Trials Sponsors Conditions Drugs Pricing

Conditions

Inherited Retinal Diseases Caused by RPE65 Mutations Leber Congenital Amaurosis

Keywords

IRDLCALCA2

Outcome Measures

Primary Outcomes (1)

Incidence of ocular and non-ocular adverse events
52 weeks

Secondary Outcomes (2)

Change from baseline in Best Corrected Visual Acuity (BCVA) of letters based on the Early Treatment Diabetic Retionpathy Study (ETDRS) chart
52 weeks
Change from baseline in visual fields of full-field stimulus threshold test in log cd.s/m2.
52 weeks

Study Arms (1)

HG004

EXPERIMENTAL

Drug: HG004

Interventions

HG004Drug

Low dose Medium dose High dose

HG004

Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.

Eligibility Criteria

Age6 Years - 50 Years

Sexall

Healthy VolunteersNo

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

You may qualify if:

Male or females between 6 and 50 years of age at the time of signing the informed consent form.
Willing to adhere to protocol as evidenced by written informed consent or parental permission and subject assent.

Sponsors & Collaborators

HuidaGene Therapeutics Co., Ltd.
Industrylead

Study Sites (3)

Research Site

Sacramento, California, 95817, United States

RECRUITING

Study DirectorCONTACTHG00402@huidagene.com

Research Site

Houston, Texas, 77707, United States

RECRUITING

Study DirectorCONTACTHG00402@huidagene.com

Research Site

MeSH Terms

Conditions

Leber Congenital Amaurosis

Condition Hierarchy (Ancestors)

Eye Diseases, HereditaryEye DiseasesRetinal Diseases

Study Officials

Study Director
HuidaGene Therapeutics Co., Ltd.
STUDY DIRECTOR

Central Study Contacts

Study Director

CONTACT

732-318-9873 HG00402@huidagene.com

Study Design

Study Type: interventional
Phase: phase 1 2
Allocation: NA
Masking: NONE
Purpose: TREATMENT
Intervention Model: SINGLE GROUP
Sponsor Type: INDUSTRY
Responsible Party: SPONSOR

Study Record Dates

First Submitted

May 9, 2023

First Posted

June 18, 2023

Study Start

October 31, 2023

Actual (reported)

Primary Completion

December 2025

Estimated (date passed)

This estimate has passed. The registry has not confirmed an actual date for this milestone.

Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (2)

Study Arms (1)

HG004

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (3)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Study Officials

Central Study Contacts

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

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (2)

Study Arms (1)

HG004

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (3)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Study Officials

Central Study Contacts

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations