Duchenne Muscular DystrophyPhase 1NCT02530905

Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients

A Phase 1 study testing SRP-4045 in people with Duchenne Muscular Dystrophy, run by Sarepta Therapeutics, Inc. at 3 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

This is a first-in-human dose-titration and open-label extension study to assess safety, tolerability, and pharmacokinetics of SRP-4045 in advanced-stage Duchenne muscular dystrophy (DMD) patients with deletions amenable to exon 45 skipping. Read more about this study

Who is it for?: Ages 7 to 21 years, men only
Conditions the study is for: Duchenne Muscular Dystrophy
What is being tested?: SRP-4045
Type of treatment: Injection or infusion
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 12 participants
Where: 3 sitesUnited States
Study dates: 8 Oct 2015to 3 Oct 2018
Phase: Phase 1
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 7 to 21 years
Men only
No healthy volunteers
Child, Adult

Who may be able to join

Genotypically confirmed DMD (amenable to exon 45 skipping).
Stable cardiac and pulmonary function.
Limited or no ambulation.
On a stable dose of oral corticosteroids for at least 24 weeks OR has not received corticosteroids for at least 24 weeks.

Who cannot join

Current or previous treatment with the experimental agents SMT C1100 (BMN-195) or PRO045.
Other experimental treatment in the past 12 weeks.
If on cardiac medication, must be on a stable dose for the past 12 weeks.
Major surgery within the past 3 months.
Other inclusion/exclusion criteria apply.

Trial locations and contacts

3 sites in 1 country.

United States3 sites

Kennedy Krieger Institute
Baltimore, Maryland, 21205
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
David Geffen School of Medicine at UCLA
Los Angeles, California, 90095

Other trials in United States

About this study

Official title: A Randomized, Double-Blind, Placebo-Controlled, Dose-Titration, Safety, Tolerability, and Pharmacokinetics Study Followed by an Open-Label Safety and Efficacy Evaluation of SRP-4045 in Advanced-Stage Patients With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping

Read the detailed description

This is a randomized, placebo-controlled dose-titration study to assess safety, tolerability, and pharmacokinetics of 4 dose levels of SRP-4045 in genotypically confirmed advanced-stage DMD patients with deletions amenable to exon 45 skipping.

After completion of the dose-titration portion of the study and SRP-4045 is determined to be safe, all patients will be evaluated on open-label SRP-4045 for the duration of the study.

Safety, including adverse event monitoring, routine laboratory assessments, and cardiac testing will be monitored through the duration of the dose-titration and open-label portions of the study.

Clinical efficacy will be assessed at regularly scheduled study visits via quality of life questionnaires and tests of pulmonary and upper extremity function through the duration of the dose-titration and open-label portions of the trial.

Conditions: Duchenne Muscular Dystrophy
Being tested: SRP-4045, Placebo
Keywords: Duchenne muscular dystrophy, Exon Skipping
Medical subject headings: Muscular Dystrophy, Duchenne

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

SRP-4045Drug
SRP-4045 solution for IV infusion.
PlaceboDrug
SRP-4045 placebo-matching solution for IV infusion.

Study groups in NCT02530905
Study group	Role	What they receive
Placebo (double-blind dose titration)	Placebo comparator	Participants with genotypically confirmed Duchenne muscular dystrophy (DMD) characterized by deletions amenable to exon 45 skipping will receive placebo-matching to casimersen intravenous (IV) infusions, once weekly over approximately 12 weeks in the double-blind period. Receives: Drug: Placebo
SRP-4045 (double-blind dose titration)	Experimental	Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping will receive weekly IV infusions of casimersen at four escalating dose levels, each for at least 2 weeks: 4 milligrams per kilograms (mg/kg) during Week 1 to Week 2, followed by 10 mg/kg during Week 3 to Week 4, followed by 20 mg/kg during Week 5 to Week 6, followed by 30 mg/kg beginning at Week 7 and continue over approximately Week 12 in the double-blind period. Receives: Drug: SRP-4045
SRP-4045 (open label extension period)	Experimental	All participants who completed double blind period will be enrolled to receive casimersen 30 mg/kg once weekly, for up to Week 144 in the open label extension period. Receives: Drug: SRP-4045

What is being measured

The primary outcome is the main question the study is designed to answer.

Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Adverse event (AE) was any untoward medical occurrence in a clinical trial participant, which does not necessarily have a causal relationship with the investigational drug. AEs also included abnormal physical examination findings (Physical examination were conducted per protocol and any clinically significant abnormal findings were recorded in medical history if pre-existing or addressed as an AE if new or worsening). TEAEs was defined as AEs that started, worsened, or became serious on or after the start of first infusion through 148 weeks. Number of participants with TEAEs were reported.
Measured: Baseline up to Week 148
Number of Participants With Potentially Clinically Significant (PCS) Laboratory Abnormalities Reported as TEAEs
Laboratory parameters included serum chemistry (hepatic chemistry and renal chemistry), hematology, coagulation, and urinalysis. Number of participants with potentially clinically significant abnormal finding were reported as TEAEs. The Investigator determined whether abnormal assessment results were potentially clinically significant or not. Potentially clinical significance was defined as any variation in assessment results that had medical relevance resulting in an alteration in medical care.
Measured: Baseline up to Week 148
Number of Participants With Potentially Clinically Significant Abnormalities in Vital Signs Reported as TEAEs
Vital sign parameters included systolic blood pressure (SBP), diastolic blood pressure (DBP), heart rate (HR), and body temperature. Number of participants with at least one potentially clinically significant abnormal vital signs findings were reported as TEAEs. The Investigator determined whether abnormal assessment results were potentially clinically significant or not. Potential clinical significance was defined as any variation in assessment results that had medical relevance resulting in an alteration in medical care.
Measured: Baseline up to Week 148
Number of Participants With Potentially Clinically Significant Abnormalities in Electrocardiogram (ECG) Reported as TEAEs
Twelve-lead ECGs were performed at a consistent time of day throughout the study. Electrocardiograms were performed only after the participant was in the supine position, resting, and quiet for a minimum of 15 minutes. The ECG was manually reviewed and interpreted by medically qualified personnel. Number of participants with potentially clinically significant abnormalities in ECG reported as TEAEs were presented here. The Investigator determined whether abnormal assessment results were potentially clinically significant or not.
Measured: Baseline up to Week 148
Number of Participants With Potentially Clinically Significant Abnormalities in Echocardiograms (ECHO)
Standard, 2-dimensional ECHOs were performed at a consistent time of day throughout the study.The ECHO was reviewed and interpreted by medically qualified personnel. Number of participants with potentially clinically significant abnormalities in ECHO were reported.
Measured: Baseline up to Week 148

Secondary outcomes (10)

