Mucopolysaccharidosis Type III APhase 1/2NCT02053064
Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy
A Phase 1/2 study testing SAF-301 in people with Mucopolysaccharidosis Type III A and Sanfilippo Disease Type A, run by LYSOGENE at 1 site in France.
Trial summary
P2-SAF-301 is an open-label interventional study without administration of investigational product, evaluating the long-term safety and tolerability of intracerebral SAF-301 previously administered to 4 patients with Sanfilippo type A syndrome. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Mucopolysaccharidosis Type III A, Sanfilippo Disease Type A
- What is being tested?
- SAF-301
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteFrance
- Study dates
- May 2013to Jun 2017
- Phase
- Phase 1/2
- 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
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients having completed the previous phase I/II study (P1-SAF-301),
- Family understanding the follow-up procedures and the informed consent,
- Patient's parents having signed the informed consent form.
Who cannot join
- Participation in any other clinical trial.
Trial locations and contacts
1 site in 1 country.
France1 site
Hôpitaux Universitaires Paris Sud (Bicêtre)
Le Kremlin-Bicêtre, 94275
About this study
Official title: Long-term Follow-up of Patients With Sanfilippo Type A Syndrome Who Have Previously Been Treated in the P1-SAF-301 Clinical Study Evaluating the Tolerability and Safety of the Intracerebral Administration of SAF-301.
P2-SAF-301 is an open-label interventional study without administration of investigational product, evaluating the long-term safety and tolerability of intracerebral SAF-301 previously administered to 4 patients with Sanfilippo type A syndrome.
The primary objective is to collect additional safety and tolerability data on intracerebral SAF-301 previously administered to 4 patients with Sanfilippo type A syndrome.
The secondary objective is to further collect data to assess the effects of SAF-301 on neurological and psychological status, and potential biological markers.
- Being tested
- SAF-301
- Keywords
- Neurodegenerative diseases, Nervous systems diseases, Genetic diseases, Inborn, Metabolic diseases, Gene therapy, Adeno Associated Virus (AAV)
- Medical subject headings
- Mucopolysaccharidosis III, Neurodegenerative Diseases, Genetic Diseases, Inborn, Metabolic Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SAF-301Genetic
| Study group | Role | What they receive |
|---|---|---|
| SAF-301 | Experimental | Not described Receives: Genetic: SAF-301 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Checking of adverse events
Measured: until 60 months after SAF-301 administration
Secondary outcomes (3)
Information on neurological and cognitive changes based on clinical status, standardized neurocognitive and behavioral assessments
Measured: 5 years after the injection of SAF-301
Information on changes in potential biomarkers of the disease
Measured: 5 years after the injection of SAF-301
Information to further evaluation of immune response
Measured: 5 years after the injection of SAF-301
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Feb 2014 (estimated)
- Study startMay 2013
- Primary completionMay 2017
- Study completionJun 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Mucopolysaccharidosis Type III AStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- LYSOGENE
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Kumaran DEIVA, Dr, Principal investigator, AP-HP Hôpital Bicêtre
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02053064
- Sponsor's protocol ID
- P2-SAF-301
- Status last verified
- Jun 2017
- First submitted
- 16 Jan 2014
- Last updated
- 20 Jun 2017
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 20 Jun 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long-term Follow-up of Patients With Sanfilippo Type A Syndrome Who Have Previously Been Treated in the P1-SAF-301 Clinical Study Evaluating the Tolerability and Safety of the Intracerebral Administration of SAF-301.
- ClinicalTrials.gov ID
- NCT02053064
- Acronym / sponsor ID
- P2-SAF-301
- Keywords
- Neurodegenerative diseases, Nervous systems diseases, Genetic diseases, Inborn, Metabolic diseases, Gene therapy, Adeno Associated Virus (AAV)
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 4 participants (actual)
- Lead sponsor
- LYSOGENEIndustry (pharma, biotech, devices)
- Study start
- May 2013
- Primary completion
- May 2017 (actual)
- Study completion
- Jun 2017 (actual)
- First posted
- 3 Feb 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.
P2-SAF-301 is an open-label interventional study without administration of investigational product, evaluating the long-term safety and tolerability of intracerebral SAF-301 previously administered to 4 patients with Sanfilippo type A syndrome.
The primary objective is to collect additional safety and tolerability data on intracerebral SAF-301 previously administered to 4 patients with Sanfilippo type A syndrome.
The secondary objective is to further collect data to assess the effects of SAF-301 on neurological and psychological status, and potential biological markers.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| SAF-301 | Experimental | Genetic: SAF-301 |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Patients having completed the previous phase I/II study (P1-SAF-301),
- Family understanding the follow-up procedures and the informed consent,
- Patient's parents having signed the informed consent form.
Key exclusion criteria (1 listed)
- Participation in any other clinical trial.
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 |
|---|---|---|
| SAF-301 | Genetic | SAF-301 |
Primary outcome
Checking of adverse events
Time frame: until 60 months after SAF-301 administration
3 secondary outcomes
Information on neurological and cognitive changes based on clinical status, standardized neurocognitive and behavioral assessments
Time frame: 5 years after the injection of SAF-301
Information on changes in potential biomarkers of the disease
Time frame: 5 years after the injection of SAF-301
Information to further evaluation of immune response
Time frame: 5 years after the injection of SAF-301
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 20 Jun 2017.
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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.
Kumaran DEIVA, Dr principal investigator
AP-HP Hôpital Bicêtre
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing SAF-301 in people with Mucopolysaccharidosis Type III A and Sanfilippo Disease Type A, run by LYSOGENE at 1 site in France.
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 1 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 20 Jun 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 France. 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
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?
Checking of adverse events (time frame: until 60 months after SAF-301 administration). 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 20 Jun 2017; AzTrials last refreshed its copy on 7 Nov 2026.