Mucopolysaccharidosis (MPS)NCT02095015
Mucopolysaccharidosis (MPS) I, II, and VI Screening in a High-Risk Population With Previous Surgical Repair or Presence of Inguinal and/or Umbilical Hernia in Combination With Pediatric ENT Surgery (The HATT Project)
An observational study of Mucopolysaccharidosis (MPS), run by Shire at 14 sites in 7 countries.
Trial summary
Mucopolysaccharidosis (MPS) type II (MPS II; Hunter syndrome) is a rare, X-linked disease caused by a deficiency of the lysosomal enzyme iduronate-2-sulfatase (I2S) and occurs almost exclusively in boys, with an incidence of approximately 1.3 per 100,000 live male births.1 Early identification of MPS II is challenging… Read more about this study
- Who is it for?
- Ages Up to 7 years, men only
- Conditions the study is for
- Mucopolysaccharidosis (MPS)
- How many people
- 159 participants
- Where
- 14 sitesin 7 countries
- Study dates
- 21 May 2014to 14 Aug 2015
- 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 Up to 7 years
- Men only
- No healthy volunteers
- Child
Study population: Males of any ethnic background or race, <7 years of age or under, who have had or are scheduled for adenoidectomy and/or tonsillectomy and/or tympanostomy (alone or in combination), will be enrolled. In addition, the subjects must have previous surgical repair or presence of inguinal and/or umbilical hernia.
Who may be able to join
- Each subject must meet the following criteria to be enrolled in this study:
- The subject is male.
- The subject is <7 years of age.
- The subject has had or is scheduled for ENT surgery for any of the following, alone or in combination: adenoidectomy, tonsillectomy, and tympanostomy.
- The subject has record of previous surgical repair or presence of inguinal and/or umbilical hernia.
- The subject's parent(s) or the subject's legally authorized representative(s) has provided written informed consent that has been approved by the Institutional Review Board/Independent Ethics Committee (IRB/IEC).
Who cannot join
- Subjects who meet any of the following criteria will be excluded from the study:
- The subject has a current confirmed diagnosis of any MPS disorder.
- The subject was born prematurely (defined as born before 37 weeks gestation).
- The subject has received a blood transfusion within the past 6 weeks.
- The subject has received a bone marrow transplant.
Trial locations and contacts
14 sites in 7 countries.
14 sites in 7 countries
United States7 sites
Nemours Children's Clinic
Jacksonville, Florida, 32207
Children's Hospital Los Angeles
Los Angeles, California, 90027
Children's Hospital and Clinic
Minneapolis, Minnesota, 55404
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
Intermountain Ear Nose and Throat Center
Salt Lake City, Utah, 84102
Saint Louis Children's Hospital
St Louis, Missouri, 63110
Italy2 sites
Ospedale San Gerardo
Monza, 20900
Ospedale Pediatrico Bambino Gesù
Passoscuro, 00050
Turkey (Türkiye)1 site
Hacettepe Universitesi Tip Fakultesi Hastanesi
Ankara, 06100
United Kingdom1 site
Royal Manchester Children's Hospital
Manchester, M13 9WL
About this study
Official title: Mucopolysaccharidosis (MPS) I, II, and VI Screening in a High Risk Population With Previous Surgical Repair or Presence of Inguinal and/or Umbilical Hernia in Combination With Pediatric Ear, Nose and Throat (ENT) Surgery (Adenoidectomy and/or Tonsillectomy and/or Tympanostomy) (The HATT Project)
Mucopolysaccharidosis (MPS) type II (MPS II; Hunter syndrome) is a rare, X-linked disease caused by a deficiency of the lysosomal enzyme iduronate-2-sulfatase (I2S) and occurs almost exclusively in boys, with an incidence of approximately 1.3 per 100,000 live male births.1 Early identification of MPS II is challenging because some initial features, such as chronic runny nose, otitis media, and hernias, are commonly seen in the general population. As a result, even though the signs and symptoms of MPS II typically appear early in childhood, the diagnosis may lag behind by several years.
The primary objective of this international multi-center study is to evaluate the positive screening rate of MPS II subjects by screening a high-risk male pediatric population who have had or are scheduled for 1 or more specific ENT surgical procedures (adenoidectomy and/or tonsillectomy and/or tympanostomy) and who have a previously repaired or present evidence of an inguinal and/or umbilical hernia.
Read the detailed description
With evidence-based information from MPS registries regarding the types of surgical interventions that are most prevalent in MPS, this screening study is expected to provide the understanding of the role pediatric ENT surgeons can play in identifying young children with MPS.
- Conditions
- Mucopolysaccharidosis (MPS)
- Keywords
- MPS II, iduronate-2-sulfatase, Mucopolysaccharidosis VI, MPS, MPS VI, ear, nose and throat, Mucopolysaccharidosis II, Hunter syndrome, ENT, Mucopolysaccharidosis I, Hurler, Hurler-Scheie, or Scheie syndrome, Mucopolysaccharidosis, MPS I, Maroteaux-Lamy syndrome
- Medical subject headings
- Mucopolysaccharidoses, Sudden Infant Death, Mucopolysaccharidosis VI, Mucopolysaccharidosis II, Mucopolysaccharidosis I
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 |
|---|---|---|
| Analysis population | All subjects enrolled in the study who meet the eligibility criteria |
What is being measured
The primary outcome is the main question the study is designed to answer.
The status of diagnosis of MPS II (either positive or negative) of each subject
To evaluate the positive screening rate of MPS II subjects by screening a high risk male pediatric population.
