Back to resultsA Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients
Primary Purpose
Sanfilippo Syndrome Type A (MPS IIIA)
Status
Completed
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
SOBI003
Sponsored by
About this trial
This is an interventional treatment trial for Sanfilippo Syndrome Type A (MPS IIIA)
Eligibility Criteria
Inclusion Criteria:
- Completion of study SOBI003-001
- Informed consent obtained from the patient´s legally authorized representative
Exclusion Criteria:
- If, in the opinion of the investigator, there are patient specific safety concerns that contraindicates further treatment with SOBI003
Sites / Locations
- Children´s Hospital and research center
- University of North Carolina hospitals
- Gazi University Hospital
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
SOBI003
Arm Description
SOBI003 solution, 20 mg/mL, is mixed with NaCl 0.9% infusion solution prior to administration. For a bodyweight < 25 kg, the total infusion volume is 100 mL. For a bodyweight ≥ 25 kg, the total infusion volume is 250 mL. SOBI003 is administered as i.v. infusions given once weekly for a duration of 80 weeks (from Week 25 until Week 104 following the first 24 weeks of SOBI003 administration in the FIH study (SOBI003-001) study. The SOBI003 dose will be adjusted to the highest dose that has been declared safe by the safety review committee on the FIH study.Hence, dose adjustments may occur a couple of times on the extension study until the final decided dose has been determined.
Outcomes
Primary Outcome Measures
Safety as Measured by Adverse Events Frequencies (by Type and Severity)
Number of adverse events, by type and severity, from week 25 up to week 104
Secondary Outcome Measures
The Observed SOBI003 Serum Concentration Immediately Before the Start of Infusion of SOBI003
The observed SOBI003 serum concentration immediately before the start of infusion of SOBI003 (CPre-dose) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture.
The Observed SOBI003 Serum Concentration at the End of Infusion of SOBI003
The observed SOBI003 serum concentration at the end of infusion of SOBI003 (CEnd of inf) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture.
The Time of the End of the Infusion of SOBI003
The time of the end of infusion of SOBI003 (tEnd of inf) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture.
The Maximum Observed Serum Concentration of SOBI003
The maximum observed serum concentration of SOBI003 (Cmax) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture.
The Time at Which the Maximum Serum Concentration of SOBI003 is Observed
The time after start of infusion at which the maximum serum concentration is observed (tmax) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture.
The Minimum Observed Serum Concentration of SOBI003
The minimum observed serum concentration of SOBI003 (CTrough) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture.
Clearance
Clearance (CL) of SOBI003 Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture.
Area Under the SOBI003 Serum Concentration-time Curve From Time 0 to168 Hours
Area under the SOBI003 serum concentration-time curve from time 0 to 168 hours (AUC 0-168h) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture.
The Half-life
The half-life of SOBI003 in serum (T1/2) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture.
SOBI003 Concentration in Cerebrospinal Fluid
Concentration of SOBI003 in cerebrospinal fluid
Number of Patients Having Anti-drug Antibodies in Serum
Number of patients in each dose group having anti-drug antibodies in serum
Number of Patients Having Anti-drug Antibodies in Cerebrospinal Fluid
Number of patients in each dose group having anti-drug antibodies cerebrospinal fluid
Change From Baseline in Heparan Sulfate Concentration in Cerebrospinal Fluid
Change from baseline, in percent, of Heparan Sulfate levels in cerebrospinal fluid
Change From Baseline in Heparan Sulfate Levels in Serum
Change from baseline in Heparan sulfate, in mg/L, levels in serum
Change From Baseline in Heparan Sulfate Levels in Urine
Change from baseline in Heparan sulfate levels, in g/mol, in urine
Change From Baseline in Neurocognitive Development Quotient
Quotient between age equivalent score and age, 0 - 100%, where high values are desirable. The age equivalent score represent the age of the typical and normal individual who would achieve the same result as the one who was tested The age equivalent scores are assessed by the Bayley Scales of Infant and Toddler Development®, third edition cognitive subtest or the Kaufman Assessment Battery for Children, Second edition.
