AzTrials
AzTrials

Charcot-Marie-Tooth DiseasePhase 3PREMIERNCT04762758

Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients

A Phase 3 study testing (RS)-baclofen, naltrexone hydrochloride and D-sorbitol in people with Charcot-Marie-Tooth Disease, run by Pharnext S.C.A. at 50 sites in 9 countries.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
Search all trials

Trial summary

The study will consist of 2 periods: Double-blind Treatment and Open-Label Extension(OLE) Period. -Double-blind Treatment Period - This will be randomized, double-blind, placebo-controlled part of the study which will be conducted in parallel groups, ie,1 group receiving the active treatment (PXT3003) and the other… Read more about this study

Who is it for?
Ages 16 to 65 years
Conditions the study is for
Charcot-Marie-Tooth Disease
What is being tested?
(RS)-baclofen, naltrexone hydrochloride and D-sorbitol
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
350 participants
Where
50 sitesin 9 countries
Study dates
30 Mar 2021to 19 Apr 2024
Phase
Phase 3
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 16 to 65 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Male and non-pregnant female subjects, aged 16 to 65 years with a genetically proven diagnosis of CMT1A. Notes: a) A report of a genetic test confirming PMP22 duplication and therefore a diagnosis of CMT1A must be available in the subject's record at the clinical site. b) In the absence of a report of a genetic test confirming PMP22 duplication in the subject's medical record, a confirmatory genetic test must be conducted via the central laboratory as part of Screening. c) In the exceptional case wherein subject was randomized into the study without meeting(a) or (b), an unscheduled confirmatory genetic test will be performed. In the event of a negative genetic test result, the subject will be withdrawn from the study.
  • Able to provide written informed consent/assent and comply with study procedures.
  • Mild-to-moderate severity assessed by a CMTNS-V2 score >2 and ≤18.
  • Muscle weakness in at least foot dorsiflexion on clinical assessment.
  • Ulnar nerve motor conduction time of at least 15 m/s.
  • If taking prescribed psychoactive drugs(eg, antidepressants, stimulants, tranquilizers, anti-epileptics) for CMT1A, should be on a stable dose for at least 4 weeks prior to randomization, which is not planned to be changed.
Show all 38Show fewer
  • If taking prescribed or 'over-the-counter' analgesic medications (eg, paracetamol/acetaminophen, nonsteroidal anti-inflammatory drugs) for CMT1A, should be on a stable dose for at least 2 weeks prior to randomization, which is not planned to be changed.
  • If female, subject must be: (a) surgically sterilized via hysterectomy, bilateral oophorectomy, or bilateral tubal ligation; or (b) of childbearing potential and using a birth control method such as:
  • Combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation:
  • Oral
  • Intravaginal
  • Transdermal
  • Progestogen-only hormonal contraception associated with inhibition of ovulation:
  • Oral
  • Injectable
  • Implantable
  • Intrauterine device
  • Intrauterine hormone-releasing system
  • Bilateral tubal occlusion
  • Vasectomized partner
  • Sexual abstinence or (c) Of non-childbearing potential (i.e., no menses for ≥ 12 consecutive months without any other underlying medical cause)
  • If male, the subject must have had a vasectomy or must use a reliable method of birth control with their partner or total abstinence from sexual intercourse. The subject must agree to continue using their selected method of birth control with their sexual partner during the study and for 120 days after study completion.
  • Able to provide written informed consent/assent and comply with study procedures.
  • If female, subject must be (a) surgically sterilized via hysterectomy, bilateral oophorectomy, or bilateral tubal ligation; or (b) of childbearing potential and using a birth control method such as:
  • Combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation:
  • Oral
  • Intravaginal
  • Transdermal
  • Progestogen-only hormonal contraception associated with inhibition of ovulation:
  • Oral
  • Injectable
  • Implantable
  • IUD
  • IUS
  • Bilateral tubal occlusion
  • Vasectomized partner
  • Sexual abstinence or (c) of non-childbearing potential (ie, no menses for ≥12 consecutive months without any other underlying medical cause).
  • If male, the subject must have had a vasectomy or must use a reliable method of birth control with their partner or total abstinence from sexual intercourse. The subject must agree to continue using their selected method of birth control with their sexual partner during the study and for 120 days after study completion.

