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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05524883




Registration number
NCT05524883
Ethics application status
Date submitted
30/08/2022
Date registered
1/09/2022

Titles & IDs
Public title
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
Scientific title
A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-251 Administered to Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
Secondary ID [1] 0 0
2023-510351-31-00
Secondary ID [2] 0 0
DYNE251-DMD-201
Universal Trial Number (UTN)
Trial acronym
DELIVER
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Duchenne Muscular Dystrophy (DMD) 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - DYNE-251
Treatment: Drugs - Placebo

Experimental: Placebo-Controlled MAD Period - DYNE-251 - DYNE-251 will be administered once every 4 weeks (Q4W) or once every 8 weeks (Q8W) over 24 weeks.

Experimental: Placebo-Controlled MAD Period - Placebo - Placebo will be administered Q4W or Q8W over 24 weeks.

Experimental: Open-Label and Long-Term Extension Period - DYNE-251 - DYNE-251 will be administered Q4W or Q8W for up to 192 weeks after participants complete the Placebo-Controlled MAD Period of the study.


Treatment: Drugs: DYNE-251
Administered by IV infusion

Treatment: Drugs: Placebo
Administered by IV infusion
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Timepoint [1] 0 0
Through study completion, up to Week 241
Primary outcome [2] 0 0
Change From Baseline in Dystrophin Protein Levels in Muscle Tissue at Week 25
Timepoint [2] 0 0
Baseline, Week 25
Secondary outcome [1] 0 0
Change From Baseline in Muscle Tissue Exon 51 Skipping Levels at Week 25 For Participants Dosed at Q4W or Q8W Interval With a Second Biopsy Performed at Week 25
Timepoint [1] 0 0
Baseline, Week 25
Secondary outcome [2] 0 0
Change From Baseline in Muscle Tissue Percent Dystrophin-Positive Fiber (PDPF) at Week 25 For Participants Dosed at Q4W or Q8W Interval With a Second Biopsy Performed at Week 25
Timepoint [2] 0 0
Baseline, Week 25
Secondary outcome [3] 0 0
Change From Baseline in Blood Creatine Kinase (CK) Levels up to Week 241 For Participants Dosed at Q4W or Q8W Interval With a Second Biopsy Performed at Week 25
Timepoint [3] 0 0
Baseline, up to Week 241
Secondary outcome [4] 0 0
Change From Baseline in Dystrophin Protein Level in Muscle Tissue as Determined by Western Blot at Week 49 For Participants Dosed at Q8W Interval With a Second Biopsy Performed at Week 49
Timepoint [4] 0 0
Baseline, Week 49
Secondary outcome [5] 0 0
Change From Baseline in Muscle Tissue Exon 51 Skipping Levels at Week 49 For Participants Dosed at Q8W Interval With a Second Biopsy Performed at Week 49
Timepoint [5] 0 0
Baseline, Week 49
Secondary outcome [6] 0 0
Change From Baseline in Muscle Tissue PDPF at Week 49 For Participants Dosed at Q8W Interval With a Second Biopsy Performed at Week 49
Timepoint [6] 0 0
Baseline, Week 49
Secondary outcome [7] 0 0
Change From Baseline in Blood CK Levels up to Week 241 For Participants Dosed at Q8W Interval With a Second Biopsy Performed at Week 49
Timepoint [7] 0 0
Baseline, up to Week 241
Secondary outcome [8] 0 0
Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score in Ambulatory Participants up to Week 241
Timepoint [8] 0 0
Baseline, up to Week 241
Secondary outcome [9] 0 0
Change From Baseline in Time to Rise From Floor in Ambulatory Participants up to Week 241
Timepoint [9] 0 0
Baseline, up to Week 241
Secondary outcome [10] 0 0
Change From Baseline in 10-Meter Run/Walk (10MRW) Time in Ambulatory Participants up to Week 241
Timepoint [10] 0 0
Baseline, up to Week 241
Secondary outcome [11] 0 0
Change From Baseline in Performance Upper Limb (PUL) Scale Version 2.0 Score up to Week 241
Timepoint [11] 0 0
Baseline, up to Week 241
Secondary outcome [12] 0 0
Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) up to Week 241
Timepoint [12] 0 0
Baseline, up to Week 241
Secondary outcome [13] 0 0
Change From Baseline in Stride Velocity 95th Centile (SV95C) in Ambulatory Participants up to Week 241
Timepoint [13] 0 0
Baseline, up to Week 241
Secondary outcome [14] 0 0
Maximum Observed Plasma Drug Concentration of DYNE-251 (Cmax)
Timepoint [14] 0 0
Through study completion, up to Week 241
Secondary outcome [15] 0 0
Time to Maximum Observed Plasma Drug Concentration of DYNE-251 (tmax)
Timepoint [15] 0 0
Through study completion, up to Week 241
Secondary outcome [16] 0 0
Area Under the Plasma Drug Concentration-Time Curve From Time 0 to the Last Quantifiable Concentration of DYNE-251 in Plasma (AUC0-tlast)
Timepoint [16] 0 0
Through study completion, up to Week 241
Secondary outcome [17] 0 0
Area Under the Plasma Drug Concentration Versus Time Curve From Time 0 (Dosing) Extrapolated to Time Infinity of DYNE-251 (AUC8)
Timepoint [17] 0 0
Through study completion, up to Week 241
Secondary outcome [18] 0 0
Apparent Terminal Phase Elimination Rate Constant of DYNE-251 in Plasma (?z)
Timepoint [18] 0 0
Through study completion, up to Week 241
Secondary outcome [19] 0 0
Apparent Terminal Elimination Half-Life of DYNE-251 in Plasma (t½)
Timepoint [19] 0 0
Through study completion, up to Week 241
Secondary outcome [20] 0 0
Total Body Clearance (CL) of DYNE-251
Timepoint [20] 0 0
Through study completion, up to Week 241
Secondary outcome [21] 0 0
Volume of Distribution at the Terminal Phase of DYNE-251 in Plasma (Vz)
Timepoint [21] 0 0
Through study completion, up to Week 241
Secondary outcome [22] 0 0
Volume of Distribution at Steady State of DYNE-251 in Plasma (Vss)
Timepoint [22] 0 0
Through study completion, up to Week 241
Secondary outcome [23] 0 0
Tissue Phosphorodiamidate Morpholino Oligomer (PMO) Concentration of DYNE-251 in Muscle Tissue
Timepoint [23] 0 0
Through study completion, up to Week 241
Secondary outcome [24] 0 0
Percentage of Participants With Antidrug Antibodies (ADAs)
Timepoint [24] 0 0
Through study completion, up to Week 241