The Bayley Scales of Infant and Toddler Development-Third Edition is an individually administered test designed to assess developmental functioning of infants and toddlers. The Bayley-III assesses development in five areas: cognitive, language, motor,social-emotional, and adaptive behavior.
The Kaufman Assessment Battery for Children (K-ABC) is a clinical instrument for assessing cognitive development.
These results are truly "unitless"/"dimensionless" because they represents quotients of values from the same scale, which means that the units in the denominator and
Change From Baseline in Age-equivalence Score
The age equivalent score represent the age in months of the typical and normal individual who would achieve the same result as the one who was tested. The age equivalent scores are assessed by the Bayley Scales of Infant and Toddler Development®, third edition cognitive subtest or the Kaufman Assessment Battery for Children, Second edition. The Bayley Scales of Infant and Toddler Development-Third Edition is an individually administered test designed to assess developmental functioning of infants and toddlers. The Bayley-III assesses development in five areas: cognitive, language, motor, social-emotional, and adaptive behavior. The Kaufman Assessment Battery for Children (K-ABC) is a clinical instrument for assessing cognitive development.
Age-equivalence Score as Assessed Either by the BSID-III, Cognitive Subtest, or the KABC-II.
The age equivalent score represent the age in months of the typical and normal individual who would achieve the same result as the one who was tested. The age equivalent scores are assessed by Vineland™ Adaptive Behavior Scales, Expanded Interview Form, Second edition (VABS-II). The Vineland is designed to measure adaptive behavior of individuals from birth to age 90. The Vineland-II contains 5 domains each with 2-3 subdomains. The main domains are: Communication, Daily Living Skills, Socialization, Motor Skills, and Maladaptive Behavior.
Change From Baseline in Age-equivalence Score as Assessed Either by the BSID-III, Cognitive Subtest, or the KABC-II.
The age equivalent score represents the age in months of the typical and normal individual who would achieve the same result as the one who was tested.
The age equivalent scores are assessed either by the Bayley Scales of Infant and Toddler Development®, third edition, (BSID-III) cognitive subtest or the Kaufman Assessment Battery for Children, Second edition (KABC-II) depending on chronological age of the subject. Quotient between age equivalent score and age, 0 - 100%, where high values are desirable.
The BSID-III is an individually administered test designed to assess developmental functioning of infants and toddlers. The BSID-III assesses development in five areas: cognitive, language, motor, social-emotional, and adaptive behavior.
The KABC-II is a clinical instrument for assessing cognitive development. The unit and measurement is the same in both scales (BSID-III and KABC-II): Age-equivalent score.
Age-equivalence Score as Assessed by VABS-II
The age equivalent score represent the age in months of the typical and normal individual who would achieve the same result as the one who was tested.
The age equivalent scores are assessed by Vineland™ Adaptive Behavior Scales, Expanded Interview Form, Second edition (VABS-II). The Vineland is designed to measure adaptive behavior of individuals from birth to age 90.
The Vineland-II contains 5 domains each with 2-3 subdomains. The main domains are: Communication, Daily Living Skills, Socialization, Motor Skills, and Maladaptive Behavior.
Change From Baseline in Age-equivalence Score as Assessed by VABS-II
The age equivalent score represent the age in months of the typical and normal individual who would achieve the same result as the one who was tested.
The age equivalent scores are assessed by Vineland™ Adaptive Behavior Scales, Expanded Interview Form, Second edition (VABS-II). The Vineland is designed to measure adaptive behavior of individuals from birth to age 90.
The Vineland-II contains 5 domains each with 2-3 subdomains. The main domains are: Communication, Daily Living Skills, Socialization, Motor Skills, and Maladaptive Behavior.
