Study of an Investigational Drug, RO7239361 (BMS-986089), in Ambulatory Boys With DMD | DuchenneXchange

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active, not recruiting

Study of an Investigational Drug, RO7239361 (BMS-986089), in Ambulatory Boys With DMD

key information

study id #: NCT02515669

condition: Muscular Dystrophy (DMD)

status: active, not recruiting

purpose:

The purpose of this study is to determine the safety and tolerability of RO7239361 in boys with Duchenne Muscular Dystrophy with any genetic mutation.

intervention: RO7239361, Placebo

mechanism of action: Anti-myostatin adnectin to promote muscle growth

results: https://clinicaltrials.gov/ct2/show/results/NCT02515669

last updated: July 08, 2019

study details

start date: December 2, 2015

estimated completion: February 8, 2018

phase of development: Phase 1/Phase 2

size / enrollment: 43

primary outcomes:

  • Safety Summary for the 24 Week Double-Blind Phase [ Time Frame: 24 weeks ]
    Percentage of participants with fatalities, adverse event (AEs) and serious adverse events (SAEs) up to Week 24.
    Participants in Panel 1 received 4 mg RO7239361; participants in Panel 2 received either 12.5 mg (weight between 15 and 45 kg) or 20 mg (weight >45 kg) RO7239361, and participants in Panel 3 and the Expansion Panel received either 35 mg (weight between 15 and 45 kg) or 50 mg (weight >45 kg) RO7239361.
  • Safety Summary for the Whole Study [ Time Frame: Baseline to 72 weeks ]
    Percentage of participants with fatalities, adverse event (AEs) and serious adverse events (SAEs) for the whole study.
    Participants in Panel 1 received 4 mg RO7239361; participants in Panel 2 received either 12.5 mg (weight between 15 and 45 kg) or 20 mg (weight >45 kg) RO7239361, and participants in Panel 3 and the Expansion Panel received either 35 mg (weight between 15 and 45 kg) or 50 mg (weight >45 kg) RO7239361.

secondary outcomes:

