A Phase 2 interventional study of PXT3003 Low dose and PXT3003 Intermediate Dose in Charcot-Marie-Tooth Disease, Hereditary Neuropathy With Liability to Pressure Palsies and Genetic Disorders, sponsored by Pharnext S.C.A.. Completed at 6 sites in France. Open to participants aged 18 Years to 65 Years. Per ClinicalTrials.gov, last updated 2017-11-22.
Sponsored by Pharnext S.C.A. · Phase 2, Interventional, and Treatment
The present trial is a randomized, placebo-controlled study evaluating 3 different doses of PXT3003 in patients with CMT1A disease.
In addition to the safety and tolerability of the treatment, clinical, electrophysiological and biological endpoints (PMP22 mRNA, skin biopsy histology and plasma biomarkers) will be assessed. Standard laboratory tests and drug plasma concentrations will also be measured. Because of the slow progression of the disease and the nature of the observed symptoms, a minimum duration of 12 months of treatment is required in order to observe a potential improvement in any of the efficacy parameters.
113 studies on the registry are indexed under Charcot-Marie-Tooth Disease; 38 are open to participants now.
This study's enrollment of 80 is above the median of 41 across 62 interventional studies indexed under Charcot-Marie-Tooth Disease.
Browse Charcot-Marie-Tooth Disease studies →Pharnext S.C.A. is the lead sponsor of 6 studies on the registry; none are open to participants now.
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Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECG (electrocardiogram)
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Oral Liquid formulation, 1/100, bid, 12 months
Drug: PXT3003 Low dose
Oral Liquid formulation, 1/50, bid, 12 months
Drug: PXT3003 Intermediate Dose
Oral Liquid formulation, 1/10, bid, 12 months
Drug: PXT3003 High Dose
Oral Liquid formulation, bid, 12 months
Other: Placebo
Liquid,5 ml, twice a day, 12-month treatment
Also known as: Pleocompound PXT3003
Liquid,5 ml, twice a day, 12-month treatment
Also known as: Pleocompound PXT3003
Liquid,5 ml, twice a day, 12-month treatment
Also known as: Pleocompound PXT3003
Liquid,5 ml, twice a day, 12-month treatment
Also known as: Pleocompound PXT3003
Safety and Tolerability of PXT3003
The Primary Objective is to assess the clinical and laboratory safety and tolerability of three doses of PXT3003 administered orally for 12 months to CMT1A patients versus placebo. Number of participants with adverse events in each arm.
Time frame: Screening, randomization, 1-, 3-, 6-, 9-, 12-month treatment and 1-month follow-up
To Obtain Preliminary Data on the Efficacy of PXT3003 on Clinical Scores and Functional Tests
Efficacy scores and functional tests will be assessed CMTNS/CMTES:ONLS, VAS, fatigue, pain, six minute walk test (6MWT), nine-hole peg test, quantified muscular testing (QMT; hand grip and foot dorsiflexion), CGI. For each test or score, change from baseline after 3-,6-, 9- and 12-months of treatment.
Time frame: Screening, randomization, 3-, 6-, 9- and 12-months treatment
To Assess the Pharmacodynamic Effect of PXT3003 on PMP22 mRNA Levels and Intra-epidermal Axon Density in Cutaneous Biopsy
A cutaneous biopsy (consisting in 2 small punch biopsies) will be performed to assess PMP22 mRNA expression and intra-epidermal axon density. Change from baseline after 12-month of treatment.
Time frame: Randomization and 12-month treatment
To Assess the Pharmacodynamic Effect of PXT3003 on Selected Neurophysiological Parameters
Electrophysiological examination will be performed to assess sensory and motor responses of the median and ulnar nerves (non-dominant side) including: NCV, compound muscle action potential (CMAP) and SNAP. Change from baseline after 3-,6-, 9- and 12-months of treatment.
Time frame: Screening, randomization, 3-, 6-, 9- and 12-month treatment
To Assess the Pharmacodynamic Effect of PXT3003 on a Series of Biochemical Biomarkers
Dosages of biochemical biomarkers in plasma. Change from baseline after 3-month of treatment.
Time frame: Randomization and 3-month treatment
To Assess the Plasma Concentrations of PXT3003
PXT3003 plasmatic concentrations after one administration (randomization) and after 1-,6-and 12-months of treatment.
