Peripheral Arterial Occlusive DiseasePhase 1NCT01157143
Gene Expression and Tolerability Study of NV1FGF in Patients With Peripheral Artery Occlusive Disease Planned to Undergo Major Amputation
A Phase 1 study testing XRP0038 (NV1FGF) in people with Peripheral Arterial Occlusive Disease, run by Sanofi at 2 sites in 2 countries.
Trial summary
The primary objective is to evaluate the transgene expression (synthesis of FGF-1 mRNA) in injected tissue, at injection site, after Intra Muscular (IM) administration of increasing single doses of NV1FGF. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Peripheral Arterial Occlusive Disease
- What is being tested?
- XRP0038 (NV1FGF)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 2 sitesin 2 countries
- Study dates
- Jan 2002to Oct 2003
- Phase
- Phase 1
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Subjects with prior decision for amputation above the ankle because of severe PAOD
- Males or females above 18 years
- Females must be either:
- Non pregnant, non lactating , having practicing a medically accepted method of birth control for more than 2 months prior screening visit;
- or surgically sterilized (tubal ligation or hysterectomy)
- or post menopausal for at least one year
Who cannot join
- Subjects with urgent need for amputation that cannot await until completion of the screening period (up to 4 weeks), added to the minimum required 48 hours between study test medication administration and tissue sample collection
- Previous or current history of malignant disease (subjects with successful tumor resection more than 5 years -without any recurrence- prior to study start could be enrolled)
- Abnormal Chest X-ray or mammography with suspicion of malignant disease
- Positive stool hemoccult (except in case of hemorrhoids or any other identified cause with no malignancy origin)
- Men with positive Prostate Specific Antigen (PSA) (above 2.5 ng/ml in subjects < 50 years and above 5 ng/ml in subjects above 50 years)
- Females with Papanicolaou smear of Class IV or Class V characterization
Show all 16Show fewer
- Serious concomitant medical conditions not adequately controlled
- Alcohol or drug abuse
- Active proliferate retinopathy defined by the presence of new vessel formation and scarring
- Participation in clinical trials of non-approved experimental agents within four weeks before study entry;
- Positive serology for HIV1 or 2
- Creatinine above 2.0 mg/dl (176 µmol/l), unless the subject is on hemodialysis / peritoneal dialysis and diagnosed with complete and irreversible renal failure or end-stage renal disease (ESRD)
- Subjects who had a stroke or a neurological deficit presumably due to a stroke, within 3 months prior to study treatment (Amendment #1)
- Alpha-fetoprotein (AFP) in serum > 15 µg/l, unless liver ultrasound ruled out any malignant disease
- Positive serology for hepatitis B or C, unless liver ultrasound ruled out any malignant disease.
- The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
About this study
Official title: Phase I, Double Blind, Parallel-Group, Multi-center, Gene Expression (Synthesis of FGF-1 mRNA) and Tolerability Study of Increasing Single Dose of NV1FGF Administered by Intra-Muscular Injection in Subjects With Severe Peripheral Artery Occlusive Disease (PAOD) Planned to Undergo Amputation Above the Ankle
The primary objective is to evaluate the transgene expression (synthesis of FGF-1 mRNA) in injected tissue, at injection site, after Intra Muscular (IM) administration of increasing single doses of NV1FGF.