Eligibility
Key inclusion criteria
* Age 4 to 16 years inclusive, at the time of informed consent/assent.
* Male with a confirmed diagnosis of DMD and with a mutation in the dystrophin gene characterized by exon deletion amenable to exon 51 skipping.
* Upper extremity muscle group that is amenable to muscle biopsy.
* Brooke Upper Extremity Scale score of 1 or 2.
* Ambulatory or non-ambulatory. A non-ambulatory participant must have been non-ambulatory for <2 years before enrollment.
* Receiving a stable dosage of glucocorticoids for at least 12 weeks prior to the start of study drug administration, with the expectation of maintaining a stable dose during the Placebo-Controlled and Open-Label Periods of the study (unless dose adjustment is required by weight change).
* Left ventricular ejection fraction of =50% by echocardiogram or =55% by cardiac magnetic resonance imaging (MRI).
Minimum age
4 Years
Maximum age
16 Years
Sex
Males
Can healthy volunteers participate?
No
Key exclusion criteria
* Uncontrolled clinical symptoms and signs of congestive heart failure (CHF).
* Any change in prophylaxis/treatment for CHF within 3 months prior to the start of study treatment.
* History of major surgical procedure within 12 weeks prior to the start of study drug administration or an expectation of a major surgical procedure during the study.
* Requirement of daytime ventilator assistance.
* Percent predicted FVC <40 % (applies only for participants who are age =7 years).
* Receipt of eteplirsen, or alternative exon-skipping/dystrophin-modifying therapy, within 12 weeks of randomization.
* Receipt of non-exon skipping investigational drug within 4 months before the start of study drug administration.
* Receipt of gene therapy at any time.

Other inclusion and exclusion criteria may apply.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Other
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
12/08/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/11/2029
Actual
Sample size
Target
88
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Children's Hospital at Westmead - Westmead
Recruitment hospital [2] 0 0
Murdoch Children's Research Institute - Parkville
Recruitment postcode(s) [1] 0 0
02145 - Westmead
Recruitment postcode(s) [2] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
Georgia
Country [4] 0 0
United States of America
State/province [4] 0 0
Massachusetts
Country [5] 0 0
United States of America
State/province [5] 0 0
Ohio
Country [6] 0 0
United States of America
State/province [6] 0 0
Oregon
Country [7] 0 0
United States of America
State/province [7] 0 0
Pennsylvania
Country [8] 0 0
United States of America
State/province [8] 0 0
Tennessee
Country [9] 0 0
United States of America
State/province [9] 0 0
Utah
Country [10] 0 0
United States of America
State/province [10] 0 0
Virginia
Country [11] 0 0
Belgium
State/province [11] 0 0
Gent
Country [12] 0 0
Belgium
State/province [12] 0 0
Leuven
Country [13] 0 0
Belgium
State/province [13] 0 0
Liège
Country [14] 0 0
Canada
State/province [14] 0 0
British Columbia
Country [15] 0 0
Canada
State/province [15] 0 0
Ontario
Country [16] 0 0
Ireland
State/province [16] 0 0
Dublin
Country [17] 0 0
Italy
State/province [17] 0 0
Lazio
Country [18] 0 0
Italy
State/province [18] 0 0
Liguria
Country [19] 0 0
Italy
State/province [19] 0 0
Lombardia
Country [20] 0 0
Spain
State/province [20] 0 0
Barcelona
Country [21] 0 0
United Kingdom
State/province [21] 0 0
Merseyside
Country [22] 0 0
United Kingdom
State/province [22] 0 0
Northumberland
Country [23] 0 0
United Kingdom
State/province [23] 0 0
West Yorkshire
Country [24] 0 0
United Kingdom
State/province [24] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Dyne Therapeutics
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Dyne Clinical Trials
Address 0 0
Country 0 0
Phone 0 0
+1-781-317-1919
Fax 0 0
Email 0 0
clinicaltrials@dyne-tx.com
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05524883