Change From Baseline in Gray Matter Volume
Grey matter contains most of the brain's neuronal cell bodies. The grey matter includes regions of the brain involved in muscle control, and sensory perception such as seeing and hearing, memory, emotions, speech, decision making, and self-control. The gray matter volume will be measured by volumetric magnetic resonance imaging (MRI) at weeks 52 and 104.
Pediatric Quality of Life Inventory (PedsQL™) Total Score
Pediatric Quality of Life Inventory (PedsQL™) is a modular approach to measuring health-related quality of life in healthy children and adolescents and those with acute and chronic health conditions. Lower scores indicate better functioning. Min score = 0, and max score = 144.
Change From Baseline in Pediatric Quality of Life Inventory (PedsQL™) Total Score
Pediatric Quality of Life Inventory (PedsQL™) is a modular approach to measuring health-related quality of life (HRQOL) in healthy children and adolescents and those with acute and chronic health conditions. Higher scores indicate better functioning. Min score = 0, and max score = 144.
PedsQL™ Family Impact Module Total Score
Pediatric Quality of Life Inventory (PedsQL™) is a modular approach to measuring health-related quality of life in healthy children and adolescents and those with acute and chronic health conditions. The Total Score is the sum of all 36 items in the test divided by the number of items answered. Higher scores indicate better functioning. Min score = 0, and max score = 144.
Change From Baseline in PedsQL™ Family Impact Module Total Score
Pediatric Quality of Life Inventory (PedsQL™) is a modular approach to measuring health-related quality of life in healthy children and adolescents and those with acute and chronic health conditions. The Total Score is the sum of all 36 items in the test divided by the number of items answered. Higher scores indicate better functioning.
Full Information
NCT ID
NCT03811028
First Posted
December 17, 2018
Last Updated
February 2, 2022
Sponsor
Swedish Orphan Biovitrum
1. Study Identification
Unique Protocol Identification Number
NCT03811028
Brief Title
A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients
Official Title
An Open, Single-arm, Multicenter Extension Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients
Study Type
Interventional
2. Study Status
Record Verification Date
December 2021
Overall Recruitment Status
Completed
Study Start Date
January 19, 2019 (Actual)
Primary Completion Date
April 30, 2021 (Actual)
Study Completion Date
May 7, 2021 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Swedish Orphan Biovitrum
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
MPS IIIA, also known as Sanfilippo A, is an inherited lysosomal storage disease (LSD). MPS IIIA is caused by a deficiency in sulfamidase, one of the enzymes involved in the lysosomal degradation of the glycosaminoglycan (GAG) heparan sulfate (HS). The natural course of MPS IIIA is characterized by devastating neurodegeneration with initially mild somatic involvement. The aim of the present study is to assess the safety, tolerability and efficacy of long-term SOBI003 treatment. SOBI003 is a chemically modified recombinant human (rh) Sulfamidase developed as an enzyme replacement therapy (ERT).
Detailed Description
This is an open, single-arm, multicenter extension study to assess the safety, tolerability and efficacy of long-term SOBI003 treatment in pediatric MPS IIIA patients. The study is an extension of the First in Human (FIH) SOBI003-001 study, allowing continuous treatment of SOBI003 for up to 2 years. Study patients who complete Week 24 of the FIH study (SOBI003-001) will be invited to continue to Week 25 in the extension study.
When entering the extension study, these patients will receive the highest dose that has been declared safe in the ongoing FIH study (SOBI003-001). Upon completion of the FIH study, an analysis aimed at selecting the dose for forthcoming studies will take place. Once the dose has been selected, this dose will be applied to all patients enrolled in the extension study. The total duration of the extension study for an individual patient is 80 weeks, resulting in a total of 104 weeks (2 years) of SOBI003 treatment.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Sanfilippo Syndrome Type A (MPS IIIA)
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
6 (Actual)
8. Arms, Groups, and Interventions
Arm Title
SOBI003
Arm Type
Experimental
Arm Description
SOBI003 solution, 20 mg/mL, is mixed with NaCl 0.9% infusion solution prior to administration. For a bodyweight < 25 kg, the total infusion volume is 100 mL. For a bodyweight ≥ 25 kg, the total infusion volume is 250 mL.
