Familial Amyloid Polyneuropathy (FAP) Clinical Trial
Official title:
Study of SOM0226 in Familial Amyloid Polyneuropathy (FAP) Patients and Asymptomatic Carriers to Evaluate Protein Stabilization Activity
| Verified date | November 2016 |
| Source | SOM Biotech SL |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | Spain: Spanish Agency of Medicines |
| Study type | Interventional |
Clinical proof of concept study to evaluate SOM0226 efficacy in TTR Amyloidosis.
| Status | Completed |
| Enrollment | 17 |
| Est. completion date | November 2015 |
| Est. primary completion date | November 2015 |
| Accepts healthy volunteers | Accepts Healthy Volunteers |
| Gender | Both |
| Age group | 18 Years and older |
| Eligibility |
Inclusion Criteria: - 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 Exclusion Criteria: - 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 |
Allocation: Non-Randomized, Endpoint Classification: Pharmacodynamics Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
| Country | Name | City | State |
|---|---|---|---|
| Spain | Hospital Vall d'Hebron | Barcelona | Catalonia |
| Lead Sponsor | Collaborator |
|---|---|
| SOM Biotech SL | Hospital Vall d'Hebron |
Spain,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | 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. | 24 hours and 32 hours | No |
| Secondary | Pharmacodynamics assessment | Determine the minimal molar ratio SOM0226:TTR that must be reached in plasma to confer maximal TTR stabilization | 24 hours and 32 hours | No |
| Secondary | Safety | Drug safety will be assessed by blood biochemistry and hematology analysis, patient exploration and adverse event reporting | 24 hours | Yes |