Sanfilippo SyndromePhase 2NCT02350816
An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093.
A Phase 2 study testing HGT-1410 in people with Sanfilippo Syndrome and Mucopolysaccharidosis (MPS), run by Shire at 8 sites in 7 countries.
Trial summary
This extension study will allow participants to continue receiving treatment with HGT-1410 and to initiate treatment in patients who received no-treatment in Study HGT-SAN-093, and will evaluate the long-term safety and efficacy of the study drug. Read more about this study
- Who is it for?
- Ages 12 to 48 months
- Conditions the study is for
- Sanfilippo Syndrome, Mucopolysaccharidosis (MPS)
- What is being tested?
- HGT-1410
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 17 participants
- Where
- 8 sitesin 7 countries
- Study dates
- 8 Apr 2015to 12 Apr 2019
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 12 to 48 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Patients must meet all of the following criteria to be considered eligible for enrollment:
- Patient has completed through at least the Week 48 visit of Study HGT-SAN-093
- The patient's parent(s) or legally authorized guardian(s) must have voluntarily signed an Institutional Review Board- (IRB-)/ Independent Ethics Committee- (IEC-) approved informed consent form after all relevant aspects of the study have been explained and discussed. Consent of the patient's parent(s) or legally authorized guardian(s) and the patient's assent, as relevant, must be obtained
Who cannot join
- Patients will be excluded from the study if any of the following criteria are met:
- The patient, if randomized to treatment in Study HGT-SAN-093, has experienced a decline of more than 20 points in the BSID-III cognitive DQ score between Baseline and the Week 48 visit in Study HGT-SAN-093, AND, upon individual evaluation by the Investigator, has been deemed a treatment failure*
- The patient has experienced, in the opinion of the Investigator, a safety or medical issue that contraindicates treatment with HGT-1410, including but not limited to clinically relevant intracranial hypertension, severe infusion-related reactions after treatment with HGT-1410, uncontrollable seizure disorder
- The patient has a known hypersensitivity to any of the components of HGT-1410
- The patient is enrolled in another clinical study, other than HGT-SAN-093, that involves clinical investigations or use of any investigational product (drug or [intrathecal/spinal] device) within 30 days prior to study enrollment or at any time during the study
- The patient has any known or suspected hypersensitivity to anesthesia or is thought to be at an unacceptably high risk for anesthesia due to airway compromise or other conditions
Show all 17Show fewer
- The patient has a condition that is contraindicated as described in the SOPH-A-PORT® Mini S IDDD Instructions for Use, including:
- The patient has had, or may have, an allergic reaction to the materials of construction of the SOPH-A-PORT ® Mini S device
- The patient's body size is too small to support the size of the SOPH-A-PORT ® Mini S Access Port, as judged by the Investigator
- The patient's drug therapy requires substances known to be incompatible with the materials of construction
- The patient has a known or suspected local or general infection
- The patient is at risk of abnormal bleeding due to a medical condition or therapy
- The patient has one or more spinal abnormalities that could complicate safe implantation or fixation
- The patient has a functioning CSF shunt device
- The patient has shown an intolerance to an implanted device
- The patient is unable to comply with the protocol (eg, is unable to return for safety evaluations, or is otherwise unlikely to complete the study) as determined by the Investigator
- All treated patients in Study HGT-SAN-093 will have their cognitive development assessed at the Week 48 Visit in Study HGT-SAN-093. If a decline from Baseline of 20 points or less in the BSID-III DQ score is observed, then the patient may proceed into the Study SHP-610-201 without further evaluation. If a decline from Baseline of more than 20 points in DQ score is observed, then an individual evaluation by the Investigator will occur to determine if the patient is a treatment failure. This individual evaluation will take into account the DQ scores, VABS-II score, physical status, and any other information available for that patient at that time. If the Investigator deems the patient to be a treatment failure, then the patient may not enter the Study SHP-610-201
Trial locations and contacts
8 sites in 7 countries.
