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Charcot-Marie-Tooth Type 1APhase 3NCT05092841

A Study to Assess the Efficacy and Safety of PXT3003 in Charcot-Marie-Tooth Type 1A

A Phase 3 study testing PXT3003 in people with Charcot-Marie-Tooth Type 1A, run by Tasly GeneNet Pharmaceuticals Co., Ltd at 25 sites in China.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This is a randomized, double-blind, placebo-controlled and multicenter 3 phase trial evaluating the therapeutic effect and safety of CMT1A by PXT3003. This double-blind study will assess in parallel groups 1 dose of PXT3003 compared to Placebo in CMT1A patients treated for 15 months. Read more about this study

Who is it for?
Ages 16 to 65 years
Conditions the study is for
Charcot-Marie-Tooth Type 1A
What is being tested?
PXT3003
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
176 participants
Where
25 sitesChina
Study dates
28 Sep 2021to 18 Mar 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

  • Patients aged 16 to 65 years (included boundary value), of either sex;
  • Patients with CMT1A (PMP22 duplication on chromosome 17p11.2) confirmed by gene diagnosis; 3.2 < CMTNS-v2 score ≤ 18;
  • 4.Patients are dorsalis pedis flexor weakness at least (clinical evaluation); 5.Ulnar nerve motor nerve conductance velocity > 15 m/s; 6.Subjects participate in clinical trials and sign informed consent voluntarily , and they have the ability to understand as will as abide by research procedures.

Who cannot join

  • Being allergic to RS-baclofen, naltrexone HCL, D-sorbitol or any component in pxt3003 excipients or having other serious prior allergic reaction;
  • Existence contraindications of baclofen, naltrexone or sorbitol, such as porphyria;
  • Any other associated cause of peripheral neuropathy such as diabetes mellitus (including diabetes history and glycosylated hemoglobin >6.5%)
  • Subjects with other neurological diseases affecting the evaluation of study treatment;
  • Patients with the score of ONLS score is 0;
  • A history of unstable medical diseases with clinically significant unstable medical diseases (unstable angina pectoris, tumor, blood disease, hepatitis or liver failure, renal failure, etc.) that may cause harm to the subjects participating in this study in the past 1 year;
Show all 19Show fewer
  • Limb surgery had implemented within the first six months of randomization or will be planned before the completion of the clinical trial;
  • Hepatic or renal dysfunction:
  • TBIL>1.5×ULN,ALT>3×ULN,AST>3×ULN;
  • Cr>1.5×ULN;
  • Syphilis antibody and HIV antibody positive subjects;
  • Subjects with tumors indicated by chest radiograph or B-ultrasound;
  • Subjects with alcohol dependence in recent 3 months;
  • Females that are of childbearing potential, pregnant, or are breast-feeding;Subjects who are unable to use appropriate contraceptives during the trial;
  • Subjects with concomitant treatment 4 weeks before enrollment, including but not limited to baclofen, naltrexone, sorbitol, opioids, vitamin C, levothyroxine and potentially neurotoxic drugs (such as amiodarone and chloroquine);
  • Subjects unable to complete the follow-up of study;
  • Participated in another clinical trial and used the test drug within the last 30 days;
  • Different subjects from the same family and living in the same residence can only include one subject, so as to avoid treatment confusion, affect blind treatment and affect the interpretation of the research results;
  • Investigators affirm the compliance in a certain subject is poor or there are some other factors that are not suitable to participate in this clinical trial.

Trial locations and contacts

25 sites in 1 country.

25 sites in 1 country

China25 sites

  • Peking University Third Hospital

    Beijing

  • Xuanwu Hospital Capital Medical University

    Beijing

  • The First Bethune Hospital of Jilin University

    Changchun

  • The Third Xiangya Hospital of Central South University

    Changsha

  • The First Affiliated Hospital of Fujian Medical University

    Fuzhou

  • Nanfang Hospital Southern Medical University

    Guangzhou

  • The First Affiliated Hospital，Sun Yat-sen University

    Guangzhou

  • The Affiliated Hospital of Guizhou Medical University

    Guiyang

  • The Second Affiliated Hospital Zhejiang University School of Medicine

    Hangzhou

  • The First Affiliated Hospital of Harbin Medical University

    Harbin

  • The First Affiliated Hospital of Anhui Medical University

    Hefei

  • Inner Mongolia People's Hospital

    Hohhot

  • Qilu Hospital of Shandong University

    Jinan

  • The Second Affiliated Hospital of Nanchang University

    Nanchang

  • The Affiliated Hospital of Nanjing University Medical School

    Nanjing

  • Zhongda Hospital Southeast University

    Nanjing

  • Qilu Hospital of Shandong University（Qingdao）

    Qingdao

  • Shanghai Jiaotong University School of Medicine Ruijin Hospital

    Shanghai

  • The First Hospital of China Medical University

    Shenyang

  • The Second Hospital of Hebei Medical University

    Shijiazhuang

  • The First Affiliated Hospital of Soochow University

    Suzhou

  • Renmin Hospital of Wuhan University（Hubei General Hospital）

    Wuhan

  • The First Affiliated Hospital of Xi'an Jiaotong University

    Xi'an

  • Henan Provincial People's Hospital

    Zhengzhou

  • The First Affiliated Hospital of Zhengzhou University

    Zhengzhou

Other trials in China

About this study

Official title: A Multi-Center, Randomized, Double-Blind, Placebo-Controlled Phase III Study to Assess the Efficacy and Safety of PXT3003 in Charcot-Marie-Tooth Type 1A (CMT1A) Treated 15 Months

This is a randomized, double-blind, placebo-controlled and multicenter 3 phase trial evaluating the therapeutic effect and safety of CMT1A by PXT3003. This double-blind study will assess in parallel groups 1 dose of PXT3003 compared to Placebo in CMT1A patients treated for 15 months.

