Phase
Condition
Muscular Dystrophy
Treatment
Placebo
Vamorolone
Clinical Study ID
Ages 18-64 Male
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
Subject or Subject's parent(s) or legal guardian (s) has (have) provided writteninformed consent and Health Insurance Portability and Accountability Act (HIPAA)authorization, where applicable, prior to any study-related procedures;
Subject is a male and has a confirmed diagnosis of Becker dystrophy as defined as:
Identifiable mutation within the DMD gene (deletion/duplication of one or moreexons), where reading frame can be predicted as 'in-frame', and clinicalpicture consistent with Becker dystrophy, OR
Complete dystrophin gene sequencing showing an alteration (small mutation,duplication, other) that is expected to allow production of an internallydeleted dystrophin protein, with a typical clinical picture of Beckerdystrophy;
Subject is ≥ 18 years of age and <65 years of age at time of informed consent;
Subject is able to perform the timed run/walk 10 meters assessment (TTRW) in ≤ 30sec at screening; assistive devices, cane or walker, are allowed.
Subject has an NSAA score ≤ 32 at screening.
Clinical laboratory test results are within the normal range at the Screening Visit,or if abnormal, are not clinically significant, in the opinion of the Investigator. (Note: Serum gamma glutamyl transferase [GGT], creatinine, and total bilirubin allmust be ≤ upper limit of the normal range at the Screening Visit). While AST and ALTcan be elevated due to disease of muscle or liver, the study PI will review anyincreases of AST or ALT. If above upper limit of normal (ULN), then study PI willassess whether the increases are likely of muscle origin to determine inclusion.
Subject has not received oral glucocorticoids or other oral immunosuppressive agentsfor at least 3 months prior to first administration of study medication. [Note:Inhaled and/or topical glucocorticoids are permitted if last use is at least 4 weeksprior to first administration of study medication or if administered at stable dosebeginning at least 4 weeks prior to first administration of study medication andanticipated to be used at the stable dose regimen for the duration of the study];
Subject has evidence of chicken pox immunity as determined by:
Presence of IgG antibodies to varicella, as documented by a positive testresult from the local laboratory from blood collected during the ScreeningPeriod; OR
Documentation, provided at the Screening Visit, that the subject has received 2doses of varicella vaccine, with or without serologic evidence of immunity,with the second of the 2 immunizations given at least 14 days prior to firstadministration of study medication;
Subject is willing and able to comply with scheduled visits, study medicationadministration plan, and study procedures.
Subject agrees to use barrier contraception methods during his participation in thisstudy and for 30 days after the tapering dose is completed.
Exclusion
Exclusion Criteria:
Subject has current or history of major renal or hepatic impairment, uncontrolleddiabetes mellitus (defined as a diagnosis of diabetes with random glucose more than 1.5x ULN at screening and the patient has symptoms of polyuria or polydipsia) orimmunosuppression;
Subject has current or history of chronic systemic fungal or viral infections;
Subject has had an acute illness within 4 weeks prior to the first dose of studymedication
Subject has used mineralocorticoid receptor agents, such as spironolactone,eplerenone, canrenone (canrenoate potassium), prorenone (prorenoate potassium), ormexrenone (mexrenoate potassium) within 4 weeks prior to administration of studymedication;
Subject has evidence of symptomatic cardiomyopathy [Note: Asymptomatic cardiacabnormality on investigation would not be exclusionary unless cardiac ejectionfraction is less than 40%];
Subject has an allergy or hypersensitivity to the study medication or to any of itsconstituents;
Subject has severe behavioral or cognitive problems that preclude participation inthe study, in the opinion of the Investigator;
Subject has previous or ongoing medical condition, medical history, physicalfindings or laboratory abnormalities that could affect safety, make it unlikely thattreatment and follow-up will be correctly completed or impair the assessment ofstudy results, in the opinion of the Investigator;
Subject is taking (or has taken within 4 weeks prior to first dose of studymedication) herbal remedies and supplements which can impact muscle strength andfunction (e.g., Co-enzyme Q10, creatine, etc);
Subject has been administered a live attenuated vaccine within 14 days prior to thefirst dose of study medication;
Subject is currently taking any other investigational drug or has taken any otherinvestigational drug within 3 months prior to first dose of study medication; or
Subject has previously been enrolled in the VBP15-BMD-001 study or any othervamorolone study.
Study Design
Study Description
Connect with a study center
Azienda Ospedale Universita Padova
Padova, 35129
ItalySite Not Available
Azienda Ospedale Universita Padova
Padua 3171728, 35129
ItalySite Not Available
University of Pittsburgh
Pittsburgh, Pennsylvania 15261
United StatesSite Not Available
University of Pittsburgh
Pittsburgh 5206379, Pennsylvania 6254927 15261
United StatesSite Not Available

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