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- || FDA event, Review, Journal: Comprehensive review of adverse reactions and toxicology in ASO-based therapies for Duchenne Muscular Dystrophy: From FDA-approved drugs to peptide-conjugated ASO. (Pubmed Central) - Jul 10, 2024
This article provides a comprehensive review and discussion of the available data pertaining to adverse reactions and toxicology associated with FDA-approved ASO drugs for DMD. Furthermore, it offers insights into the emerging category of peptide-conjugated ASO drugs those are clinical and preclinical trials, shedding light on their potential benefits and challenges.
- | vesleteplirsen (SRP-5051) / Sarepta Therap, Exondys 51 (eteplirsen) / Sarepta Therap
Trial completion date: MOMENTUM: Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Efficacy (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (clinicaltrials.gov) - Dec 1, 2023
P2, N=62, Active, not recruiting, Sponsor: Sarepta Therapeutics, Inc.
Furthermore, it offers insights into the emerging category of peptide-conjugated ASO drugs those are clinical and preclinical trials, shedding light on their potential benefits and challenges. Trial completion date: Mar 2025 --> Jan 2029
- || vesleteplirsen (SRP-5051) / Sarepta Therap, Exondys 51 (eteplirsen) / Sarepta Therap
Enrollment closed, Trial completion date: MOMENTUM: Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Efficacy (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (clinicaltrials.gov) - May 1, 2023
P2, N=62, Active, not recruiting, Sponsor: Sarepta Therapeutics, Inc.
Trial completion date: Mar 2025 --> Jan 2029 Recruiting --> Active, not recruiting | Trial completion date: Aug 2024 --> Mar 2025
- || Review, Journal: Recent Trends in Antisense Therapies for Duchenne Muscular Dystrophy. (Pubmed Central) - Mar 30, 2023
These upcoming therapies often utilize novel drug chemistries to address limitations of existing therapies, and their development could herald the next generation of antisense therapy. This review article aims to summarize the current state of development for antisense-based therapies for the treatment of Duchenne muscular dystrophy, exploring candidates designed for both exon skipping and gene knockdown.
- || SRP-5051 / Sarepta Therap, Exondys 51 (eteplirsen) / Sarepta Therap
e.g. the SRP-5051 trials (vesleteplirsen) and the AAV delivery of U7 antisense genes - here they mention only the early work done in mdx mice and not the work ongoing by @KevinMFlanigan in patients with exon 2 duplications... (Twitter) - Aug 10, 2022
- || SRP-5051 / Sarepta Therap, Exondys 51 (eteplirsen) / Sarepta Therap
Review, Journal: Pharmacology and toxicology of eteplirsen and SRP-5051 for DMD exon 51 skipping: an update. (Pubmed Central) - Apr 5, 2022
Issues faced by eteplirsen and SRP-5051, including efficacy and safety, are identified. Lastly, the current state of eteplirsen and exon-skipping therapy in general as a strategy for the treatment of DMD are discussed.
- ||| vesleteplirsen (SRP-5051) / Sarepta Therap, Exondys 51 (eteplirsen) / Sarepta Therap
Enrollment open: MOMENTUM: Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Efficacy (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (clinicaltrials.gov) - Jan 5, 2022
P2, N=60, Recruiting, Sponsor: Sarepta Therapeutics, Inc.
Lastly, the current state of eteplirsen and exon-skipping therapy in general as a strategy for the treatment of DMD are discussed. Active, not recruiting --> Recruiting
- || vesleteplirsen (SRP-5051) / Sarepta Therap, Exondys 51 (eteplirsen) / Sarepta Therap
Trial completion date, Trial primary completion date: MOMENTUM: Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Efficacy (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (clinicaltrials.gov) - Nov 24, 2021
P2, N=60, Active, not recruiting, Sponsor: Sarepta Therapeutics, Inc.
Active, not recruiting --> Recruiting Trial completion date: May 2022 --> Aug 2024 | Trial primary completion date: May 2022 --> Sep 2023
- | vesleteplirsen (SRP-5051) / Sarepta Therap, Exondys 51 (eteplirsen) / Sarepta Therap
Enrollment change, Trial completion date, Trial termination, Trial primary completion date: An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (Vesleteplirsen) (clinicaltrials.gov) - Nov 24, 2021
P1/2, N=15, Terminated, Sponsor: Sarepta Therapeutics, Inc.
N=60 --> 15 | Trial completion date: Jul 2024 --> Aug 2021 | Active, not recruiting --> Terminated | Trial primary completion date: Jul 2024 --> Aug 2021; The sponsor has decided to integrate 5051-102 into 5051-201. Participants from 5051-102 will be eligible to enroll in 5051-201 Part B.
- || vesleteplirsen (SRP-5051) / Sarepta Therap, Exondys 51 (eteplirsen) / Sarepta Therap
Enrollment closed: An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (Vesleteplirsen) (clinicaltrials.gov) - May 6, 2021
P1/2, N=60, Active, not recruiting, Sponsor: Sarepta Therapeutics, Inc.
Participants from 5051-102 will be eligible to enroll in 5051-201 Part B. Enrolling by invitation --> Active, not recruiting
- || vesleteplirsen (SRP-5051) / Sarepta Therap, Exondys 51 (eteplirsen) / Sarepta Therap
Enrollment closed: MOMENTUM: Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Efficacy (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (clinicaltrials.gov) - May 4, 2021
P2, N=70, Active, not recruiting, Sponsor: Sarepta Therapeutics, Inc.