Who cannot join

  • Subjects previously enrolled in any PXT3003 study.
  • Subjects living in the same household and enrolled in a PXT3003 study (due to potential lack of adequate storage for study material, risk of mixing treatments and potential unblinding).
  • CMT of any subtype other than 1A.
  • ONLS score of 0.
  • Known clinically significant motor or sensory abnormalities secondary to a different neurological cause (eg, diabetes, alcohol, vascular, autoimmune, neoplastic, neurodegenerative, human immunodeficiency virus, etc.). Note: subjects with diagnosis of unilateral carpal tunnel syndrome at least 1 year prior to Screening Visit, that is asymptomatic at the time of Screening Visit, will not be excluded from participating in this study.
  • Subjects who have had any surgery or have a concomitant disorder (eg, severe arthrosis) that reduces the mobility of the ankle or wrist making it, in the opinion of the investigator, difficult to assess the efficacy of the treatment. Note: subjects with surgical repair of unilateral carpel tunnel syndrome will not be excluded from participating in this study.
Show all 30Show fewer
  • Known peripheral neuropathy, myopathy, or neuromuscular disorder of any other kind. Note: subjects with diagnosis of unilateral carpal tunnel syndrome at least 1year prior to Screening Visit, that is asymptomatic at the time of Screening Visit, will not be excluded from participating in this study.
  • Any other clinically significant and/or uncontrolled medical condition that, in the opinion of the investigator, could be a confound, may increase subject's risk, or may preclude successful participation or completion of the study.
  • Known hypersensitivity or intolerance to PXT3003( or matching placebo), including any of its active ingredients( baclofen, naltrexone, or sorbitol), and/or any of its excipients( acetate buffer, sodium methyl parahydroxybenzoate, sodium propyl parahydroxybenzoate, or isoamyl acetate).
  • Concomitant treatments including but not limited to baclofen, naltrexone, sorbitol (pharmaceutical form), opioids, potent central nervous system depressants (such as barbiturates, long-acting benzodiazepines, and neuroleptics), and potentially neurotoxic drugs such as amiodarone, chloroquine, and chemotherapeutics capable of inducing peripheral neuropathy. Subjects able to stop these medications at least 2 weeks before randomization and for the study duration may be included. Subjects with positive urine drug screen at Baseline Visit will be excluded, except for permitted use of codeine and benzodiazepines.
  • History of porphyria.
  • Diagnosis or history of substance use disorder by Diagnostic and Statistical Manual of Mental Disorders-5th Edition criteria within the past 12 months.
  • Medical or recreational use of marijuana in the 3 months prior to the Screening Visit.
  • Active suicidality (eg, any suicide attempts within the past 12 months or any current suicidal intent, including a plan, as assessed by the C SSRS score of "YES" on questions 4 or 5; and/or based on clinical evaluation by the investigator).
  • Currently active major depression, as determined by a Beck Depression Inventory-II (BDI-II) score ≥20.
  • Currently lactating, pregnant, or planning on becoming pregnant during the study.
  • Alanine aminotransferase or aspartate aminotransferase levels greater than 2 times the upper limit of normal.
  • Significant renal impairment as determined by glomerular filtration rate of less than 50 mL/min.
  • Subject has participated in an investigational drug or device study within 30 days prior to the Screening Visit or plans to participate in an investigational drug or device study during the course of this study.
  • Subject is a dependent and/or relative of the Sponsor or Principal Investigator.
  • OLE Period
  • Any clinically significant and/or uncontrolled medical condition that, in the opinion of the investigator, could be a confound factor, may increase subject's risk, or may preclude successful participation or completion of the study.
  • Concomitant treatments including but not limited to baclofen, naltrexone, sorbitol (pharmaceutical form) other than PXT3003 taken in the Double-blind Treatment Period of this study, opioids, potent CNS depressants (such as barbiturates, long-acting benzodiazepines, and neuroleptics), and potentially neurotoxic drugs such as amiodarone, chloroquine, and chemotherapeutics capable of inducing peripheral neuropathy. Subjects able to stop these medications at least 2 weeks before randomization and for the study duration may be included.
  • Diagnosis or history of substance use disorder by Diagnostic and Statistical Manual of Mental Disorders-5ᵗʰ Edition criteria within the past 12 months.
  • Active suicidality (eg, any suicide attempts within the past 12 months or any current suicidal intent, including a plan, as assessed by the C-SSRS score of "YES" on questions 4 or 5; and/or based on clinical evaluation by the investigator).
  • Currently active major depression, as determined by a BDI-II score ≥20.
  • Currently lactating, pregnant, or planning on becoming pregnant during the study.
  • ALT or AST levels greater than 2 × ULN relative to Baseline.
  • Estimated GFR of less than 50 mL/min.
  • Note: PXT3003 will be dispensed to all subjects before laboratory results are available, and if the laboratory results are out of range, ie, subject meeting the exclusion criteria, the subject will be called immediately to stop taking PXT3003. One retest within 4 weeks may be performed in consultation with the Medical Monitor if any of the above laboratory abnormalities are found. In case of eligibility determination after the retest, subjects will restart taking 2 weeks of half dose of PXT3003.

