Familial Amyloid Polyneuropathy (FAP)Phase 1/2NCT02191826
Study of SOM0226 in Familial Amyloid Polyneuropathy
A Phase 1/2 study testing SOM0226 in people with Familial Amyloid Polyneuropathy (FAP), run by SOM Innovation Biotech SA at 1 site in Spain.
Trial summary
Clinical proof of concept study to evaluate SOM0226 efficacy in TTR Amyloidosis. Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Familial Amyloid Polyneuropathy (FAP)
- What is being tested?
- SOM0226
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 17 participants
- Where
- 1 siteSpain
- Study dates
- Jul 2014to Nov 2015
- 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 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Participant is willing and able to give informed consent for participation in the study
- Male or Female, aged 18 years or above at the time of consent
- Two cohorts defined by TTR variant (wild type: healthy volunteers; TTR mutant: asymptomatic carriers with documented mutation in TTR or diagnosed TTR-FAP patients (stage 1 or 2) not undergoing pharmacological treatment with Vyndaqel
- Body Mass Index (BMI) > 17.5 kg/m2
- Female participants of child bearing potential and male participants whose partner is of child bearing potential must be willing to ensure that they or their partner use effective contraception during the study and for 2 month thereafter
- Able (in the Investigators opinion) and willing to comply with all study requirements
Who cannot join
- Female participant who is pregnant, lactating or planning pregnancy during the course of the study
- Evidence of history of clinically significant hepatic disease
- An ALT or AST measurement > 2 times the ULN (Upper Limit of Normal)
- Scheduled elective surgery or other procedures requiring general anaesthesia during the study
- Donation of blood during the study or within the past 4 weeks
- Treatment (during the study or within the past 4 weeks) with a prescription or investigational drug for the treatment of TTR amyloidosis
- Treatment with NSAIDs (nonsteroidal antiinflammatory drug) during the study or within the past 4 weeks. The following NSAID are allowed: acetylsalicylic acid, etodolac, ibuprofen, indomethacin, ketoprofen, nabumetone, naproxen, nimesulide, piroxicam and sulindac
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Study of SOM0226 in Familial Amyloid Polyneuropathy (FAP) Patients and Asymptomatic Carriers to Evaluate Protein Stabilization Activity
Clinical proof of concept study to evaluate SOM0226 efficacy in TTR Amyloidosis.
Read the detailed description
This is an open label interventional Phase IIa proof of concept clinical trial designed in two phases separated by a washout period of 6 weeks (± 2 weeks) , to evaluate the TTR stabilization activity of SOM0226 in healthy volunteers (wild type), TTR-FAP patients and asymptomatic carriers.
- Phase A (24 hours): SOM0226 single dose
- Phase B (32 hours): SOM0226 multiple dose
- Conditions
- Familial Amyloid Polyneuropathy (FAP)
- Being tested
- SOM0226
- Keywords
- Transthyretin, TTR, Amyloidosis, SOM0226, Familiar Amyloid Polyneuropathy
- Medical subject headings
- Amyloid Neuropathies, Familial, Amyloidosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SOM0226Drug
Oral
| Study group | Role | What they receive |
|---|---|---|
| SOM0226 single dose | Experimental | Not described Receives: Drug: SOM0226 |
| SOM0226 multiple doses | Experimental | Not described Receives: Drug: SOM0226 |
What is being measured
The primary outcome is the main question the study is designed to answer.
TTR stabilization
TTR stabilization will be measured by determining the concentration of tetrameric TTR after denaturation. The ratio of TTR tetramer after denaturation / TTR tetramer before denaturation of plasma samples from treated subjects will be compared to their baseline ratio. TTR stabilization will be determined as percentage of the baseline ratio.
Measured: 24 hours and 32 hours
Secondary outcomes (2)
Pharmacodynamics assessment
Determine the minimal molar ratio SOM0226:TTR that must be reached in plasma to confer maximal TTR stabilization
Measured: 24 hours and 32 hours
Safety
Drug safety will be assessed by blood biochemistry and hematology analysis, patient exploration and adverse event reporting
Measured: 24 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Jul 2014 (estimated)
- Study startJul 2014
- Primary completionNov 2015
- Study completionNov 2015
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 Familial Amyloid Polyneuropathy (FAP)Studies 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
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 17 participants (actual)
Sponsor and investigators
- Lead sponsor
- SOM Innovation Biotech SA
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Hospital Vall d'Hebron
- Responsible party
- Sponsor
- Investigators
- Josep Gámez, MD, Principal investigator, Hospital Vall d'Hebron
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02191826
- Sponsor's protocol ID
- SOMCT01-C
- Other IDs
- 2014-001586-27, 2014
- Status last verified
- Nov 2016
- First submitted
- 9 Jul 2014
- Last updated
- 30 Nov 2016
- Results posted
- No
- Data monitoring committee
- No
Publications
Gamez J, Salvado M, Reig N, Sune P, Casasnovas C, Rojas-Garcia R, Insa R. Transthyretin stabilization activity of the catechol-O-methyltransferase inhibitor tolcapone (SOM0226) in hereditary ATTR amyloidosis patients and asymptomatic carriers: proof-of-concept study. Amyloid. 2019 Jun;26(2):74-84. doi: 10.1080/13506129.2019.1597702. Epub 2019 May 23.
