Sanfilippo Syndrome Type A (MPS IIIA)Phase 1/2NCT03423186
A Study to Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA Patients
A Phase 1/2 study testing SOBI003 in people with Sanfilippo Syndrome Type A (MPS IIIA), run by Swedish Orphan Biovitrum at 4 sites in 3 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 12 to 72 months
- Conditions the study is for
- Sanfilippo Syndrome Type A (MPS IIIA)
- What is being tested?
- SOBI003
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 4 sitesin 3 countries
- Study dates
- 19 Jun 2018to 25 Oct 2019
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 72 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Informed consent obtained from the patient's legally authorized representative(s)
- Patients with MPS IIIA, as confirmed by both:
- A documented deficiency in sulfamidase enzyme activity in concordance with a diagnosis of MPS IIIA, and
- Normal enzyme activity level of at least one other sulfatase measured in leukocytes
- Chronological age of ≥12 and ≤72 months (i.e., 1 to 6 years) at the time of the first SOBI003 infusion and a developmental age ≥12 months at screening as assessed by the Vineland Adaptive Behavior Scales, Second Edition (VABS-II)
- Medically stable patient who is expected to be able to comply with study procedures
Who cannot join
- At least one S298P mutation in the SGSH gene
- Contraindications for anesthetic procedures, surgical procedure (venous access port) MRI scans and/or lumbar punctures
- History of poorly controlled seizures
- Patients is currently receiving psychotropic or other medications which in the investigator's opinion, would be likely to substantially confound test results
- Significant non-MPS IIIA-related central nervous system (CNS) impairment or behavioral disturbances, which in the investigator's opinion, would confound the scientific integrity or interpretation of study assessments
- Prior administration of stem cell or gene therapy, or ERT for MPS IIIA
- Concurrent or prior (within 30 days of enrolment into this study) participation in a study involving invasive procedures
Trial locations and contacts
4 sites in 3 countries.
4 sites in 3 countries
United States2 sites
University of North Carolina Hospitals
Chapel Hill, North Carolina, 27599
Childrens's Hospital and Research Center
Oakland, California, 94609
About this study
Official title: An Open, Non-controlled, Parallel, Ascending Multiple-dose, Multicenter Study to Assess Safety and Tolerability, Pharmacokinetics and Pharmacodynamics of SOBI003 in Pediatric MPS IIIA Patients
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 aims of the present study is to assess the dose related safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of SOBI003, a chemically modified recombinant human (rh) Sulfamidase developed as an enzyme replacement therapy (ERT).
Read the detailed description
This is an open-label, non-controlled, parallel, sequential ascending multiple-dose, multicenter study to assess the dose related safety, tolerability, PK and PD of SOBI003 in pediatric MPS IIIA patients. Patients between 1 and 6 years of age who have not received previous treatment for MPS IIIA with an ERT, gene- or stem cell therapy will be eligible to participate in the study. The study is planned to consist of 3 dose cohorts, each comprising 3 patients. Treatment initiations will be staggered within each cohort in order to be able to observe, interpret and treat possible adverse reactions. SOBI003 is administered as weekly i.v. infusions over a period of 24 weeks. Upon completion of the 24-week treatment period with satisfactory tolerability, the patient is offered to receive continued SOBI003 treatment by participation in an extension study.
- Conditions
- Sanfilippo Syndrome Type A (MPS IIIA)
- Being tested
- SOBI003
- Medical subject headings
- Mucopolysaccharidosis III
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SOBI003Drug
Also called Modified recombinant human sulphamidase
Weekly i.v.infusion
| Study group | Role | What they receive |
|---|---|---|
| Dose group 1 | Experimental | SOBI003 dose 3 mg/kg once weekly for 24 weeks Receives: Drug: SOBI003 |
| Dose group 2 | Experimental | SOBI003 dose 10 mg/kg once weekly for 24 weeks Receives: Drug: SOBI003 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety as Measured by Adverse Events Frequencies (by Type and Severity)
Number of adverse events, by type and severity, from start of infusion up to 24 weeks
Measured: From start of first infusion up to Week 24
Secondary outcomes (21)
The Observed Serum Concentration Immediately Before the Start of Infusion of SOBI003
The observed serum concentration immediately before the start of infusion of SOBI003 (CPre-dose).
