Charcot-Marie-Tooth disease type 1A

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ORPHA:101081OMIM:118220G60.0
Who is this for?
Show terms as
5Active trials33Specialists8Treatment centers

Where are you in your journey?

UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

Also known as:

Clinical phenotype terms— hover any for plain English:

Decreased motor nerve conduction velocityHP:0003431Decreased sensory nerve conduction velocityHP:0003448Demyelinating peripheral neuropathyHP:0007108Sensory ataxiaHP:0010871Acute demyelinating polyneuropathyHP:0007131Diaphragmatic weaknessHP:0009113Spontaneous pain sensationHP:0010833Shoulder painHP:0030834Hyperactive deep tendon reflexesHP:0006801
Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

6 events
Apr 2027Phase I/IIa Trial of scAAV1.tMCK.NTF3 for Treatment of CMT1A

Nationwide Children's Hospital — PHASE1, PHASE2

TrialNOT YET RECRUITING
Sep 2025A First in Human Study to Assess the Safety, Tolerability, and Pharmacokinetics of EDK060 in Adults With CMT1A.

Novartis Pharmaceuticals — PHASE1

TrialRECRUITING
Jun 2025Identification of Novel Biomarkers in Early Charcot-Marie-Tooth 1A Disease

Assistance Publique Hopitaux De Marseille — NA

TrialRECRUITING
Mar 2025A Multi-omic Approach to the Identification of Novel Biomarkers in Early Charcot-Marie-Tooth 1A Disease (CMT1A)

University Medical Center Goettingen

TrialRECRUITING
Nov 2024Longitudinal Biomarkers With Selected Outcome Measures In CMT

University Medical Center Goettingen

TrialRECRUITING
Jun 2016Tools for Therapeutic Evaluation in Charcot-Marie-Tooth Disease Type 1A: Outcome Measures and Biomarkers

Assistance Publique Hopitaux De Marseille — NA

TrialACTIVE NOT RECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

No FDA-approved treatments are currently listed for Charcot-Marie-Tooth disease type 1A.

5 clinical trialsare actively recruiting — trials can provide access to cutting-edge therapies.

View clinical trials →

Clinical Trials

5 recruitingView all trials with filters →
Phase 11 trial
A First in Human Study to Assess the Safety, Tolerability, and Pharmacokinetics of EDK060 in Adults With CMT1A.
Phase 1
Actively Recruiting
· Sites: Ottawa, Ontario; Montreal, Quebec +1 more · Age: 1860 yrs
N/A2 trials
Identification of Novel Biomarkers in Early Charcot-Marie-Tooth 1A Disease
N/A
Actively Recruiting
PI: François CREMIEUX (Assistance Publique - Hôpitaux de Marseille) · Sites: Marseille · Age: 1030 yrs
Tools for Therapeutic Evaluation in Charcot-Marie-Tooth Disease Type 1A: Outcome Measures and Biomarkers
N/A
Active
PI: Urielle Desalbres (Assistance Publique Hôpitaux de Marseille) · Sites: Marseille; Montpellier · Age: 1870 yrs
Other2 trials
Longitudinal Biomarkers With Selected Outcome Measures In CMT
Actively Recruiting
· Sites: Göttingen, Lower Saxony · Age: 1865 yrs
A Multi-omic Approach to the Identification of Novel Biomarkers in Early Charcot-Marie-Tooth 1A Disease (CMT1A)
Actively Recruiting
PI: Michael W Sereda (University Medical Centre Göttingen) · Sites: Göttingen · Age: 1030 yrs

Specialists

Showing 25 of 33View all specialists →
MS
Michael W Sereda
Specialist
PI on 1 active trial2 Charcot-Marie-Tooth disease type 1A publications
MN
Michael W. Sereda, MD, Professor of Neurology
Specialist
PI on 1 active trial
SM
Shahram Attarian, MD
Specialist
PI on 1 active trial
SM
Sharam Attarian, MD
Specialist
PI on 1 active trial
SM
Shahram ATTARIAN, MD
Specialist
PI on 1 active trial
CM
C. Verhamme, MD
Specialist
PI on 1 active trial
PM
Peter Young, MD
Specialist
PI on 1 active trial
UD
Urielle Desalbres
Specialist
PI on 21 active trials1 Charcot-Marie-Tooth disease type 1A publication
MM
Mario Saporta, MD
MIAMI, FL
Specialist
PI on 1 active trial
VP
Viviane BERTRAND, PhD
Specialist
PI on 1 active trial
EC
Emmanuel COUDEYRE
Specialist
PI on 1 active trial
RM
Richard A Lewis, MD
Specialist
PI on 1 active trial
EC
Emmanuel Coudeyre
Specialist
PI on 1 active trial
TM
Teresa Sevilla, MD
Specialist
PI on 1 active trial
JM
Jack Puymirat, MD
Specialist
PI on 1 active trial
PM
Philip Van Damme, MD
Specialist
PI on 1 active trial
FM
Florian Thomas, MD
Specialist
PI on 1 active trial
MM
Mark Roberts, MD
Specialist
PI on 1 active trial
MM
Marianne De Visser, MD
Specialist
PI on 1 active trial
MP
M. de Visser, MD, PhD
Specialist
PI on 1 active trial
FP
F. Baas, MD, PhD
Specialist
PI on 1 active trial
RP
R. de Haan, MD, PhD
Specialist
PI on 1 active trial

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Travel Grants

No travel grants are currently matched to Charcot-Marie-Tooth disease type 1A.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Charcot-Marie-Tooth disease type 1A

Disease timeline:

New recruiting trial: A First in Human Study to Assess the Safety, Tolerability, and Pharmacokinetics of EDK060 in Adults With CMT1A.

A new clinical trial is recruiting patients for Charcot-Marie-Tooth disease type 1A

New recruiting trial: A Multi-omic Approach to the Identification of Novel Biomarkers in Early Charcot-Marie-Tooth 1A Disease (CMT1A)

A new clinical trial is recruiting patients for Charcot-Marie-Tooth disease type 1A

Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Common questions about Charcot-Marie-Tooth disease type 1A

Are there clinical trials for Charcot-Marie-Tooth disease type 1A?

Yes — 5 recruiting clinical trials are currently listed for Charcot-Marie-Tooth disease type 1A on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Charcot-Marie-Tooth disease type 1A?

25 specialists and care centers treating Charcot-Marie-Tooth disease type 1A are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.