Health Condition 1: 3- Administration Health Condition 2: G710- Muscular dystrophy
Conditions
Interventions
Intervention1: 2O Methyl Antisense Oligonucleotide: Two arms in the study. One with underlying steroid therapy plus the active 2O Antisense Oligonucleotide and the second arm with underlying steroid t
Sponsors
Dystrophy Annihilation Research Trust
Eligibility
Inclusion criteria
Inclusion criteria: A patient must meet all of the following criteria to be eligible for this study. 1) An established clinical diagnosis of DMD with mutation amenable for Exon 45 or 51 or 53 skipping confirmed by state-of-the-art DNA diagnostic technique like Next Generation Sequencing (NGS) 2) Male, at least 5 years of age at randomization 3) Able to rise from floor in ââ?°Â¤7 seconds (without aids/orthoses) 4) Able to complete the 6MWD test with a distance of at least 75m 5) should be on glucocorticoids for a minimum of six months immediately prior to screening, with no significant change in total daily dosage or dosing regimen for a minimum of 3 months immediately prior to screening and a reasonable expectation that total daily dosage and dosing regimen will not change significantly for the duration of the study (unless clinically indicated) 6) Has stable pulmonary function (FVC % of predicted ââ?°Â¥50% and no requirement for nocturnal ventilation) that, in Investigatorââ?¬•s opinion, is unlikely to decompensate over the duration of the study 7) Echo with EF >45% 8) Life expectancy of at least 1 year 9) No previous treatment with investigational medicinal treatment within six months prior to the study 10) Willing and able to comply with all study requirements and (self or through parent/ guardian) procedures (with the exception of those assessments requiring a subject to be ambulant, for those subjects who have lost ambulation) 11) Able to give informed assent and/or consent in writing by the subject and/or parent(s)/legal guardian (according to local regulations)
Exclusion criteria
Exclusion criteria: � A patient who meets any of the following criteria will be excluded from this study. � Aberrant RNA splicing and/or aberrant response to Exon Skipping � FVC � Current or history of liver or renal disease � Acute illness within 4 weeks prior to treatment which may interfere with the measurements � Severe mental retardation which in the opinion of the investigator prohibits participation in this study � Severe cardiac myopathy which in the opinion of the investigator prohibits participation in this study � Need for mechanical ventilation � Creatinine concentration above 1.5 times the upper limit of normal (age corrected) � Serum ASAT and/or ALAT concentration(s) which suggest hepatic impairment � Use of anticoagulants, anti-thrombotic or anti-platelet agents � Positive hepatitis B surface antigen, hepatitis C antibody test, or human immunodeficiency virus (HIV) test at screening, � History of significant medical disorder which may confound the interpretation of safety data (e.g. current or history of renal or liver disease/impairment, history of inflammatory illness) � Symptomatic cardiomyopathy. If subject has a left ventricular ejection fraction � A platelet count under the lower limit of normal (LLN) at start of this study. A re-test is possible at a later stage, and if within normal range, the subject may enter the study � Current or history of drug and/or alcohol abuse
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Primary Endpoints: Efficacy ââ?¬Â¢ Change from Baseline at Week 24 in 6MWT. ââ?¬Â¢ NSAA total score Safety The safety and tolerability of 2ââ?¬â?¢O Methyl Antisense Oligonucleotide will be assessed through a review and evaluation of AEs, serious adverse events (SAEs), deaths and discontinuations due to AEs; laboratory testing including hematology, coagulation, chemistry and urinalysis, Echo, ECG; vital signs; and physical examination findings. Timepoint: 24 weeks safety and efficacy 48 weeks safety and efficacy 52 weeks safety follow up | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary Endpoints: ââ?¬Â¢ Ability to rise independently from the floor (without external support) at Week 24 ââ?¬Â¢ Time to Loss of Ambulation (LOA) from randomization through Week 24. ââ?¬Â¢ Change from Baseline at Week 24 in: o forced vital capacity percent (FVC%) predicted o Frequency of falls o LVEF Timepoint: Baseline vs 24 weeks and 48 weeks | — |
Countries
India
Contacts
Public ContactDr Arun Shastry
Vibrance Clinical Research
Outcome results
None listed