Drugs / DIRLOCTOCOGENE SAMOPARVOVEC
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT03003533 | Jan 2017 → Dec 2023 | hemophilia A | Spark Therapeutics, Inc. | Completed | No outcome recorded |
| Phase 31 trial | ||||||
| Phase 3 | NCT06297486 | Mar 2024 → May 2027 | hemophilia A | Spark Therapeutics, Inc. | Withdrawn | No outcome recorded Stop: Business |
News releases announcing trial results or a regulatory action · 2
| Date | Issuer | Release |
|---|---|---|
| 2021-07-02 | Hoffmann-La Roche | Results Roche to present new data at the ISTH 2021 Congress highlighting long-standing commitment to advancing haemophilia A standard of care roche.com ↗
Spark Therapeutics will share updated data from the ongoing phase I/II clinical trial of investigational gene therapy SPK-8011, demonstrating that hepatocyte expression of factor VIII can be stable and durable up to four years following vector administration, with an acceptable safety profile |
| 2020-06-29 | Hoffmann-La Roche | Results Roche announces new data at the ISTH 2020 Congress, demonstrating ongoing commitment to advancing care for people with haemophilia A roche.com ↗
Spark Therapeutics will present data from the initial dose cohorts of its phase I/II SPK-8011 gene therapy study, showing stable and durable factor VIII expression, substantial improvement in annualised bleed rate for more than two years, and an acceptable safety profile after more than two years of follow-up1 |
All press releases naming this drug 2 releases
| Date | Issuer | Release |
|---|---|---|
| 2021-07-02 | Hoffmann-La Roche | Roche to present new data at the ISTH 2021 Congress highlighting long-standing commitment to advancing haemophilia A standard of care roche.com ↗ |
| 2020-06-29 | Hoffmann-La Roche | Roche announces new data at the ISTH 2020 Congress, demonstrating ongoing commitment to advancing care for people with haemophilia A roche.com ↗ |
Evidence & citations 6 cited values
Every value below carries the sentence it was read from. 4 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | DIRLOCTOCOGENE SAMOPARVOVEC | ChEMBL registry synonym — accepted as the source's own label CHEMBL4650253 ↗ |
| Known as | AAV-Spark 200-hFVIII | ChEMBL registry synonym — accepted as the source's own label CHEMBL4650253 ↗ |
| Known as | SPK-8011 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03003533 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “investigational adeno-associated viral (AAV) vector (SPK-8011)” PMID 34788507 ↗ Nov 2021 |
| Route | Intravenous | “single intravenous (IV) infusion of SPK-8011” NCT03003533 ↗ |
| Target | F8 | “B-domain Deleted Human Factor VIII Gene” NCT06297486 ↗ |