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Tests treatment safety and results for Duchenne Muscular Dystrophy (DMD)
Official title Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
ClinicalTrials.gov ID: NCT07188012
What this study is testing
What is SPOT-03?
SPOT-03 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for duchenne muscular dystrophy (dmd).
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
- The primary objective of this study is to evaluate the safety and tolerability of SPOT-03 administered by intravenous (IV) infusion to DMD patients. In addition, this study will preliminarily investigate the changes in dystrophin nucleic acid concentration, dystrophin protein expression and engraftment, anti-dystrophin antibodies and cytokine profiles, as well as fat tissue mas and lean tissue mass following SPOT-03 administrations.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 7, men only
You may be able to join if
- According to the requirements of the region/country and/or IRB/IEC, the patient and/or legal guardian have signed a written informed consent form and...
- Boys aged ≥ 2 years to \< 8 years and capable of walking independently for at least 10 meters.
- The medical history includes clinical diagnosis of DMD and confirmed Duchenne mutations using validated genetic testing (MLPA and whole genome...
- Able to tolerate muscle biopsy under anesthesia and have no contraindications to biopsy.
- Heart, liver, lung, and kidney functions are sufficient:
You likely can't join if
- Complications other than DMD that may cause muscle weakness and/or motor dysfunction.
- There are severe intellectual disabilities (such as severe autism, severe cognitive impairment, and severe behavioral disorders) that, according to...
- Hospitalization for respiratory failure within 8 weeks prior to screening.
- Asthma or underlying lung diseases that are poorly controlled, such as bronchitis, bronchiectasis, emphysema, or recurrent infectious pneumonia that...
- Severe uncontrolled heart failure (NYHA III-IV), including any of the following conditions：
- Intravenous administration of diuretics or positive inotropic drugs is required within 8 weeks prior to screening.
See the full eligibility criteria
- According to the requirements of the region/country and/or IRB/IEC, the patient and/or legal guardian have signed a written informed consent form and are aware of all relevant study content.
- Boys aged ≥ 2 years to \< 8 years and capable of walking independently for at least 10 meters.
- The medical history includes clinical diagnosis of DMD and confirmed Duchenne mutations using validated genetic testing (MLPA and whole genome sequencing).
- Able to tolerate muscle biopsy under anesthesia and have no contraindications to biopsy.
- Heart, liver, lung, and kidney functions are sufficient:
- The left ventricular ejection fraction (LVEF) should be ≥ 50%;
- Forced vital capacity (FVC) \> 50% of the expected value, and do not require nighttime ventilation;
- Patient's glomerular filtration rate (GFR)\>30 mL/min/1.73 m2
- Complications other than DMD that may cause muscle weakness and/or motor dysfunction.
- There are severe intellectual disabilities (such as severe autism, severe cognitive impairment, and severe behavioral disorders) that, according to the investigator's judgment, can affect the study.
- Hospitalization for respiratory failure within 8 weeks prior to screening.
- Asthma or underlying lung diseases that are poorly controlled, such as bronchitis, bronchiectasis, emphysema, or recurrent infectious pneumonia that investigator believes may affect respiratory function.
- Severe uncontrolled heart failure (NYHA III-IV), including any of the following conditions：
- Intravenous administration of diuretics or positive inotropic drugs is required within 8 weeks prior to screening.
- Hospitalization due to worsening heart failure or arrhythmia within 8 weeks prior to screening.
- Abnormal laboratory values considered clinically significant:
- GGT \> 3 × upper limit of normal
- Bilirubin ≥ 3.0 mg/dL
- Creatinine ≥ 1.8 mg/dL
- Hemoglobin \ 18 g/dL
- White blood cell count \> 18,500/μL
- Arrhythmias that require anti-arrhythmic treatment.
- people who are undergoing immunosuppressive therapy.
- Has used other gene therapy, investigational drugs, or any treatment aimed at increasing dystrophin expression.
- people with a history of major surgeries within 12 weeks prior to the initial infusion or planning to undergo major surgeries (such as scoliosis surgery) during this study.
- people who are allergic to investigational products or local aesthetic drugs or have a history of severe allergies or genetic allergic reactions.
- Within 6 months prior to the initial infusion, the people are exposed to another investigational drug or have participated in an intervention clinical trial.
- people with positive hepatitis B core antibody or hepatitis C antibody or HIV antibody during screening.
- Investigator believes that the presence of any other serious diseases, medical conditions, or chronic drug treatment needs can pose unnecessary risks to gene transfer.
The study team makes the final eligibility decision.
Where it's taking place
- Shanghai, Shanghai Municipality, China
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 male, 2 years to 7 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Shanghai, Shanghai Municipality, China. 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.