Read the detailed description

This multi-center, randomized, double-blind, placebo-controlled, Phase III clinical study is designed to evaluate PXT3003 versus placebo in subjects with genetically confirmed CMT1A of mild-to-moderate severity (CMTNS-V2 score >2 and ≤18) aged 16 to 65 years.

Genetically confirmed CMT1A subjects will be screened and randomized in a 1:1 ratio to receive either oral PXT3003 daily or matching placebo for 15 months. A total of approximately 176 subjects will be enrolled.

Visits will take place at Screening (up to -30 days), Baseline , and Months 3, 6, 9, 12, and 15.

Being tested
PXT3003, PXT3003 placebo

What participants receive

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

  • PXT3003Drug

    Also called (RS)-baclofen, naltrexone hydrochloride and D-sorbitol oral fixed dose combination

    Patients will be administered PXT3003 twice daily (bid) at 10mL.

  • PXT3003 placeboDrug

    Also called liquid oral solution

    Patients will be administered PXT3003 placebo twice daily (bid) at 10mL.

Study groups in NCT05092841
Study groupRoleWhat they receive
PXT3003ExperimentalLiquid oral solution, 10 mL twice a day, morning and evening with food
Receives: Drug: PXT3003
PXT3003 PlaceboPlacebo comparatorLiquid oral solution, 10 mL twice a day, morning and evening with food
Receives: Drug: PXT3003 placebo

What is being measured

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

  1. Overall Neuropathy Limitation Scale (ONLS) score

    The primary efficacy endpoint will be the main effect of the studied treatment on the improvement of disability measured by the Overall Neuropathy Limitation Scale (ONLS) score, summarized at 15 months defined by: the change of the ONLS from baseline to the 15 months.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 in 5 points) and the lower limbs (rated on 7 points) . The total score goes from 0 ( no disability) to 12 (maximum disability).

    Measured: 15 months

Secondary outcomes (8)

  1. Treatment responders rate of PXT3003;

    Responders Rate to PXT3003 therapy defined as a patients improving on ONLS at end of treatment.

    Measured: 15 months

  2. The sub-item of Arm and leg scores in Overall Neuropathy Limitation Scale (ONLS)

    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 in 5 points) and the lower limbs (rated on 7 points) . The total score goes from 0 ( no disability) to 12 (maximum disability).

    Measured: 15 months

  3. Total and sub-item score of Charcot-Marie-Tooth neuropathy score second version (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).

    Measured: 15 months

  4. 10-Meter Walk Test (10MWT);

    Record the time for walk 10 meters . 10m WT is simple to administer, standardized, reliable and valid evaluation of functional exercise capacity and gait that has been proven reliable in neurologic disorders and in CMT patients. Results recorded are the time to walk 10 meters and the number of steps performed.

    Measured: 15 months

  5. Nine-hole peg test (9HPT) for non-dominant hand ;

    The 9-HPT is a simple timed test of fine motor coordination of extremities in the upper limbs.

    Measured: 15 months

  6. Quantified Muscular Testing (QMT) (grip strength and bilateral foot dorsiflexion dynamometry) ;

    QMT is used to evaluate motor strength in CMT1A. The following muscles will be evaluated: hand grip (right and left).

    Measured: 15 months

  7. Electrophysiological parameters Sensory responses measured at ulnar and radial nerves on the non-dominant side:

    The assessment parameters including: Distal motor latency (DML) .

    Measured: 15 months

  8. Electrophysiological parameters motor responses measured at ulnar and radial nerves on the non-dominant side

    The assessment parameters including: Nerve conduction velocity (NCV) .

    Measured: 15 months

Timeline

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

  1. First posted26 Oct 2021
  2. Study start28 Sep 2021
  3. Primary completion18 Mar 2024
  4. Study completion18 Mar 2024

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
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
176 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05092841
Sponsor's protocol ID
TSL-CM-PXT3003-Ⅲ
Status last verified
Jan 2025
First submitted
26 Sep 2021
Last updated
9 Jan 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing PXT3003 in people with Charcot-Marie-Tooth Type 1A, run by Tasly GeneNet Pharmaceuticals Co., Ltd at 25 sites in China.

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 3 inclusion and 19 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: finished, as of the sponsor's last update on 9 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 25 sites in China. 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?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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?

Overall Neuropathy Limitation Scale (ONLS) score (time frame: 15 months). 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 NCT05092841.

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 9 Jan 2025.

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