Drugs / G1XCGD
G1XCGD
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT02234934 | Oct 2015 → Sep 2024 | granulomatous disease, chronic, X-linked | University of California, Los Angeles | Completed | No outcome recorded |
| Phase 1/2 | NCT01855685 | Jun 2013 → Jun 2021 | granulomatous disease, chronic, X-linked | Genethon | Terminated | No outcome recorded |
News releases announcing trial results or a regulatory action · 3
| Date | Issuer | Release |
|---|---|---|
| 2020-01-30 | Genethon | Results Genethon welcomes the conclusive results of a gene therapy trial in Chronic Septic Granulomatosis, a rare disease of the immune system genethon.com ↗
Six of the nine patients are free of treatments related to complications generated by the disease. |
| 2019-02-25 | Orchard Therapeutics | Results Orchard Therapeutics Presents Clinical Proof-of-Concept Data for OTL-102 for the Treatment of X-CGD orchard-tx.com ↗
Six of seven eligible patients showed greater than 10% (ranging from 16%-46%) functioning, oxidase-positive neutrophils in circulation at 12 months, which is the minimum threshold of oxidase-positive neutrophils necessary to demonstrate potential clinical benefit |
| 2018-12-05 | Orchard Therapeutics | Results Donald B. Kohn, MD, Orchard Therapeutics Scientific Advisory Board Member, Presents Clinical Proof-of-Concept Data for OTL-102 Gene Therapy for the Treatment of Patients with X-CGD at the Presidential Symposium at the 2018 ASH Annual Meeting orchard-tx.com ↗
The clinical proof-of-concept data are from seven patients (ages 2-27) severely affected by X-CGD treated with OTL-102, an autologous ex vivo lentiviral gene therapy which utilizes a self-inactivating lentiviral vector (G1XCGD). |
All press releases naming this drug 3 releases
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 2 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | G1XCGD | “G1XCGD Lentiviral Vector” NCT01855685 ↗ |
| Known as | G1XCGD (pCCLChimGp91/VSVg lentiviral vector) | “G1XCGD (pCCLChimGp91/VSVg lentiviral vector)” NCT02234934 ↗ |
| Known as | G1XCGD Modified Autologous BM CD34 cells | “The therapeutic product to be evaluated is autologous CD34+ hematopoietic stem cells (HSC) modified by ex vivo transduction using the pCCLchimGP91WPRE lentiviral vector (G1XCGD...” NCT02234934 ↗ |
| Action | Restore | “to effectively restore NADPH-oxidase activity in granulocytes” NCT02234934 ↗ |
| Modality | Cell therapy | “autologous CD34+ hematopoietic stem cells (HSC) modified by ex vivo transduction” NCT02234934 ↗ |