Enrolling by invitation --> Active, not recruiting Recruiting --> Active, not recruiting
- SRP-5051 / Sarepta Therap
[VIRTUAL] Biological Efficacy of the Peptide-Conjugated Phosphorodiamidate Morpholino Oligomer SRP-5051 in Preclinical Models of Duchenne Muscular Dystrophy () - Mar 4, 2021 - Abstract #MDA2021MDA_100;
SRP-5051 showed dose-dependent efficacy in preclinical models. These data justify the monthly SRP-5051 dosing regimen used in ongoing clinical studies, and support further clinical investigation of this PPMO.
- ||| vesleteplirsen (SRP-5051) / Sarepta Therap, Exondys 51 (eteplirsen) / Sarepta Therap
Enrollment change, Trial completion date, Trial primary completion date: MOMENTUM: Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Efficacy (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (clinicaltrials.gov) - Jan 7, 2021
P2, N=70, Recruiting, Sponsor: Sarepta Therapeutics, Inc.
These data justify the monthly SRP-5051 dosing regimen used in ongoing clinical studies, and support further clinical investigation of this PPMO. N=45 --> 70 | Trial completion date: Aug 2021 --> May 2022 | Trial primary completion date: Aug 2021 --> May 2022
- || vesleteplirsen (SRP-5051) / Sarepta Therap, Exondys 51 (eteplirsen) / Sarepta Therap
Enrollment status: An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (Vesleteplirsen) (clinicaltrials.gov) - Sep 2, 2020
P1/2, N=60, Enrolling by invitation, Sponsor: Sarepta Therapeutics, Inc.
N=45 --> 70 | Trial completion date: Aug 2021 --> May 2022 | Trial primary completion date: Aug 2021 --> May 2022 Recruiting --> Enrolling by invitation
- ||| vesleteplirsen (SRP-5051) / Sarepta Therap, Exondys 51 (eteplirsen) / Sarepta Therap
Enrollment change, Trial completion date, Trial primary completion date: MOMENTUM: Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Efficacy (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (clinicaltrials.gov) - Jan 18, 2020
P2, N=45, Recruiting, Sponsor: Sarepta Therapeutics, Inc.
Recruiting --> Enrolling by invitation N=24 --> 45 | Trial completion date: Mar 2021 --> Aug 2021 | Trial primary completion date: Mar 2021 --> Aug 2021
- | vesleteplirsen (SRP-5051) / Sarepta Therap, Exondys 51 (eteplirsen) / Sarepta Therap
Trial completion, Enrollment change: A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 (Vesleteplirsen) in Patients With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) - Aug 28, 2019
P1, N=15, Completed, Sponsor: Sarepta Therapeutics, Inc.
N=24 --> 45 | Trial completion date: Mar 2021 --> Aug 2021 | Trial primary completion date: Mar 2021 --> Aug 2021 Recruiting --> Completed | N=30 --> 15
- | vesleteplirsen (SRP-5051) / Sarepta Therap, Exondys 51 (eteplirsen) / Sarepta Therap
Enrollment change, Trial completion date, Trial primary completion date: An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (Vesleteplirsen) (clinicaltrials.gov) - Jul 31, 2019
P1/2, N=54, Recruiting, Sponsor: Sarepta Therapeutics, Inc.
Recruiting --> Completed | N=30 --> 15 N=30 --> 54 | Trial completion date: Nov 2020 --> Jul 2024 | Trial primary completion date: Nov 2020 --> Jul 2024
- || vesleteplirsen (SRP-5051) / Sarepta Therap, Exondys 51 (eteplirsen) / Sarepta Therap
New P2 trial: MOMENTUM: Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Efficacy (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (clinicaltrials.gov) - Jun 30, 2019
P2, N=24, Recruiting, Sponsor: Sarepta Therapeutics
- || vesleteplirsen (SRP-5051) / Sarepta Therap, Exondys 51 (eteplirsen) / Sarepta Therap
Enrollment open: An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (Vesleteplirsen) (clinicaltrials.gov) - Jan 17, 2019
P1/2, N=30, Recruiting, Sponsor: Sarepta Therapeutics
N=30 --> 54 | Trial completion date: Nov 2020 --> Jul 2024 | Trial primary completion date: Nov 2020 --> Jul 2024 Not yet recruiting --> Recruiting
- | vesleteplirsen (SRP-5051) / Sarepta Therap, Exondys 51 (eteplirsen) / Sarepta Therap
Trial primary completion date: An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (Vesleteplirsen) (clinicaltrials.gov) - Nov 26, 2018
P1/2, N=30, Not yet recruiting, Sponsor: Sarepta Therapeutics
Not yet recruiting --> Recruiting Trial primary completion date: Apr 2020 --> Nov 2020
- | vesleteplirsen (SRP-5051) / Sarepta Therap, Exondys 51 (eteplirsen) / Sarepta Therap
Trial completion date, Trial primary completion date: A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 (Vesleteplirsen) in Patients With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) - Oct 16, 2018
P1, N=30, Recruiting, Sponsor: Sarepta Therapeutics
Trial primary completion date: Apr 2020 --> Nov 2020 Trial completion date: Jan 2019 --> Sep 2019 | Trial primary completion date: Jan 2019 --> Sep 2019
- | vesleteplirsen (SRP-5051) / Sarepta Therap, Exondys 51 (eteplirsen) / Sarepta Therap
New P1/2 trial: An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (Vesleteplirsen) (clinicaltrials.gov) - Sep 18, 2018
P1/2, N=30, Not yet recruiting, Sponsor: Sarepta Therapeutics
- | vesleteplirsen (SRP-5051) / Sarepta Therap, Exondys 51 (eteplirsen) / Sarepta Therap
New P1 trial: A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 (Vesleteplirsen) in Patients With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) - Dec 18, 2017
P1, N=30, Recruiting, Sponsor: Sarepta Therapeutics