Measured: Screening visit
Secondary outcomes (1)
The status of diagnosis of MPS I or VI (either positive or negative) of each subject
Measured: Screening visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Mar 2014 (estimated)
- Study start21 May 2014
- Primary completion14 Aug 2015
- Study completion14 Aug 2015
Why it stopped: terminated for not meeting enrollment goals
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Mucopolysaccharidosis (MPS)Studies 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
- Other
- Time perspective
- Prospective
- Enrolment
- 159 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02095015
- Sponsor's protocol ID
- SHP-001-801
- Status last verified
- Mar 2021
- First submitted
- 20 Mar 2014
- Last updated
- 17 Mar 2021
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Mucopolysaccharidosis (MPS) I, II, and VI Screening in a High Risk Population With Previous Surgical Repair or Presence of Inguinal and/or Umbilical Hernia in Combination With Pediatric Ear, Nose and Throat (ENT) Surgery (Adenoidectomy and/or Tonsillectomy and/or Tympanostomy) (The HATT Project)
- ClinicalTrials.gov ID
- NCT02095015
- Acronym / sponsor ID
- SHP-001-801
- Conditions
- Mucopolysaccharidosis (MPS)
- Keywords
- MPS II, iduronate-2-sulfatase, Mucopolysaccharidosis VI, MPS, MPS VI, ear, nose and throat, Mucopolysaccharidosis II, Hunter syndrome
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: terminated for not meeting enrollment goals
- Enrolment
- 159 participants (actual)
- Lead sponsor
- ShireIndustry (pharma, biotech, devices)
- Study start
- 21 May 2014 (actual)
- Primary completion
- 14 Aug 2015 (actual)
- Study completion
- 14 Aug 2015 (actual)
- First posted
- 24 Mar 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.
Mucopolysaccharidosis (MPS) type II (MPS II; Hunter syndrome) is a rare, X-linked disease caused by a deficiency of the lysosomal enzyme iduronate-2-sulfatase (I2S) and occurs almost exclusively in boys, with an incidence of approximately 1.3 per 100,000 live male births.1 Early identification of MPS II is challenging because some initial features, such as chronic runny nose, otitis media, and hernias, are commonly seen in the general population. As a result, even though the signs and symptoms of MPS II typically appear early in childhood, the diagnosis may lag behind by several years.
The primary objective of this international multi-center study is to evaluate the positive screening rate of MPS II subjects by screening a high-risk male pediatric population who have had or are scheduled for 1 or more specific ENT surgical procedures (adenoidectomy and/or tonsillectomy and/or tympanostomy) and who have a previously repaired or present evidence of an inguinal and/or umbilical hernia.
Detailed description
With evidence-based information from MPS registries regarding the types of surgical interventions that are most prevalent in MPS, this screening study is expected to provide the understanding of the role pediatric ENT surgeons can play in identifying young children with MPS.
Study design
- Observational model
- Other
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Analysis population | – | –All subjects enrolled in the study who meet the eligibility criteria |
Who may qualify
- Ages
- Up to 7 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Males of any ethnic background or race, <7 years of age or under, who have had or are scheduled for adenoidectomy and/or tonsillectomy and/or tympanostomy (alone or in combination), will be enrolled. In addition, the subjects must have previous surgical repair or presence of inguinal and/or umbilical hernia.
Key inclusion criteria (6 listed)
- The subject is male.
- The subject is <7 years of age.
- The subject has had or is scheduled for ENT surgery for any of the following, alone or in combination: adenoidectomy, tonsillectomy, and tympanostomy.
- The subject has record of previous surgical repair or presence of inguinal and/or umbilical hernia.
- The subject's parent(s) or the subject's legally authorized representative(s) has provided written informed consent that has been approved by the Institutional Review Board/Independent Ethics Committee (IRB/IEC).
Key exclusion criteria (5 listed)
- The subject has a current confirmed diagnosis of any MPS disorder.
- The subject was born prematurely (defined as born before 37 weeks gestation).
- The subject has received a blood transfusion within the past 6 weeks.
- The subject has received a bone marrow transplant.
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 status of diagnosis of MPS II (either positive or negative) of each subject
Time frame: Screening visit
To evaluate the positive screening rate of MPS II subjects by screening a high risk male pediatric population.
1 secondary outcome
The status of diagnosis of MPS I or VI (either positive or negative) of each subject
Time frame: Screening visit
Results and safety
The study was stopped early (reason given: terminated for not meeting enrollment goals). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Mar 2021.
- To obtain more information on the study, click here/on this link.Link provided by the sponsor.
Related trials for Mucopolysaccharidosis (MPS)
Active, not recruitingObservational study
Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.
Hematopoietic Cell Transplantation, Mucopolysaccharidosis Type I
RecruitingPhase 1/2
Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)
Mucopolysaccharidosis Type IIIA
Enrolling by invitationPhase 2/3
An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007
Mucopolysaccharidosis II
RecruitingObservational study
Cardiac Structure and Function in MPS
MPS I, MPS IV A
Not yet recruitingObservational study
MPS IIIA Disease Monitoring Program
MPS IIIA, Sanfilippo Syndrome
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.
Study Director study director
Takeda
14 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Mucopolysaccharidosis (MPS), run by Shire at 14 sites in 7 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 6 inclusion and 5 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 17 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in United States, Italy, Canada, Mexico, Spain and 2 more countries. 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: terminated for not meeting enrollment goals). 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?
The status of diagnosis of MPS II (either positive or negative) of each subject (time frame: Screening visit). 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 17 Mar 2021.