Home » Publications (page 2)

Publications

Concluded/Terminated Studies-Trials

Assessment of long-term safety of deferiprone in children (DEEP-3 Study)     Update: 20 December 2018 The study has concluded.   Update: 02 October 2018 The safety of deferiprone, either alone or in combination with deferoxamine, was evaluated in 297 patients from 16 hospitals in 6 Mediterranean countries, who had ...

Read More »

Gene Therapy

ZYNTEGLO (ex-Lentiglobin) gene therapy for patients with TDT   Update: 27 September 2019 No update available   Update: 30 July 2019 Updated results from the completed Phase 1/2 Northstar (HGB-204) study, and new data from the Phase 3 Northstar-2 (HGB-207) and Phase 3 Northstar-3 (HGB-212) clinical studies of its LentiGlobin® ...

Read More »

Gene Editing

CRISPR gene editing   Update: 27 September 2019 Enrollment in Phase 1/2 study of CTX001 in patients with TDT is ongoing. Based on the progression of the program, CRISPR Therapeutics expects to obtain preliminary safety and efficacy data in late 2019. The first patient treated with CTX001 in a Phase ...

Read More »

Hepcidin

Hepcidin trials – TRANSCEND   Update: 27 September 2019 No update available   Update: 30 July 2019 No update available.   Update: 30 May 2019 No update available.   Update: 01 February 2019 Hepcidin is a hormone that performs various functions related to iron absorption and distribution in the body.  ...

Read More »

Improving safety of blood transfusion

    INTERCEPT-treated RBCs   Update: 27 September 2019 No update available   Update: 30 July 2019 No update available.   Update: 30 May 2019 No update available.   Update: 01 February 2019 The SPARC study was a randomized, double blinded cross-over study evaluating the efficacy and safety of INTERCEPT ...

Read More »