  • Maximum Observed Serum Concentrations (Cmax) of RO7239361 at Steady State for 4 mg QW, 12.5 mg QW and 35 mg QW Doses. [ Time Frame: Baseline to Week 24 ]
    PK parameter estimates at steady state. Steady state was achieved following approximately 12 weeks QW administration.
    Panel: 1 = 4mg QW, 2 = 12.5mg QW, 3 = 35mg QW
    Participants in Panel 1 received 4 mg RO7239361; participants in Panel 2 received either 12.5 mg (weight between 15 and 45 kg) or 20 mg (weight >45 kg) RO7239361, and participants in Panel 3 and the Expansion Panel received either 35 mg (weight between 15 and 45 kg) or 50 mg (weight >45 kg) RO7239361. No participants received the Panel 2 20mg dose.
  • Maximum Observed Serum Concentrations (Cmax) of RO7239361 at Steady State for 50 mg QW Dose. [ Time Frame: Baseline to Week 24 ]
    PK parameter estimates at steady state following approximately 12 weeks QW administration.
    Panel 3 = 50 mg QW
    Participants in Panel 1 received 4 mg RO7239361; participants in Panel 2 received either 12.5 mg (weight between 15 and 45 kg) or 20 mg (weight >45 kg) RO7239361, and participants in Panel 3 and the Expansion Panel received either 35 mg (weight between 15 and 45 kg) or 50 mg (weight >45 kg) RO7239361.
  • Time of Maximum Observed Serum Concentrations (Tmax) of RO7239361 at Steady State for 4 mg QW, 12.5 mg QW and 35 mg QW Doses. [ Time Frame: Baseline to Week 24 ]
    PK parameter estimates at steady state. Steady state was achieved following approximately 12 weeks QW administration.
    Panel: 1 = 4mg QW, 2 = 12.5mg QW, 3 = 35mg QW.
    Results for the Panel 3 50mg QW dose level are represented in Outcome Measure 6. Participants in Panel 1 received 4 mg RO7239361; participants in Panel 2 received either 12.5 mg (weight between 15 and 45 kg) or 20 mg (weight >45 kg) RO7239361, and participants in Panel 3 and the Expansion Panel received either 35 mg (weight between 15 and 45 kg) or 50 mg (weight >45 kg) RO7239361.
  • Time of Maximum Observed Serum Concentrations (Tmax) of RO7239361 at Steady State for 50 mg QW Dose. [ Time Frame: Baseline to Week 24 ]
    PK parameter estimates at steady state following approximately 12 weeks QW administration.
    Panel 3 = 50 mg QW
    Participants in Panel 1 received 4 mg RO7239361; participants in Panel 2 received either 12.5 mg (weight between 15 and 45 kg) or 20 mg (weight >45 kg) RO7239361, and participants in Panel 3 and the Expansion Panel received either 35 mg (weight between 15 and 45 kg) or 50 mg (weight >45 kg) RO7239361.
  • Area Under the Concentration-Time Curve From Time Zero to Time of Next Dosing (AUCtau) of RO7239361 at Steady State for 4 mg QW, 12.5 mg QW and 35 mg QW Doses. [ Time Frame: Baseline to Week 24 ]
    PK parameter estimates at steady state. Steady state was achieved following approximately 12 weeks QW administration.
    Panel: 1 = 4mg QW, 2 = 12.5mg QW, 3 = 35mg QW
    Results for the Panel 3 50mg QW dose level are represented in Outcome Measure 8. Participants in Panel 1 received 4 mg RO7239361; participants in Panel 2 received either 12.5 mg (weight between 15 and 45 kg) or 20 mg (weight >45 kg) RO7239361, and participants in Panel 3 and the Expansion Panel received either 35 mg (weight between 15 and 45 kg) or 50 mg (weight >45 kg) RO7239361.
  • Area Under the Concentration-Time Curve From Time Zero to Time of Next Dosing (AUCtau) of RO7239361 at Steady State for 50 mg QW Dose. [ Time Frame: Baseline to Week 24 ]
    PK parameter estimates at steady state following approximately 12 weeks QW administration.
    Panel 3 = 50 mg QW
    Participants in Panel 1 received 4 mg RO7239361; participants in Panel 2 received either 12.5 mg (weight between 15 and 45 kg) or 20 mg (weight >45 kg) RO7239361, and participants in Panel 3 and the Expansion Panel received either 35 mg (weight between 15 and 45 kg) or 50 mg (weight >45 kg) RO7239361.
  • RO7239361 Trough Concentrations [ Time Frame: Baseline to Week 24 ]
    Trough concentrations of RO7239361 at different dose levels.
    Panel 1 = 4mg, Panel 2 = 12.5mg and 20mg, Panel 3 = 35mg, Expansion Panels = 35mg and 50mg.
    Participants in Panel 1 received 4 mg RO7239361; participants in Panel 2 received either 12.5 mg (weight between 15 and 45 kg) or 20 mg (weight >45 kg) RO7239361, and participants in Panel 3 and the Expansion Panel received either 35 mg (weight between 15 and 45 kg) or 50 mg (weight >45 kg) RO7239361.
  • Frequency of Subjects With Positive Anti-RO7239361 Antibodies (ADA) Assessment, Double-Blind Phase [ Time Frame: Day 8 through Week 24, baseline and on-study information represented in table. ]
