Trials Sponsors Conditions Drugs Pricing

Conditions

X-linked Retinoschisis

Keywords

gene therapyAAVRS1

Outcome Measures

Primary Outcomes (2)

Incidence of adverse events and serious adverse events
An adverse event (AE) is any untoward medical occurrence in a clinical investigation participant administered a product; the event will not need to have a causal relationship with the treatment. A serious adverse event (SAE) is any untoward medical occurrence at any dose that leading to the following: Results in death; Life-threatening, refers to an event in which the patient is at risk of death at the time of the event; it does not refer to an event which hypothetically might have caused death if it were more severe; Significant or permanent disability/incapacity, where disability refers to a serious disruption and damage of a person's ability to perform normal life functions; Requires inpatient hospitalization or prolongation of existing hospitalization; Congenital anomaly or birth defect; Other medically important events.
baseline to day 7, month 1, 2
Change in best corrected visual acuity (BCVA)
BCVA of both eyes will be assessed using the early treatment of diabetic retinopathy study (ETDRS) chart or tumbling "E" chart. This approach was chosen to facilitate visual acuity testing in children who cannot recognize letters, which was more appropriate for this study.
baseline to day 7, month 1, 2

Secondary Outcomes (7)

Incidence of adverse events and serious adverse events
baseline to month 3, 4, 6, 9, 12
Change in Quality of Life
baseline to month 9, 12
Change in best corrected visual acuity (BCVA)
baseline to month 3, 4, 6, 9, 12
Change in visual field
baseline to month 1, 2, 3, 4, 6, 9, 12
Change in electrophysiology result
baseline to month 1, 2, 3, 4, 6, 9, 12
+2 more secondary outcomes

Study Arms (2)

group 1

EXPERIMENTAL

IVT administration of a single low dose ZM-01 injection

Drug: ZM-01-L

group 2

EXPERIMENTAL

IVT administration of a single high dose ZM-01 injection

Drug: ZM-01-H

Interventions

ZM-01-LDrug

rAAV-hRS1 intravitreal injection of low dose

Also known as: rAAV-hRS1

group 1

ZM-01-HDrug

rAAV-hRS1 intravitreal injection of high dose

Also known as: rAAV-hRS1

Eligibility Criteria

Age3 Years - 18 Years

Sexmale

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

Subjects who meet all of the following criteria will be enrolled into the study
Diagnosis of X-linked retinoschisis consistent with the presence of RS1 gene mutation

Sponsors & Collaborators

Zhongmou Therapeutics
Industrylead

Study Sites (1)

Wuhan University Renmin Hospital affiliated with Hanchuan Hospital

Xiaogan, Hubei, China

MeSH Terms

Conditions

Retinoschisis

Condition Hierarchy (Ancestors)

Retinal DegenerationRetinal DiseasesEye Diseases

Study Officials

Yin Shen, PhD
Zhongmou Theraputics
STUDY CHAIR

Enrollment

2 participants (actual enrollment reported by the registry).

Study Design

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

Study Record Dates

First Submitted

September 25, 2023

First Posted

October 4, 2023

Study Start

September 25, 2022

Actual (reported)

Safety and Efficacy Study of Novel Gene Therapy ZM-01 for X-linked Retinoschisis Patients

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

Study Start

First Submitted

First Posted

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (2)

Secondary Outcomes (7)

Study Arms (2)

group 1

group 2

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (1)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Study Officials

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

Study Start

First Submitted

First Posted

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (2)

Secondary Outcomes (7)

Study Arms (2)

group 1

group 2

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (1)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Study Officials

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations