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Small Fiber NeuropathyPhase 2NCT03339336

Efficacy and Safety Study of BIIB074 in Participants With Small Fiber Neuropathy

A Phase 2 study testing BIIB074 in people with Small Fiber Neuropathy and Diabetes Mellitus, run by Biogen at 97 sites in 14 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

The primary objective of this study is to evaluate the efficacy of BIIB074 in treating pain experienced by participants with confirmed small fiber neuropathy (SFN) that is idiopathic or associated with diabetes mellitus. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Small Fiber Neuropathy, Diabetes Mellitus
What is being tested?
BIIB074
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
265 participants
Where
97 sitesin 14 countries
Study dates
31 May 2018to 12 Apr 2021
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • This study will be conducted in subjects who have had a diagnosis of at least probable SFN, length-dependent distribution, for 6 months and ≤10 years prior to screening, defined as a history of the symptoms and clinical signs based on discussions at the ACTTION CONCEPPT meeting on diagnosis of SFN, Washington, DC March 2018, and confirmed by intraepidermal nerve fiber density (IENFD) values, and weekly mean average daily pain (ADP) score of ≥5 and ≤9 on an 11-point Pain Intensity Numeric Rating Scale (PI-NRS) over the last 7 days of prior to the Screening visit.
  • In addition to these criteria, subjects with diabetes will be required to have HbA1c ≤11%, treated with oral hypoglycemics and/or subcutaneous insulin or diet, no evidence of ulcers, advanced retinopathy (defined as greater than State 3 [moderate non-proliferative diabetic retinopathy]) (DCCT/EDIC Research Group 2017), severe nephropathy, or clinically significant obstructive atherosclerotic disease or current class IV heart failure to be eligible for the study.

Who cannot join

  • Previous exposure to BIIB074 (formerly known as CNV1014802 or GSK1014802).
  • Use of capsaicin patch within 3 months prior to Screening.
  • Unable or unwilling to discontinue concomitant medications for SFN pain prior to Day 1.
  • Unable or unwilling to comply with the prohibited concomitant medication restrictions, including but not limited to UDP-glucuronosyltransferase (UGT) inducers and inhibitors, monoamine oxidase inhibitors (MAOIs), and Nav blockers.
  • Use of over-the-counter medications, vitamin and mineral supplements, herbal remedies (including St. John's wort), dietary supplements, or foods (including grapefruit juice) that affect and UGTs.
  • Unable or unwilling to discontinue medications that are P-glycoprotein substrates with a narrow therapeutic index, including but not limited to digoxin.
Show all 9Show fewer
  • History of hemophilia or Von Willebrand's disease, or use of anticoagulants that may result in bleeding risk during the skin biopsy.
  • Any contraindication, as determined by the Investigator, to performing a skin biopsy for intraepidermal nerve fiber analysis.
  • NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Trial locations and contacts

97 sites in 14 countries.

