Mucopolysaccharidosis III, Type B (MPS IIIB)Phase 1/2CL01-TNCT02618512

A Open Label Study in Previously Studied, SBC-103 Treatment Naïve MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously

A Phase 1/2 study testing SBC-103 in people with Mucopolysaccharidosis III, Type B (MPS IIIB) and Sanfilippo B, run by Alexion Pharmaceuticals, Inc. at 1 site in United Kingdom.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial summary

Who is it for?: Ages 5 years and older
Conditions the study is for: Mucopolysaccharidosis III, Type B (MPS IIIB), Sanfilippo B
What is being tested?: SBC-103
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 3 participants
Where: 1 siteUnited Kingdom
Study dates: 15 Oct 2015to 18 Aug 2017
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 5 years and older
All sexes
No healthy volunteers
Child, Adult, Older adult

Participants from NGU-CL01 study were enrolled into the NGLU-CL01-T study. Subjects who met all of the inclusion criteria and none of the exclusion criteria were eligible to participate in this study.

Trial locations and contacts

1 site in 1 country.

United Kingdom1 site

Study site
Birmingham, B46NH

Other trials in United Kingdom

About this study

Official title: A Phase I/II Open Label Study in Previously Studied, SBC-103 Treatment Naïve MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously

This study evaluated the safety and tolerability of intravenous (IV) administration of SBC-103 in previously studied, SBC-103 treatment naïve patients with mucopolysaccharidosis III, type B (MPS IIIB, Sanfilippo B) who participated in the NGLU-CL01 study. The NGLU-CL01 study was a non-interventional study that evaluated structural brain abnormalities and blood brain barrier (BBB) integrity by magnetic resonance imaging (MRI) and cerebrospinal fluid/serum albumin index.

Conditions: Mucopolysaccharidosis III, Type B (MPS IIIB), Sanfilippo B
Being tested: SBC-103
Medical subject headings: Mucopolysaccharidosis III

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

SBC-103Drug
SBC-103 is a recombinant human alpha-N-acetylglucosaminidase (rhNAGLU). Patients were started with low dose of SBC-103 for 12 weeks and then will escalate to a higher dose.

Study groups in NCT02618512
Study group	Role	What they receive
SBC-103	Experimental	Patients were administered 1 mg/kg by IV infusion once every other week (qow) for at least 12 weeks. After evaluation of 12-week safety, tolerability, and pharmacodynamic data in individual patients, the dose was increased to 3 mg/kg qow. Infusions were to be at least 10 days apart and were administered every 14 days ±5 days. Receives: Drug: SBC-103

What is being measured

The primary outcome is the main question the study is designed to answer.

Safety and Tolerability of SBC-103
The planned primary endpoint of this study was safety and tolerability of SBC-103 in patients with MPS IIIB, as measured by Number of participants with treatment-emergent adverse events, including serious adverse events; infusion-associated reactions; incidence of antidrug antibodies, clinical laboratory tests, cerebrospinal fluid findings, vital signs, and prior and concomitant medications
Measured: Planned duration was baseline to 164 weeks but due to early termination of the study, actual is 96 weeks.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

15 Oct 201518 Aug 2017

First posted1 Dec 2015 (estimated)
Study start15 Oct 2015
Primary completion18 Aug 2017
Study completion18 Aug 2017

Why it stopped: The Sponsor's decision to terminate the SBC-103 program was reached after review of the data from all interventional clinical studies of SBC-103.

