Extension Study to Study PQ-110-001 (NCT03140969)

NCT03913130 | Terminated Phase 1 Results DMC FDA Drug

• 2 other identifiers: PQ-110-0022018-003500-40
• Study Type: interventional
• Target: 9
• Locations: 2 countries
• Sites: 3

Brief Summary

Subjects completing participation in study PQ-110-001 (EudraCT 2017-000813-22 / NCT03140969) will be given the opportunity to enroll into the extension study for continued dosing if available data support current and/or future benefits for the subject. Study PQ-110-002 will provide long-term safety, tolerability, pharmacokinetic (PK), and efficacy data of QR-110.

Conditions

Leber Congenital Amaurosis 10; Blindness; Leber Congenital Amaurosis; Vision Disorders; Sensation Disorders; Neurologic Manifestations; Eye Diseases; Eye Diseases, Hereditary; Eye Disorders Congenital; Retinal Disease

Keywords

LCA10; CEP290; p.Cys998X; c.2991+1655A>G; Leber's Congenital Amaurosis; Antisense oligonucleotide; RNA therapy

Outcome Measures

Primary Outcomes (2)

• Frequency of Ocular AEs

  Frequency of ocular adverse events (AEs)

  24 months

• Frequency of Non-ocular AEs

  Frequency of non-ocular AEs

  24 months

Secondary Outcomes (12)

• Change in BCVA in First Treated Eye

  24 months

• Change in Mobility Course Score

  24 months

• Change in Photoreceptor Outer Segment Layer Thickness

  24 months

• Change in OCI

  24 months

• Change in FST Blue

  24 months

Study Arms (1)

Drug QR-110

EXPERIMENTAL

First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration

Drug: QR-110

Interventions

QR-110 DRUG

First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration

Drug QR-110

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Subjects who completed participation in the study PQ-110-001 and who may derive benefit from continued treatment with QR 110, as assessed by the Investigator, in consultation with the Medical Monitor
• Persistence of detectable outer nuclear layer (ONL) in the area of the macula in the opinion of the Investigator, as determined by OCT.
• Clear ocular media and adequate pupillary dilation to permit good quality retinal imaging, as assessed by the Investigator.
• An adult (≥ 18 years) willing and able to provide informed consent for participation OR a minor (6 to \< 18 years) with a parent or legal guardian willing and able to provide written permission for the subject's participation prior to performing any study related procedures, and pediatric subjects able to provide age-appropriate assent for study participation.
• Female subjects who have reached menarche and male subjects must either practice true abstinence in accordance with their preferred and usual lifestyle, or agree to use acceptable, highly effective methods of contraception for up to 3 months following their last dose QR-110. Acceptable methods of contraception are defined in the protocol. Women of non-childbearing potential may be included without the use of adequate birth control, provided they meet the criteria in the protocol.

You may not qualify if:

• Any contraindication to IVT injection according to the Investigator's clinical judgment and international guidelines (Avery 2014).
• Safety issue during study PQ-110-001 that may compromise subject safety when continued dosing, as determined by the Investigator, and in consultation with the Medical Monitor.
• Any ocular or systemic disease or condition (including medications and laboratory test abnormalities) that could compromise subject safety or interfere with assessment of efficacy and safety, as determined by the Investigator and in consultation with the Medical Monitor.
• Pregnant or breast-feeding female.

Sponsors & Collaborators

• Laboratoires Thea: lead
• Sepul Bio: collaborator

Study Sites (3)

University of Iowa

Iowa City, Iowa, 52242, United States

Location

Scheie Eye Institute, University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

Ghent University Hospital and Ghent University

Ghent, B-9000, Belgium

Location

MeSH Terms

Conditions

Leber Congenital Amaurosis 10; Blindness; Leber Congenital Amaurosis; Vision Disorders; Sensation Disorders; Neurologic Manifestations; Eye Diseases; Eye Diseases, Hereditary; Eye Abnormalities; Retinal Diseases; Meckel Syndrome, Type 4; Optic Atrophy, Hereditary, Leber

Condition Hierarchy (Ancestors)

Nervous System Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Congenital Abnormalities; Optic Atrophies, Hereditary; Optic Atrophy; Optic Nerve Diseases; Cranial Nerve Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Mitochondrial Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Zuhal Butuner - Chief Medical Officer
• Organization: Sepul Bio

contact@sepulbio.com

(905) 599 7887

Study Officials

• Sepul Bio Chief Medical Officer

  Sepul Bio

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 21, 2019
• First Posted: April 12, 2019
• Study Start: May 13, 2019
• Primary Completion: October 3, 2022
• Study Completion: October 3, 2022
• Last Updated: October 15, 2024
• Results First Posted: January 4, 2023

Record last verified: 2024-10

Data Sharing

• IPD Sharing: Will not share

Locations

BE (1); US (2)