. Participant was >8 months of age at the time of dosing.
2. Confirmation of LAL-D diagnosis as determined by the central laboratory or, for participants with prior hematopoietic stem cell transplant or liver transplant, historical enzyme activity or molecular genetic testing confirming a diagnosis of LAL-D.
3. Participants >8 months but <4 years of age at Screening had at least 1 of the following documented clinical manifestations of LAL-D:

* Dyslipidemia
* Elevated transaminases
* Impaired growth
* Suspected malabsorption
* Other clinical manifestation of LAL-D
4. Participants =4 years of age at Screening had at least 1 of the following documented clinical manifestations of LAL-D:

* Evidence of advanced liver disease
* Histologically confirmed disease recurrence in participants with past liver or hematopoietic transplant
* Persistent dyslipidemia
* Suspected malabsorption
* Other clinical manifestation of LAL-D

Key
Minimum age
8 Months
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Participant had known causes of active liver disease other than LAL-D, which had not been adequately treated.
2. Participant received a hematopoietic stem cell or liver transplant <2 years from the time of dosing.
3. Participant with co-morbidities other than complications due to LAL-D, which were irreversible or associated with a high mortality risk within 6 months or would interfere with study compliance or data interpretation.

Study design
Purpose of the study
Treatment
Allocation to intervention
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
24/06/2014
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
28/12/2017
Sample size
Target
Accrual to date
Final
31
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
- Westmead
Recruitment postcode(s) [1] 0 0
NSW 2145 - Westmead
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Illinois
Country [2] 0 0
United States of America
State/province [2] 0 0
Louisiana
Country [3] 0 0
United States of America
State/province [3] 0 0
Ohio
Country [4] 0 0
Belgium
State/province [4] 0 0
Brussels
Country [5] 0 0
Brazil
State/province [5] 0 0
Sao Paulo
Country [6] 0 0
Canada
State/province [6] 0 0
Nova Scotia
Country [7] 0 0
Croatia
State/province [7] 0 0
Zagreb
Country [8] 0 0
Denmark
State/province [8] 0 0
Copenhagen
Country [9] 0 0
Germany
State/province [9] 0 0
Freiburg
Country [10] 0 0
Italy
State/province [10] 0 0
Padova
Country [11] 0 0
Mexico
State/province [11] 0 0
Mexico City
Country [12] 0 0
Netherlands
State/province [12] 0 0
Amsterdam
Country [13] 0 0
Russian Federation
State/province [13] 0 0
Moscow
Country [14] 0 0
Spain
State/province [14] 0 0
A Coruna
Country [15] 0 0
Spain
State/province [15] 0 0
Barcelona
Country [16] 0 0
Spain
State/province [16] 0 0
Madrid
Country [17] 0 0
Turkey
State/province [17] 0 0
Balcali
Country [18] 0 0
United Kingdom
State/province [18] 0 0
Birmingham

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Alexion Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results are available at https://clinicaltrials.gov/study/NCT02112994