PubMed 31119947
Study at a glance
From the registry record, last updated by the sponsor on 30 Nov 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of SOM0226 in Familial Amyloid Polyneuropathy (FAP) Patients and Asymptomatic Carriers to Evaluate Protein Stabilization Activity
- ClinicalTrials.gov ID
- NCT02191826
- Acronym / sponsor ID
- SOMCT01-C
- Conditions
- Familial Amyloid Polyneuropathy (FAP)
- Keywords
- Transthyretin, TTR, Amyloidosis, SOM0226, Familiar Amyloid Polyneuropathy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 17 participants (actual)
- Lead sponsor
- SOM Innovation Biotech SAIndustry (pharma, biotech, devices)
- Collaborators
- Hospital Vall d'Hebron
- Study start
- Jul 2014
- Primary completion
- Nov 2015 (actual)
- Study completion
- Nov 2015 (actual)
- First posted
- 16 Jul 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.
Clinical proof of concept study to evaluate SOM0226 efficacy in TTR Amyloidosis.
Detailed description
This is an open label interventional Phase IIa proof of concept clinical trial designed in two phases separated by a washout period of 6 weeks (± 2 weeks) , to evaluate the TTR stabilization activity of SOM0226 in healthy volunteers (wild type), TTR-FAP patients and asymptomatic carriers.
- Phase A (24 hours): SOM0226 single dose
- Phase B (32 hours): SOM0226 multiple dose
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| SOM0226 single dose | Experimental | Drug: SOM0226 |
| SOM0226 multiple doses | Experimental | Drug: SOM0226 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Participant is willing and able to give informed consent for participation in the study
- Male or Female, aged 18 years or above at the time of consent
- Two cohorts defined by TTR variant (wild type: healthy volunteers; TTR mutant: asymptomatic carriers with documented mutation in TTR or diagnosed TTR-FAP patients (stage 1 or 2) not undergoing pharmacological treatment with Vyndaqel
- Body Mass Index (BMI) > 17.5 kg/m2
- Female participants of child bearing potential and male participants whose partner is of child bearing potential must be willing to ensure that they or their partner use effective contraception during the study and for 2 month thereafter
Key exclusion criteria (7 listed)
- Female participant who is pregnant, lactating or planning pregnancy during the course of the study
- Evidence of history of clinically significant hepatic disease
- An ALT or AST measurement > 2 times the ULN (Upper Limit of Normal)
- Scheduled elective surgery or other procedures requiring general anaesthesia during the study
- Donation of blood during the study or within the past 4 weeks
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 |
|---|---|---|
| SOM0226Oral | Drug | SOM0226 multiple doses, SOM0226 single dose |
Primary outcome
TTR stabilization
Time frame: 24 hours and 32 hours
TTR stabilization will be measured by determining the concentration of tetrameric TTR after denaturation. The ratio of TTR tetramer after denaturation / TTR tetramer before denaturation of plasma samples from treated subjects will be compared to their baseline ratio. TTR stabilization will be determined as percentage of the baseline ratio.
2 secondary outcomes
Pharmacodynamics assessment
Time frame: 24 hours and 32 hours
Safety
Time frame: 24 hours
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 30 Nov 2016.
- Derived from this study: PubMed 31119947Gamez J, Salvado M, Reig N, Sune P, Casasnovas C, Rojas-Garcia R, Insa R. Transthyretin stabilization activity of the catechol-O-methyltransferase inhibitor tolcapone (SOM0226) in hereditary ATTR amyloidosis patients and asymptomatic carriers: proof-of-concept study. Amyloid. 2019 Jun;26(2):74-84. doi: 10.1080/13506129.2019.1597702. Epub 2019 May 23.
- Related InfoLink provided by the sponsor.
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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.
Josep Gámez, MD principal investigator
Hospital Vall d'Hebron
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 SOM0226 in people with Familial Amyloid Polyneuropathy (FAP), run by SOM Innovation Biotech SA at 1 site in Spain.
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 7 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 30 Nov 2016. 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 Spain. 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?
TTR stabilization (time frame: 24 hours and 32 hours). 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 NCT02191826, 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 30 Nov 2016.