Myotonic dystrophy type 1 (DM1)
Conditions
Interventions
CGB1001 multiple dose:CGB1001 IV infusion, Q6W, 3 administrations total
Sponsors
Affiliated hangzhou first people's hospital, zhejiang university school of medicine
Eligibility
Sex/Gender
All
Age
18 Years to 50 Years
Inclusion criteria
Inclusion criteria: 1) Ages >=18 and = 100; 3) Age of onset of DM1 symptoms >= 12 years; 4) Presence of handgrip myotonia; 5) Ability to walk(Orthotics allowed, canes and walkers not allowed), able to walk at least 25 meters independently at the time of screening; 6) Reproductive status: female patients of reproductive age or male patients whose sexual partner is a female of reproductive age are willing to use medically approved highly effective contraceptive methods, such as intrauterine devices or condoms, from the signing of informed consent to 12 months after the use of the study drug (women of reproductive age include premenopausal women and postmenopausal women within 24 months); 7)Willing and able to provide signed and dated informed consent prior to any study-related procedures, willing and able to comply with all study procedures.
Exclusion criteria
Exclusion criteria: 1) Pregnant and lactating women; 2) Congenital DM1; 3) Known human immunodeficiency virus (HIV), hepatitis C test positive, hepatitis B surface antigen positiven; 4) Clinically significant abnormalities in the screening of laboratory values, rendering subjects unsuitable for inclusion; 5) Laboratory tests during the screening period showed Hb=2.5 ULN, or AST>=2.5 ULN; If the investigator considers that the subject's laboratory abnormalities may be non-pathological, the test may be repeated; 6) Have any of the following conditions: uncontrolled diabetes, congestive heart failure, symptomatic cardiomyopathy, symptomatic coronary artery disease, cancer (other than skin cancer) within five years, multiple sclerosis, or another serious condition; 7) History of major surgical procedures within 12 weeks prior to initiation of the study product administration or plans to undergo major surgical procedures (such as implantation of a cardiac defibrillator) during the study period; 8) Health conditions other than DM1 can seriously affect walking or participating in functional assessments; 9) There is an active infection that requires systemic antiviral or antimicrobial treatment and cannot be completed the day before study dosing; 10) A history of bleeding tendency or ongoing oral anticoagulant therapy; 11) Unwillingness or inability to comply with the research procedures (such as muscle biopsies) prescribed in this protocol, including follow-up, or unwillingness to cooperate fully with the investigator; 12) treatment with another investigational drug, biologic or device within one month of screening, or within the 5 half-life of the investigational drug, whichever is older; Any history of previous treatment with oligonucleotides, including siRNA; 13) A recent or current history of drug or alcohol abuse; 14) Developmental delay, intellectual disability, or significant behavioral neuropsychiatric manifestations; 15) Clinically significant electrocardiogram or echocardiogram abnormalities, such as atrioventricular block of more than 2 degrees, malignant ventricular arrhythmia, or obvious symptoms of cardiac insufficiency at screening; 16) Treatment with anti-myotonic drugs within 30 days prior to the first study drug administration for a cumulative period of more than 2 weeks. This may include, but is not limited to: phenytoin, carbamazepine, procainamide, propylpyramine, Nifedipine, acetazolamide, Clomipramine, imipramine, amitriptyline, taurine, quinine, Meciletine; 17) A history of allergy to local anesthetics used in the biopsy procedure or its components; 18) There is any condition in which the investigator considers the subject unsuitable for inclusion or likely to interfere with the subject's participation or completion of the study;
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence and severity of dose-limiting toxicity (DLT) within 28 days after CGB1001 administration; | — |
Secondary
| Measure | Time frame |
|---|---|
| Efficacy endpoints;Changes in drug concentrations; | — |
Countries
China
Contacts
Public ContactWang Ying
Affiliated hangzhou first people's hospital, zhejiang university school of medicine
Outcome results
None listed