MPS IIIB (Sanfilippo B Syndrome)NCT02293408
Natural History Study to Characterise the Course of Disease Progression in Participants With Mucopolysaccharidosis Type IIIB
An observational study of MPS IIIB (Sanfilippo B Syndrome), run by Alexion Pharmaceuticals, Inc. at 7 sites in 6 countries.
Trial 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) Read more about this study
- Who is it for?
- Ages 1 year and older
- Conditions the study is for
- MPS IIIB (Sanfilippo B Syndrome)
- How many people
- 30 participants
- Where
- 7 sitesin 6 countries
- Study dates
- 14 Sep 2014to 11 Jul 2017
- Phase
- Observational
- Are there results?
- None posted in the registry
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 1 year and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: 27 participants with MPS IIIB were enrolled and analyzed in Component 1. Component 1 (27 participants): The participant has a definitive diagnosis of MPS IIIB. Component 2 (15 participants from Component 1): The participant is considered to be at risk of rapid disease progression.
Who may be able to join
- A participant must meet all of the following inclusion criteria to be eligible for this study:
- 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.
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- The participant is willing and able to comply with protocol requirements to the extent that may be expected of a participant with cognitive impairment.
- In addition to the eligibility criteria above, a participant must meet all of the following criteria for Component 2:
- The participant meets criterion a or criterion b below.
- The participant is considered to be at risk of rapid disease progression based on at least 1 of the following criteria:
- The participant has documented mutations of the NAGLU gene that are reported to be linked to rapid disease progression (for example, disease onset before 6 years of age), or
- The participant has a sibling, or other first- or second-degree relative with rapidly progressing MPS IIIB (for example, disease onset before 6 years of age).
- The participant had disease onset prior to 6 years of age (biological age), as defined by:
- Cognitive delay evaluated by Bayley Scales of Infant Development, Third Edition (BSID-III) or Kaufman Assessment Battery for Children, Second Edition (KABC-II), or
- Language delay, plateauing, or regression of language skills as determined by the Investigator (for example, participant uses isolated words, associated words such as 2-word combinations, sentences, poor or reduced language, and/or difficult to understand).
- The participant has an age equivalent of ≥1 year on the Vineland Adaptive Behavior Scales, Second Edition (Vineland II).
Who cannot join
- A participant who meets any of the following exclusion criteria will be ineligible for this study:
- The participant has visual or hearing impairments sufficient to preclude cooperation with neurodevelopmental testing.
- The participant has a history of poorly-controlled seizure disorder.
- The participant is currently receiving medication, which, in the Investigator's opinion, would be likely to substantially confound interpretation of the results (for example, the participant has been on the current dose of psychotropic medication for less than 3 months).
- The participant is receiving a newly increased dose of melatonin (for example, less than 3 months on current dose).
- The participant has previously received an investigational therapy for MPS IIIB (with the exception of high dose Genistein >150 milligram/kilogram (mg/kg)/day, which will require a minimum of 3 months wash-out before entering the study) or has had hematopoietic stem cell transplant (HSCT).
- The participant has any other prior or ongoing medical condition that may present a safety risk, interfere with study compliance, or confound data interpretation.
Trial locations and contacts
7 sites in 6 countries.
7 sites in 6 countries
United States2 sites
Study site
Minneapolis, Minnesota
Study site
Pittsburgh, Pennsylvania
About this study
Official title: A Prospective Cross-Sectional and Longitudinal Study With Additional Retrospective Chart Review to Evaluate Clinical and Biochemical Characteristics and Disease Progression in Patients With Mucopolysaccharidosis Type IIIB
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)
- Conditions
- MPS IIIB (Sanfilippo B Syndrome)
- Keywords
- MPS IIIB, Mucopolysaccharidosis, Mucopolysaccharidosis type IIIB, Sanfilippo Syndrome Type B
- Medical subject headings
- Mucopolysaccharidosis III, Mucopolysaccharidoses
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Component 1 | Component 1 involved an evaluation of the clinical characteristics of MPS IIIB in participants based on a retrospective chart review to collect information on demographics, clinical history, diagnostic tests, treatments, clinical chemistry and hematology test results, physical examination findings, anthropometric data, radiology results, and supportive interventions performed over a period of up to 6 weeks. | |
| Component 2 | Component 2 involved a longitudinal evaluation of the course of disease progression in a subset of participants considered to be at risk of rapid disease progression, who, after completing Component 1, were to be prospectively followed for a period of at least 1 year (Longitudinal Follow-Up) and up to 3 years total (Extended Follow-Up). |
What is being measured
The primary outcome is the main question the study is designed to answer.
Collection And Analysis Of Clinical Characteristics Of MPS IIIB
Component 1 involved an evaluation of the clinical characteristics of MPS IIIB in participants based on a retrospective chart review to collect information on demographics, clinical history, diagnostic tests, treatments, clinical chemistry and hematology test results, physical examination findings, anthropometric data, radiology results, and supportive interventions performed over a period of up to 6 weeks.