Measured: Weeks 1, 2, 3, 4, 8, 12, and 24
The Observed Serum Concentration at the End of Infusion of SOBI003
The observed serum concentration at the end of infusion of SOBI003 (CEnd of inf)
Measured: Weeks 1, 2, 3, 4, 8, 12, and 24
The Time of the End of the Infusion of SOBI003
The time of the end of infusion of SOBI003 (tEnd of inf)
Measured: Weeks 1, 2, 3, 4, 8, 12, and 24
The Maximum Observed Serum Concentration of SOBI003
The Maximum Observed Serum Concentration of SOBI003 (Cmax)
Measured: 0, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96 hours post-dose on Weeks 1, 4, 12, and 24
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)
Measured: Weeks 1, 4, 12, and 24
The Minimum Observed Serum Concentration of SOBI003
The minimum observed serum concentration of SOBI003 (CTrough)
Measured: Weeks 1, 4, 12, and 24
Clearance
Clearance (CL) of SOBI003
Measured: Weeks 1, 4, 12, and 24
Area Under the Serum Concentration-time Curve From Time 0 to 168 Hours
Area under the serum concentration-time curve from time 0 to 168 hours (AUC 0-168h)
Measured: 0,1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 120, 144, 168 hours post-dose on Weeks 1, 4,12, and 24
The Half-life
The half-life of SOBI003 in serum (T1/2)
Measured: Weeks 1, 4, 12, and 24
SOBI003 Concentration in Cerebrospinal Fluid
SOBI003 concentration in cerebrospinal fluid
Measured: Weeks 12 and 24
Number of Patients Having Anti-drug Antibodies in Serum
Number of patients in each dose group having anti-drug antibodies in serum
Measured: Weeks 2,4,8,12 and 24
Patients Having Anti-drug Antibodies in Cerebrospinal Fluid
Percent of patients having anti-drug antibodies in cerebrospinal fluid
Measured: Weeks 12 and 24
Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid
Change from baseline, in percent, of Heparan Sulfate levels in cerebrospinal fluid
Measured: Baseline, weeks 12, and 24
Change From Baseline in Heparan Sulfate Levels in Serum
Change from baseline in Heparan sulfate levels in serum
Measured: Weeks 2, 3, 4, 8, 12 and 24
Change From Baseline in Heparan Sulfate Levels in Urine
Change from baseline in Heparan sulfate levels in urine
Measured: Weeks 2, 3, 4, 8, 12 and 24
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.
Measured: Week 24
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.
Measured: Week 24
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.
Measured: Week 24
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).
Measured: Week 24
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. Lower scores indicate better functioning. Min score = 0, and max score = 144.
Measured: Week 24
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 measure includes a scale, from where the categorical score "4", "3", "2", "1", and "0" was reversed and linearly transformed to a 0-100 scale to 4=0, 3=25, 2=50, 1=75 and 0=100, where 100 = minimum and 0 = maximum. 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.
Measured: Week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Feb 2018
- Study start19 Jun 2018
- Primary completion25 Oct 2019
- Study completion25 Oct 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Sanfilippo Syndrome Type A (MPS IIIA)Studies of the same condition, open and under way.
- More trials run by Swedish Orphan BiovitrumThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Model description
- The study was designed to include three arms, up to 20 mg/kg. After a company decision to end the development of the compound it was decided to not start a third cohort, but if stated safe the dose could increase up to 20 mg/kg in cohort 1 and 2.
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Swedish Orphan Biovitrum
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Paul Harmatz, MD, Principal investigator, Childrens's Hospital and Research Center Oakland
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03423186
- Sponsor's protocol ID
- SOBI003-001
- Status last verified
- Nov 2021
- First submitted
- 29 Jan 2018
- Last updated
- 19 Nov 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: Undecided
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.
PubMed 35835061
Study at a glance
From the registry record, last updated by the sponsor on 19 Nov 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open, Non-controlled, Parallel, Ascending Multiple-dose, Multicenter Study to Assess Safety and Tolerability, Pharmacokinetics and Pharmacodynamics of SOBI003 in Pediatric MPS IIIA Patients
- ClinicalTrials.gov ID
- NCT03423186
- Acronym / sponsor ID
- SOBI003-001
- Conditions
- Sanfilippo Syndrome Type A (MPS IIIA)
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Swedish Orphan BiovitrumIndustry (pharma, biotech, devices)
- Study start
- 19 Jun 2018 (actual)
- Primary completion
- 25 Oct 2019 (actual)
- Study completion
- 25 Oct 2019 (actual)
- First posted
- 6 Feb 2018 (actual)
- Results
- Posted 22 Apr 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 aims of the present study is to assess the dose related safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of SOBI003, a chemically modified recombinant human (rh) Sulfamidase developed as an enzyme replacement therapy (ERT).
