A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients

• ClinicalTrials.gov Identifier: NCT03811028
• Recruitment Status: Completed
• First Posted: January 22, 2019
• Results First Posted: February 25, 2022
• Last Update Posted: February 25, 2022
• Sponsor: Swedish Orphan Biovitrum
• Information provided by (Responsible Party): Swedish Orphan Biovitrum

Tracking Information

• First Submitted Date: December 17, 2018
• First Posted Date: January 22, 2019
• Results First Submitted Date: December 7, 2021
• Results First Posted Date: February 25, 2022
• Last Update Posted Date: February 25, 2022
• Actual Study Start Date: January 19, 2019
• Actual Primary Completion Date: April 30, 2021 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: December 7, 2021)

Safety as Measured by Adverse Events Frequencies (by Type and Severity) [ Time Frame: From infusion week 25 up to week 104 ]

Number of adverse events, by type and severity, from week 25 up to week 104

• Original Primary Outcome Measures (submitted: January 18, 2019): Safety will be measured by adverse events frequencies (by type and severity) [ Time Frame: Continuously from the informed consent is signed through study completion, summarized over 18 months. ]

Current Secondary Outcome Measures (submitted: February 2, 2022)

• The Observed SOBI003 Serum Concentration Immediately Before the Start of Infusion of SOBI003 [ Time Frame: Weeks 38, 52, 78 and 104 ]
  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 [ Time Frame: Weeks 38, 52, 78 and 104 ]
  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 [ Time Frame: Weeks 38, 52, 78 and 104 ]
  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 [ Time Frame: Weeks 38, 52, 78 and 104 ]
  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 [ Time Frame: Weeks 38, 52, 78 and 104 ]
  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 [ Time Frame: Weeks 38, 52, 78 and 104 ]
  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 [ Time Frame: Weeks 38, 52, 78 and 104 ]
  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 [ 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 ]
  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 [ Time Frame: Weeks 38, 52, 78 and 104 ]
  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 [ Time Frame: Weeks 52 and 104 ]
  Concentration of SOBI003 in cerebrospinal fluid
• Number of Patients Having Anti-drug Antibodies in Serum [ Time Frame: Weeks 38, 52, 78 and 104 ]
  Number of patients in each dose group having anti-drug antibodies in serum
• Number of Patients Having Anti-drug Antibodies in Cerebrospinal Fluid [ Time Frame: Weeks 52 and 104 ]
  Number of patients in each dose group having anti-drug antibodies cerebrospinal fluid
• Change From Baseline in Heparan Sulfate Concentration in Cerebrospinal Fluid [ Time Frame: Weeks 52 and 104 ]
  Change from baseline, in percent, of Heparan Sulfate levels in cerebrospinal fluid
• Change From Baseline in Heparan Sulfate Levels in Serum [ Time Frame: Weeks 38, 52, 78 and 104 ]
  Change from baseline in Heparan sulfate, in mg/L, levels in serum
• Change From Baseline in Heparan Sulfate Levels in Urine [ Time Frame: Weeks 38, 52, 78 and 104 ]
  Change from baseline in Heparan sulfate levels, in g/mol, in urine
• Change From Baseline in Neurocognitive Development Quotient [ Time Frame: Weeks 52 and 104 ]
  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 [ Time Frame: Week 52 and 104 ]
  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. [ Time Frame: Week 52 and 104 ]
  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. [ Time Frame: Week 52 and 104 ]
  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 [ Time Frame: Week 52 and 104 ]
  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 [ Time Frame: Week 52 and 104 ]
  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 [ Time Frame: Week 52 and 104 ]
  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 [ Time Frame: Week 52 and 104 ]
  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 [ Time Frame: Week 52 and 104 ]
  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 [ Time Frame: Week 52 and 104 ]
  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 [ Time Frame: Week 52 and 104 ]
  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.

