Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy

Muscle And Peripheral Nerve Disease
Duchenne Muscular Dystrophy (DMD)

For Medical Professional
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Basic Details

Sponsor Hoffmann-La Roche

Phase Phase 2/Phase 3

Study Identifier NCT03039686, CN001-016, 2016-001654-18,WN40227

Condition Duchenne Muscular Dystrophy, Duchenne Muscular Dystrophy

Official Title A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy

Study Summary

This is a multi-center, randomized, double-blind, placebo-controlled study to assess the efficacy, safety and tolerability of two different weekly doses of RO7239361 in ambulatory boys with Duchenne Muscular Dystrophy (DMD).

Eligibility Criteria

Gender

Male

Age

≥6 Years & ≤ 11 Years

Healthy Volunteers

Inclusion Criteria

Diagnosed with DMD by confirmed medical history and genetic testing
Able to walk without assistance
Minimum North Star Ambulatory Assessment score of 15 at screening
Able to walk up 4 stairs in 8 seconds or less
Weigh at least 15 kg (33 lbs)
Taking corticosteroids for DMD

Exclusion Criteria

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
Concomitant or previous participation at any time in a gene therapy study

Other protocol defined Inclusion/Exclusion Criteria could apply.

The source of the below information is public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc.. It has been summarised and edited into simpler language. For more information about this clinical study see the For Expert tab on the specific ForPatients page or follow these links to https://clinicaltrials.gov and/or https://euclinicaltrials.eu and/or https://www.isrctn.com.

The information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc., and has not been edited.

Results Disclaimer

What is Clinical Research?

In clinical research, volunteers, researchers, and medical professionals work together toward a shared goal: better treatment outcomes for patients. Clinical trials are vital to their process. They are carefully designed and follow approved protocols.

Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy

Basic Details

Study Summary

Eligibility Criteria

What is Clinical Research?

Study results

SPITFIRE_final-results-summary_Nov2020_English (PDF, 0.7 MB)

What is Clinical Research?

For the latest version of this information please go to www.forpatients.roche.com

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Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy

For the latest version of this information please go to www.forpatients.roche.com