Mucopolysaccharidosis IIIANCT02746341
Natural History Study of Patients With MPS IIIA
An observational study of Mucopolysaccharidosis IIIA, run by LYSOGENE at 5 sites in 5 countries.
Trial summary
Evaluate the clinical progression in patients with MPS IIIA who are untreated with any investigational product and to obtain standardized assessments: neurocognitive, behavioral, sleep-wake habits and effect of MPS IIIA on the quality of life of patients and their families. Read more about this study
- Who is it for?
- Ages 0 to 9 years
- Conditions the study is for
- Mucopolysaccharidosis IIIA
- How many people
- 23 participants
- Where
- 5 sitesin 5 countries
- Study dates
- Apr 2016to May 2019
- 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 0 to 9 years
- All sexes
- No healthy volunteers
- Child
Study population: Subjects diagnosed with MPS IIIA
Who may be able to join
- Documented MPS IIIA diagnosis
- Children up to and including 9 years of age
- The patient is sufficiently able, in the opinion of the Investigator, to adhere to the study visit schedule and other protocol requirements
- The patient's parent(s) or legal guardian(s) has signed written informed consent, according to the local regulations and after all relevant aspects of the -study have been explained and discussed
Who cannot join
- The patient is participating in a clinical trial of any potential disease-modifying investigational medicinal product or taking high dose (>100 mg/kg/day) synthetic genistein (patients on low dose or naturally derived genistein can be included in this study).
- The patient has received a hematopoietic stem cell or bone marrow transplant or gene therapy.
- The patient has received enzyme replacement therapy in the last 6 months.
- Homozygous or compound heterozygous for the S298P mutation or the investigator and/or trial steering committee considers the patient not to have the classical severe form of MPS IIIA.
- Individuals with rare and unrelated serious comorbidities e.g. Down syndrome, intraventricular hemorrhage in the new-born period, or extreme low birth weight (<1500 grams).
- Visual or hearing impairment sufficient, in the clinical judgment of the investigator, to preclude cooperation with neurodevelopmental testing. Use of hearing aids is permitted.
Trial locations and contacts
5 sites in 5 countries.
5 sites in 5 countries
Netherlands1 site
Academic Medical Center, Emma Children's Hospital
Amsterdam
United Kingdom1 site
Great Ormond Street Hospital NHS Foundation Trust
London
About this study
Official title: An Observational, Prospective, Multi-center, Natural History Study of Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)
Evaluate the clinical progression in patients with MPS IIIA who are untreated with any investigational product and to obtain standardized assessments: neurocognitive, behavioral, sleep-wake habits and effect of MPS IIIA on the quality of life of patients and their families.
Read the detailed description
This is a multicenter, multinational, longitudinal, observational study in children aged up to and including 9 years, who have been diagnosed with MPS IIIA. The study will detail the natural course of MPS IIIA via standardized clinical, biochemical, neurocognitive, developmental, behavioral and imaging measures. This information is anticipated to inform the design and interpretation of future interventional studies.
- Conditions
- Mucopolysaccharidosis IIIA
What is being measured
The primary outcome is the main question the study is designed to answer.
The change from baseline in cognitive function using the Bayley scales of infant and toddler development third edition
Measured: Baseline, and every 6 months, for up to 24 months
Secondary outcomes (4)
Change from baseline in the adaptive behavior composite standard score as measured by the Vineland Adaptive Behavior scale
Measured: Baseline and every 6 months up to 24 months
Sleep disturbances measured by Actigraphy
Measured: Baseline and every 3 months up to 24 months
Patient Quality of Life Questionnaires
Measured: Baseline and every 6 months up to 24 months
Change from baseline in total cortical grey matter volume
Measured: Baseline, 12 months, 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Apr 2016 (estimated)
- Study startApr 2016
- Primary completionMay 2019
- Study completionMay 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Mucopolysaccharidosis IIIAStudies of the same condition, open and under way.
- 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 23 participants (actual)
Sponsor and investigators
- Lead sponsor
- LYSOGENE
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02746341
- Sponsor's protocol ID
- P3-LYS-SAF
- Status last verified
- Aug 2021
- First submitted
- 15 Apr 2016
- Last updated
- 31 Aug 2021
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 31 Aug 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Observational, Prospective, Multi-center, Natural History Study of Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)
- ClinicalTrials.gov ID
- NCT02746341
- Acronym / sponsor ID
- P3-LYS-SAF
- Conditions
- Mucopolysaccharidosis IIIA
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 23 participants (actual)
- Lead sponsor
- LYSOGENEIndustry (pharma, biotech, devices)
- Study start
- Apr 2016
- Primary completion
- May 2019 (actual)
- Study completion
- May 2019 (actual)
- First posted
- 21 Apr 2016 (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.
Evaluate the clinical progression in patients with MPS IIIA who are untreated with any investigational product and to obtain standardized assessments: neurocognitive, behavioral, sleep-wake habits and effect of MPS IIIA on the quality of life of patients and their families.
Detailed description
This is a multicenter, multinational, longitudinal, observational study in children aged up to and including 9 years, who have been diagnosed with MPS IIIA. The study will detail the natural course of MPS IIIA via standardized clinical, biochemical, neurocognitive, developmental, behavioral and imaging measures. This information is anticipated to inform the design and interpretation of future interventional studies.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 0 to 9 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Subjects diagnosed with MPS IIIA
Key inclusion criteria (4 listed)
- Documented MPS IIIA diagnosis
- Children up to and including 9 years of age
- The patient is sufficiently able, in the opinion of the Investigator, to adhere to the study visit schedule and other protocol requirements
- The patient's parent(s) or legal guardian(s) has signed written informed consent, according to the local regulations and after all relevant aspects of the -study have been explained and discussed
Key exclusion criteria (6 listed)
- The patient is participating in a clinical trial of any potential disease-modifying investigational medicinal product or taking high dose (>100 mg/kg/day) synthetic genistein (patients on low dose or naturally derived genistein can be included in this study).
- The patient has received a hematopoietic stem cell or bone marrow transplant or gene therapy.
- The patient has received enzyme replacement therapy in the last 6 months.
- Homozygous or compound heterozygous for the S298P mutation or the investigator and/or trial steering committee considers the patient not to have the classical severe form of MPS IIIA.
- Individuals with rare and unrelated serious comorbidities e.g. Down syndrome, intraventricular hemorrhage in the new-born period, or extreme low birth weight (<1500 grams).
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
The change from baseline in cognitive function using the Bayley scales of infant and toddler development third edition
Time frame: Baseline, and every 6 months, for up to 24 months
4 secondary outcomes
Change from baseline in the adaptive behavior composite standard score as measured by the Vineland Adaptive Behavior scale
Time frame: Baseline and every 6 months up to 24 months
Sleep disturbances measured by Actigraphy
Time frame: Baseline and every 3 months up to 24 months
Patient Quality of Life Questionnaires
Time frame: Baseline and every 6 months up to 24 months
Change from baseline in total cortical grey matter volume
Time frame: Baseline, 12 months, 24 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Aug 2021.
- Individual participant dataSharing planned: Undecided
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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.
5 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Mucopolysaccharidosis IIIA, run by LYSOGENE at 5 sites in 5 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 4 inclusion and 6 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: finished, as of the sponsor's last update on 31 Aug 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in Brazil, France, Germany, Netherlands, 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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
The change from baseline in cognitive function using the Bayley scales of infant and toddler development third edition (time frame: Baseline, and every 6 months, for up to 24 months). 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 31 Aug 2021.