Time frame: Randomization, 1-, 6- and 12-month treatment
| Milestone | PXT3003 Low Dose | PXT3003 Intermediate Dose | PXT3003 High Dose | Placebo |
|---|---|---|---|---|
| Started | 21 | 21 | 19 | 19 |
| Completed | 21 | 19 | 16 | 17 |
| Not completed | 0 | 2 | 3 | 2 |
The Primary Objective is to assess the clinical and laboratory safety and tolerability of three doses of PXT3003 administered orally for 12 months to CMT1A patients versus placebo. Number of participants with adverse events in each arm.
| Participants | PXT3003 Low Dose | PXT3003 Intermediate Dose | PXT3003 High Dose | Placebo |
|---|---|---|---|---|
| Safety and Tolerability of PXT3003 | 21 | 21 | 19 | 19 |
Efficacy scores and functional tests will be assessed CMTNS/CMTES:ONLS, VAS, fatigue, pain, six minute walk test (6MWT), nine-hole peg test, quantified muscular testing (QMT; hand grip and foot dorsiflexion), CGI. For each test or score, change from baseline after 3-,6-, 9- and 12-months of treatment.
Results for this outcome have not been posted.
A cutaneous biopsy (consisting in 2 small punch biopsies) will be performed to assess PMP22 mRNA expression and intra-epidermal axon density. Change from baseline after 12-month of treatment.
Results for this outcome have not been posted.
Electrophysiological examination will be performed to assess sensory and motor responses of the median and ulnar nerves (non-dominant side) including: NCV, compound muscle action potential (CMAP) and SNAP. Change from baseline after 3-,6-, 9- and 12-months of treatment.
Results for this outcome have not been posted.
Dosages of biochemical biomarkers in plasma. Change from baseline after 3-month of treatment.
Results for this outcome have not been posted.
PXT3003 plasmatic concentrations after one administration (randomization) and after 1-,6-and 12-months of treatment.
Results for this outcome have not been posted.
Collected over 13 months. Non-serious events are listed at a 2% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| PXT3003 Low Dose | — | 2/21 (9.5%) | 12/21 (57.1%) |
| PXT3003 Intermediate Dose | — | 0/21 (0%) | 10/21 (47.6%) |
| PXT3003 High Dose | — | 0/19 (0%) | 13/19 (68.4%) |
| Placebo | — | 0/19 (0%) | 11/19 (57.9%) |
| Event | PXT3003 Low Dose | PXT3003 Intermediate Dose | PXT3003 High Dose | Placebo |
|---|---|---|---|---|
| osteoarthritisMusculoskeletal and connective tissue disorders | 1/21 | 0/21 | 0/19 | 0/19 |
| HemorrhoidsGastrointestinal disorders | 1/21 | 0/21 | 0/19 | 0/19 |
| Event | PXT3003 Low Dose | PXT3003 Intermediate Dose | PXT3003 High Dose | Placebo |
|---|---|---|---|---|
| Musculoskeletal and connective tissue disordersMusculoskeletal and connective tissue disorders | 12/21 | 8/21 | 13/19 | 7/19 |
| Infections and infestationsInfections and infestations | 11/21 | 10/21 | 11/19 | 8/19 |
| Nervous system disordersNervous system disorders | 8/21 | 8/21 | 11/19 | 9/19 |
| Gastrointestinal disordersGastrointestinal disorders | 7/21 | 5/21 | 10/19 | 11/19 |
| Age, Categorical(Participants) | PXT3003 Low Dose | PXT3003 Intermediate Dose | PXT3003 High Dose | Placebo | Total |
|---|---|---|---|---|---|
| <=18 years | 0 | 0 | 0 | 0 | 0 |
| Between 18 and 65 years | 21 | 21 | 19 | 19 | 80 |
| >=65 years | 0 | 0 | 0 | 0 | 0 |
| Age, Continuous(years) | PXT3003 Low Dose | PXT3003 Intermediate Dose | PXT3003 High Dose | Placebo | Total |
|---|---|---|---|---|---|
| Mean | 47.9 (18 to 63) | 44.3 (23 to 64) | 44.6 (24 to 63) | 43.2 (21 to 65) | 45.1 (18 to 65) |
| Sex: Female, Male(Participants) | PXT3003 Low Dose | PXT3003 Intermediate Dose | PXT3003 High Dose | Placebo | Total |
|---|---|---|---|---|---|
| Female | 14 | 13 | 10 | 11 | 48 |
| Male | 7 | 8 | 9 | 8 | 32 |
| Region of Enrollment(participants) | PXT3003 Low Dose | PXT3003 Intermediate Dose | PXT3003 High Dose | Placebo | Total |
|---|---|---|---|---|---|
| France | 21 | 21 | 19 | 19 | 80 |
Plan to share: No
The record gives a start of Dec 2010, a primary completion of Oct 2011 and a completion of Dec 2012. It does not say whether these dates are actual or estimated.
Results were first posted in Feb 2017, 5 years 4 months after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Oct 2017. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: PXT3003
Pharnext S.C.A.