SOBI003 is administered as i.v. infusions given once weekly for a duration of 80 weeks (from Week 25 until Week 104 following the first 24 weeks of SOBI003 administration in the FIH study (SOBI003-001) study. The SOBI003 dose will be adjusted to the highest dose that has been declared safe by the safety review committee on the FIH study.Hence, dose adjustments may occur a couple of times on the extension study until the final decided dose has been determined.
Intervention Type
Drug
Intervention Name(s)
SOBI003
Other Intervention Name(s)
Modified recombinant human sulphamidase
Intervention Description
weekly i.v. infusion
Primary Outcome Measure Information:
Title
Safety as Measured by Adverse Events Frequencies (by Type and Severity)
Description
Number of adverse events, by type and severity, from week 25 up to week 104
Time Frame
From infusion week 25 up to week 104
Secondary Outcome Measure Information:
Title
The Observed SOBI003 Serum Concentration Immediately Before the Start of Infusion of SOBI003
Description
The observed SOBI003 serum concentration immediately before the start of infusion of SOBI003 (CPre-dose) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture.
Time Frame
Weeks 38, 52, 78 and 104
Title
The Observed SOBI003 Serum Concentration at the End of Infusion of SOBI003
Description
The observed SOBI003 serum concentration at the end of infusion of SOBI003 (CEnd of inf) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture.
Time Frame
Weeks 38, 52, 78 and 104
Title
The Time of the End of the Infusion of SOBI003
Description
The time of the end of infusion of SOBI003 (tEnd of inf) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture.
Time Frame
Weeks 38, 52, 78 and 104
Title
The Maximum Observed Serum Concentration of SOBI003
Description
The maximum observed serum concentration of SOBI003 (Cmax) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture.
Time Frame
Weeks 38, 52, 78 and 104
Title
The Time at Which the Maximum Serum Concentration of SOBI003 is Observed
Description
The time after start of infusion at which the maximum serum concentration is observed (tmax) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture.
Time Frame
Weeks 38, 52, 78 and 104
Title
The Minimum Observed Serum Concentration of SOBI003
Description
The minimum observed serum concentration of SOBI003 (CTrough) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture.
Time Frame
Weeks 38, 52, 78 and 104
Title
Clearance
Description
Clearance (CL) of SOBI003 Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture.
Time Frame
Weeks 38, 52, 78 and 104
Title
Area Under the SOBI003 Serum Concentration-time Curve From Time 0 to168 Hours
Description
Area under the SOBI003 serum concentration-time curve from time 0 to 168 hours (AUC 0-168h) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture.
Time Frame
0,1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 120, 144, 168 hours post-dose on Weeks 38, 52, 78 and 104
Title
The Half-life
Description
The half-life of SOBI003 in serum (T1/2) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture.
Time Frame
Weeks 38, 52, 78 and 104
Title
SOBI003 Concentration in Cerebrospinal Fluid
Description
Concentration of SOBI003 in cerebrospinal fluid
Time Frame
Weeks 52 and 104
Title
Number of Patients Having Anti-drug Antibodies in Serum
Description
Number of patients in each dose group having anti-drug antibodies in serum
Time Frame
Weeks 38, 52, 78 and 104
Title
Number of Patients Having Anti-drug Antibodies in Cerebrospinal Fluid
Description
Number of patients in each dose group having anti-drug antibodies cerebrospinal fluid
Time Frame
Weeks 52 and 104
Title
Change From Baseline in Heparan Sulfate Concentration in Cerebrospinal Fluid
Description
Change from baseline, in percent, of Heparan Sulfate levels in cerebrospinal fluid
Time Frame
Weeks 52 and 104
Title
Change From Baseline in Heparan Sulfate Levels in Serum
Description
Change from baseline in Heparan sulfate, in mg/L, levels in serum
Time Frame
Weeks 38, 52, 78 and 104
Title
Change From Baseline in Heparan Sulfate Levels in Urine
Description
Change from baseline in Heparan sulfate levels, in g/mol, in urine
Time Frame
Weeks 38, 52, 78 and 104
Title
Change From Baseline in Neurocognitive Development Quotient
Description
Quotient between age equivalent score and age, 0 - 100%, where high values are desirable. The age equivalent score represent the age of the typical and normal individual who would achieve the same result as the one who was tested The age equivalent scores are assessed by the Bayley Scales of Infant and Toddler Development®, third edition cognitive subtest or the Kaufman Assessment Battery for Children, Second edition.