Original Secondary Outcome Measures (submitted: January 18, 2019)

• The observed serum concentration immediately before the start of the next infusion of SOBI003 (CPre-infusion) [ Time Frame: Weeks 38, 52, 78 and 104 ]
• The observed serum concentration at the end of infusion of SOBI003 (CEnd of inf) [ Time Frame: Weeks 38, 52, 78 and 104 ]
  The study is an extension study, thus there might be serum concentrations from the previous study at the first assessment (Week 38) in the study
• The time of the end of the infusion of SOBI003 (tEnd of inf) [ Time Frame: Weeks 38, 52, 78 and 104 ]
• The maximum observed serum concentration (Cmax) [ Time Frame: Weeks 38, 52, 78 and 104 ]
• The time at which the maximum serum concentration is observed (tmax) [ Time Frame: Weeks 38, 52, 78 and 104 ]
• The minimum observed serum concentration (CTrough) [ Time Frame: Weeks 38, 52, 78 and 104 ]
• Clearance (CL) [ Time Frame: Weeks 38, 52, 78 and 104 ]
• The area under the plasma concentration-time curve from time 0 to last sample (AUC0-168h) [ Time Frame: Weeks 38, 52, 78 and 104 ]
• The half-life (t1/2) [ Time Frame: Weeks 38, 52, 78 and 104 ]
• SOBI003 concentration in cerebrospinal fluid [ Time Frame: Weeks 52 and 104 ]
• Proportion of patients having anti-drug antibodies in serum [ Time Frame: Weeks 38, 52, 78 and 104 ]
• Proportion of patients having anti-drug antibodies in cerebrospinal fluid [ Time Frame: Weeks 52 and 104 ]
• Change from baseline in Heparan Sulfate concentration in cerebrospinal fluid [ Time Frame: Weeks 52 and 104 ]
• Change from baseline in Heparan sulfate concentration in serum [ Time Frame: Weeks 38, 52, 78 and 104 ]
• Change from baseline in Heparan sulfate concentration levels in urine [ Time Frame: Weeks 38, 52, 78 and 104 ]
• Neurocognitive Development Quotient [ Time Frame: Weeks 52 and 104 ]
  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.
• Change from baseline in Neurocognitive Development Quotient [ Time Frame: Week 52 and 104 ]
  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.
• Age-equivalence score as assessed either by the BSID-III, cognitive subtest, or the KABC-II. [ Time Frame: Week 52 and 104 ]
  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.
• Change from baseline in Age-equivalence score as assessed either by the BSID-III, cognitive subtest, or the KABC-II. [ Time Frame: Week 52 and 104 ]
  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 [ Time Frame: Week 52 and 104 ]
  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 [ Time Frame: Week 52 and 104 ]
  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.
• Gray matter volume [ Time Frame: Week 52 and 104 ]
  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).
• Change from baseline in gray matter volume [ Time Frame: Week 52 and 104 ]
  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).
• Pediatric Quality of Life Inventory (PedsQL™) total score [ Time Frame: Week 52 and 104 ]
  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 [ Time Frame: Week 52 and 104 ]
  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. Lower scores indicate better functioning. Min score = 0, and max score = 144.
• PedsQL™ Family Impact Module total score [ Time Frame: Week 52 and 104 ]
  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.
• Change from baseline in PedsQL™ Family Impact Module total score [ Time Frame: Week 52 and 104 ]
  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.

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• 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
• 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.

• Study Type: Interventional
• Study Phase: Phase 1; Phase 2
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Sanfilippo Syndrome Type A (MPS IIIA)

Intervention

Drug: SOBI003

weekly i.v. infusion

Other Name: Modified recombinant human sulphamidase

Study Arms

Experimental: SOBI003

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: Drug: SOBI003

• Publications *: 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.

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: June 1, 2021): 6
• Original Estimated Enrollment (submitted: January 18, 2019): 9
• Actual Study Completion Date: May 7, 2021
• Actual Primary Completion Date: April 30, 2021 (Final data collection date for primary outcome measure)

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Months to 78 Months (Child)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Turkey, United States

Administrative Information

• NCT Number: NCT03811028
• Other Study ID Numbers: SOBI003-002
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: Swedish Orphan Biovitrum
• Original Responsible Party: Same as current
• Current Study Sponsor: Swedish Orphan Biovitrum
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Principal Investigator: Paul Harmatz, MD; Children´s Hospital and Research Center, Oakland

• PRS Account: Swedish Orphan Biovitrum
• Verification Date: December 2021