Detailed description
This is an open-label, non-controlled, parallel, sequential ascending multiple-dose, multicenter study to assess the dose related safety, tolerability, PK and PD of SOBI003 in pediatric MPS IIIA patients. Patients between 1 and 6 years of age who have not received previous treatment for MPS IIIA with an ERT, gene- or stem cell therapy will be eligible to participate in the study. The study is planned to consist of 3 dose cohorts, each comprising 3 patients. Treatment initiations will be staggered within each cohort in order to be able to observe, interpret and treat possible adverse reactions. SOBI003 is administered as weekly i.v. infusions over a period of 24 weeks. Upon completion of the 24-week treatment period with satisfactory tolerability, the patient is offered to receive continued SOBI003 treatment by participation in an extension study.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- The study was designed to include three arms, up to 20 mg/kg. After a company decision to end the development of the compound it was decided to not start a third cohort, but if stated safe the dose could increase up to 20 mg/kg in cohort 1 and 2.
| Arm | Type | Receives |
|---|---|---|
| Dose group 1 | Experimental | Drug: SOBI003SOBI003 dose 3 mg/kg once weekly for 24 weeks |
| Dose group 2 | Experimental | Drug: SOBI003SOBI003 dose 10 mg/kg once weekly for 24 weeks |
Who may qualify
- Ages
- 12 to 72 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Informed consent obtained from the patient's legally authorized representative(s)
- Patients with MPS IIIA, as confirmed by both:
- Chronological age of ≥12 and ≤72 months (i.e., 1 to 6 years) at the time of the first SOBI003 infusion and a developmental age ≥12 months at screening as assessed by the Vineland Adaptive Behavior Scales, Second Edition (VABS-II)
- Medically stable patient who is expected to be able to comply with study procedures
Key exclusion criteria (7 listed)
- At least one S298P mutation in the SGSH gene
- Contraindications for anesthetic procedures, surgical procedure (venous access port) MRI scans and/or lumbar punctures
- History of poorly controlled seizures
- Patients is currently receiving psychotropic or other medications which in the investigator's opinion, would be likely to substantially confound test results
- Significant non-MPS IIIA-related central nervous system (CNS) impairment or behavioral disturbances, which in the investigator's opinion, would confound the scientific integrity or interpretation of study assessments
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SOBI003Also: Modified recombinant human sulphamidaseWeekly i.v.infusion | Drug | Dose group 1, Dose group 2 |
Primary outcome
Safety as Measured by Adverse Events Frequencies (by Type and Severity)
Time frame: From start of first infusion up to Week 24
Number of adverse events, by type and severity, from start of infusion up to 24 weeks
21 secondary outcomes
The Observed Serum Concentration Immediately Before the Start of Infusion of SOBI003
Time frame: Weeks 1, 2, 3, 4, 8, 12, and 24
The Observed Serum Concentration at the End of Infusion of SOBI003
Time frame: Weeks 1, 2, 3, 4, 8, 12, and 24
The Time of the End of the Infusion of SOBI003
Time frame: Weeks 1, 2, 3, 4, 8, 12, and 24
The Maximum Observed Serum Concentration of SOBI003
Time frame: 0, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96 hours post-dose on Weeks 1, 4, 12, and 24
The Time at Which the Maximum Serum Concentration of SOBI003 is Observed
Time frame: Weeks 1, 4, 12, and 24
The Minimum Observed Serum Concentration of SOBI003
Time frame: Weeks 1, 4, 12, and 24
Clearance
Time frame: Weeks 1, 4, 12, and 24
Area Under the Serum Concentration-time Curve From Time 0 to 168 Hours
Time frame: 0,1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 120, 144, 168 hours post-dose on Weeks 1, 4,12, and 24
The Half-life
Time frame: Weeks 1, 4, 12, and 24
SOBI003 Concentration in Cerebrospinal Fluid
Time frame: Weeks 12 and 24
Number of Patients Having Anti-drug Antibodies in Serum
Time frame: Weeks 2,4,8,12 and 24
Patients Having Anti-drug Antibodies in Cerebrospinal Fluid
Time frame: Weeks 12 and 24
Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid
Time frame: Baseline, weeks 12, and 24
Change From Baseline in Heparan Sulfate Levels in Serum
Time frame: Weeks 2, 3, 4, 8, 12 and 24
Change From Baseline in Heparan Sulfate Levels in Urine
Time frame: Weeks 2, 3, 4, 8, 12 and 24
Change From Baseline in Neurocognitive Development Quotient
Time frame: Week 24
Change From Baseline in Age-equivalence Score
Time frame: Week 24
Change From Baseline in Age-equivalence Score as Assessed by VABS-II
Time frame: Week 24
Change From Baseline in Gray Matter Volume
Time frame: Week 24
Change From Baseline in Pediatric Quality of Life Inventory (PedsQL™) Total Score
Time frame: Week 24
Change From Baseline in PedsQL™ Family Impact Module Total Score
Time frame: Week 24
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Nov 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 35835061Harmatz 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.
- Individual participant dataSharing planned: No. Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Paul Harmatz, MD principal investigator
Childrens's Hospital and Research Center Oakland
4 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing SOBI003 in people with Sanfilippo Syndrome Type A (MPS IIIA), run by Swedish Orphan Biovitrum at 4 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 Nov 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States, Germany, Turkey (Türkiye). Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 22 Apr 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety as Measured by Adverse Events Frequencies (by Type and Severity) (time frame: From start of first infusion up to Week 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03423186, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Nov 2021.