The Bayley Scales of Infant and Toddler Development-Third Edition is an individually administered test designed to assess developmental functioning of infants and toddlers. The Bayley-III assesses development in five areas: cognitive, language, motor,social-emotional, and adaptive behavior.
The Kaufman Assessment Battery for Children (K-ABC) is a clinical instrument for assessing cognitive development.
These results are truly "unitless"/"dimensionless" because they represents quotients of values from the same scale, which means that the units in the denominator and
Time Frame
Weeks 52 and 104
Title
Change From Baseline in Age-equivalence Score
Description
The age equivalent score represent the age in months of the typical and normal individual who would achieve the same result as the one who was tested. The age equivalent scores are assessed by the Bayley Scales of Infant and Toddler Development®, third edition cognitive subtest or the Kaufman Assessment Battery for Children, Second edition. The Bayley Scales of Infant and Toddler Development-Third Edition is an individually administered test designed to assess developmental functioning of infants and toddlers. The Bayley-III assesses development in five areas: cognitive, language, motor, social-emotional, and adaptive behavior. The Kaufman Assessment Battery for Children (K-ABC) is a clinical instrument for assessing cognitive development.
Time Frame
Week 52 and 104
Title
Age-equivalence Score as Assessed Either by the BSID-III, Cognitive Subtest, or the KABC-II.
Description
The age equivalent score represent the age in months of the typical and normal individual who would achieve the same result as the one who was tested. The age equivalent scores are assessed by Vineland™ Adaptive Behavior Scales, Expanded Interview Form, Second edition (VABS-II). The Vineland is designed to measure adaptive behavior of individuals from birth to age 90. The Vineland-II contains 5 domains each with 2-3 subdomains. The main domains are: Communication, Daily Living Skills, Socialization, Motor Skills, and Maladaptive Behavior.
Time Frame
Week 52 and 104
Title
Change From Baseline in Age-equivalence Score as Assessed Either by the BSID-III, Cognitive Subtest, or the KABC-II.
Description
The age equivalent score represents the age in months of the typical and normal individual who would achieve the same result as the one who was tested.
The age equivalent scores are assessed either by the Bayley Scales of Infant and Toddler Development®, third edition, (BSID-III) cognitive subtest or the Kaufman Assessment Battery for Children, Second edition (KABC-II) depending on chronological age of the subject. Quotient between age equivalent score and age, 0 - 100%, where high values are desirable.
The BSID-III is an individually administered test designed to assess developmental functioning of infants and toddlers. The BSID-III assesses development in five areas: cognitive, language, motor, social-emotional, and adaptive behavior.
The KABC-II is a clinical instrument for assessing cognitive development. The unit and measurement is the same in both scales (BSID-III and KABC-II): Age-equivalent score.
Time Frame
Week 52 and 104
Title
Age-equivalence Score as Assessed by VABS-II
Description
The age equivalent score represent the age in months of the typical and normal individual who would achieve the same result as the one who was tested.
The age equivalent scores are assessed by Vineland™ Adaptive Behavior Scales, Expanded Interview Form, Second edition (VABS-II). The Vineland is designed to measure adaptive behavior of individuals from birth to age 90.
The Vineland-II contains 5 domains each with 2-3 subdomains. The main domains are: Communication, Daily Living Skills, Socialization, Motor Skills, and Maladaptive Behavior.