Maximum Plasma Concentration (Cmax) of Casimersen
Maximum Concentration (Cmax) of casimersen in plasma was evaluated.
Measured: Pre-infusion, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16, and 24 hours post-dose at Weeks 1 (for 4 mg/kg ), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm), 7 (for 30 mg/kg arm) in DBP and Week 60 (for 30 mg/kg arm) in OLEP
Time to Reach Maximum Plasma Concentration (Tmax) of Casimersen
Time to reach maximum plasma concentration (Tmax) of casimersen was evaluated.
Measured: Pre-infusion, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16, and 24 hours post-dose at Weeks 1 (for 4 mg/kg ), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm), 7 (for 30 mg/kg arm) in DBP and Week 60 (for 30 mg/kg arm) in OLEP
Area Under Concentration-time Curve From Time of Dosing to the Last Measurable Concentration (AUClast) of Casimersen in Plasma
Area under the concentration-time curve from time zero to the last quantifiable concentration of casimersen in plasma were evaluated.
Measured: Pre-infusion, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16, and 24 hours post-dose at Weeks 1 (for 4 mg/kg ), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm), 7 (for 30 mg/kg arm) in DBP and Week 60 (for 30 mg/kg arm) in OLEP
Area Under Concentration-Time Curve From Time Zero Pre-dose to Twenty-Four Hours Post-dose (AUC0-24) of Casimersen in Plasma
Area under concentration-time curve from time zero pre-dose to twenty-four hours post-dos of casimersen in plasma were evaluated.
Measured: Pre-infusion, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16, and 24 hours post-dose at Weeks 1 (for 4 mg/kg ), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm), 7 (for 30 mg/kg arm) in DBP and Week 60 (for 30 mg/kg arm) in OLEP
Area Under the Concentration-Time Curve From Time Zero Extrapolated to the Infinity (AUCinf) of Casimersen in Plasma
Area under the concentration-time curve from time zero extrapolated to the infinity of casimersen in plasma was evaluated.
Measured: Pre-infusion, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16, and 24 hours post-dose at Weeks 1 (for 4 mg/kg ), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm), 7 (for 30 mg/kg arm) in DBP and Week 60 (for 30 mg/kg arm) in OLEP
Apparent Volume of Distribution at Steady State (Vss) of Casimersen
Volume of distribution was defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of a drug. Apparent volume of distribution at steady state of casimersen was evaluated.
Measured: Pre-infusion, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16, and 24 hours post-dose at Weeks 1 (for 4 mg/kg ), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm), 7 (for 30 mg/kg arm) in DBP and Week 60 (for 30 mg/kg arm) in OLEP
Elimination Half-life (T1/2) of Casimersen
T1/2 is the time measured for the plasma concentration of drug to decrease by one half. T1/2 of casimersen was evaluated.
Measured: Pre-infusion, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16, and 24 hours post-dose at Weeks 1 (for 4 mg/kg ), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm), 7 (for 30 mg/kg arm) in DBP and Week 60 (for 30 mg/kg arm) in OLEP
Total Clearance (CL) of Casimersen
Drug clearance is a quantitative measure of the rate at which a drug substance is removed from the blood. CL of casimersen was evaluated.
Measured: Pre-infusion, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16, and 24 hours post-dose at Weeks 1 (for 4 mg/kg ), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm), 7 (for 30 mg/kg arm) in DBP and Week 60 (for 30 mg/kg arm) in OLEP
Mean Residence Time Extrapolated to Infinity (MRTinf) of Casimersen
MRTinf = AUMCinf/AUCinf - T/2, where T was the infusion duration, and AUMCinf was the area under the first moment curve from time 0 extrapolated to infinite time, calculated using the linear/log trapezoidal method. MRTinf of casimersen was evaluated.
Measured: Pre-infusion, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16, and 24 hours post-dose at Weeks 1 (for 4 mg/kg ), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm), 7 (for 30 mg/kg arm) in DBP and Week 60 (for 30 mg/kg arm) in OLEP
Double-Blind Period: Renal Clearance (CLR) of Casimersen
Renal clearance was calculated using the partial AUC0-24 from the non-compartmental analysis in plasma and AE0-24. AUC0-24 was defined as area under the plasma concentration-time curve, from time 0 to 24 hours after completion of dosing. AE0-24 was defined as total cumulative amount excreted from 0 to 24 hours. Summarized data of all urine collection intervals are reported.
Measured: 0 to 4, 4 to 8, 8 to 12, and 12 to 24 hours post-dose at Weeks 1 (for 4 mg/kg ), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm), 7 and 12 (for 30 mg/kg arm) in double-blind period

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

8 Oct 20153 Oct 2018

First posted21 Aug 2015 (estimated)
Study start8 Oct 2015
Primary completion3 Oct 2018
Study completion3 Oct 2018

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
More trials run by Sarepta Therapeutics, Inc.The sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 1
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 12 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02530905
Sponsor's protocol ID: 4045-101
Status last verified: Apr 2021
First submitted: 10 Aug 2015
Last updated: 17 May 2021
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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A Phase 2 Trial to Investigate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tegacorat in Male Participants 4 to <7 Years of Age With Duchenne Muscular Dystrophy
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Multimodal Study About Absence of Dp140 in Becker Muscular Dystrophy: Brain-muscle Imaging, Cognition and Muscle Function
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A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
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All Duchenne Muscular Dystrophy trials

Study at a glance

From the registry record, last updated by the sponsor on 17 May 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Randomized, Double-Blind, Placebo-Controlled, Dose-Titration, Safety, Tolerability, and Pharmacokinetics Study Followed by an Open-Label Safety and Efficacy Evaluation of SRP-4045 in Advanced-Stage Patients With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping
ClinicalTrials.gov ID: NCT02530905
Acronym / sponsor ID: 4045-101
Conditions: Duchenne Muscular Dystrophy
Keywords: Duchenne muscular dystrophy, Exon Skipping
Phase: Phase 1
Study type: Interventional
Recruitment status: Completed
Enrolment: 12 participants (actual)
Lead sponsor: Sarepta Therapeutics, Inc.Industry (pharma, biotech, devices)
Study start: 8 Oct 2015 (actual)
Primary completion: 3 Oct 2018 (actual)
Study completion: 3 Oct 2018 (actual)
First posted: 21 Aug 2015 (estimated)
Results: Posted 17 May 2021

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

After completion of the dose-titration portion of the study and SRP-4045 is determined to be safe, all patients will be evaluated on open-label SRP-4045 for the duration of the study.

Safety, including adverse event monitoring, routine laboratory assessments, and cardiac testing will be monitored through the duration of the dose-titration and open-label portions of the study.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 3

Arm	Type	Receives
Placebo (double-blind dose titration)	Placebo comparator	Drug: PlaceboParticipants with genotypically confirmed Duchenne muscular dystrophy (DMD) characterized by deletions amenable to exon 45 skipping will receive placebo-matching to casimersen intravenous (IV) infusions, once weekly over approximately 12 weeks in the double-blind period.
SRP-4045 (double-blind dose titration)	Experimental	Drug: SRP-4045Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping will receive weekly IV infusions of casimersen at four escalating dose levels, each for at least 2 weeks: 4 milligrams per kilograms (mg/kg) during Week 1 to Week 2, followed by 10 mg/kg during Week 3 to Week 4, followed by 20 mg/kg during Week 5 to Week 6, followed by 30 mg/kg beginning at Week 7 and continue over approximately Week 12 in the double-blind period.
SRP-4045 (open label extension period)	Experimental	Drug: SRP-4045All participants who completed double blind period will be enrolled to receive casimersen 30 mg/kg once weekly, for up to Week 144 in the open label extension period.

Who may qualify

Ages: 7 to 21 years
Sex: Male only
Healthy volunteers: Not accepted
Age groups: Child, Adult

Key inclusion criteria (4 listed)

Genotypically confirmed DMD (amenable to exon 45 skipping).
Stable cardiac and pulmonary function.
Limited or no ambulation.
On a stable dose of oral corticosteroids for at least 24 weeks OR has not received corticosteroids for at least 24 weeks.

Key exclusion criteria (5 listed)

Current or previous treatment with the experimental agents SMT C1100 (BMN-195) or PRO045.
Other experimental treatment in the past 12 weeks.
If on cardiac medication, must be on a stable dose for the past 12 weeks.
Major surgery within the past 3 months.
Other inclusion/exclusion criteria apply.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
SRP-4045SRP-4045 solution for IV infusion.	Drug	SRP-4045 (double-blind dose titration), SRP-4045 (open label extension period)
PlaceboSRP-4045 placebo-matching solution for IV infusion.	Drug	Placebo (double-blind dose titration)

Primary outcome

Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Time frame: Baseline up to Week 148
Adverse event (AE) was any untoward medical occurrence in a clinical trial participant, which does not necessarily have a causal relationship with the investigational drug. AEs also included abnormal physical examination findings (Physical examination were conducted per protocol and any clinically significant abnormal findings were recorded in medical history if pre-existing or addressed as an AE if new or worsening). TEAEs was defined as AEs that started, worsened, or became serious on or after the start of first infusion through 148 weeks. Number of participants with TEAEs were reported.
Number of Participants With Potentially Clinically Significant (PCS) Laboratory Abnormalities Reported as TEAEs
Time frame: Baseline up to Week 148
Laboratory parameters included serum chemistry (hepatic chemistry and renal chemistry), hematology, coagulation, and urinalysis. Number of participants with potentially clinically significant abnormal finding were reported as TEAEs. The Investigator determined whether abnormal assessment results were potentially clinically significant or not. Potentially clinical significance was defined as any variation in assessment results that had medical relevance resulting in an alteration in medical care.
Number of Participants With Potentially Clinically Significant Abnormalities in Vital Signs Reported as TEAEs
Time frame: Baseline up to Week 148
Vital sign parameters included systolic blood pressure (SBP), diastolic blood pressure (DBP), heart rate (HR), and body temperature. Number of participants with at least one potentially clinically significant abnormal vital signs findings were reported as TEAEs. The Investigator determined whether abnormal assessment results were potentially clinically significant or not. Potential clinical significance was defined as any variation in assessment results that had medical relevance resulting in an alteration in medical care.
Number of Participants With Potentially Clinically Significant Abnormalities in Electrocardiogram (ECG) Reported as TEAEs
Time frame: Baseline up to Week 148
Twelve-lead ECGs were performed at a consistent time of day throughout the study. Electrocardiograms were performed only after the participant was in the supine position, resting, and quiet for a minimum of 15 minutes. The ECG was manually reviewed and interpreted by medically qualified personnel. Number of participants with potentially clinically significant abnormalities in ECG reported as TEAEs were presented here. The Investigator determined whether abnormal assessment results were potentially clinically significant or not.
Number of Participants With Potentially Clinically Significant Abnormalities in Echocardiograms (ECHO)
Time frame: Baseline up to Week 148
Standard, 2-dimensional ECHOs were performed at a consistent time of day throughout the study.The ECHO was reviewed and interpreted by medically qualified personnel. Number of participants with potentially clinically significant abnormalities in ECHO were reported.

