Mucopolysaccharidosis Type III APhase 1/2NCT01474343
Intracerebral Gene Therapy for Sanfilippo Type A Syndrome
A Phase 1/2 study testing SAF-301 in people with Mucopolysaccharidosis Type III A and Sanfilippo Disease Type A, run by LYSOGENE at 2 sites in France.
Trial summary
The clinical trial P1-SAF-301 is an open-label, single arm, monocentric, phase I/II clinical study evaluating the tolerance and the safety of intracerebral administration of adeno-associated viral vector serotype 10 carrying the human SGSH and SUMF1 cDNAs for the treatment of Sanfilippo type A syndrome The treatment… Read more about this study
- Who is it for?
- Ages 18 months to 6 years
- Conditions the study is for
- Mucopolysaccharidosis Type III A, Sanfilippo Disease Type A
- What is being tested?
- SAF-301
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 2 sitesFrance
- Study dates
- Aug 2011to May 2013
- 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 18 months to 6 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Age: 18 (eighteen) months to end of 6 (six) years
- Onset of clinical manifestations related to MPSIIIA during the first 5 years of life
- SGSH activity in peripheral blood cell and / or cultured fibroblast extracts of less than 10% of controls.
- Patient affiliated to the French social security or assimilated regimens
- Family understanding the procedure and the informed consent
- Signed informed consent
- Vital laboratory parameters within normal range
Who cannot join
- Presence of brain atrophy on inclusion MRI judged on a cortico-dural distance of more than 1cm
- No independent walking (Ability to walk without help)
- Any condition that would contraindicate permanently anaesthesia
- Any other permanent medical condition not related to MPSIIIA
- Any vaccination 1 month before investigational drug administration
- Intake of aspirin within one month
- Any medication aiming at modifying the natural course of MPSIIIA given during the 6 months before vector injection
- Any condition that would contraindicate treatment with Prograf®, Modigraf®, Cellcept® and Solupred®
Trial locations and contacts
2 sites in 1 country.
France2 sites
Hôpital Bicêtre - Assistance Publique des Hôpitaux de Paris
Le Kremlin-Bicêtre, 94275
Hôpital Necker, Assistance Publique des Hôpitaux de Paris
Paris, 75015
About this study
Official title: An Open-label, Single Arm, Monocentric, Phase I/II Clinical Study of Intracerebral Administration of Adeno-associated Viral Vector Serotype 10 Carrying the Human SGSH and SUMF1 cDNAs for the Treatment of Sanfilippo Type A Syndrome.
The clinical trial P1-SAF-301 is an open-label, single arm, monocentric, phase I/II clinical study evaluating the tolerance and the safety of intracerebral administration of adeno-associated viral vector serotype 10 carrying the human SGSH and SUMF1 cDNAs for the treatment of Sanfilippo type A syndrome The treatment plan consists on a direct injection of the investigational medicinal product SAF-301 to both sides of the brain through 6 image-guided tracks, with 2 deposits per track, in a single neurosurgical session.
The primary objective is to assess the tolerance and the safety associated to the proposed treatment through a one-year follow up.
Read more
The secondary objective is to collect data to define exploratory tests that could become evaluation criteria for further clinical phase III efficacy studies.
Four patients will be included in the clinical trial and will be followed during one year.
The enrollment and the follow-up of the patients will take place at Bicêtre Hospital. The Neurosurgery will be performed at Necker-Enfants Malades Hospital.
Safety will be evaluating on clinical, radiological and biological parameters.
- Being tested
- SAF-301
- Keywords
- Neurodegenerative Diseases, Nervous System Diseases, Genetic Diseases, Inborn, Metabolic Diseases, Gene Therapy, Adenovirus Associated Vector
- Medical subject headings
- Mucopolysaccharidosis III, Neurodegenerative Diseases, Nervous System 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
The treatment plan consists on a direct injection of the investigational medicinal product SAF-301 to both sides of the brain through 6 image-guided tracks, with 2 deposits per track, in a single neurosurgical session.
| 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.