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.
Other trials for Mucopolysaccharidosis III, Type B (MPS IIIB)Studies of the same condition, open and under way.
More trials run by Alexion Pharmaceuticals, Inc.The 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: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 3 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02618512
Sponsor's protocol ID: NGLU-CL01-T
Other IDs: 2015-001983-20, 2015
Acronym: CL01-T
Status last verified: Mar 2018
First submitted: 21 Oct 2015
Last updated: 17 Apr 2018
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated device: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Active, not recruitingPhase 1/2
An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB
Mucopolysaccharidosis III-B
Not yet recruitingPhase 3
Intracerebroventricular Tralesinidase Alfa in Participants With MPS IIIB (Sanfilippo Syndrome Type B)
MPS IIIB
Enrolling by invitationObservational study
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2
Enrolling by invitationPhase 4
Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE
MPS III B

All Mucopolysaccharidosis III, Type B (MPS IIIB) trials

Study at a glance

From the registry record, last updated by the sponsor on 17 Apr 2018. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase I/II Open Label Study in Previously Studied, SBC-103 Treatment Naïve MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously
ClinicalTrials.gov ID: NCT02618512
Acronym / sponsor ID: CL01-T / NGLU-CL01-T
Conditions: Mucopolysaccharidosis III, Type B (MPS IIIB), Sanfilippo B
Phase: Phase 1/2
Study type: Interventional
Recruitment status: TerminatedReason stopped: The Sponsor's decision to terminate the SBC-103 program was reached after review of the data from all interventional clinical studies of SBC-103.
Enrolment: 3 participants (actual)
Lead sponsor: Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
Study start: 15 Oct 2015 (actual)
Primary completion: 18 Aug 2017 (actual)
Study completion: 18 Aug 2017 (actual)
First posted: 1 Dec 2015 (estimated)
Results: Posted 17 Apr 2018

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
SBC-103	Experimental	Drug: SBC-103Patients were administered 1 mg/kg by IV infusion once every other week (qow) for at least 12 weeks. After evaluation of 12-week safety, tolerability, and pharmacodynamic data in individual patients, the dose was increased to 3 mg/kg qow. Infusions were to be at least 10 days apart and were administered every 14 days ±5 days.

Who may qualify

Ages: 5 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

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
SBC-103SBC-103 is a recombinant human alpha-N-acetylglucosaminidase (rhNAGLU). Patients were started with low dose of SBC-103 for 12 weeks and then will escalate to a higher dose.	Drug	SBC-103

Primary outcome

Safety and Tolerability of SBC-103
Time frame: Planned duration was baseline to 164 weeks but due to early termination of the study, actual is 96 weeks.
The planned primary endpoint of this study was safety and tolerability of SBC-103 in patients with MPS IIIB, as measured by Number of participants with treatment-emergent adverse events, including serious adverse events; infusion-associated reactions; incidence of antidrug antibodies, clinical laboratory tests, cerebrospinal fluid findings, vital signs, and prior and concomitant medications

Results and safety

Results posted to the registry on 17 Apr 2018, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
SBC-103	3	3

Primary outcome: Safety and Tolerability of SBC-103

Time frame: Planned duration was baseline to 164 weeks but due to early termination of the study, actual is 96 weeks.. Count of participants. Unit: Participants

Measure	SBC-103 n = 3
Value	3

Adverse events

Time frame: Baseline to 96 Weeks.. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
SBC-103	2 of 3 (66.7%)	3 of 3 (100%)	0 of 3

Limitations and caveats (from the sponsor): Due to early termination of the study only safety data are reported.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 17 Apr 2018.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

Related trials for Mucopolysaccharidosis III, Type B (MPS IIIB)

Active, not recruitingPhase 1/2
An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB
Mucopolysaccharidosis III-B
Not yet recruitingPhase 3
Intracerebroventricular Tralesinidase Alfa in Participants With MPS IIIB (Sanfilippo Syndrome Type B)
MPS IIIB
Enrolling by invitationObservational study
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2
Enrolling by invitationPhase 4
Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE
MPS III B

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.

The record lists no contact. Use the official record to reach the sponsor.

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing SBC-103 in people with Mucopolysaccharidosis III, Type B (MPS IIIB) and Sanfilippo B, run by Alexion Pharmaceuticals, Inc. at 1 site in United Kingdom.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

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: stopped early, as of the sponsor's last update on 17 Apr 2018. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United Kingdom. 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 17 Apr 2018). 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 and Tolerability of SBC-103 (time frame: Planned duration was baseline to 164 weeks but due to early termination of the study, actual is 96 weeks.). 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 NCT02618512.

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 17 Apr 2018.