97 sites in 14 countries

Germany14 sites
  • Research Site

    Aschaffenburg, Bavaria

  • Zentrum fur Klinische Forschung

    Bad Homburg

  • Gemeinschaftspraxis für Neurologie

    Berlin

  • Clinical Research

    Böblingen, Baden-Wurttemberg

  • Gemeinschaftspraxis Diabeteszentrum Dortmund Dr.med. Klaus Busch

    Dortmund, North Rhine-Westphalia

  • Research Site

    Essen

  • Diabetologische Schwerpunktpraxis Harburg

    Hamburg

  • Research Site

    Künzing, Bavaria

  • Hausarzt- und Diabetologische Schwerpunktpraxis

    Lage, Saxony-Anhalt

  • Research Site

    Mainz

  • Clinical Research

    Münster, Nord Rhein Westfalen

  • Clinical Research

    Westerstede, Lower Saxony

  • DKD Helios Klinik Wiesbaden

    Wiesbaden

  • Research Site

    Würzburg

Other trials in Germany

France12 sites
  • Hôpital Ambroise Paré - Boulogne-Billancourt

    Boulogne-Billancourt

  • Research Site

    Brest, Finistere

  • CHU Clermond Ferrand - Hopital Gabriel Montpied

    Clermont-Ferrand, Puy De Dome

  • Research Site

    Corbeil-Essonnes

  • Hopital Henri Mondor

    Créteil

  • Research Site

    Le Creusot

  • Groupement Hospitalier Sud - Hôpital Bicêtre

    Le Kremlin-Bicêtre

  • Hopital Salengro - CHRU de Lille

    Lille, Nord

  • CHU Nice - Hôpital de l'Archet 1

    Nice

  • Hopital Lariboisiere

    Paris

  • CHU Saint Etienne - Hôpital Nord

    Saint Priest En Jarez, Pays de la Loire Region

  • Research Site

    Vénissieux, Rhone

Other trials in France

United Kingdom11 sites
  • Clinical Reseach

    Bath

  • Research Site

    Ipswich

  • Research Site

    Liverpool

  • Guy's Hospital

    London

  • King's College Hospital

    London

  • St Pancras Clinical Research

    London

  • The Royal London Hospital

    London

  • Research Site

    Manchester

  • John Radcliffe Hospital

    Oxford

  • Royal Hallamshire Hospital

    Sheffield, South Yorkshire

  • Research Site

    Swansea

Other trials in United Kingdom

Poland8 sites
  • Research Site

    Bydgoszcz

  • Research Site

    Chorzów

  • PRATIA MCM Kraków

    Krakow

  • Research Site

    Lublin

  • Research Site

    Oświęcim

  • Praktyka Lekarska Ewa Krzyzagorska

    Poznan

  • Research Site

    Warsaw

  • Regionalna Poradnia Diabetologiczna Zytkiewicz-Jaruga,Stasinska

    Wroclaw

Other trials in Poland

Spain8 sites
  • Research Site

    A Coruña

  • Hospital General Universitario de Alicante

    Alicante

  • Hospital Universitari de Bellvitge

    Barcelona

  • Research Site

    Barcelona

  • Hospital Universitario Reina Sofia

    Córdoba

  • Research Site

    Madrid

  • Hospital Universitario Puerta de Hierro Majadahonda

    Majadahonda

  • Hospital Universitari i Politecnic La Fe

    Valencia

Other trials in Spain

Canada7 sites
  • Research Site

    Greenfield Park, Quebec

  • Research Site

    Kingston

  • Research Site

    Montreal, Quebec

  • Recherche Médicale St-Jérôme Inc.

    Saint-Jérôme, Quebec

  • Toronto General Hospital

    Toronto, Ontario

  • Vancouver General Hospital

    Vancouver, British Columbia

  • Research Site

    Winnipeg

Other trials in Canada

Hungary7 sites
  • Research Site

    Baja

  • Bekes Megyei Kozponti Korhaz Dr. Rethy Pal Tagkorhaz

    Békéscsaba

  • UNO Medical Trials Kft.

    Budapest

  • Somogy Megyei Kaposi Mor Oktato Korhaz

    Kaposvár

  • Research Site

    Nyíregyháza

  • Research Site

    Pécs

  • Research Site

    Szeged

Other trials in Hungary

Czechia6 sites
  • Fakultni Nemocnice Brno

    Brno

  • Fakultni Nemocnice u sv. Anny v Brne

    Brno

  • Research Site

    Hradec Králové

  • Fakultni nemocnice Ostrava

    Ostrava-Poruba

  • Nemocnice Pardubickeho kraje a.s. Pardubicka nemocnice

    Pardubice

  • Fakultni nemocnice v Motole

    Prague

Other trials in Czechia

Italy6 sites
  • Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia (Presidio Spedali Civili)

    Brescia

  • Research Site

    Genova

  • Research Site

    Milan

  • Azienda Ospedaliero Univeraitaria Pisana

    Pisa

  • Università Campus Bio-Medico di Roma

    Roma

  • Research Site

    Telese Terme

Other trials in Italy

Bulgaria4 sites
  • Research Site

    Byala

  • UMHAT 'Dr Georgi Stranski' EAD

    Pleven

  • Research Site

    Plovdiv

  • Research Site

    Sofia

Other trials in Bulgaria

Denmark4 sites
  • Research Site

    Aarhus

  • Research Site

    Copenhagen

  • Research Site

    Herlev

  • OUH

    Odense

Other trials in Denmark

Greece4 sites
  • Research Site

    Athens

  • Research Site

    Heraklion

  • Research Site

    Pátrai

  • AHEPA General Hospital of Thessaloniki

    Thessaloniki

Other trials in Greece

Switzerland4 sites
  • CHUV - Centre Hospitalier Universitaire Vaudois

    Lausanne

  • Ospedale Regionale di Lugano

    Lugano

  • Kantonspital St. Gallen

    Sankt Gallen

  • Research Site

    Zurich

Other trials in Switzerland

Netherlands2 sites
  • Amsterdam UMC, Locatie AMC

    Amsterdam

  • Maastricht UMC+

    Maastricht

Other trials in Netherlands

About this study

Official title: A Phase 2 Placebo-Controlled, Double-Blind, Enriched Enrollment Randomized Withdrawal Study to Evaluate the Efficacy and Safety of BIIB074 (Vixotrigine) in Treating Pain Experienced by Subjects With Confirmed Small Fiber Neuropathy That is Idiopathic or Associated With Diabetes Mellitus

The primary objective of this study is to evaluate the efficacy of BIIB074 in treating pain experienced by participants with confirmed small fiber neuropathy (SFN) that is idiopathic or associated with diabetes mellitus. A secondary endpoint that relates to the primary objective is the change from Randomization to Week 12 of the double-blind period in mean average daily pain score.

