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Tests treatment safety and results for MECP2 Duplication Syndrome
Official title A First-in-Human Clinical Trial to Evaluate the Safety, Tolerability, and Efficacy of a Novel CRISPR RNA-editing Therapy in Patients with Mecp2 Duplication Syndrome, a Rare Orphan Disease (HERO)
ClinicalTrials.gov ID: NCT06615206
What this study is testing
What is HG204?
HG204 is an investigational medicine, given as an injectable medicine, being studied as a potential treatment for mecp2 duplication syndrome.
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
- Methyl-CpG binding protein 2 (MECP2) is a dosage-sensitive, X-linked gene critical for central nervous system development and functional maintenance, which gain-of-function causes MECP2 duplication syndrome (MDS). Affecting primarily in males, this disorder is characterized by severe intellectual disability, motor dysfunction, infantile hypotonia, epilepsy, respiratory tract infections, and premature death before 25 years of age with no curative therapy.
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 18, men only
You may be able to join if
- Males ≥ 2 and ≤18 years at the time of signing informed consent;
- Genetic test and clinical confirmed diagnosis of MDS;
- Stable pattern of seizures, or has had no seizures while currently receiving medical treatment (including antiepileptics) and physical therapy are...
- Willing to adhere to protocol, including biological samples collection and hospitalization for intracerebroventricular injection surgery;
- Acceptable hematology, clinical chemistry, and urine laboratory parameters.
You likely can't join if
- MECP2 gene triplication;
- Concurrent genetic syndromes other than MDS;
- Significant brain or cerebellar atrophy, or other significant degenerative changes as shown in cranial MRI at screening;
- Prior or current hypertension, cardiomyopathy, myocardial ischemia or atrial fibrillation and other cardiovascular diseases;
- Prior central nervous system surgery within 6 months before enrolment;
- Systemic use of immunosuppressive drugs within 3 months before enrolment;
See the full eligibility criteria
- Males ≥ 2 and ≤18 years at the time of signing informed consent;
- Genetic test and clinical confirmed diagnosis of MDS;
- Stable pattern of seizures, or has had no seizures while currently receiving medical treatment (including antiepileptics) and physical therapy are stable for at least 2 months before screening;
- Willing to adhere to protocol, including biological samples collection and hospitalization for intracerebroventricular injection surgery;
- Acceptable hematology, clinical chemistry, and urine laboratory parameters.
- MECP2 gene triplication;
- Concurrent genetic syndromes other than MDS;
- Significant brain or cerebellar atrophy, or other significant degenerative changes as shown in cranial MRI at screening;
- Prior or current hypertension, cardiomyopathy, myocardial ischemia or atrial fibrillation and other cardiovascular diseases;
- Prior central nervous system surgery within 6 months before enrolment;
- Systemic use of immunosuppressive drugs within 3 months before enrolment;
- Prior gene therapy or oligonucleotide therapy treatments;
- Any other conditions that would not allow the potential subject to complete follow-up examinations during the study and would, in the opinion of the investigator, make the potential subject unsuitable for the study.
The study team makes the final eligibility decision.
Where it's taking place
- Beijing, Beijing 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 18 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Beijing, Beijing 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.