    A positive ADA sample is defined as one in which the presence of detectable ADAs is confirmed to be specific to RO7239361. A participant is considered as ADA positive if, after initiation of treatment, they have an ADA positive relative to baseline sample.
    Participants in Panel 1 received 4 mg RO7239361; participants in Panel 2 received either 12.5 mg (weight between 15 and 45 kg) or 20 mg (weight >45 kg) RO7239361, and participants in Panel 3 and the Expansion Panel received either 35 mg (weight between 15 and 45 kg) or 50 mg (weight >45 kg) RO7239361.
  • Frequency of Subjects With Positive Anti-RO7239361 Antibodies (ADA) Assessment, Whole Study [ Time Frame: Day 8 through Week 72, baseline and on-study information represented in table. ]
    A positive ADA sample is defined as one in which the presence of detectable ADAs is confirmed to be specific to RO7239361. A participant is considered as ADA positive if, after initiation of treatment, they have an ADA positive relative to baseline sample.
    Double-blind phase data for placebo participants is not included. Placebo participants in each arm moved on to RO7239361 upon entering the open label phase.
    Participants in Panel 1 received 4 mg RO7239361; participants in Panel 2 received either 12.5 mg (weight between 15 and 45 kg) or 20 mg (weight >45 kg) RO7239361, and participants in Panel 3 and the Expansion Panel received either 35 mg (weight between 15 and 45 kg) or 50 mg (weight >45 kg) RO7239361.
  • Serum Concentration of Free Myostatin in the Double-blind Phase [ Time Frame: Baseline through week 24 ]
    Participants in Panel 1 received 4 mg RO7239361; participants in Panel 2 received either 12.5 mg (weight between 15 and 45 kg) or 20 mg (weight >45 kg) RO7239361, and participants in Panel 3 and the Expansion Panel received either 35 mg (weight between 15 and 45 kg) or 50 mg (weight >45 kg) RO7239361.
  • Percent Inhibition of Free Myostatin [ Time Frame: Baseline through week 24 ]
    Participants in Panel 1 received 4 mg RO7239361; participants in Panel 2 received either 12.5 mg (weight between 15 and 45 kg) or 20 mg (weight >45 kg) RO7239361, and participants in Panel 3 and the Expansion Panel received either 35 mg (weight between 15 and 45 kg) or 50 mg (weight >45 kg) RO7239361.
  • Serum Concentration of Drug-myostatin Complex [ Time Frame: Baseline through week 24 ]
    Participants in Panel 1 received 4 mg RO7239361; participants in Panel 2 received either 12.5 mg (weight between 15 and 45 kg) or 20 mg (weight >45 kg) RO7239361, and participants in Panel 3 and the Expansion Panel received either 35 mg (weight between 15 and 45 kg) or 50 mg (weight >45 kg) RO7239361.
  • Fold Change From Baseline in Contractile Versus Non-contractile Content for Muscles in the Right Thigh [ Time Frame: Baseline through Week 24 ]
    Ratio of Contractile vs Non-contractile Content is Contractile Content / Non-contractile Content. Fold change from Baseline of the ratio is defined as the ratio of Fold change from baseline of Contractile content vs Fold change from baseline of Non-contractile content.
    Right thigh measurements. W12 = Week 12, W24 = Week 24.
    Participants in Panel 1 received 4 mg RO7239361; participants in Panel 2 received either 12.5 mg (weight between 15 and 45 kg) or 20 mg (weight >45 kg) RO7239361, and participants in Panel 3 and the Expansion Panel received either 35 mg (weight between 15 and 45 kg) or 50 mg (weight >45 kg) RO7239361.
  • Change From Baseline in Thigh Muscle Maximal Cross Sectional Area (CSAmax) [ Time Frame: Baseline through Week 24 ]
    Right thigh measurements. W12 = Week 12, W24 = Week 24.
    Participants in Panel 1 received 4 mg RO7239361; participants in Panel 2 received either 12.5 mg (weight between 15 and 45 kg) or 20 mg (weight >45 kg) RO7239361, and participants in Panel 3 and the Expansion Panel received either 35 mg (weight between 15 and 45 kg) or 50 mg (weight >45 kg) RO7239361.

inclusion criteria:
• Diagnosed with DMD
• Able to walk without assistance
• Able to walk up 4 stairs in 8 seconds or less
• Weigh at least 15 kg
• Taking corticosteroids for DMD

exclusion criteria:
• Ejection fraction < 55% on echocardiogram, based on central read
• Any behavior or mental issue that will affect the ability to complete the required study procedures
• Previously or currently taking medications like androgens or human growth hormone
• Use of a ventilator during the day
• Unable to have blood samples collected or receive an injection under the skin

study contacts

sponsor: Hoffmann-La Roche

investigators: Clinical Trials, Hoffmann-La Roche

locations: United States, Canada

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