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Exon Skipping Agent

High-Dose Eteplirsen for DMD

Phase 3
Waitlist Available
Research Sponsored by Sarepta Therapeutics, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be a male with an established clinical diagnosis of DMD and an out-of-frame deletion mutation of the DMD gene amenable to exon 51 skipping.
Ambulatory participant, able to perform TTRISE in 10 seconds or less at the time of screening visit.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline, postdose (at week 24, week 48, or week 144)
Awards & highlights

Study Summary

This trial will test if eteplirsen is safe and effective in treating Duchenne Muscular Dystrophy. There will be two parts to the study. In the first part, around 10 people will test two different doses of the drug to see if it is safe and tolerable. In the second part, around 144 people will test different doses of the drug to see if it is effective.

Who is the study for?
This trial is for boys with Duchenne Muscular Dystrophy who can walk independently and perform a timed test quickly. They must have specific genetic mutations treatable by skipping exon 51, stable breathing function, and be on steady corticosteroid doses. Boys using other DMD treatments or with kidney issues, recent major surgery, heart problems, or other serious diseases cannot join.Check my eligibility
What is being tested?
The study tests two high doses of Eteplirsen (100 mg/kg and 200 mg/kg) against a standard dose (30 mg/kg) in boys with Duchenne Muscular Dystrophy. It's divided into two parts: first to see if the high doses are safe and then to find out which one works best compared to the standard dose.See study design
What are the potential side effects?
Eteplirsen may cause side effects like injection site reactions, balance problems, nausea or vomiting. Since it's being tested at higher than usual doses, there might be new side effects that doctors will watch for carefully.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I am a male diagnosed with DMD and have a specific genetic mutation treatable by exon 51 skipping.
Select...
I can walk and complete a specific task in 10 seconds or less.
Select...
I can walk on my own without help from devices.
Select...
Both of my biceps muscles are healthy and intact.
Select...
I am 7 or older with stable lung function, or I am 4-6 years old and don't need a ventilator.
Select...
I am a male with DMD due to a specific genetic mutation treatable by exon 51 skipping.
Select...
I can walk and complete a specific physical task in 10 seconds or less.
Select...
I can walk on my own without needing help or devices.
Select...
My upper arm muscles are healthy and intact.
Select...
I am 7 or older with stable lung function and don't need help breathing at night.
Select...
I am between 4 to 6 years old and do not need a ventilator.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline, postdose (at week 24, week 48, or week 144)
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, postdose (at week 24, week 48, or week 144) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Part 1: Incidence of Adverse Events (AEs)
Part 2: Change From Baseline in the NSAA Total Score at Week 144
Secondary outcome measures
Part 2: Change From Baseline in Skeletal Muscle Dystrophin Expression
Part 2: Change From Baseline in Time to Rise From the Floor, Time to Complete 10-Meter Walk/Run, and the Timed Stair Ascend Test
Part 2: Change From Baseline in the Total Distance Walked During 6-Minute Walk Test (6MWT)
+4 more

Side effects data

From 2021 Phase 2 trial • 15 Patients • NCT03218995
100%
Pyrexia
83%
Nasopharyngitis
83%
Cough
67%
Rhinitis
67%
Vomiting
50%
Ear infection
50%
Rash maculo-papular
50%
Gastroenteritis
50%
Rhinorrhoea
50%
Diarrhoea
33%
Head injury
33%
Teething
33%
Bronchitis
33%
Influenza
33%
Lethargy
33%
Upper respiratory tract infection
33%
Fall
33%
Ear pain
33%
Iron deficiency
17%
Laceration
17%
Abdominal pain
17%
Otitis media acute
17%
Faeces discoloured
17%
Bronchiolitis
17%
Constipation
17%
Chalazion
17%
Rash
17%
Rash generalised
17%
Dermatitis diaper
17%
Dermatitis contact
17%
Procedural pain
17%
Vaccination complication
17%
Eczema
17%
Iron deficiency anaemia
17%
Allergy to chemicals
17%
Dysuria
17%
Conjunctivitis
17%
Autoimmune neutropenia
17%
Hypochromic anaemia
17%
Genital cyst
17%
Body temperature
17%
Body temperature increased
17%
Post-traumatic pain
17%
Tracheal obstruction
17%
Pharyngitis
17%
Eye infection
17%
Gastrointestinal viral infection
17%
Hand-foot-and-mouth disease
17%
Hordeolum
17%
Lower respiratory tract infection
17%
Varicella
17%
Catheter site swelling
17%
Infusion site extravasation
17%
Localised oedema
17%
Cerumen impaction
17%
External ear inflammation
17%
Inner ear inflammation
17%
Tympanic membrane hyperaemia
17%
Hyposideraemia
17%
Initial insomnia
100%
80%
60%
40%
20%
0%
Study treatment Arm
Cohort 2: Age 6 to <24 Months
Cohort 1: Age 24 to 48 Months