8 sites in 7 countries
United States2 sites
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27514
University of Minnesota
Minneapolis, Minnesota, 55455
About this study
Official title: An Open-Label Extension of Study HGT-SAN-093 Evaluating the Safety and Efficacy Study of HGT-1410 (Recombinant Human Heparan N Sulfatase) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Mucopolysaccharidosis Type IIIA Disease
This extension study will allow participants to continue receiving treatment with HGT-1410 and to initiate treatment in patients who received no-treatment in Study HGT-SAN-093, and will evaluate the long-term safety and efficacy of the study drug.
- Conditions
- Sanfilippo Syndrome, Mucopolysaccharidosis (MPS)
- Being tested
- HGT-1410
- 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.
HGT-1410Drug
Also called Recombinant Human Heparan N Sulfatase
HGT-1410 administered according to Patient Group assignment.
| Study group | Role | What they receive |
|---|---|---|
| HGT-1410 Q2W in Study HGT-SAN-093 randomized to HGT-1410 Q2W | Active comparator | Patients in Group 1 will continue HGT-1410 treatment at a dose of 45 mg administered every 2 weeks (Q2W) starting at Week 50, with a cumulative treatment period of up to 42 months (168 weeks) . HGT-1410 will be administered intrathecally (IT) by an indwelling intrathecal drug delivery device (IDDD).
HGT-SAN-093 = NCT02060526 Receives: Drug: HGT-1410 |
| HGT-1410 Q4W in Study HGT-SAN-093 randomized to HGT-1410 Q4W | Active comparator | Patients in Group 2 will continue HGT-1410 treatment at a dose of 45 mg administered every 4 weeks (Q4W) starting at Week 52, with a cumulative treatment period of up to 42 months (168 weeks). HGT-1410 will be administered intrathecally (IT) by an indwelling intrathecal drug delivery device (IDDD). Receives: Drug: HGT-1410 |
| no-treatment in Study HGT-SAN-093 randomized to HGT-1410 Q2W | Active comparator | Patients in Group 3A will receive an IDDD following informed consent and will be randomized in a 1:1 allocation ratio to begin HGT-1410 treatment at a dose of 45 mg administered every 2 weeks (Q2W) starting at Week 0 of the extension study, with a cumulative treatment period of up to 30 months (120 weeks). HGT-1410 will be administered intrathecally (IT) by an indwelling intrathecal drug delivery device (IDDD). Receives: Drug: HGT-1410 |
| no-treatment in Study HGT-SAN-093 randomized to HGT-1410 Q4W | Active comparator | Patients in Group 3B will receive an IDDD following informed consent and will be randomized in a 1:1 allocation ratio to begin HGT-1410 treatment at a dose of 45 mg administered every 4 weeks (Q4W) starting at Week 0 of the extension study, with a cumulative treatment period of up to 30 months (120 weeks). HGT-1410 will be administered intrathecally (IT) by an indwelling intrathecal drug delivery device (IDDD). Receives: Drug: HGT-1410 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Based on Type, Severity and Relationship to Treatment Drug
An AE was defined as any untoward medical occurrence in a clinical investigation participant administered as a pharmaceutical product that did not necessarily have a causal relationship with this treatment. TEAEs was defined as all AEs from the time of initial IDDD implantation (or first dose if earlier) in either Study NCT02060526 (HGT-SAN-093) or Study NCT02350816 (SHP-610-210) to the data cutoff date (28 Jun 2017), or 30 days after the date of the last dose or 2 weeks after the date of device explant (whichever was later) if early termination occurred. Treatment-emergent AEs were summarized by type (serious, life-threatening), severity (mild, moderate, severe) and degree of relationship to investigational product (Intrathecal Drug Delivery Device (IDDD), device surgical procedure, or intraThecal administration of HGT-1410).
