Study of SOM0226 in Familial Amyloid Polyneuropathy

NCT02191826 | Completed Phase 1

• 2 other identifiers: SOMCT01-C2014-001586-27
• Study Type: interventional
• Target: 17
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Familial Amyloid Polyneuropathy (FAP)

Keywords

Transthyretin; TTR; Amyloidosis; SOM0226; Familiar Amyloid Polyneuropathy

Outcome Measures

Primary Outcomes (1)

• 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

Secondary Outcomes (2)

• Pharmacodynamics assessment

  24 hours and 32 hours

• Safety

  24 hours

Study Arms (2)

SOM0226 single dose

EXPERIMENTAL

Drug: SOM0226

SOM0226 multiple doses

EXPERIMENTAL

Drug: SOM0226

Interventions

SOM0226 DRUG

Oral

SOM0226 multiple doses; SOM0226 single dose

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• SOM Innovation Biotech SA: lead
• Hospital Vall d'Hebron: collaborator

Study Sites (1)

Hospital Vall d'Hebron

Barcelona, Catalonia, 08035, Spain

Location

Related Publications (1)

• 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.

  PMID: 31119947 DERIVED

Related Links

• Related Info

MeSH Terms

Conditions

Amyloid Neuropathies, Familial; Amyloidosis

Interventions

Tolcapone

Condition Hierarchy (Ancestors)

Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Nervous System Diseases; Amyloid Neuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Amyloidosis, Familial; Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases; Proteostasis Deficiencies

Intervention Hierarchy (Ancestors)

Benzophenones; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Organic Chemicals; Nitrophenols; Phenols; Ketones; Nitro Compounds

Study Officials

• Josep Gámez, MD

  Hospital Vall d'Hebron

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 9, 2014
• First Posted: July 16, 2014
• Study Start: July 1, 2014
• Primary Completion: November 1, 2015
• Study Completion: November 1, 2015
• Last Updated: November 30, 2016

Record last verified: 2016-11

Locations

ES (1)