The secondary objectives of this study are to evaluate the effect on worst pain, neuropathic pain quality, sleep interference due to pain, patient global impression, use of rescue medication, and SFN symptoms in participants treated with BIIB074; to investigate the safety and tolerability of BIIB074 in participants with SFN; and to characterize the pharmacokinetics (PK) of BIIB074 in participants with SFN.

Being tested
BIIB074, Placebo
Medical subject headings
Small Fiber Neuropathy, Diabetes Mellitus

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • BIIB074Drug

    Administered as specified in the treatment arm.

  • PlaceboDrug

    Administered as specified in the treatment arm.

Study groups in NCT03339336
Study groupRoleWhat they receive
BIIB074 350 mgExperimentalTaper Period (if applicable) from neuropathic pain medication, followed by a washout period, then BIIB074 350 mg tablets orally twice daily (BID) Open-Label Run-In Period, then BIIB074 350 mg tablets orally BID Double-Blind Treatment Period.
Receives: Drug: BIIB074
BIIB074 200 mgExperimentalTaper Period (if applicable) from neuropathic pain medication, followed by a washout period, then BIIB074 350 mg tablets orally twice daily (BID) Open-Label Run-In Period, then BIIB074 200 mg tablets orally BID Double-Blind Treatment Period.
Receives: Drug: BIIB074
PlaceboPlacebo comparatorTaper Period (if applicable) from neuropathic pain medication, followed by a washout period, then BIIB074 350 mg tablets orally twice daily (BID) Open-Label Run-In Period, then BIIB074 placebo-matching tablets orally BID Double-Blind Treatment Period.
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change from Baseline in Weekly Mean Average Daily Pain (ADP) Score

    Participants will rate their ADP using an 11-point Numerical Rating Scale (NRS) (0=no pain and 10=worst possible pain) and record their score in an electronic diary (eDiary). Weekly mean ADP scores for Baseline (the 5 days prior to the first dose of study treatment in the open-label run-in period) and Week 12 (the 7 days prior to the visit at the end of Week 12) will be derived from the ADP scores and calculated as the mean of the daily scores over the last 7 days.

    Measured: Baseline and Week 12 of the Double-Blind Period

  2. Change from Randomization in Weekly Mean ADP Score

    Participants will rate their ADP using an 11-point NRS (0=no pain and 10=worst possible pain) and record their score in an eDiary. Weekly mean ADP scores for Randomization (the 7 days prior to the first dose of study treatment in the double-blind period) and Week 12 (the 7 days prior to the visit at the end of Week 12) will be derived from the ADP scores and calculated as the mean of the daily scores over the last 7 days.

    Measured: Randomization and Week 12 of the Double-Blind Period

Secondary outcomes (11)
  1. Change from Baseline in Weekly Mean Worst Daily Pain (WDP) Score

    Participants will rate their WDP using an 11-point NRS (0=no pain and 10=worst possible pain) and record their score in an eDiary. Weekly mean WDP scores for Baseline (the 5 days prior to the first dose of study treatment in the open-label run-in period) and Week 12 (the 7 days prior to the visit at the end of Week 12) will be derived from the WDP scores and calculated as the mean of the daily scores over the last 7 days.

    Measured: Baseline and Week 12 of the Double-Blind Period

  2. Change from Baseline in Weekly Mean Sleep Interference Numerical Rating Scale (S-NRS)

    Participants will rate their S-NRS using an 11-point NRS (0=pain does not interfere with sleep and 10= pain completely interferes with sleep) and record their score in an eDiary. Weekly mean S-NRS scores for Baseline (the 5 days prior to the first dose of study treatment in the open-label run-in period) and Week 12 (the 7 days prior to the visit at the end of Week 12) will be derived from the daily S-NRS scores and calculated as the mean of the daily scores over the last 7 days.

    Measured: Baseline and Week 12 of the Double-Blind Period

  3. Change from Baseline in Neuropathic Pain Symptom Inventory (NPSI) Total Score and Sum Score

    Participant will use the NPSI questionnaire to rate different symptoms of neuropathic pain. The NPSI includes ten items related to different pain descriptors (e.g. burning and pressure) that are rated on an 11-point NRS (0=no symptoms to 10=worst symptoms imaginable). A score in each dimension and also a total score (from 0-100) is generated using data from the questionnaire.