Trial locations and contacts

50 sites in 9 countries.

50 sites in 9 countries

United States20 sites

  • National Neuromuscular Research Institute

    Austin, Texas, 78759

  • Massachusetts General Hospital Neuromuscular Diagnostic Center

    Boston, Massachusetts, 02114

  • UNC Department of Neurology Peripheral Neuropathy Center

    Chapel Hill, North Carolina, 27514

  • Atrium Health Neurosciences Institute

    Charlotte, North Carolina, 28207

  • MU Health Care Neurology and Sleep Disorders Clinic

    Columbia, Missouri, 65212

  • The Ohio State University Wexner Medical Center

    Columbus, Ohio, 43210

  • University of Kansas Medical Center Research Institute

    Fairway, Kansas, 66205

  • University of Florida Clinical Research Center

    Gainesville, Florida, 32610

  • Hackensack Meridian Health Hackensack University Medical Center

    Hackensack, New Jersey, 07601

  • Cedars-Sinai Medical Center

    Los Angeles, California, 90048

  • UCLA Department of Psychiatry and Biobehavioral Sciences

    Los Angeles, California, 90095

  • University of Miami Leonard M. Miller School of Medicine

    Miami, Florida, 33136

  • University of Minnesota Health

    Minneapolis, Minnesota, 55414

  • Hospital for Special Care

    New Britain, Connecticut, 06053

  • Colombia University Department of Neurology

    New York, New York, 10032

  • Advent Health Medical Group Neurology Orlando

    Orlando, Florida, 32803

  • UC Davis Health Department of Physical Medicine and Rehabilitation

    Sacramento, California, 95817

  • Neurology Clinic at University of Washington Medical Center

    Seattle, Washington, 98195

  • Providence St. Luke's Rehabilitation Medical Center

    Spokane, Washington, 99202

  • Oregon Neurology

    Springfield, Oregon, 97477

Other trials in United States

France6 sites

  • Centre de Reference des Maladies Neuromusculaires AOC Service de Neurologie, CHU d'Angers

    Angers, 49933

  • Centre de reference des maladies neuromusculaires AOC Hopital Pellegrin CHU de Bordeaux

    Bordeaux, 33076

  • CHU de Lille Hôpital Salengro

    Lille, 59037

  • Service de Neurologie et Maladies Neuromusculaires, CHU de Marseille - Hopital La Timone

    Marseille, 13385

  • Association lnstitut de Myologie Hopital Pitie-Salpetriere Service de Neuro-Myologie