Measured: From start of study drug administration up to follow-up (Week 276)
Number of Participants With Positive Anti-Recombinant Human Heparan N-Sulfatase (rhHNS) Antibody Status in Serum
Number of participants with positive anti-rhHNS antibody status in serum were reported.
Measured: Up to 120 weeks
Area Under Curve (AUC) of Recombinant Human Heparan N-Sulfatase (rhHNS) Concentration in Cerebro Spinal Fluid (CSF)
No sufficient pharmacokinetic (PK) samples were collected and analyzed due to early termination of the study.
Measured: Week 0 and 48
Area Under Curve (AUC) of Recombinant Human Heparan N-Sulfatase (rhHNS) Concentration in Serum
No sufficient PK samples were collected and analyzed due to early termination of the study.
Measured: Week 0, 48 and 96
Levels of Glycosaminoglycan (GAG) Concentration in Cerebro Spinal Fluid (CSF)
Levels of GAG concentration in CSF was reported. Last measurable data was presented for respective participant up to their last observed time point.
Measured: Up to Week 120
Levels of Glycosaminoglycan (GAG) Concentration in Urine
Levels of GAG concentration in Urine were reported. Last measurable data was presented for respective participant up to their last available time point.
Measured: Up to Week 120
Secondary outcomes (3)
Change From Baseline Vineland Adaptive Behavior Scales Second Edition (VABS-II)
VABS-II measured adaptive behaviors, including the ability to cope with environmental changes, to learn new everyday skills, and to demonstrate independence. It was an instrument that supports the diagnosis of intellectual and developmental disabilities in participants. This test measured 5 key domains: communication, daily living skills, socialization, motor skills, and the adaptive behavior composite ((a composite of the other four domains). Scoring was 'Usually' = 2, 'Sometimes'/Partially' = 1 or 'Never' = 0. The raw scores was converted to domain standard scores (mean 100, SD 15). Higher scores indicate undesirable behavior. Due to the premature termination of the treatment period, efficacy data were not analyzed and no efficacy conclusions were drawn.
Measured: Baseline, Week 120
Change From Baseline in the Developmental Quotient (DQ) Assessed by Neurocognitive Tests
The development quotient (DQ) was to express a neurodevelopmental/cognitive delay which was computed as a ratio and expressed as a percentage using the age equivalent score divided by the age at testing ([age-equivalent score/chronological age] × 100; range: 0, 100). Higher scores are indicative of decreased development. Neurocognitive tests included Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III), Kaufman Assessment Battery for Children, Second Edition (KABC-II), Vineland Adaptive Behavior Scales, Second Edition (VABS-II). Due to the premature termination of the treatment period, efficacy data were not analyzed and no efficacy conclusions were drawn.
Measured: Baseline, Week 120
Change From Baseline in Total Cortical Grey Matter Volume
The total cortical grey matter volume was assessed by volumetric magnetic resonance imaging (MRI) of the brain. Due to the premature termination of the treatment period, efficacy data were not analyzed and no efficacy conclusions were drawn.
