Phase 2
N=6
A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients
Sanfilippo Syndrome Type A (MPS IIIA)
Bottom Line
View on ClinicalTrials.gov: NCT03811028 ↗Enrolled (actual)
6
Serious AEs
66.7%
Results posted
Feb 2022
Primary outcome: Primary: Safety as Measured by Adverse Events Frequencies (by Type and Severity) — 174; 355; 0; 1 events
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- SOBI003 (Drug)
- Age
- Pediatric · 0+ yrs
- Sex
- All
- Sponsor
- Swedish Orphan Biovitrum
- Primary completion
- Apr 2021
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Safety as Measured by Adverse Events Frequencies (by Type and Severity) |
174; 355; 0; 1; 174; 351 | — |
| SECONDARY The Observed SOBI003 Serum Concentration Immediately Before the Start of Infusion of SOBI003 |
120.0; 47.0; 75.0; 2610; 49; 30 | — |
| SECONDARY The Observed SOBI003 Serum Concentration at the End of Infusion of SOBI003 |
210000; 14370; 109500; 7900; 203000; 72000 | — |
| SECONDARY The Time of the End of the Infusion of SOBI003 |
6.20; 4.50; 4.170; 4.47; 4.5; 4.825 | — |
| SECONDARY The Maximum Observed Serum Concentration of SOBI003 |
105000; 203000; 136.0; 75.0; 79700; 7690 | — |
| SECONDARY The Time at Which the Maximum Serum Concentration of SOBI003 is Observed |
87.96; 4.50; 4.170; 168; 0; 4.47 | — |
| SECONDARY The Minimum Observed Serum Concentration of SOBI003 |
68.0; 47.0; 75.0; 2610; 34; 30 | — |
| SECONDARY Clearance |
208; 1.6; 0.661; 3.51; 2.21; 3.61 | — |
| SECONDARY Area Under the SOBI003 Serum Concentration-time Curve From Time 0 to168 Hours |
15400; 2040000; 2880000; 1110000; 4390000; 2770000 | — |
| SECONDARY The Half-life |
— | — |
| SECONDARY SOBI003 Concentration in Cerebrospinal Fluid |
15.7; 92.3; 118.35; 57.1 | — |
| SECONDARY Number of Patients Having Anti-drug Antibodies in Serum |
3; 3; 3; 3; 3; 2 | — |
| SECONDARY Number of Patients Having Anti-drug Antibodies in Cerebrospinal Fluid |
2; 3; 3; 3 | — |
| SECONDARY Change From Baseline in Heparan Sulfate Concentration in Cerebrospinal Fluid |
-1.19; -3.47; -6.07; -3.59 | — |
| SECONDARY Change From Baseline in Heparan Sulfate Levels in Serum |
-1.811; -1.88; -1.76; -2.25; -1.729; -2.01 | — |
| SECONDARY Change From Baseline in Heparan Sulfate Levels in Urine |
-447.2; -692.78; -464.4; -698.79; -512.19; -494.610 | — |
| SECONDARY Change From Baseline in Neurocognitive Development Quotient |
-8.97; -31.74; -16.28; -24.94 | — |
| SECONDARY Change From Baseline in Age-equivalence Score |
-1.0; -1.0; -3.0; 5.0 | — |
| SECONDARY Age-equivalence Score as Assessed Either by the BSID-III, Cognitive Subtest, or the KABC-II. |
14.0; 16.0; 13.0; 19.0 | — |
| SECONDARY Change From Baseline in Age-equivalence Score as Assessed Either by the BSID-III, Cognitive Subtest, or the KABC-II. |
-1.0; -1.0; -3.0; 5.0 | — |
| SECONDARY Age-equivalence Score as Assessed by VABS-II |
15; 28; 16; 23 | — |
| SECONDARY Change From Baseline in Age-equivalence Score as Assessed by VABS-II |
-1; 1; -6; 8 | — |
| SECONDARY Change From Baseline in Gray Matter Volume |
-24.629; 19.485; -53.584; 13.387 | — |
| SECONDARY Pediatric Quality of Life Inventory (PedsQL™) Total Score |
53.4; 72.2; 55.9; 76.5 | — |
| SECONDARY Change From Baseline in Pediatric Quality of Life Inventory (PedsQL™) Total Score |
-17.0; -3.7; -14.5; 0.6 | — |
| SECONDARY PedsQL™ Family Impact Module Total Score |
66.7; 70.0; 73.3; 70.0 | — |
| SECONDARY Change From Baseline in PedsQL™ Family Impact Module Total Score |
16.7; 0.0; 23.3; 0.0 | — |
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).
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
Data sourced from ClinicalTrials.gov (NCT03811028). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.