    Paris, 75013

  • Centre d'investigation Clinique CHU de Strasbourg Hopital de Hautepierre

    Strasbourg, 67098

Other trials in France

Canada5 sites

  • CIUSS de Saguenay-Lac-Saint-Jean Centre d'etudes Cliniques

    Chicoutimi, Quebec, G7H 5H6

  • Montreal Neurological Institute and Hospital-Clinical Research Unit

    Montreal, Quebec, H3A 2B4

  • Ottawa Hospital Research Institute- Neuromuscular Research Centre

    Ottawa, Ontario, K1Y 4E9

  • CHU de Quebec-Universite Laval- Hopital Enfant-Jesus

    Québec, Quebec, G1J 1Z4

  • UHN Toronto General Hospital Krembil Neuroscience Centre

    Toronto, Ontario, M5G 2C4

Other trials in Canada

Germany5 sites

  • University Hospital RWTH Aachen, Department of Neurology and Institute for Neuropathology

    Aachen, D-52074

  • University Medical Centre Goettingen, Dept. of Clinical Neurology

    Göttingen, D-37075

  • Friedrich-Baur-Institut, Neurologische Klinik und Poliklinik Ludwig-Maximilians-Universität

    München, D-80336

  • University Hospital Muenster UKM Department of Neurology

    Münster, 48149

  • Universitätsklinikum Tübingen Crona Kliniken Neuromuskuläres Zentrum

    Tübingen, 72076

Other trials in Germany

Italy5 sites

  • Azienda Ospedaliera Universitaria San Martino Universita Degli Studi di Genova Clinica Neurologica

    Genova, 16132

  • Azienda Ospedaliera Universitaria Policlinico "G. Martino" di Messina

    Messina, 98125

  • University of Naples Federico II

    Naples, 80131

  • Tor Vergata University of Rome

    Rome, 00133

  • University Hospital GB Rossi UOC Neurologia B, AOUI Verona Department of Neuroscience, Biomedicine and Movement Sciences

    Verona, 37134

Other trials in Italy

Spain4 sites

  • Hospital Universitario Clinico San Carlos

    Madrid, 28040

  • Complejo Hospitalario Universitario de Santiago

    Santiago de Compostela, 15706

  • Hospital Universitario Virgen del Rocío

    Seville, 41013

  • Hospital Universitario y Politécnico La Fé

    Valencia, 46026

Other trials in Spain

Israel3 sites

  • Hadassah Ein Kerem University Medical Center Department of Neurology

    Jerusalem, 91120

  • Sheba Medical Center

    Ramat Gan, 52662

  • Tel Aviv Sourasky Medical Center

    Tel Aviv, 6423906

Other trials in Israel

Belgium1 site

  • Universitaire Ziekenhuizen Leuven

    Leuven, 3000

Other trials in Belgium

Denmark1 site

  • Rigshospitalet, University of Copenhagen Copenhagen Neuromuscular Center

    Copenhagen, DK-1200

Other trials in Denmark

About this study

Official title: A Multi-center, Randomized, Double-blind, Placebo Controlled Phase III Study to Assess the Efficacy, Safety, and Tolerability of PXT3003 in Charcot-Marie-Tooth Type 1A (CMT1A)

The study will consist of 2 periods: Double-blind Treatment and Open-Label Extension(OLE) Period.

-Double-blind Treatment Period - This will be randomized, double-blind, placebo-controlled part of the study which will be conducted in parallel groups, ie,1 group receiving the active treatment (PXT3003) and the other group receiving placebo.

Read more

Primary endpoint of the study will be assessed at Month 15.

-Open-label Extension (OLE) Period - All subjects completing Double-blind Treatment Period will be given an opportunity to enter the OLE Period of the study and receive the active treatment (PXT3003). The duration of the OLE Period will be based on Sponsor discretion, ie, Sponsor intends to keep the study open until the study drug PXT3003 is commercially available.

During this period, the long-term safety and efficacy of PXT3003 will be assessed as an exploratory objective.

Double-blind Treatment Period Objectives:

Primary:

To evaluate the efficacy of treatment with PXT3003 (a fixed-dose combination of [RS]-baclofen, naltrexone hydrochloride [HCl], and D-sorbitol) compared to placebo in subjects with Charcot-Marie-Tooth disease type 1A (CMT1A).

Secondary:

To evaluate the safety and tolerability of PXT3003 treatment in subjects with CMT1A.

