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SPK-3006 · 1 trial · 7 indications
Adverse events.
| Arm | Type | Description |
|---|---|---|
| SPK-3006 | EXPERIMENTAL | All participants who meet the eligibility criteria will receive a single intravenous (i.v.) administration of SPK-3006. |
| Name | Type | Description |
|---|---|---|
| SPK-3006 | GENETIC | adeno-associated viral (AAV) vector |
Inclusion Criteria: * Provide written informed consent; * Males and Females ≥18 years of age with late-onset Pompe disease; * Received ERT for at least the previous 24 months * Have clinically moderate, late-onset Pompe disease characteristics; * Agree to use reliable contraception. Exclusion Crit...
SPK-3006 is an investigational gene therapy being developed for the treatment of Pompe disease, a rare lysosomal storage disorder. It is specifically being studied in patients with late-onset Pompe disease, also known as glycogen storage disease type 2 or acid maltase deficiency. The therapy is currently in Phase 1 clinical development.
SPK-3006 is a gene therapy designed to address the underlying genetic cause of Pompe disease. The therapy is intended to deliver a functional gene to produce the enzyme acid alpha-glucosidase, which is deficient in patients with Pompe disease. This enzyme is responsible for breaking down glycogen in cells, and its deficiency leads to the accumulation of glycogen, particularly in muscle tissue.
SPK-3006 is being developed by Roche Holding AG, a multinational healthcare company. Roche's stock is traded on the OTC market under the ticker symbol RHHBY. The company is conducting clinical trials to evaluate the safety and efficacy of SPK-3006 in patients with late-onset Pompe disease.
SPK-3006 is currently in Phase 1 clinical development. It is an investigational gene therapy that has not yet been approved by regulatory authorities. The ongoing Phase 1 trial is designed to evaluate the safety, tolerability, and preliminary efficacy of SPK-3006 in patients with late-onset Pompe disease.
SPK-3006 is being evaluated in a Phase 1 clinical trial with the identifier NCT04093349, titled "A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)." This trial is currently active but not recruiting participants. The study is enrolling approximately 4 adult patients with late-onset Pompe disease across multiple countries, including the United States, Canada, and several European nations.
SPK-3006 is a specific gene therapy candidate developed by Roche for late-onset Pompe disease. It is distinct from other gene therapies in development for this condition, as each therapy uses different viral vectors and delivery methods. The RESOLUTE trial is evaluating SPK-3006 as a potential one-time treatment to address the root cause of the disease.