Secondary objectives :
- To evaluate the safety and tolerability of IM administration of increasing single doses of NV1FGF
- To evaluate the transgene expression (FGF-1 protein) in injected tissues (injection site and remote site)
- To evaluate the presence of FGF-1 receptors in injected tissues (injection site and remote site)
- To evaluate the NV1FGF biodistribution in injected tissues (injection site and remote site), in multiple organs/tissues when appropriate, and plasma
- To evaluate the transgene expression (synthesis of FGF-1 mRNA) in injected tissue at remote site
- To collect data from plasma NV1FGF pharmacokinetics
- To evaluate healing of the amputation site
Read the detailed description
Screening of 1 to 4 weeks before study drug administration; Single study drug administration 3 to 8 days before planned amputation; 6 months of follow up
- Conditions
- Peripheral Arterial Occlusive Disease
- Being tested
- XRP0038 (NV1FGF)
- Medical subject headings
- Peripheral Arterial Occlusive Disease 1
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
XRP0038 (NV1FGF)Drug
Pharmaceutical form : solution
Route of administration : intramuscular
| Study group | Role | What they receive |
|---|---|---|
| NV1FGF 500 μg | Experimental | 8 intramuscular injections for a total of 500 μg administered in one single administration 3 to 8 days before major amputation Receives: Drug: XRP0038 (NV1FGF) |
| NV1FGF 2000 μg | Experimental | 8 intramuscular injections for a total of 2000 μg administered in one single administration 3 to 8 days before major amputation Receives: Drug: XRP0038 (NV1FGF) |
| NV1FGF 4000 μg | Experimental | 8 intramuscular injections for a total of 4000 μg administered in one single administration 3 to 8 days before major amputation Receives: Drug: XRP0038 (NV1FGF) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Detection of FGF-1 mRNA (by real-time RT-PCR) in injected tissues at injection site
Measured: 3 to 8 days after amputation
Secondary outcomes (5)
Detection of FGF-1 protein (by immunohistochemistry) in injected tissues (injection and remote site)
Measured: 3 to 8 days after amputation
Detection of FGF-1 mRNA (by real-time RT-PCR) in injected tissues at remote site
Measured: 3 to 8 days after amputation
Detection of FGF-1 receptor (by immunohistochemistry) in injected tissues (injection and remote site)
Measured: 3 to 8 days after amputation
Detection of NV1FGF (by real-time PCR) in injected tissues (injection and remote site), in multiple organs/tissues when appropriate, and in plasma
Measured: 2 months
Evaluation of healing of the amputation site
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jul 2010 (estimated)
- Study startJan 2002
- Primary completionOct 2003
- Study completionOct 2003
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Peripheral Arterial Occlusive DiseaseStudies of the same condition, open and under way.
- More trials run by SanofiThe sponsor's other registered studies.
- 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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sanofi
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- International Clinical Development Clinical Study Director, Study director, Sanofi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01157143
- Sponsor's protocol ID
- TED10105
- Other IDs
- PM105
- Status last verified
- Jul 2010
- First submitted
- 2 Jul 2010
- Last updated
- 5 Jul 2010
- Results posted
- No
- Data monitoring committee
- No
Publications
Niebuhr A, Henry T, Goldman J, Baumgartner I, van Belle E, Gerss J, Hirsch AT, Nikol S. Long-term safety of intramuscular gene transfer of non-viral FGF1 for peripheral artery disease. Gene Ther. 2012 Mar;19(3):264-70. doi: 10.1038/gt.2011.85. Epub 2011 Jun 30.
PubMed 21716303
Study at a glance
From the registry record, last updated by the sponsor on 5 Jul 2010. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I, Double Blind, Parallel-Group, Multi-center, Gene Expression (Synthesis of FGF-1 mRNA) and Tolerability Study of Increasing Single Dose of NV1FGF Administered by Intra-Muscular Injection in Subjects With Severe Peripheral Artery Occlusive Disease (PAOD) Planned to Undergo Amputation Above the Ankle
- ClinicalTrials.gov ID
- NCT01157143
- Acronym / sponsor ID
- TED10105
- Conditions
- Peripheral Arterial Occlusive Disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- SanofiIndustry (pharma, biotech, devices)
- Study start
- Jan 2002
- Primary completion
- Oct 2003 (actual)
- Study completion
- Oct 2003 (actual)
- First posted
- 5 Jul 2010 (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 primary objective is to evaluate the transgene expression (synthesis of FGF-1 mRNA) in injected tissue, at injection site, after Intra Muscular (IM) administration of increasing single doses of NV1FGF.
