Mucopolysaccharidosis Type 7NCT02097251
An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient
A study testing UX003 in people with Mucopolysaccharidosis Type 7, run by Joyce Fox at 1 site in United States.
Trial summary
Emergency access granted to treat a single patient with advanced Mucopolysaccharidosis Type 7 with UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy via IV administration every other week (QOW) for up to 144 weeks. Read more about this study
- Who is it for?
- Any age, men only
- Conditions the study is for
- Mucopolysaccharidosis Type 7
- What is being tested?
- UX003
- How many people
- Not given
- Where
- 1 siteUnited States
- Study dates
- Start not givento not given
- Phase
- Expanded access
- 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 Any age
- Men only
- Child, Adult, Older adult
Who may be able to join
- 1 specific patient
- Confirmed diagnosis of MPS 7 based on leukocyte or fibroblast glucuronidase enzyme assay or genetic testing confirming diagnosis.
- Written informed consent of parent(s) or legal guardian(s)
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
United States1 site
Cohen Children's Medical Center
New Hyde Park, New York, 11040
About this study
Official title: An Open-Label Treatment Protocol With UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy for an Advanced Stage Mucopolysaccharidosis Type 7 (MPS 7) Patient
Emergency access granted to treat a single patient with advanced Mucopolysaccharidosis Type 7 with UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy via IV administration every other week (QOW) for up to 144 weeks.
- Conditions
- Mucopolysaccharidosis Type 7
- Being tested
- UX003
- Keywords
- Mucopolysaccharidosis Type 7, Sly syndrome, UX003, Enzyme Replacement Therapy, rare disease, Lysosomal Storage Disease, metabolic disorder
- Medical subject headings
- Mucopolysaccharidosis VII, Rare Diseases, Lysosomal Storage Diseases, Metabolic Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
UX003Drug
Also called recombinant human beta glucuronidase, rhGUS
Open Label
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Mar 2014 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Mucopolysaccharidosis Type 7Studies 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
- Expanded access
Sponsor and investigators
- Lead sponsor
- Joyce Fox
- Sponsor type
- University, hospital or other
- Collaborators
- Ultragenyx Pharmaceutical Inc
- Responsible party
- Sponsor-investigator, Joyce Fox, Northwell Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02097251
- Sponsor's protocol ID
- 13-606A
- Status last verified
- Feb 2017
- First submitted
- 24 Mar 2014
- Last updated
- 6 Feb 2017
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Feb 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Treatment Protocol With UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy for an Advanced Stage Mucopolysaccharidosis Type 7 (MPS 7) Patient
- ClinicalTrials.gov ID
- NCT02097251
- Acronym / sponsor ID
- 13-606A
- Conditions
- Mucopolysaccharidosis Type 7
- Keywords
- Mucopolysaccharidosis Type 7, Sly syndrome, UX003, Enzyme Replacement Therapy, rare disease, Lysosomal Storage Disease, metabolic disorder
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- Joyce FoxUniversity, hospital or other
- Collaborators
- Ultragenyx Pharmaceutical Inc
- First posted
- 27 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.
Emergency access granted to treat a single patient with advanced Mucopolysaccharidosis Type 7 with UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy via IV administration every other week (QOW) for up to 144 weeks.
Study design
Who may qualify
- Sex
- Male only
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- 1 specific patient
- Confirmed diagnosis of MPS 7 based on leukocyte or fibroblast glucuronidase enzyme assay or genetic testing confirming diagnosis.
- Written informed consent of parent(s) or legal guardian(s)
Key exclusion criteria (0 listed)
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 |
|---|---|---|
| UX003Also: recombinant human beta glucuronidase, rhGUSOpen Label | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Feb 2017.
Related trials for Mucopolysaccharidosis Type 7
RecruitingObservational study
Mucopolysaccharidosis VII Disease Monitoring Program
Mucopolysaccharidosis VII, MPS VII
Active, not recruitingObservational study
Early Check: Expanded Screening in Newborns
Spinal Muscular Atrophy, Fragile X Syndrome
RecruitingObservational study
Registry of Patients Diagnosed With Lysosomal Storage Diseases
Mucopolysaccharidosis I, Mucopolysaccharidosis II
RecruitingPhase 1
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
MPS I, MPS II
Active, not recruitingPhase 2
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
Mucopolysaccharidosis Disorders, Hurler 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.
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 study testing UX003 in people with Mucopolysaccharidosis Type 7, run by Joyce Fox at 1 site in United States.
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 3 inclusion and 0 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: status not confirmed, as of the sponsor's last update on 6 Feb 2017. 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 States. 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?
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.
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 6 Feb 2017.