Exploratory:

To characterize the relationship between plasma biomarkers and response to PXT3003 treatment.

OLE Period Objective:

Exploratory:

To evaluate the long-term safety and efficacy of PXT3003.

Read the detailed description

This is an international, multi-center, randomized, double-blind, placebo-controlled, parallel-group, Phase III study of PXT3003 in subjects with CMT1A. The study will be conducted at approximately 52 sites worldwide.

This study consists of Double-blind Treatment and OLE Periods.

Double-blind Treatment Period:

Eligible subjects will be screened and randomized in a 1:1 ratio to receive either oral PXT3003 or matching placebo, 10 mL, twice daily (BID) for 15 months. In order to maximize the tolerability of (RS)-baclofen for all randomized subjects, treatment will start with a half-dose (5 mL), taken BID (morning and evening with food) during the first 2 weeks, and then will be increased to a full-dose (10 mL), taken BID (morning and evening with food) until completion of the Treatment Period at Month 15.

A total of approximately 350 subjects will be enrolled. Visits will take place at Screening (up to -35 days), Baseline (Day 1), and Months 3, 6, 9, 12, and 15. Telephone contacts will take place at weeks 2 or 3, Month 1 and Month 2, and then monthly between subsequent in-person visits. A genotyping test for the Peripheral Myelin Protein 22 (PMP22) gene duplication will take place at the Screening Visit if it is not already documented for the subject. All subjects completing the Double-blind Treatment Period of the study will be given an opportunity to enter the OLE Period at Month 15 (Visit 6). Subjects not consenting to enter the OLE Period will have their last study visit (ie, Safety Follow-up Visit, Visit 7), 30 days after their last dosing day.

The primary outcome measures modified Overall Neuropathy Limitations Score(mONLS) and the 10-Meter Walk Test (10mWT), along with the Columbia Suicide Severity Rating Scale (C-SSRS) will be evaluated at each post-randomization in-person visit. The other secondary outcome measures, exploratory outcome, and safety/tolerability assessments will be evaluated as per Schedule of Activities (SOA). A Data Safety and Monitoring Board (DSMB) will meet on a scheduled basis throughout the study to review safety data and will reconvene on an ad hoc basis as necessary.

Planned duration for sites to enroll subjects: approximately 12 months, Subject Screening Period: 35 days, Subject Treatment Period: up to 15 months, Safety Follow-up Period (for subjects not entering the OLE Period): 30 days

OLE Period:

A subject entering the OLE Period (whether the subject was randomized to oral PXT3003 or matching placebo in the Double-blind Treatment Period) will start taking a half-dose of PXT3003 (5 mL) BID (morning and evening with food) during the first 2 weeks, and then a full dose of PXT3003 (10 mL) BID (morning and evening with food) throughout the OLE Period. The visits and assessments during the OLE Period are described in the SOA.

For subjects entering the OLE Period, Screening Day will occur on the same day as Visit 6 (Month 15) of the Double-blind Treatment Period. The duration of the Treatment Period will be based on Sponsor discretion. Sponsor intends to keep the study open until the study drug PXT3003 is commercially available. Safety Follow-up Period: 30 days

Being tested
(RS)-baclofen, naltrexone hydrochloride and D-sorbitol, Placebo
Keywords
Charcot Marie Tooth Type 1, Peripheral Neuropathy, PXT3003
Medical subject headings
Charcot-Marie-Tooth Disease, Peripheral Nervous System Diseases

What participants receive

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

  • (RS)-baclofen, naltrexone hydrochloride and D-sorbitolDrug

    Also called PXT3003

    oral fixed dose combination

  • PlaceboDrug

    liquid oral solution

Study groups in NCT04762758
Study groupRoleWhat they receive
PXT3003ExperimentalLiquid oral solution, 10 mL twice a day, morning and evening with food
Receives: Drug: (RS)-baclofen, naltrexone hydrochloride and D-sorbitol
PlaceboPlacebo comparatorLiquid oral solution, 10 mL twice a day, morning and evening with food
Receives: Drug: Placebo

What is being measured

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

  1. modified Overall Neuropathy Limitation Scale (mONLS)

    The modified ONLS is a disability scale that was derived and improved from the Overall Disability Sum Score to measure limitations in the everyday activities of the upper limbs (rated on 5 points) and the lower limbs (rated on 7 points).38 The total score goes from 0 (no disability) to 12 (maximum disability). Lower values in the ONLS indicate a better clinical condition.