Measured: Baseline, Week 120
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jan 2015 (estimated)
- Study start8 Apr 2015
- Primary completion12 Apr 2019
- Study completion12 Apr 2019
Why it stopped: Completion of follow-up period
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Sanfilippo SyndromeStudies 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 17 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
- NCT02350816
- Sponsor's protocol ID
- SHP610-201
- Other IDs
- 2014-003960-20, 2014
- Status last verified
- May 2021
- First submitted
- 21 Jan 2015
- Last updated
- 11 Jun 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- Yes: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
Study at a glance
From the registry record, last updated by the sponsor on 11 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Extension of Study HGT-SAN-093 Evaluating the Safety and Efficacy Study of HGT-1410 (Recombinant Human Heparan N Sulfatase) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Mucopolysaccharidosis Type IIIA Disease
- ClinicalTrials.gov ID
- NCT02350816
- Acronym / sponsor ID
- SHP610-201
- Conditions
- Sanfilippo Syndrome, Mucopolysaccharidosis (MPS)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Completion of follow-up period
- Enrolment
- 17 participants (actual)
- Lead sponsor
- ShireIndustry (pharma, biotech, devices)
- Study start
- 8 Apr 2015 (actual)
- Primary completion
- 12 Apr 2019 (actual)
- Study completion
- 12 Apr 2019 (actual)
- First posted
- 30 Jan 2015 (estimated)
- Results
- Posted 20 Apr 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This extension study will allow participants to continue receiving treatment with HGT-1410 and to initiate treatment in patients who received no-treatment in Study HGT-SAN-093, and will evaluate the long-term safety and efficacy of the study drug.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| HGT-1410 Q2W in Study HGT-SAN-093 randomized to HGT-1410 Q2W | Active comparator | Drug: HGT-1410Patients in Group 1 will continue HGT-1410 treatment at a dose of 45 mg administered every 2 weeks (Q2W) starting at Week 50, with a cumulative treatment period of up to 42 months (168 weeks) . HGT-1410 will be administered intrathecally (IT) by an indwelling intrathecal drug delivery device (IDDD). HGT-SAN-093 = NCT02060526 |
| HGT-1410 Q4W in Study HGT-SAN-093 randomized to HGT-1410 Q4W | Active comparator | Drug: HGT-1410Patients in Group 2 will continue HGT-1410 treatment at a dose of 45 mg administered every 4 weeks (Q4W) starting at Week 52, with a cumulative treatment period of up to 42 months (168 weeks). HGT-1410 will be administered intrathecally (IT) by an indwelling intrathecal drug delivery device (IDDD). |
| no-treatment in Study HGT-SAN-093 randomized to HGT-1410 Q2W | Active comparator | Drug: HGT-1410Patients in Group 3A will receive an IDDD following informed consent and will be randomized in a 1:1 allocation ratio to begin HGT-1410 treatment at a dose of 45 mg administered every 2 weeks (Q2W) starting at Week 0 of the extension study, with a cumulative treatment period of up to 30 months (120 weeks). HGT-1410 will be administered intrathecally (IT) by an indwelling intrathecal drug delivery device (IDDD). |
| no-treatment in Study HGT-SAN-093 randomized to HGT-1410 Q4W | Active comparator | Drug: HGT-1410Patients in Group 3B will receive an IDDD following informed consent and will be randomized in a 1:1 allocation ratio to begin HGT-1410 treatment at a dose of 45 mg administered every 4 weeks (Q4W) starting at Week 0 of the extension study, with a cumulative treatment period of up to 30 months (120 weeks). HGT-1410 will be administered intrathecally (IT) by an indwelling intrathecal drug delivery device (IDDD). |
Who may qualify
- Ages
- 12 to 48 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Patients must meet all of the following criteria to be considered eligible for enrollment:
- Patient has completed through at least the Week 48 visit of Study HGT-SAN-093
- The patient's parent(s) or legally authorized guardian(s) must have voluntarily signed an Institutional Review Board- (IRB-)/ Independent Ethics Committee- (IEC-) approved informed consent form after all relevant aspects of the study have been explained and discussed. Consent of the patient's parent(s) or legally authorized guardian(s) and the patient's assent, as relevant, must be obtained
Key exclusion criteria (17 listed)
- The patient, if randomized to treatment in Study HGT-SAN-093, has experienced a decline of more than 20 points in the BSID-III cognitive DQ score between Baseline and the Week 48 visit in Study HGT-SAN-093, AND, upon individual evaluation by the Investigator, has been deemed a treatment failure*
