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Enzyme Replacement Therapy
A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients
Phase 1 & 2
Waitlist Available
Led By Paul Harmatz, MD
Research Sponsored by Swedish Orphan Biovitrum
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 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
Awards & highlights
No Placebo-Only Group
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).
Eligible Conditions
- Sanfilippo Syndrome
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 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
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~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
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Safety as Measured by Adverse Events Frequencies (by Type and Severity)
Secondary study objectives
Age-equivalence Score as Assessed Either by the BSID-III, Cognitive Subtest, or the KABC-II.
Age-equivalence Score as Assessed by VABS-II
Area Under the SOBI003 Serum Concentration-time Curve From Time 0 to168 Hours
+23 moreSide effects data
From 2019 Phase 1 & 2 trial • 6 Patients • NCT03423186100%
Upper respiratory tract infection
67%
Urticaria
33%
Conjunctivitis
33%
Gastroenteritis
33%
Viral infection
33%
Tachycardia
33%
Vascular access site occlusion
33%
Tremor
33%
Device damage
33%
Anxiety
33%
Cough
33%
Hyperaemia
33%
Iron deficiency anaemia
33%
Vomiting
33%
Medical device site haemorrhage
33%
Device malfunction
33%
Pyrexia
100%
80%
60%
40%
20%
0%
Study treatment Arm
Dose Group 1
Dose Group 2
Awards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Trial Design
1Treatment groups
Experimental Treatment
Group I: SOBI003Experimental Treatment1 Intervention
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.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
SOBI003
2019
Completed Phase 2
~20
Find a Location
Who is running the clinical trial?
Swedish Orphan BiovitrumLead Sponsor
100 Previous Clinical Trials
13,142 Total Patients Enrolled
Paul Harmatz, MDPrincipal InvestigatorChildren´s Hospital and Research Center, Oakland
5 Previous Clinical Trials
54 Total Patients Enrolled