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Duchenne Clinical Trials Enrolling Or Soon to Be Enrolling Since 2001, Jett Foundation has met the needs of the Duchenne muscular dystrophy community. While we were founded out of one family’s desperate need to save their son, we have transformed dramatically. Once a non-profit dedicated to raising money for biomedical research, today we are meeting our families and young people where they are with direct service and educational programs that improve and enrich the lives of individuals impacted by Duchenne. We empower patients and families with the knowledge needed to be their own best advocates, spread awareness about Duchenne, and help accelerate research and development. We believe for Duchenne patients, education equals survival. Please use this sheet only as an outline when learning about potiential clinical trial options for you, your child, or your patient. DUCHENNE CLINICAL TRIALS ENROLLING OR SOON TO BE ENROLLING STUDY NAME & SPONSOR STUDY DESCRIPTION AGE CRITERIA AMBULATION METHOD STEROIDS RECRUITMENT WVE-210201 Phase 1 Clinical Trial -Exon-skipping 51 ≥ 5 and ≤ 18 years *Both Infusion Yes Enrolling (US) (WVE-DMDX51-001) -Phase 1 [Wave Life Sciences] -Double-blind, placebo controlled, single ascending dose Study of SRP-4045 and SRP-4053 in DMD Patients -Exon-skipping 45 and 53 ≥ 7 and ≤ 13 years Ambulatory Infusion Yes Enrolling (US) (ESSENCE) - NCT02500381 -Phase III [Sarepta Therapeutics, Inc.] -Double-blind, placebo-controlled Safety Study of Eteplirsen to Treat Early Stage -Exon-skipping 51 EXONDYS 51 ≥ 4 and ≤ 6 years Ambulatory Infusion Enrolling (US) Duchenne - NCT02420379 -Phase II [Sarepta Therapeutics, Inc.] -Open-label Long-term Outcomes of Ataluren with Duchenne - -Nonsense mutation (stop codon) ≥ 5 years Ambulatory Oral Enrolling (US) NCT03179631 [PTC Therapeutics, Inc.] -Phase III -Double-blind, placebo controlled Systemic Gene Delivery Clinical Trial NCT03375164 -Gene Therapy Cohort A: Ambulatory Infusion Yes Enrolling (US) [Nationwide Children’s Hospital] -Phase I/II ≥ 3 months ≤ 3 years -Open-label, single-dose Cohort B: ≥ 4 s ≤ 7 years A Study to Evaluate the Safety and -Gene Therapy ≥ 5 and ≤ 12 years Ambulatory Infusion Yes Soon to be Tolerability of PF-06939926 -Phase I Enrolling (US) Gene Therapy in Duchenne - NCT03362502 -Open-label [Pfizer, Inc.] A Study to Assess the Efficacy and Safety of MNK- -Cosyntropin vs. Placebo ≥ 4 and ≤ 8 years Ambulatory Injection Yes Enrolling (US) 1411 in Duchenne Muscular Dystrophy (BRAVE) -Phase II NCT03400852 [Mallinckrodt] -Double-blind, multiple dose, placebo controlled Microdystrophin Gene Transfer Study in Adoles- -Microdystrophin Gene Transfer ≥ 4 and ≤ 17 years *Both Infusion Yes Enrolling (US) cents and Children With DMD (IGNITE DMD) -Phase I/II [Solid Biosciences] -Open-label, randomized Oral Ifetroban in Subjects With Duchenne -Oral Ifetroban ≥ 7 years *Both Oral *Both Soon to be NCT03340675 [Cumberland Pharmaceuticals] -Phase II Enrolling (US) -Open-label, dose-ranging Plus Epicatechin Duchenne Muscular Dystrophy in -Epicatechin on pre-clinical cardio- ≥ 8 years ≤ 17 years Non-Ambulatory Oral Yes Enrolling (US) Non-Ambulatory Adolescents- NCT02964377 myopathy [Craig McDonald, University of California, -Phase I/II Davis] -Open-label Transplantation of Myoblasts to Duchenne -Transplantation of myoblasts ≥ 6 and ≤ 16 years *Both Injection Enrolling (Canada) Patients - NCT02196467 [Centre Hospitalier -Phase I/II Universitaire de Québec] -Double-blind, placebo-controlled All data is as of 01/18/2018 *Non-Ambulatory & Ambulatory patients permitted Mutation Specific Clinical Trial Every effort has been made to ensure the accuracy, completeness and reliability of the information provided, Jett Foundation assumes no responsibility therefore and the user of the information agrees that the information is subject to change without notice. 