- The patient has experienced, in the opinion of the Investigator, a safety or medical issue that contraindicates treatment with HGT-1410, including but not limited to clinically relevant intracranial hypertension, severe infusion-related reactions after treatment with HGT-1410, uncontrollable seizure disorder
- The patient has a known hypersensitivity to any of the components of HGT-1410
- The patient is enrolled in another clinical study, other than HGT-SAN-093, that involves clinical investigations or use of any investigational product (drug or [intrathecal/spinal] device) within 30 days prior to study enrollment or at any time during the study
- The patient has any known or suspected hypersensitivity to anesthesia or is thought to be at an unacceptably high risk for anesthesia due to airway compromise or other conditions
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 |
|---|---|---|
| HGT-1410Also: Recombinant Human Heparan N SulfataseHGT-1410 administered according to Patient Group assignment. | Drug | HGT-1410 Q2W in Study HGT-SAN-093 randomized to HGT-1410 Q2W, HGT-1410 Q4W in Study HGT-SAN-093 randomized to HGT-1410 Q4W, no-treatment in Study HGT-SAN-093 randomized to HGT-1410 Q2W, no-treatment in Study HGT-SAN-093 randomized to HGT-1410 Q4W |
Primary outcome
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Based on Type, Severity and Relationship to Treatment Drug
Time frame: From start of study drug administration up to follow-up (Week 276)
An AE was defined as any untoward medical occurrence in a clinical investigation participant administered as a pharmaceutical product that did not necessarily have a causal relationship with this treatment. TEAEs was defined as all AEs from the time of initial IDDD implantation (or first dose if earlier) in either Study NCT02060526 (HGT-SAN-093) or Study NCT02350816 (SHP-610-210) to the data cutoff date (28 Jun 2017), or 30 days after the date of the last dose or 2 weeks after the date of device explant (whichever was later) if early termination occurred. Treatment-emergent AEs were summarized by type (serious, life-threatening), severity (mild, moderate, severe) and degree of relationship to investigational product (Intrathecal Drug Delivery Device (IDDD), device surgical procedure, or intraThecal administration of HGT-1410).
Number of Participants With Positive Anti-Recombinant Human Heparan N-Sulfatase (rhHNS) Antibody Status in Serum
Time frame: Up to 120 weeks
Number of participants with positive anti-rhHNS antibody status in serum were reported.
Area Under Curve (AUC) of Recombinant Human Heparan N-Sulfatase (rhHNS) Concentration in Cerebro Spinal Fluid (CSF)
Time frame: Week 0 and 48
No sufficient pharmacokinetic (PK) samples were collected and analyzed due to early termination of the study.
Area Under Curve (AUC) of Recombinant Human Heparan N-Sulfatase (rhHNS) Concentration in Serum
Time frame: Week 0, 48 and 96
No sufficient PK samples were collected and analyzed due to early termination of the study.
Levels of Glycosaminoglycan (GAG) Concentration in Cerebro Spinal Fluid (CSF)
Time frame: Up to Week 120
Levels of GAG concentration in CSF was reported. Last measurable data was presented for respective participant up to their last observed time point.
Levels of Glycosaminoglycan (GAG) Concentration in Urine
Time frame: Up to Week 120
Levels of GAG concentration in Urine were reported. Last measurable data was presented for respective participant up to their last available time point.
3 secondary outcomes
Change From Baseline Vineland Adaptive Behavior Scales Second Edition (VABS-II)
Time frame: Baseline, Week 120
Change From Baseline in the Developmental Quotient (DQ) Assessed by Neurocognitive Tests
Time frame: Baseline, Week 120
Change From Baseline in Total Cortical Grey Matter Volume
Time frame: Baseline, Week 120
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Jun 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Yes. Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
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Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
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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.
Study Director study director
Takeda
8 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing HGT-1410 in people with Sanfilippo Syndrome and Mucopolysaccharidosis (MPS), run by Shire at 8 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 3 inclusion and 17 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 11 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in United States, France, Germany, Italy, Netherlands 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 20 Apr 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Based on Type, Severity and Relationship to Treatment Drug (time frame: From start of study drug administration up to follow-up (Week 276)). The study lists 6 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 11 Jun 2021.