10 secondary outcomes

Maximum Plasma Concentration (Cmax) of Casimersen
Time frame: Pre-infusion, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16, and 24 hours post-dose at Weeks 1 (for 4 mg/kg ), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm), 7 (for 30 mg/kg arm) in DBP and Week 60 (for 30 mg/kg arm) in OLEP
Time to Reach Maximum Plasma Concentration (Tmax) of Casimersen
Time frame: Pre-infusion, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16, and 24 hours post-dose at Weeks 1 (for 4 mg/kg ), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm), 7 (for 30 mg/kg arm) in DBP and Week 60 (for 30 mg/kg arm) in OLEP
Area Under Concentration-time Curve From Time of Dosing to the Last Measurable Concentration (AUClast) of Casimersen in Plasma
Time frame: Pre-infusion, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16, and 24 hours post-dose at Weeks 1 (for 4 mg/kg ), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm), 7 (for 30 mg/kg arm) in DBP and Week 60 (for 30 mg/kg arm) in OLEP
Area Under Concentration-Time Curve From Time Zero Pre-dose to Twenty-Four Hours Post-dose (AUC0-24) of Casimersen in Plasma
Time frame: Pre-infusion, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16, and 24 hours post-dose at Weeks 1 (for 4 mg/kg ), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm), 7 (for 30 mg/kg arm) in DBP and Week 60 (for 30 mg/kg arm) in OLEP
Area Under the Concentration-Time Curve From Time Zero Extrapolated to the Infinity (AUCinf) of Casimersen in Plasma
Time frame: Pre-infusion, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16, and 24 hours post-dose at Weeks 1 (for 4 mg/kg ), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm), 7 (for 30 mg/kg arm) in DBP and Week 60 (for 30 mg/kg arm) in OLEP
Apparent Volume of Distribution at Steady State (Vss) of Casimersen
Time frame: Pre-infusion, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16, and 24 hours post-dose at Weeks 1 (for 4 mg/kg ), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm), 7 (for 30 mg/kg arm) in DBP and Week 60 (for 30 mg/kg arm) in OLEP
Elimination Half-life (T1/2) of Casimersen
Time frame: Pre-infusion, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16, and 24 hours post-dose at Weeks 1 (for 4 mg/kg ), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm), 7 (for 30 mg/kg arm) in DBP and Week 60 (for 30 mg/kg arm) in OLEP
Total Clearance (CL) of Casimersen
Time frame: Pre-infusion, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16, and 24 hours post-dose at Weeks 1 (for 4 mg/kg ), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm), 7 (for 30 mg/kg arm) in DBP and Week 60 (for 30 mg/kg arm) in OLEP
Mean Residence Time Extrapolated to Infinity (MRTinf) of Casimersen
Time frame: Pre-infusion, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16, and 24 hours post-dose at Weeks 1 (for 4 mg/kg ), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm), 7 (for 30 mg/kg arm) in DBP and Week 60 (for 30 mg/kg arm) in OLEP
Double-Blind Period: Renal Clearance (CLR) of Casimersen
Time frame: 0 to 4, 4 to 8, 8 to 12, and 12 to 24 hours post-dose at Weeks 1 (for 4 mg/kg ), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm), 7 and 12 (for 30 mg/kg arm) in double-blind period

Results and safety

Results posted to the registry on 17 May 2021, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Double-Blind Period: Placebo	4	4
Double-Blind Period: Casimersen	8	8
Open Label Extension Period: Casimersen	0	0

