Clinical trial NCT02310763 (phase 2, status: terminated)
Conditions: duchenne muscular dystrophy
ICD codes: G71.01
Drugs: placebo
SMILES: CN1C(=O)C=C(N2CCC[C@@H](N)C2)N(CC2=C(C=CC=C2)C#N)C1=O
Reason stopped: 
    termination date 30aug2018: reason for termination: lack of efficacy
  
Eligibility criteria:

        Inclusion Criteria:

          1. Ambulatory boys age 6 to <16 years old (at the time of randomization), diagnosed with
             DMD. Diagnosis must be confirmed in subject's medical history and by genetic testing
             obtained during routine clinical care for diagnostic purposes as reported from an
             appropriate regulated laboratory using a clinically validated genetic test (genetic
             testing is not provided by the sponsor).

          2. Subjects who are able to perform the 4 stair climb in > or = 0.33 but < or =1.6
             stairs/second.

          3. Subjects must be receiving glucocorticosteroids for a minimum of 6 months prior to
             signing informed consent. There should be no significant change (>0.2 mg/kg) in dosage
             or dose regimen (not related to body weight change) for at least 3 months immediately
             prior to signing the informed consent and a reasonable expectation that dosage and
             dosing regimen will not change significantly for the duration of the study.

          4. Adequate hepatic and renal function on screening laboratory assessments.

          5. No underlying disposition for iron accumulation on screening laboratory assessments.

          6. Iron content estimate on the screening liver MRI is within the normal range.

        Exclusion Criteria:

          1. Subjects with known cognitive impairment or behavioral issues that would impede the
             ability to follow instructions.

          2. History of major surgical procedure within 6 weeks of signing the informed consent or
             planned surgery during the study.

          3. Any injury which may impact functional testing. Previous injuries must be fully healed
             prior to consenting. Prior lower limb fractures must be fully healed and at least 3
             months from injury date.

          4. Presence or history of other musculoskeletal or neurologic disease or somatic disorder
             not related to DMD including pulmonary and cardiac disease.

          5. Compromised cardiac function (left ventricular ejection fraction <55% as determined on
             a screening cardiac MRI or echocardiogram). Subjects may be receiving ACE (angiotensin
             converting enzyme) inhibitors or beta blockers, ARB (angiotensin II receptor
             antagonist) or aldosterone blocker/thiazide diuretic; however they must have initiated
             treatment more than 3 months prior to screening to ensure stable therapy.

          6. Evidence or history of clinically significant hematological, renal, endocrine,
             pulmonary, gastrointestinal, cardiovascular (including uncontrolled hypertension),
             hepatic, neurologic, or allergic disease (including drug allergies, but excluding
             untreated, asymptomatic, seasonal allergies at time of dosing).

          7. Documented history of iron overload including hemochromatosis, beta thalassemia major,
             beta thalassemia intermedia or hemolytic anemia.

          8. Unwilling or unable (eg, metal implants, requires sedation) to undergo examination
             with closed MRI without sedation.

          9. Participation in other studies involving investigational drug(s) for a minimum of 30
             days or within 5 half lives (whichever is longer) prior to signing the informed
             consent and/or during study participation.

         10. Current or prior treatment with anti-myostatin, exon skipping, nonsense mutation
             targeted therapies ever or more than 30 days of treatment with utrophin modifiers and
             treatment with utrophin modifiers within 30 days prior ot signing the informed consent
             and/or during study participation.

         11. Current or prior treatment within the past 3 months with androgens or human growth
             hormone.

         12. Current treatment with immunosuppressant therapies (other than glucocorticoid
             steroids), aminoglycosides (eg, gentamicin), multi vitamins with iron and iron
             supplements and other investigational therapies (including idebenone).