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Study of SOM0226 in Familial Amyloid Polyneuropathy

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ClinicalTrials.gov Identifier: NCT02191826
Recruitment Status : Completed
First Posted : July 16, 2014
Last Update Posted : November 30, 2016
Sponsor:
Collaborator:
Hospital Vall d'Hebron
Information provided by (Responsible Party):
SOM Biotech SL

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

Condition or disease Intervention/treatment Phase
Familial Amyloid Polyneuropathy (FAP) Drug: SOM0226 Phase 1 Phase 2

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

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 17 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Study of SOM0226 in Familial Amyloid Polyneuropathy (FAP) Patients and Asymptomatic Carriers to Evaluate Protein Stabilization Activity
Study Start Date : July 2014
Actual Primary Completion Date : November 2015
Actual Study Completion Date : November 2015


Arm Intervention/treatment
Experimental: SOM0226 single dose Drug: SOM0226
Oral

Experimental: SOM0226 multiple doses Drug: SOM0226
Oral




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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

  2. Safety [ Time Frame: 24 hours ]
    Drug safety will be assessed by blood biochemistry and hematology analysis, patient exploration and adverse event reporting



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02191826


Locations
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Spain
Hospital Vall d'Hebron
Barcelona, Catalonia, Spain, 08035
Sponsors and Collaborators
SOM Biotech SL
Hospital Vall d'Hebron
Investigators
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Principal Investigator: Josep Gámez, MD Hospital Vall d'Hebron

Additional Information:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: SOM Biotech SL
ClinicalTrials.gov Identifier: NCT02191826     History of Changes
Other Study ID Numbers: SOMCT01-C
2014-001586-27 ( EudraCT Number )
First Posted: July 16, 2014    Key Record Dates
Last Update Posted: November 30, 2016
Last Verified: November 2016

Keywords provided by SOM Biotech SL:
Transthyretin
TTR
Amyloidosis
SOM0226
Familiar Amyloid Polyneuropathy

Additional relevant MeSH terms:
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Amyloidosis
Polyneuropathies
Amyloid Neuropathies
Amyloid Neuropathies, Familial
Proteostasis Deficiencies
Metabolic Diseases
Peripheral Nervous System Diseases
Neuromuscular Diseases
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Amyloidosis, Familial
Metabolism, Inborn Errors
Tolcapone
Antiparkinson Agents
Anti-Dyskinesia Agents
Catechol O-Methyltransferase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action