Tolerance and safety
Measured by
- adverse events (by type and severity)
- clinical parameters (fever, seizure, headache, abnormal somnolence or lethargy, any new neurological symptoms),
- radiological parameters (on MRI, any sign of bleeding after surgery, any hypersignal on T2 weighted images or diffusion images that are not at the points of injection, and any necrotic area evaluated through T1-weighted and diffusion imaging as well as modification of lipids in spectroscopy)
- biological parameters (in particular anemia, leucopenia, thrombopenia, liver dysfunction)
Measured: during the one year follow-up
Secondary outcomes (1)
To collect data to define exploratory tests that could become evaluation criteria for further clinical phase III efficacy studies
- Brain MRI
- Neurocognitive/behavioral tests
- Biological markers on blood, urine and CSF
Measured: during the one year follow-up
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Nov 2011 (estimated)
- Study startAug 2011
- Primary completionMay 2013
- Study completionMay 2013
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
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01474343
- Sponsor's protocol ID
- P1-SAF-301
- Status last verified
- May 2014
- First submitted
- 10 Nov 2011
- Last updated
- 6 May 2014
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 6 May 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Single Arm, Monocentric, Phase I/II Clinical Study of Intracerebral Administration of Adeno-associated Viral Vector Serotype 10 Carrying the Human SGSH and SUMF1 cDNAs for the Treatment of Sanfilippo Type A Syndrome.
- ClinicalTrials.gov ID
- NCT01474343
- Acronym / sponsor ID
- P1-SAF-301
- Keywords
- Neurodegenerative Diseases, Nervous System Diseases, Genetic Diseases, Inborn, Metabolic Diseases, Gene Therapy, Adenovirus Associated Vector
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 4 participants (actual)
- Lead sponsor
- LYSOGENEIndustry (pharma, biotech, devices)
- Study start
- Aug 2011
- Primary completion
- May 2013 (actual)
- Study completion
- May 2013 (actual)
- First posted
- 18 Nov 2011 (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.
The clinical trial P1-SAF-301 is an open-label, single arm, monocentric, phase I/II clinical study evaluating the tolerance and the safety of intracerebral administration of adeno-associated viral vector serotype 10 carrying the human SGSH and SUMF1 cDNAs for the treatment of Sanfilippo type A syndrome The treatment plan consists on a direct injection of the investigational medicinal product SAF-301 to both sides of the brain through 6 image-guided tracks, with 2 deposits per track, in a single neurosurgical session.
The primary objective is to assess the tolerance and the safety associated to the proposed treatment through a one-year follow up.
The secondary objective is to collect data to define exploratory tests that could become evaluation criteria for further clinical phase III efficacy studies.
Four patients will be included in the clinical trial and will be followed during one year.
The enrollment and the follow-up of the patients will take place at Bicêtre Hospital. The Neurosurgery will be performed at Necker-Enfants Malades Hospital.
Safety will be evaluating on clinical, radiological and biological parameters.
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
- Ages
- 18 months to 6 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- Age: 18 (eighteen) months to end of 6 (six) years
- Onset of clinical manifestations related to MPSIIIA during the first 5 years of life
- SGSH activity in peripheral blood cell and / or cultured fibroblast extracts of less than 10% of controls.
- Patient affiliated to the French social security or assimilated regimens
- Family understanding the procedure and the informed consent
Key exclusion criteria (8 listed)
- Presence of brain atrophy on inclusion MRI judged on a cortico-dural distance of more than 1cm
- No independent walking (Ability to walk without help)
- Any condition that would contraindicate permanently anaesthesia
- Any other permanent medical condition not related to MPSIIIA
- Any vaccination 1 month before investigational drug administration
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-301The treatment plan consists on a direct injection of the investigational medicinal product SAF-301 to both sides of the brain through 6 image-guided tracks, with 2 deposits per track, in a single neurosurgical session. | Genetic | SAF-301 |
Primary outcome
Tolerance and safety
Time frame: during the one year follow-up
Measured by * adverse events (by type and severity) * clinical parameters (fever, seizure, headache, abnormal somnolence or lethargy, any new neurological symptoms), * radiological parameters (on MRI, any sign of bleeding after surgery, any hypersignal on T2 weighted images or diffusion images that are not at the points of injection, and any necrotic area evaluated through T1-weighted and diffusion imaging as well as modification of lipids in spectroscopy) * biological parameters (in particular anemia, leucopenia, thrombopenia, liver dysfunction)
1 secondary outcome
To collect data to define exploratory tests that could become evaluation criteria for further clinical phase III efficacy studies
Time frame: during the one year follow-up
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 6 May 2014.
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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.
2 sites 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 2 sites 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 7 inclusion and 8 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 6 May 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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?
Tolerance and safety (time frame: during the one year follow-up). 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 6 May 2014.