Time Frame
Week 52 and 104
Title
Change From Baseline in Age-equivalence Score as Assessed by VABS-II
Description
The age equivalent score represent the age in months of the typical and normal individual who would achieve the same result as the one who was tested.
The age equivalent scores are assessed by Vineland™ Adaptive Behavior Scales, Expanded Interview Form, Second edition (VABS-II). The Vineland is designed to measure adaptive behavior of individuals from birth to age 90.
The Vineland-II contains 5 domains each with 2-3 subdomains. The main domains are: Communication, Daily Living Skills, Socialization, Motor Skills, and Maladaptive Behavior.
Time Frame
Week 52 and 104
Title
Change From Baseline in Gray Matter Volume
Description
Grey matter contains most of the brain's neuronal cell bodies. The grey matter includes regions of the brain involved in muscle control, and sensory perception such as seeing and hearing, memory, emotions, speech, decision making, and self-control. The gray matter volume will be measured by volumetric magnetic resonance imaging (MRI) at weeks 52 and 104.
Time Frame
Week 52 and 104
Title
Pediatric Quality of Life Inventory (PedsQL™) Total Score
Description
Pediatric Quality of Life Inventory (PedsQL™) is a modular approach to measuring health-related quality of life in healthy children and adolescents and those with acute and chronic health conditions. Lower scores indicate better functioning. Min score = 0, and max score = 144.
Time Frame
Week 52 and 104
Title
Change From Baseline in Pediatric Quality of Life Inventory (PedsQL™) Total Score
Description
Pediatric Quality of Life Inventory (PedsQL™) is a modular approach to measuring health-related quality of life (HRQOL) in healthy children and adolescents and those with acute and chronic health conditions. Higher scores indicate better functioning. Min score = 0, and max score = 144.
Time Frame
Week 52 and 104
Title
PedsQL™ Family Impact Module Total Score
Description
Pediatric Quality of Life Inventory (PedsQL™) is a modular approach to measuring health-related quality of life in healthy children and adolescents and those with acute and chronic health conditions. The Total Score is the sum of all 36 items in the test divided by the number of items answered. Higher scores indicate better functioning. Min score = 0, and max score = 144.
Time Frame
Week 52 and 104
Title
Change From Baseline in PedsQL™ Family Impact Module Total Score
Description
Pediatric Quality of Life Inventory (PedsQL™) is a modular approach to measuring health-related quality of life in healthy children and adolescents and those with acute and chronic health conditions. The Total Score is the sum of all 36 items in the test divided by the number of items answered. Higher scores indicate better functioning.
Time Frame
Week 52 and 104
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Months
Maximum Age & Unit of Time
78 Months
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Completion of study SOBI003-001
Informed consent obtained from the patient´s legally authorized representative
Exclusion Criteria:
If, in the opinion of the investigator, there are patient specific safety concerns that contraindicates further treatment with SOBI003
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Paul Harmatz, MD
Organizational Affiliation
Children´s Hospital and Research Center, Oakland
Official's Role
Principal Investigator
Facility Information:
Facility Name
Children´s Hospital and research center
City
Oakland
State/Province
California
ZIP/Postal Code
94609
Country
United States
Facility Name
University of North Carolina hospitals
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27599
Country
United States
Facility Name
Gazi University Hospital
City
Ankara
Country
Turkey
12. IPD Sharing Statement
Plan to Share IPD
No
Citations:
PubMed Identifier
35835061
Citation
Harmatz P, Muenzer J, Ezgu F, Dalen P, Huledal G, Lindqvist D, Gelius SS, Wiken M, Onnestam K, Broijersen A. Chemically modified recombinant human sulfamidase (SOBI003) in mucopolysaccharidosis IIIA patients: Results from an open, non-controlled, multicenter study. Mol Genet Metab. 2022 Aug;136(4):249-259. doi: 10.1016/j.ymgme.2022.06.008. Epub 2022 Jun 28.
Results Reference
derived
Learn more about this trial
A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients
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