a long-term active treatment study of mongersen (ged-0301) in subjects with crohn's disease

Conditions: Crohn's Disease

Summary: the purpose of this study is to assess long-term safety data of ged-0301 for a period of up to 208 weeks in adult subjects (i.e., ≥ 18 years of age) who participated in the core phase 3 ged-0301-cd-002 and ged-0301-cd-003 studies and adolescent subjects (i.e., 12 to 17 years of age) who participated in the core phase 3 ged-0301-cd-003 study. although all subjects will receive active treatment, this study is double-blinded for the entire 208 weeks for the purpose of preserving the blind of the subject's treatment allocation in the initial, core phase 3 ged-0301 study. the ged-0301-cd-003 trial was not initiated; see detailed description.

this is a long-term active treatment study in patients with crohn's disease (cd). subjects who met the early escape criteria in study ged-0301-cd-002, or subjects who completed study ged-0301-002 or ged-0301-003, may be eligible for this study. primary objective is to assess long-term safety of ged 0301. additional efficacy and patient reported outcomes will be explored. there are 5 possible treatment groups for ged-0301-cd-002 subjects (groups 1-5). there are 3 possible treatment groups for ged-0301-cd-003 subjects (groups 1-3). treatment is assigned based on clinical improvement achieved or not achieved from the core ged-0301 study. 1. continuous ged-0301 160 mg once daily for 12 weeks, followed by alternating placebo once daily for 4 weeks with ged-0301 160 mg once daily for 4 weeks, through week 208; 2. alternating ged-0301 160 mg once daily for 4 weeks with placebo once daily for 4 weeks, through week 208; 3. alternating placebo once daily for 4 weeks with ged-0301 160 mg once daily for 4 weeks, through week 208; 4. continuous ged-0301 40 mg once daily through week 208; 5. alternating placebo once daily for 4 weeks, followed by ged-0301 40 mg once daily for 4 weeks, through week 208. the ged-0301-cd-003 trial was not initiated; the ged-0301 program was terminated; no safety findings were noted.

Eligibility criteria:
inclusion criteria for adult subjects: subjects must satisfy the following criteria to be screened and enrolled in the study: * male or female ≥ 18 years of age. * subject must have participated in the ged-0301-cd-002 or ged 0301 cd 003 study. * subject must use protocol approved contraception. inclusion criteria for adolescent subjects: adolescent subjects must satisfy the following criteria to be screened and enrolled in the study * male or female 12 to 17 years of age. * subject must have participated in the ged 0301 cd 003 study. * subject is able to swallow the ip tablets. * subject must use protocol approved contraception. exclusion criteria for adult and adolescent subjects: the presence of any of the following will exclude a subject from screening and enrollment: * subject had experienced a serious adverse event (sae) related to the investigational product while participating in the previous phase 3 ged-0301 study. * subject has initiated biologic agents, such as tnf-α blockers or integrin antagonists. * subject is pregnant or breastfeeding. * subject has developed a known hypersensitivity to oligonucleotides, ged 0301 or any ingredient in the investigational product.

Keywords: crohn's disease ged 0301 mongersen safety irritable bowel disease