Secondary objectives :
- To evaluate the safety and tolerability of IM administration of increasing single doses of NV1FGF
- To evaluate the transgene expression (FGF-1 protein) in injected tissues (injection site and remote site)
- To evaluate the presence of FGF-1 receptors in injected tissues (injection site and remote site)
- To evaluate the NV1FGF biodistribution in injected tissues (injection site and remote site), in multiple organs/tissues when appropriate, and plasma
- To evaluate the transgene expression (synthesis of FGF-1 mRNA) in injected tissue at remote site
- To collect data from plasma NV1FGF pharmacokinetics
- To evaluate healing of the amputation site
Detailed description
Screening of 1 to 4 weeks before study drug administration; Single study drug administration 3 to 8 days before planned amputation; 6 months of follow up
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| NV1FGF 500 μg | Experimental | Drug: XRP0038 (NV1FGF)8 intramuscular injections for a total of 500 μg administered in one single administration 3 to 8 days before major amputation |
| NV1FGF 2000 μg | Experimental | Drug: XRP0038 (NV1FGF)8 intramuscular injections for a total of 2000 μg administered in one single administration 3 to 8 days before major amputation |
| NV1FGF 4000 μg | Experimental | Drug: XRP0038 (NV1FGF)8 intramuscular injections for a total of 4000 μg administered in one single administration 3 to 8 days before major amputation |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Subjects with prior decision for amputation above the ankle because of severe PAOD
- Males or females above 18 years
- Females must be either:
Key exclusion criteria (16 listed)
- Subjects with urgent need for amputation that cannot await until completion of the screening period (up to 4 weeks), added to the minimum required 48 hours between study test medication administration and tissue sample collection
- Previous or current history of malignant disease (subjects with successful tumor resection more than 5 years -without any recurrence- prior to study start could be enrolled)
- Abnormal Chest X-ray or mammography with suspicion of malignant disease
- Positive stool hemoccult (except in case of hemorrhoids or any other identified cause with no malignancy origin)
- Men with positive Prostate Specific Antigen (PSA) (above 2.5 ng/ml in subjects < 50 years and above 5 ng/ml in subjects above 50 years)
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 |
|---|---|---|
| XRP0038 (NV1FGF)Pharmaceutical form : solution Route of administration : intramuscular | Drug | NV1FGF 2000 μg, NV1FGF 4000 μg, NV1FGF 500 μg |
Primary outcome
Detection of FGF-1 mRNA (by real-time RT-PCR) in injected tissues at injection site
Time frame: 3 to 8 days after amputation
5 secondary outcomes
Detection of FGF-1 protein (by immunohistochemistry) in injected tissues (injection and remote site)
Time frame: 3 to 8 days after amputation
Detection of FGF-1 mRNA (by real-time RT-PCR) in injected tissues at remote site
Time frame: 3 to 8 days after amputation
Detection of FGF-1 receptor (by immunohistochemistry) in injected tissues (injection and remote site)
Time frame: 3 to 8 days after amputation
Detection of NV1FGF (by real-time PCR) in injected tissues (injection and remote site), in multiple organs/tissues when appropriate, and in plasma
Time frame: 2 months
Evaluation of healing of the amputation site
Time frame: 6 months
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 5 Jul 2010.
- Derived from this study: PubMed 21716303Niebuhr A, Henry T, Goldman J, Baumgartner I, van Belle E, Gerss J, Hirsch AT, Nikol S. Long-term safety of intramuscular gene transfer of non-viral FGF1 for peripheral artery disease. Gene Ther. 2012 Mar;19(3):264-70. doi: 10.1038/gt.2011.85. Epub 2011 Jun 30.
Related trials for Peripheral Arterial Occlusive Disease
Active, not recruiting
SUCCESS (SelUtion, Safety, effiCaCy, hEalth economicS and promS) PTA Study
Peripheral Artery Disease
RecruitingPhase 3
Reducing Adverse Vascular Outcomes With Factor XI Inhibition in Adult Participants With Peripheral Artery Disease
Peripheral Artery Disease (PAD)
Recruiting
The THOR IDE Study
Peripheral Artery Disease, Peripheral Artery Stenosis
Not yet recruiting
Daily Stretching to Improve Walking in Older Patients With Peripheral Artery Disease: The STRETCH PAD Study
Peripheral Artery Disease (PAD), Intermittent Claudication
Enrolling by invitationEarly Phase 1
Truway Diagnostic Tools in Primary Care
Chronic Disease, Type 2 Diabetes Mellitus (T2DM), Obesity, Overweight
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.
International Clinical Development Clinical Study Director study director
Sanofi
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing XRP0038 (NV1FGF) in people with Peripheral Arterial Occlusive Disease, run by Sanofi at 2 sites in 2 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 16 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 5 Jul 2010. 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 United States, Switzerland. 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 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.
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?
Detection of FGF-1 mRNA (by real-time RT-PCR) in injected tissues at injection site (time frame: 3 to 8 days after amputation). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01157143, which also lists 1 linked publication.
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 5 Jul 2010.