TerminatedNot Applicable

Natural History Study to Characterise the Course of Disease Progression in Participants With Mucopolysaccharidosis Type IIIB

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.

United States, Brazil, Italy30 participantsStarted 2014-09-14

Plain-language summary

The objectives of this study are to describe the clinical and biochemical characteristics and course of disease progression in participants with Mucopolysaccharidosis type IIIB (MPS IIIB)

Who can participate

Age range

1 Year

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. The participant has a definitive diagnosis of MPS IIIB, as determined by either of the following:
. Documented deficiency in alpha-N-acetyl-glucosaminidase (NAGLU) enzyme activity or
. Documented functionally-relevant mutations in both alleles of the NAGLU gene.
. The participant is at least 1 year of age (biological age).
. The participant or the participant's parent provides informed consent.
. The participant is willing and able to comply with protocol requirements to the extent that may be expected of a participant with cognitive impairment.
. The participant meets criterion a or criterion b below.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Collection And Analysis Of Clinical Characteristics Of MPS IIIB

Timeframe: Baseline to Week 43

Longitudinal Analysis Of The Course Of Disease Progression In Participants With MPS IIIB

Timeframe: Baseline to Week 43

Trial details

NCT IDNCT02293408

SponsorAlexion Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2017-07-11

View on ClinicalTrials.gov More MPS IIIB (Sanfilippo B Syndrome) trials

Who can participate

Age range

1 Year

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. The participant has a definitive diagnosis of MPS IIIB, as determined by either of the following:
. Documented deficiency in alpha-N-acetyl-glucosaminidase (NAGLU) enzyme activity or
. Documented functionally-relevant mutations in both alleles of the NAGLU gene.
. The participant is at least 1 year of age (biological age).
. The participant or the participant's parent provides informed consent.
. The participant is willing and able to comply with protocol requirements to the extent that may be expected of a participant with cognitive impairment.
. The participant meets criterion a or criterion b below.

Natural History Study to Characterise the Course of Disease Progression in Participants With Mucopolysaccharidosis Type IIIB

Plain-language summary

Who can participate

Inclusion criteria

Questions worth asking your doctor

Questions for the trial coordinator

What they're measuring

Trial details

Natural History Study to Characterise the Course of Disease Progression in Participants With Mucopolysaccharidosis Type IIIB

Plain-language summary

Who can participate

Inclusion criteria

Questions worth asking your doctor

Questions for the trial coordinator

What they're measuring

Trial details

Exclusion criteria