    Measured: Baseline and Week 12 of Double-Blind Period

  4. Proportion of Participants with at least a 2-point Reduction from Baseline in Weekly Mean ADP

    Participants will rate their ADP using an 11-point NRS (0=no pain and 10=worst possible pain) and record their score in an electronic diary (eDiary). Weekly mean ADP scores for Baseline (the 5 days prior to the first dose of study treatment in the open-label run-in period) and Week 12 (the 7 days prior to the visit at the end of Week 12) will be derived from the ADP scores and calculated as the mean of the daily scores over the last 7 days.

    Measured: Baseline and Week 12 of the Double-Blind Period

  5. Proportion of Participants with at least a 30% Reduction from Baseline in Weekly Mean ADP

    Participants will rate their ADP using an 11-point NRS) (0=no pain and 10=worst possible pain) and record their score in an electronic diary (eDiary). Weekly mean ADP scores for Baseline (the 5 days prior to the first dose of study treatment in the open-label run-in period) and Week 12 (the 7 days prior to the visit at the end of Week 12) will be derived from the ADP scores and calculated as the mean of the daily scores over the last 7 days.

    Measured: Baseline and Week 12 of Double-Blind Period

  6. Mean Weekly Amount of Rescue Medication

    Use of rescue medication (paracetamol/acetaminophen) will be monitored and dosage will be recorded on a daily basis by the participant using an electronic Diary (eDiary). Mean weekly amount of rescue medication used for neuropathic pain during the double-blind period.

    Measured: Weekly from Baseline to Week 12 of the Double-Blind Period

  7. Patient Global Impression of Change (PGIC)

    PGIC is a 7-point self-administered scale that depicts changes in a participant's overall status. Participants will rate their change as "1=very much improved," "2=much improved," "3=minimally improved," "4=no change," "5=minimally worse," "6=much worse," or "7=very much worse."

    Measured: Week 12 of the Double-Blind Period

  8. Change from Baseline in Brief Pain Inventory-Short Form (BPI-SF) Interference Score

    The BPI-SF is a 7-item self-administered questionnaire that measures how much pain has interfered with daily functioning. Participants will rate the level of pain interference on daily functioning on an 11-point NRS where 0=does not interfere and 10=completely interferes.

    Measured: Baseline and Week 12 of the Double-Blind Period

  9. Percentage of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) During Double-Blind Period

    AEs: any sign, symptom, or diagnosis/disease that is unfavorable or unintended, that is new, or if pre-existing, worsens in participants administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. SAEs: an event that results in death; an event that, in the view of the investigator, places the participant at immediate risk of death (a life-threatening event); an outcome that results in a congenital anomaly/birth defect diagnosed in a child of a participant; an event that requires or prolongs inpatient hospitalization; an event that results in persistent or significant disability/incapacity. Any other medically important event that, in the opinion of the investigator, may jeopardize the participant or may require intervention to prevent one of the other outcomes listed in the definition above.

    Measured: Week 5 to Week 17

  10. Area Under the Concentration-time Curve at Steady State

    Measured: Week 1, 3, 5, 9, 13 and 17 prior to dosing and 1 to 2 hours after dosing, and at Day 123 (Follow-up clinic visit)

  11. Maximum Observed Concentration (Cmax) at Steady State

    Measured: Week 1, 3, 5, 9, 13 and 17 prior to dosing and 1 to 2 hours after dosing, and at Day 123 (Follow-up clinic visit)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted13 Nov 2017
  2. Study start31 May 2018
  3. Primary completion12 Apr 2021
  4. Study completion12 Apr 2021

Why it stopped: Sponsor decision to close study early; not due to safety concerns

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
265 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03339336
Sponsor's protocol ID
802NP206
Other IDs
2017-000991-27, 2017
Status last verified
Apr 2021
First submitted
8 Nov 2017
Last updated
5 May 2021
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No

Publications

  • Faber CG, Attal N, Lauria G, Dworkin RH, Freeman R, Dawson KT, Finnigan H, Hajihosseini A, Naik H, Serenko M, Morris CJ, Kotecha M. Efficacy and safety of vixotrigine in idiopathic or diabetes-associated painful small fibre neuropathy (CONVEY): a phase 2 placebo-controlled enriched-enrolment randomised withdrawal study. EClinicalMedicine. 2023 Apr 27;59:101971. doi: 10.1016/j.eclinm.2023.101971. eCollection 2023 May.

    PubMed 37152360

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing BIIB074 in people with Small Fiber Neuropathy and Diabetes Mellitus, run by Biogen at 97 sites in 14 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 2 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 5 May 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 97 sites in Germany, France, United Kingdom, Poland, Spain and 9 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was stopped early (reason given: Sponsor decision to close study early; not due to safety concerns). Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change from Baseline in Weekly Mean Average Daily Pain (ADP) Score (time frame: Baseline and Week 12 of the Double-Blind Period). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03339336, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 May 2021.

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