36 CORDAGE PARK CIRCLE • SUITE 328 • PLYMOUTH, MA 02360 • O: 781-585-5566 • F: 781-585-5233 jettfoundation.org STUDY NAME & SPONSOR STUDY DESCRIPTION AGE CRITERIA AMBULATION METHOD STEROIDS RECRUITMENT Nebivolol for the Prevention of Left Ventricular -Nebivolol to prevent heart disease ≥ 10 and ≤ 15 years *Both Oral Enrolling (France) Systolic Dysfunction in Patients with Duchenne - -Phase III NCT01648634 -Double-blind, placebo-controlled [Hôpital Ambroise-ParéHôpitaux Universitaires] Ramipril Versus Carvedilol in Duchenne -Carvedilol vs Ramipril ≥ 2 and ≤ 45 years *Both Oral Yes Enrolling (Italy) and Beckers Patients - NCT00819845 -Phase IV-Open-label, randomized [Catholic University, Italy] Therapeutic Potential for Aldosterone Inhibition -Spironolactone vs. Eplerenone ≥ 10 years Non-Ambulatory Oral Enrolling (US) in Duchenne - NCT02354352 to preserve cardiac & pulmonary [Ohio State University] function -Phase III -Double-blind, randomized A Phase III Double-blind Study With Idebenone in -Idebenone on preserving respiratory ≥ 10 years *Both Oral Yes Enrolling (US) Patients with Duchenne - NCT02814019 function [Santhera Pharmaceuticals] -Phase III -Double-blind, placebo-controlled Study of Investigational Drug, BMS-986089, in -Anti-myostatin ≥ 6 and ≤ 11 years Ambulatory Injection Yes Enrolling (US, Canada) Ambulatory Boys with DMD - NCT02515669 -Phase II/III [Hoffmann-La Roche] -Double-blind, placebo-controlled A Phase 2 Study to Evaluate the Safety, Efficacy, -Anti-myostatin ≥ 6 and ≤ 15 years Ambulatory Infusion Yes Enrolling (US, Canada, Pharmacokinetics and Pharmacodynamics of -Phase II UK, Italy, Japan) PF-06252616 - NCT02310763 -Double-blind, placebo-controlled [Pfizer, Inc.] Clinical Stuy to Evaluate the Efficacy and Safety -Givinostat vs. Placebo ≥ 6 and ≤ 17 years Ambulatory Oral Enrolling (US, Canada, of Givinostat in Ambulant Patients - NCT02439216 -Phase III UK, Europe) [Italfarmaco] -Double-blind, placebo-controlled A Study to Assess Vamorolone in Boys with -Disassociative steroid ≥ 4 and ≤ 6 years Ambulatory Oral Enrolling (US, Canada) Duchenne - NCT02760264 -Phase II [ReveraGen BioPharma, Inc.] -Open-label Finding the Optimum Regimen for Duchenne -Compare 3 ways of dispensing ≥ 4 and ≤ 7 years Ambulatory Oral Enrolling (US, Canada, Muscular Dystrophy (FOR-DMD) corticosteroids Germany, Italy, UK) NCT01603407 [University of Rochester] -Phase III -Double-blind, randomized Rimeporide in Patients with Duchenne (RIM4DMD) -NHE-1 inhibition (rimeporide) ≥ 6 and ≤ 14 years Ambulatory Oral Yes Enrolling (France, Italy, NCT02710591 [EspeRare Foundation] -Phase I Spain, UK) -Open-label Trial of FG-3019, a Monoclona Antibody to -Monoclonal antibody to connective ≥12 years Non-Ambulatory Infusion Yes Enrolling (US) CTGF, in Non-Ambulatory Subjects with Duchenne tissue growth factor NCT02606136 [FibroGen, Inc.] -Phase II -Open-label Stacking Exercises Aid the Decline in FVC -Lung volume recruitment ≥ 6 and ≤ 16 years *Both Device Enrolling (Canada) and Sick Time (STEADFAST) - NCT01999075 -Phase IV [Children’s Hospital of Eastern Ontario] -Single-blind, randomized Whole Body Vibration Therapy in Boys with Duch- -Vibration therapy ≥ 8 and ≤ 14 years Ambulatory Device Yes Enrolling (Canada) enne - NCT01954940 -Open-label, randomized [Children’s Hospital of Eastern Ontario] Tamoxifen in Duchenne Muscular Dystrophy -Tamoxifen vs. Placebo ≥ 6.5 and ≤ 16 years *Both Drug Only Not Yet Enrolling Not yet Enrolling NCT03354039 -Phase III Ambula- (Switzerland) [University Hospital, Basel, Switzerland] -Double-blind, placebo-controlled tory All data is as of 01/18/2018 *Non-Ambulatory & Ambulatory patients permitted Mutation Specific Clinical Trial Every effort has been made to ensure the accuracy, completeness and reliability of the information provided, Jett Foundation assumes no responsibility therefore and the user of the information agrees that the information is subject to change without notice. 36 CORDAGE PARK CIRCLE • SUITE 328 • PLYMOUTH, MA 02360 • O: 781-585-5566 • F: 781-585-5233 jettfoundation.org.
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