UAB News, University of Alabama at Birmingham, November 04, 2015
“There
is a high demand for biomarkers because of ongoing clinical trials with
Friedreich’s ataxia patients,” said Marek Napierala, Ph.D., assistant
professor in UAB Department of Biochemistry and Molecular Genetics, UAB
Stem Cell Institute. “We need better measures of the progression of the
disease and the therapeutic response.”
Friday, November 6, 2015
Safety, Tolerability and Efficacy of ACTIMMUNE Dose Escalation in Friedreich's Ataxia Study
ClinicalTrials.gov Identifier: NCT02593773, First received: October
29, 2015
The purpose of this phase 3 multi-center, open-label extension study is to evaluate the long-term safety of ACTIMMUNE (interferon-γ 1b) in subjects with Friedreich's Ataxia (FA).
Drug: Interferon γ-1b, Other Name: ACTIMMUNE
Approximately 90 subjects will receive subcutaneous (SC) doses of ACTIMMUNE three times a week (TIW) for a total of 26 weeks. The study drug dose is planned to be escalated on a weekly basis over the first 4 weeks of treatment (from 10 µg/m² to 25, 50, and 100 µg/m²). The dose may be reduced, interrupted, or held based on tolerability. By week 13, all subjects are to be on a stable tolerated dose of study drug in order to continue study participation; the dose may not be further increased after week 13, however, it may be reduced on a case-by-case basis to manage drug-related AEs.
The purpose of this phase 3 multi-center, open-label extension study is to evaluate the long-term safety of ACTIMMUNE (interferon-γ 1b) in subjects with Friedreich's Ataxia (FA).
Drug: Interferon γ-1b, Other Name: ACTIMMUNE
Approximately 90 subjects will receive subcutaneous (SC) doses of ACTIMMUNE three times a week (TIW) for a total of 26 weeks. The study drug dose is planned to be escalated on a weekly basis over the first 4 weeks of treatment (from 10 µg/m² to 25, 50, and 100 µg/m²). The dose may be reduced, interrupted, or held based on tolerability. By week 13, all subjects are to be on a stable tolerated dose of study drug in order to continue study participation; the dose may not be further increased after week 13, however, it may be reduced on a case-by-case basis to manage drug-related AEs.
Frataxin Is Localized to Both the Chloroplast and Mitochondrion and Is Involved in Chloroplast Fe-S Protein Function in Arabidopsis.
Turowski VR, Aknin C, Maliandi MV, Buchensky C, Leaden L, Peralta DA,
Maria V. Busi, Alejandro Araya, Diego F. Gomez-Casati, PLoS ONE 10(10):
e0141443. doi:10.1371/journal.pone.0141443
A personal perspective of orphan drug development for rare diseases: A golden opportunity or an unsustainable future?
Oo, C. and Rusch, L. M. Journal of Clinical Pharma. doi: 10.1002/jcph.599
Nevertheless, there is no better opportunity than the present to develop orphan drugs in order to accelerate the treatment and alleviate the suffering of patients with rare diseases.
Nevertheless, there is no better opportunity than the present to develop orphan drugs in order to accelerate the treatment and alleviate the suffering of patients with rare diseases.
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