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New treatment option for Charcot-Marie-Tooth Disease Type 4J
Official title Study of Intrathecal ELP-02 for Charcot-Marie-Tooth Disease Type 4J (CMT4J)
ClinicalTrials.gov ID: NCT07447557
What this study is testing
What is ELP-02?
ELP-02 is an investigational medicine, being studied as a potential treatment for charcot-marie-tooth disease type 4j.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- First-in-human Phase 1/2, open-label study to evaluate the safety, tolerability, and efficacy of a single lumbar intrathecal dose of ELP-02 to individuals with CMT4J.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 3 to 20
You may be able to join if
- Male and females between the ages of 3 and 20 years at the time of screening.
- Molecularly-confirmed diagnosis of CMT4J (confirmed by a CLIA certified, CE-marked, or equivalent lab) due to bi-allelic pathogenic variants (by ACMG...
- Clinical signs and symptoms consistent with CMT4J disease/motor symptoms
- No more than moderate severity of the disease as measured by a CMTPeds score of \<35 or investigator's discretion
- Written informed consent provided by subject/parent/guardian and willingness to participate and comply with all the study related visits and...
You likely can't join if
- Prior or ongoing medical condition, medical history, physical findings, cardiovascular/ECG findings, or laboratory abnormality that, in the...
- Clinically significant abnormal laboratory values (hemoglobin \ 20 g/dL; white blood cell \> 20,000 per cmm, platelets count \ ULN; GGT, ALT, and AST...
- History of HbA1C \> 6.0%
- Contraindication or unwillingness to undergo lumbar puncture.
- Presence of a concomitant medical condition that precludes use of anesthetics for sedated procedures.
- History of hypersensitivity to sirolimus, tacrolimus, corticosteroids, gadolinium, iodine or shellfish.
See the full eligibility criteria
- Male and females between the ages of 3 and 20 years at the time of screening.
- Molecularly-confirmed diagnosis of CMT4J (confirmed by a CLIA certified, CE-marked, or equivalent lab) due to bi-allelic pathogenic variants (by ACMG criteria) in the FIG4 gene where one of the mutations must be the...
- Clinical signs and symptoms consistent with CMT4J disease/motor symptoms
- No more than moderate severity of the disease as measured by a CMTPeds score of \<35 or investigator's discretion
- Written informed consent provided by subject/parent/guardian and willingness to participate and comply with all the study related visits and procedures. Assent provided by children 10 to 17 years old based on their...
- people able to reproduce must use a barrier method of contraception for the first 12 months after dosing as well as at least one additional acceptable birth control method if sexually active
- Male people must agree not to donate sperm for the remainder of their lifetime after receiving ELP-02
- Female people must agree not to donate eggs for the remainder of their lifetime after receiving ELP-02
- Prior or ongoing medical condition, medical history, physical findings, cardiovascular/ECG findings, or laboratory abnormality that, in the investigator's opinion, could adversely affect the safety of the subject, makes...
- Clinically significant abnormal laboratory values (hemoglobin \ 20 g/dL; white blood cell \> 20,000 per cmm, platelets count \ ULN; GGT, ALT, and AST or total bilirubin \> 2x ULN, creatinine ≥ 1.5 mg/dL) prior to gene...
- History of HbA1C \> 6.0%
- Contraindication or unwillingness to undergo lumbar puncture.
- Presence of a concomitant medical condition that precludes use of anesthetics for sedated procedures.
- History of hypersensitivity to sirolimus, tacrolimus, corticosteroids, gadolinium, iodine or shellfish.
- Concomitant chronic drug treatment that would cause clinically significant interactions with immunosuppressive agents used in the study.
- The presence of significant CNS impairment or behavioral disturbances that would confound the scientific rigor or interpretation of results of the study.
- Recent or planned elective surgical procedures that would confound the scientific rigor or interpretation of results of the study.
- Reason to believe that the subject or parents of the subject will not comply with the study procedures outlined in the study protocol.
- Have received an investigational drug within 30 days prior to screening or plan to receive an investigational drug (other than gene therapy) during the study.
- Enrollment and participation in another treatment clinical trial 90 days before first visit.
- Diagnosis of a second neurodegenerative/peripheral neuropathy disease or another genetic syndrome with a progressive course.
- Advanced stage disease defined by the use of chronic invasive ventilatory support (tracheostomy with ventilator dependence) and a non-communicative status.
- Active viral infection (including HIV or serology positive for Hepatitis B or C, or COVID-19).
- Bacterial infection requiring antibiotics within the 6 weeks prior to infusion.
- Positive beta hCG pregnancy test (females of childbearing age will have pregnancy test at Screening and Day-1).
The study team makes the final eligibility decision.
Where it's taking place
- Palo Alto, California, United States
- Iowa City, Iowa, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 3 years to 20 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Palo Alto, California, United States; Iowa City, Iowa, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.