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Study of SOM0226 in Familial Amyloid Polyneuropathy

Primary Purpose

Familial Amyloid Polyneuropathy (FAP)

Status

Completed

Phase

Phase 1

Locations

Spain

Study Type

Interventional

Intervention

SOM0226

About this trial

This is an interventional treatment trial for Familial Amyloid Polyneuropathy (FAP) focused on measuring Transthyretin, TTR, Amyloidosis, SOM0226, Familiar Amyloid Polyneuropathy

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesAccepts Healthy Volunteers

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

Sites / Locations

Hospital Vall d'Hebron

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Arm Label

SOM0226 single dose

SOM0226 multiple doses

Arm Description

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

Pharmacodynamics assessment

Determine the minimal molar ratio SOM0226:TTR that must be reached in plasma to confer maximal TTR stabilization

Safety

Drug safety will be assessed by blood biochemistry and hematology analysis, patient exploration and adverse event reporting

Full Information

NCT ID

NCT02191826

First Posted

July 9, 2014

Last Updated

November 29, 2016

Sponsor

SOM Innovation Biotech SA

Collaborators

Hospital Vall d'Hebron

1. Study Identification

Unique Protocol Identification Number

NCT02191826

Brief Title

Study of SOM0226 in Familial Amyloid Polyneuropathy

Official Title

Study of SOM0226 in Familial Amyloid Polyneuropathy (FAP) Patients and Asymptomatic Carriers to Evaluate Protein Stabilization Activity

Study Type

Interventional

2. Study Status

Record Verification Date

November 2016

Overall Recruitment Status

Completed

Study Start Date

July 2014 (undefined)

Primary Completion Date

November 2015 (Actual)

Study Completion Date

November 2015 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

SOM Innovation Biotech SA

Collaborators

Hospital Vall d'Hebron

4. Oversight

Data Monitoring Committee

5. Study Description

Brief Summary

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Familial Amyloid Polyneuropathy (FAP)

Keywords

Transthyretin, TTR, Amyloidosis, SOM0226, Familiar Amyloid Polyneuropathy

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1, Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

17 (Actual)

8. Arms, Groups, and Interventions

Arm Title

SOM0226 single dose

Arm Type

Experimental

Arm Title

SOM0226 multiple doses

Arm Type

Experimental

Intervention Type

Drug

Intervention Name(s)

SOM0226

Intervention Description

Oral

Primary Outcome Measure Information:

Title

TTR stabilization

Description

Time Frame

24 hours and 32 hours

Secondary Outcome Measure Information:

Title

Pharmacodynamics assessment

Description

Determine the minimal molar ratio SOM0226:TTR that must be reached in plasma to confer maximal TTR stabilization

Time Frame

24 hours and 32 hours

Title

Safety

Description

Drug safety will be assessed by blood biochemistry and hematology analysis, patient exploration and adverse event reporting

Time Frame

24 hours

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Josep Gámez, MD

Organizational Affiliation

Hospital Vall d'Hebron

Official's Role

Principal Investigator

Facility Information:

Facility Name

Hospital Vall d'Hebron

City

Barcelona

State/Province

Catalonia

ZIP/Postal Code

08035

Country

Spain

12. IPD Sharing Statement

Citations:

PubMed Identifier

31119947

Citation

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.

Results Reference

derived

Links:

URL

http://www.sombiotech.com

Description

Related Info

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Study of SOM0226 in Familial Amyloid Polyneuropathy

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