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Study of SOM0226 in Familial Amyloid Polyneuropathy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02191826
Recruitment Status : Completed
First Posted : July 16, 2014
Last Update Posted : November 30, 2016
Hospital Vall d'Hebron
Information provided by (Responsible Party):
SOM Biotech SL

Tracking Information
First Submitted Date  ICMJE July 9, 2014
First Posted Date  ICMJE July 16, 2014
Last Update Posted Date November 30, 2016
Study Start Date  ICMJE July 2014
Actual Primary Completion Date November 2015   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 14, 2014)
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.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: July 14, 2014)
  • Pharmacodynamics assessment [ Time Frame: 24 hours and 32 hours ]
    Determine the minimal molar ratio SOM0226:TTR that must be reached in plasma to confer maximal TTR stabilization
  • Safety [ Time Frame: 24 hours ]
    Drug safety will be assessed by blood biochemistry and hematology analysis, patient exploration and adverse event reporting
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
Descriptive Information
Brief Title  ICMJE Study of SOM0226 in Familial Amyloid Polyneuropathy
Official Title  ICMJE Study of SOM0226 in Familial Amyloid Polyneuropathy (FAP) Patients and Asymptomatic Carriers to Evaluate Protein Stabilization Activity
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
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Familial Amyloid Polyneuropathy (FAP)
Intervention  ICMJE Drug: SOM0226
Study Arms  ICMJE
  • Experimental: SOM0226 single dose
    Intervention: Drug: SOM0226
  • Experimental: SOM0226 multiple doses
    Intervention: Drug: SOM0226
Publications * Gamez J, Salvadó M, Reig N, Suñé 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.

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: November 29, 2016)
Original Estimated Enrollment  ICMJE
 (submitted: July 14, 2014)
Actual Study Completion Date  ICMJE November 2015
Actual Primary Completion Date November 2015   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE Yes
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Spain
Removed Location Countries  
Administrative Information
NCT Number  ICMJE NCT02191826
Other Study ID Numbers  ICMJE SOMCT01-C
2014-001586-27 ( EudraCT Number )
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party SOM Biotech SL
Study Sponsor  ICMJE SOM Biotech SL
Collaborators  ICMJE Hospital Vall d'Hebron
Investigators  ICMJE
Principal Investigator: Josep Gámez, MD Hospital Vall d'Hebron
PRS Account SOM Biotech SL
Verification Date November 2016

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP