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Study of SOM0226 in Familial Amyloid Polyneuropathy

NCT ID: NCT02191826

Last Updated: 2016-11-30

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

17 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-07-31

Study Completion Date

2015-11-30

Brief Summary

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Clinical proof of concept study to evaluate SOM0226 efficacy in TTR Amyloidosis.

Detailed Description

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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

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Familial Amyloid Polyneuropathy (FAP)

Keywords

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Transthyretin TTR Amyloidosis SOM0226 Familiar Amyloid Polyneuropathy

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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SOM0226 single dose

Group Type EXPERIMENTAL

SOM0226

Intervention Type DRUG

Oral

SOM0226 multiple doses

Group Type EXPERIMENTAL

SOM0226

Intervention Type DRUG

Oral

Interventions

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SOM0226

Oral

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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Hospital Vall d'Hebron

OTHER

Sponsor Role collaborator

SOM Innovation Biotech SA

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Josep Gámez, MD

Role: PRINCIPAL_INVESTIGATOR

Hospital Vall d'Hebron

Locations

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Hospital Vall d'Hebron

Barcelona, Catalonia, Spain

Site Status

Countries

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Spain

References

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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.

Reference Type DERIVED
PMID: 31119947 (View on PubMed)

Related Links

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http://www.sombiotech.com

Related Info

Other Identifiers

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2014-001586-27

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

SOMCT01-C

Identifier Type: -

Identifier Source: org_study_id