QR-421a News - LARVOL Sigma

||||||||||  QR-421a / ProQR
Trial completion date, Trial termination, Trial primary completion date:  Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss (Celeste) (clinicaltrials.gov) -  Jan 20, 2023
P2/3, N=5, Terminated,  Sponsor: ProQR Therapeutics
Trial completion date: Jun 2025 --> Aug 2022 | Active, not recruiting --> Terminated | Trial primary completion date: Jun 2025 --> Aug 2022; Business decision

||||||||||  QR-421a / ProQR
Trial completion date, Trial termination, Trial primary completion date:  HELIA: An Open-Label Extension Study to Evaluate Safety & Tolerability of QR-421a in Subjects With Retinitis Pigmentosa (clinicaltrials.gov) -  Dec 27, 2022
P2, N=21, Terminated,  Sponsor: ProQR Therapeutics
Trial completion date: Jun 2025 --> Aug 2022 | Active, not recruiting --> Terminated | Trial primary completion date: Jun 2025 --> Aug 2022; Business decision Trial completion date: Jul 2026 --> Oct 2022 | Enrolling by invitation --> Terminated | Trial primary completion date: Jul 2026 --> Oct 2022; The sponsor decided to terminate the study early

|||||||||| QR-421a / ProQR
Journal: Scrutinizing pathogenicity of the USH2A c.2276 G > T; p.(Cys759Phe) variant. (Pubmed Central) - Jun 9, 2022
In conclusion, we confirmed pathogenicity of USH2A c.2276 G > T (p.(Cys759Phe)). Consequently, cases homozygous for c.2276 G > T can now receive a definite genetic diagnosis and can be considered eligible for receiving future QR-421a-mediated exon 13 skipping therapy.

|||||||||| QR-421a / ProQR
Scrutinizing pathogenicity of the USH2A c.2276G>T; p.(Cys759Phe) variant (A0068) - Apr 29, 2022 - Abstract #ARVO2022ARVO_2910;
We confirmed pathogenicity of USH2A c.2276G>T (p.(Cys759Phe)). Consequently, persons homozygous for c.2276G>T can now receive a definite genetic diagnosis and can be considered eligible for QR-421a-mediated exon 13 skipping therapy in the future.

||||||||||  QR-421a / ProQR
Enrollment closed, Enrollment change:  Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss (Celeste) (clinicaltrials.gov) -  Apr 24, 2022
P2/3, N=5, Active, not recruiting,  Sponsor: ProQR Therapeutics
Consequently, persons homozygous for c.2276G>T can now receive a definite genetic diagnosis and can be considered eligible for QR-421a-mediated exon 13 skipping therapy in the future. Recruiting --> Active, not recruiting | N=120 --> 5

||||||||||  QR-421a / ProQR
Trial completion:  Stellar: Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene (clinicaltrials.gov) -  Apr 20, 2022
P1/2, N=20, Completed,  Sponsor: ProQR Therapeutics
Recruiting --> Active, not recruiting | N=120 --> 5 Active, not recruiting --> Completed

|||||||||| QR-421a / ProQR
Journal: Antisense oligonucleotide-based treatment of retinitis pigmentosa caused by USH2A exon 13 mutations. (Pubmed Central) - Feb 5, 2022
Mouse surrogate mQR-421a reached the retinal outer nuclear layer after a single intravitreal injection, and induced a detectable level of exon skipping until at least 6 months post injection. In conclusion, QR-421a-induced exon skipping proves to be a highly promising treatment option for RP caused by mutations in USH2A exon 13.

||||||||||  QR-421a / ProQR
New P2 trial:  HELIA: An Open-Label Extension Study to Evaluate Safety & Tolerability of QR-421a in Subjects With Retinitis Pigmentosa (clinicaltrials.gov) -  Oct 19, 2021
P2, N=20, Enrolling by invitation,  Sponsor: ProQR Therapeutics

||||||||||  QR-421a / ProQR
Enrollment closed, Trial completion date:  Stellar: Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene (clinicaltrials.gov) -  Sep 16, 2021
P1/2, N=20, Active, not recruiting,  Sponsor: ProQR Therapeutics
In conclusion, QR-421a-induced exon skipping proves to be a highly promising treatment option for RP caused by mutations in USH2A exon 13. Recruiting --> Active, not recruiting | Trial completion date: Jun 2022 --> Oct 2021

|||||||||| Clinical, Review, Journal: RNA therapeutics in ophthalmology - translation to clinical trials. (Pubmed Central) - Sep 5, 2021
This review provides a detailed insight into the recent developments and clinical trials that have been conducted for several gene-silencing therapies, including ISTH0036, SYL040012, SYL1001, PF-04523655, Sirna-027, QR-110, QR-1123, QR-421a and IONIS-FB-L in glaucoma, dry eye disease, age-related macular degeneration, diabetic macular oedema and various inherited retinal diseases. Our aim is to explore the potential of these drugs whilst evaluating their associated advantages and disadvantages, and to discuss the future translation of RNA therapeutics in ophthalmology.

||||||||||  QR-421a / ProQR
Trial completion date:  Stellar: Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene (clinicaltrials.gov) -  Aug 8, 2020
P1/2, N=18, Recruiting,  Sponsor: ProQR Therapeutics
Our aim is to explore the potential of these drugs whilst evaluating their associated advantages and disadvantages, and to discuss the future translation of RNA therapeutics in ophthalmology. Trial completion date: Jan 2022 --> Jun 2022

||||||||||  QR-421a / ProQR
Trial completion date, Trial primary completion date:  Stellar: Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene (clinicaltrials.gov) -  Nov 8, 2019
P1/2, N=18, Recruiting,  Sponsor: ProQR Therapeutics
Trial completion date: Jan 2022 --> Jun 2022 Trial completion date: Jul 2020 --> Jan 2022 | Trial primary completion date: Dec 2019 --> Dec 2021

||||||||||  QR-421a / ProQR
Enrollment open:  Stellar: Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene (clinicaltrials.gov) -  Mar 7, 2019
P1/2, N=18, Recruiting,  Sponsor: ProQR Therapeutics
Trial completion date: Jul 2020 --> Jan 2022 | Trial primary completion date: Dec 2019 --> Dec 2021 Not yet recruiting --> Recruiting

||||||||||  QR-421a / ProQR
New P1/2 trial:  Stellar: Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene (clinicaltrials.gov) -  Dec 19, 2018
P1/2, N=18, Not yet recruiting,  Sponsor: ProQR Therapeutics



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