Measured: Baseline to Week 43
Longitudinal Analysis Of The Course Of Disease Progression In Participants With MPS IIIB
Component 2 involved a longitudinal evaluation of the course of disease progression in a subset of participants considered to be at risk of rapid disease progression, who, after completing Component 1, were to be prospectively followed for a period of at least 1 year (Longitudinal Follow-Up) and up to 3 years total (Extended Follow-Up).
Measured: Baseline to Week 43
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Nov 2014 (estimated)
- Study start14 Sep 2014
- Primary completion11 Jul 2017
- Study completion11 Jul 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.
- Other trials for MPS IIIB (Sanfilippo B Syndrome)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
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02293408
- Sponsor's protocol ID
- NGLU-NH02
- Status last verified
- Sep 2018
- First submitted
- 3 Nov 2014
- Last updated
- 13 Sep 2018
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Sep 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective Cross-Sectional and Longitudinal Study With Additional Retrospective Chart Review to Evaluate Clinical and Biochemical Characteristics and Disease Progression in Patients With Mucopolysaccharidosis Type IIIB
- ClinicalTrials.gov ID
- NCT02293408
- Acronym / sponsor ID
- NGLU-NH02
- Conditions
- MPS IIIB (Sanfilippo B Syndrome)
- Keywords
- MPS IIIB, Mucopolysaccharidosis, Mucopolysaccharidosis type IIIB, Sanfilippo Syndrome Type B
- Study type
- Observational
- 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
- 30 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 14 Sep 2014 (actual)
- Primary completion
- 11 Jul 2017 (actual)
- Study completion
- 11 Jul 2017 (actual)
- First posted
- 18 Nov 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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)
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Component 1 | – | –Component 1 involved an evaluation of the clinical characteristics of MPS IIIB in participants based on a retrospective chart review to collect information on demographics, clinical history, diagnostic tests, treatments, clinical chemistry and hematology test results, physical examination findings, anthropometric data, radiology results, and supportive interventions performed over a period of up to 6 weeks. |
| Component 2 | – | –Component 2 involved a longitudinal evaluation of the course of disease progression in a subset of participants considered to be at risk of rapid disease progression, who, after completing Component 1, were to be prospectively followed for a period of at least 1 year (Longitudinal Follow-Up) and up to 3 years total (Extended Follow-Up). |
Who may qualify
- Ages
- 1 year and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- 27 participants with MPS IIIB were enrolled and analyzed in Component 1. Component 1 (27 participants): The participant has a definitive diagnosis of MPS IIIB. Component 2 (15 participants from Component 1): The participant is considered to be at risk of rapid disease progression.
Key inclusion criteria (16 listed)
- A participant must meet all of the following inclusion criteria to be eligible for this study:
- The participant has a definitive diagnosis of MPS IIIB, as determined by either of the following:
- 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.
Key exclusion criteria (7 listed)
- A participant who meets any of the following exclusion criteria will be ineligible for this study:
- The participant has visual or hearing impairments sufficient to preclude cooperation with neurodevelopmental testing.
- The participant has a history of poorly-controlled seizure disorder.
- The participant is currently receiving medication, which, in the Investigator's opinion, would be likely to substantially confound interpretation of the results (for example, the participant has been on the current dose of psychotropic medication for less than 3 months).
- The participant is receiving a newly increased dose of melatonin (for example, less than 3 months on current dose).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Collection And Analysis Of Clinical Characteristics Of MPS IIIB
Time frame: Baseline to Week 43
Component 1 involved an evaluation of the clinical characteristics of MPS IIIB in participants based on a retrospective chart review to collect information on demographics, clinical history, diagnostic tests, treatments, clinical chemistry and hematology test results, physical examination findings, anthropometric data, radiology results, and supportive interventions performed over a period of up to 6 weeks.
Longitudinal Analysis Of The Course Of Disease Progression In Participants With MPS IIIB
Time frame: Baseline to Week 43
Component 2 involved a longitudinal evaluation of the course of disease progression in a subset of participants considered to be at risk of rapid disease progression, who, after completing Component 1, were to be prospectively followed for a period of at least 1 year (Longitudinal Follow-Up) and up to 3 years total (Extended Follow-Up).
Results and safety
The study was stopped early (reason given: 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.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Sep 2018.
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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.
The record lists no contact. Use the official record to reach the sponsor.
7 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of MPS IIIB (Sanfilippo B Syndrome), run by Alexion Pharmaceuticals, Inc. at 7 sites in 6 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 16 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: stopped early, as of the sponsor's last update on 13 Sep 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United States, Brazil, Italy, Portugal, Spain and 1 more country. 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.
Are results available for this study?
The study was stopped early (reason given: 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.). Results are not available in the source record.
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?
Collection And Analysis Of Clinical Characteristics Of MPS IIIB (time frame: Baseline to Week 43). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 13 Sep 2018.