Primary outcome: Number of Participants With Treatment Emergent Adverse Events (TEAEs)

Time frame: Baseline up to Week 148. Count of participants. Unit: Participants

Measure	Double-Blind Period: Placebo n = 4	Double-Blind Period: Casimersen 4 mg/kg n = 8	Double-Blind Period: Casimersen 10 mg/kg n = 8	Double-Blind Period: Casimersen 20 mg/kg n = 8	Double-Blind Period: Casimersen 30 mg/kg n = 8	Open Label Extension Period: Casimersen 30 mg/kg n = 12
Value	4	5	3	3	7	12

Population analysed: Safety set included all randomized participants who received at least 1 dose of study drug (casimersen or placebo) in both double-blind and open-label extension periods.

Primary outcome: Number of Participants With Potentially Clinically Significant (PCS) Laboratory Abnormalities Reported as TEAEs

Time frame: Baseline up to Week 148. Count of participants. Unit: Participants

Measure	Double-Blind Period: Casimersen 30 mg/kg n = 8	Open Label Extension Period: Casimersen 30 mg/kg n = 12
Serum chemistry: Hepatic chemistry	0	0
Serum chemistry: Renal chemistry	0	0
Hematolgy: Leukocytes	2	4
Hematolgy: Neutrophils	4	6
Coagulation: Platelet count	2	2
Urinalysis	0	1

Population analysed: Safety set included all randomized participants who received at least 1 dose of study drug (casimersen or placebo) in both double-blind and open-label extension periods.

Primary outcome: Number of Participants With Potentially Clinically Significant Abnormalities in Vital Signs Reported as TEAEs

Time frame: Baseline up to Week 148. Count of participants. Unit: Participants

Measure	Double-Blind Period: Placebo n = 4	Double-Blind Period: Casimersen 4 mg/kg n = 8	Double-Blind Period: Casimersen 10 mg/kg n = 8	Double-Blind Period: Casimersen 20 mg/kg n = 8	Double-Blind Period: Casimersen 30 mg/kg n = 8	Open Label Extension Period: Casimersen 30 mg/kg n = 12
Value	0	0	0	0	1	1

Population analysed: Safety set included all randomized participants who received at least 1 dose of study drug (casimersen or placebo) in both double-blind and open-label extension periods.

Primary outcome: Number of Participants With Potentially Clinically Significant Abnormalities in Electrocardiogram (ECG) Reported as TEAEs

Time frame: Baseline up to Week 148. Count of participants. Unit: Participants

Measure	Double-Blind Period: Placebo n = 4	Double-Blind Period: Casimersen 4 mg/kg n = 8	Double-Blind Period: Casimersen 10 mg/kg n = 8	Double-Blind Period: Casimersen 20 mg/kg n = 8	Double-Blind Period: Casimersen 30 mg/kg n = 8	Open Label Extension Period: Casimersen 30 mg/kg n = 12
Value	0	0	0	0	0	1

Population analysed: Safety set included all randomized participants who received at least 1 dose of study drug (casimersen or placebo) in both double-blind and open-label extension periods.

10 secondary outcomes are reported in the full results.

Adverse events

Time frame: From start of study drug administration up to Week 148. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Double-Blind Period: Placebo	0 of 4 (0%)	4 of 4 (100%)	0 of 4
Double-Blind Period: Casimersen 4 mg/kg	0 of 8 (0%)	5 of 8 (62.5%)	0 of 8
Double-Blind Period: Casimersen 10 mg/kg	0 of 8 (0%)	3 of 8 (37.5%)	0 of 8
Double-Blind Period: Casimersen 20 mg/kg	0 of 8 (0%)	3 of 8 (37.5%)	0 of 8
Double-Blind Period: Casimersen 30 mg/kg	1 of 8 (12.5%)	7 of 8 (87.5%)	0 of 8
Open Label Extension Period: Casimersen 30 mg/kg	3 of 12 (25%)	12 of 12 (100%)	0 of 12

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 17 May 2021.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Medical Director study director

Sarepta Therapeutics, Inc.

3 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing SRP-4045 in people with Duchenne Muscular Dystrophy, run by Sarepta Therapeutics, Inc. at 3 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 17 May 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 17 May 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Participants With Treatment Emergent Adverse Events (TEAEs) (time frame: Baseline up to Week 148). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02530905.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 May 2021.