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)
Recruiting
N/A
Interventional Study
MECP2 Duplication Syndrome
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
At a Glance
- Age
- 2 – 18
- Sex
- Male
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 6 (estimated)
- Sponsor
- HuidaGene Therapeutics Co., Ltd. · Industry
Think this trial could be right for you?
Answer a few quick questions to see if you may meet the eligibility requirements.
Check Your Eligibility
About This Trial
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 premat…
Trial Locations
Loading…
Loading trial locations…
Eligibility Criteria
Inclusion 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 …
Contacts