    Measured: From Baseline to Month 15

Secondary outcomes (6)

  1. 10-Meter Walk Test

    The 10mWT is simple to administer, standardized, and valid performance evaluation of functional mobility and gait that has been proven reliable in neurologic disorders, including CMT. Results recorded are the time to walk 10 meters and the number of steps performed.

    Measured: From Baseline to Month 15

  2. Quantified Muscular Testing (QMT) (bilateral foot dorsiflexion dynamometry)

    QMT is used to evaluate motor strength in CMT1A.The following muscles will be evaluated: tibialis anterior (right and left). The best value on three consecutive and reproducible tests will be collected in the electronic Case Record Form (eCRF) for each muscle.

    Measured: From Baseline to Month 15

  3. Patient Global Impression of Severity (PGI-S)

    The PGI-S is a validated tool that is used to rate the severity of a specific condition. Subjects will rate their health status over the past week. The PGI-S is a 1-item questionnaire on a 5-point scale and subjects will be asked to rate the severity of their condition from "None" (Score = 1) to "Very Severe" (Score = 5). Assessments obtained over the course of the study will be compared to baseline, prior to initiation of treatment.

    Measured: From Baseline to Month 15

  4. Patient Global Impression of Change (PGI-C)

    The PGI-C scale is a validated generic tool for assessment of overall change in the severity of illness following treatment. Subjects will rate how they feel now compared with how they felt before receiving study drug on a 7-point scale where 1 is "Very much improved" and 7 is "Very much worse".

    Measured: From Baseline to Month 15

  5. CMTNS-V2

    CMTNS is a specific scale designed to assess severity of impairment in CMT disease. Although not completely validated, it provides a single and reliable measure of CMT severity. It is a 36-point scale based on 9 items: 5 of them quantify impairment (sensory symptoms, pin sensibility, vibration, arm, and leg strength), 2 activity limitations (motor symptoms arms and legs) and 2 electrophysiological data (amplitudes of ulnar compound muscle action potential and sensory nerve action potential). Increased scores indicate a worsening of the function: the scores categorize a disability as mild (0 to10), moderate (11 to 20) and severe (21 to 36).

    A modified version 2 (CMTNS-V2) was issued in 2011 to attempt to reduce floor and ceiling effects and to standardize patient assessment.

    Measured: From Baseline to Month 15

  6. QMT (hand grip)

    QMT is used to evaluate motor strength in CMT1A. The following muscles will be evaluated: hand grip (right and left). The best value on three consecutive and reproducible tests will be collected in the eCRF for each muscle.

    Measured: From Baseline to Month 15

Timeline

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

  1. First posted21 Feb 2021
  2. Study start30 Mar 2021
  3. Primary completion19 Apr 2024 (estimated)
  4. Study completion19 Apr 2024 (estimated)

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 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Double blind
Who is masked
Participant, Investigator
Enrolment
350 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04762758
Sponsor's protocol ID
CLN-PXT3003-06
Other IDs
2020-004805-30
Acronym
PREMIER
Status last verified
Apr 2024
First submitted
17 Feb 2021
Last updated
3 Apr 2024
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing (RS)-baclofen, naltrexone hydrochloride and D-sorbitol in people with Charcot-Marie-Tooth Disease, run by Pharnext S.C.A. at 50 sites in 9 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 38 inclusion and 30 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: status not confirmed, as of the sponsor's last update on 3 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 50 sites in United States, France, Canada, Germany, Italy and 4 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

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

Are results available for this study?

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?

modified Overall Neuropathy Limitation Scale (mONLS) (time frame: From Baseline to Month 15). 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 NCT04762758.

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 3 Apr 2024.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in