Trial Design

4Treatment groups
Experimental Treatment
Active Control
Group I: Part 2: Eteplirsen 200 mg/kgExperimental Treatment1 Intervention
Randomized participants will receive eteplirsen 200 mg/kg once weekly before the selection of the high dose occurs and then will receive the selected high dose once weekly for up to 144 weeks.
Group II: Part 2: Eteplirsen 100 mg/kgExperimental Treatment1 Intervention
Randomized participants will receive eteplirsen 100 mg/kg once weekly before the selection of the high dose occurs and then will receive the selected high dose once weekly for up to 144 weeks.
Group III: Part 1: EteplirsenExperimental Treatment1 Intervention
Participants will receive eteplirsen 100 mg/kg once weekly for at least 4 weeks, followed by eteplirsen 200 mg/kg once weekly for at least 4 weeks.
Group IV: Part 2: Eteplirsen 30 mg/kgActive Control1 Intervention
Randomized participants will receive eteplirsen 30 mg/kg once weekly for up to 144 weeks.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Eteplirsen
2014
Completed Phase 2
~40

Find a Location

Who is running the clinical trial?

Sarepta Therapeutics, Inc.Lead Sponsor
49 Previous Clinical Trials
33,379 Total Patients Enrolled
Medical DirectorStudy DirectorSarepta Therapeutics, Inc.
2,777 Previous Clinical Trials
8,063,320 Total Patients Enrolled

Media Library

Eteplirsen (Exon Skipping Agent) Clinical Trial Eligibility Overview. Trial Name: NCT03992430 — Phase 3
Duchenne Muscular Dystrophy Research Study Groups: Part 1: Eteplirsen, Part 2: Eteplirsen 30 mg/kg, Part 2: Eteplirsen 100 mg/kg, Part 2: Eteplirsen 200 mg/kg
Duchenne Muscular Dystrophy Clinical Trial 2023: Eteplirsen Highlights & Side Effects. Trial Name: NCT03992430 — Phase 3
Eteplirsen (Exon Skipping Agent) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03992430 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there any available positions for volunteers in this research project?

"The latest information from clinicaltrials.gov suggests that this study is still looking for volunteers. The trial was first announced on July 13th, 2020 with the most recent update being on September 30th, 2020."

Answered by AI

Does this experiment only include adults who are over 55 years old?

"Studies like this one often have very specific age requirements for participants. In this case, the trial is only for children aged 4 to 13. Out of the 112 total studies, 62 are for patients under 18 years old and 50 are for those over 65."

Answered by AI

What sets this experiment apart from others like it?

"Eteplirsen is being trialled in 7 cities and 14 countries. The first trial was conducted in 2020 by Sarepta Therapeutics, Inc. That study completed its Phase 3 drug approval stage with the support of 154 patients. In the 2 years since 2020, 8 more studies have been completed."

Answered by AI

Has Eteplirsen undergone FDA drug approval?

"Eteplirsen is classified as safe because it is a Phase 3 trial, which means that there is evidence of its efficacy and that it has undergone multiple rounds of safety testing."

Answered by AI

Do you know of any previous research with Eteplirsen?

"As of now, there is 1 trial concerning Eteplirsen that is still ongoing. This research is in Phase 3. Additionally, there are 21 different locations running clinical trials for Eteplirsen, with some of these locations being in Praha 5 and West Yorkshire."

Answered by AI

How many people have been selected to participate in this clinical trial?

"The trial is currently ongoing, as seen on clinicaltrials.gov. This specific research project was originally posted on July 13th, 2020 and was edited September 30th, 2020. They are looking for 154 patients from 1 site."

Answered by AI

Which patients would be best suited for this kind of treatment?

"This trial is looking for 154 participants aged 4-13 who have been diagnosed with Becker muscular dystrophy. Additional inclusion criteria are as follows: participants must have intact biceps muscles or an alternative upper arm muscle group, stable pulmonary function (for ages 7 and older), no need for ventilation (for ages 4-6), be male with an out-of-frame deletion mutation of the DMD gene, be ambulatory, able to perform TTRISE in 10 seconds or less, able to walk independently, and have been on a stable dose of oral corticosteroids for at least 12 weeks prior to randomization."

Answered by AI

Who else is applying?

What state do they live in?
Georgia
How old are they?
< 18
18 - 65
What site did they apply to?
Rare Disease Research, LLC
What portion of applicants met pre-screening criteria?
Did not meet criteria
Recent research and studies
clinicaltrials.govStudy
A Study to Compare Safety and Efficacy of a High Dose of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)
2020. "A Study to Compare Safety and Efficacy of a High Dose of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03992430.
All > Muscular Dystrophy
~22 spots leftby Nov 2024
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