A Phase 1a Open-Label, Single Ascending Dose Study to Evaluate the Safety and Tolerability of Intravitreally Administered PYC-001 in Participants With Confirmed OPA1 Mutation-Associated Autosomal Dominant Optic Atrophy

Status: Recruiting
Location: See all (2) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1
SUMMARY

A First-in-Human multi-centre, prospective, Phase1a, Single Ascending Dose (SAD) interventional study of PYC-001 in participants with confirmed OPA1 mutation (haploinsufficiency) associated ADOA.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:

• Must have given written informed consent before any study-related activities are carried out and must be able to understand the full nature and purpose of the trial, including possible risks and adverse effects;

• Adult males and females, aged 18 years and above at screening;

• Body mass index (BMI) ≥18.0 and ≤32.0 kg/m2, with a body weight ≤ 100kg at screening;

• Have a molecular (genetic) diagnosis of OPA1 mutation at screening;

• Have a clinical diagnosis of OPA1 mutation-associated ADOA at screening;

• Participants with BCVA of between 20/40 (70 ETDRS letters) and 20/160 (39-43 ETDRS letters). If both eyes meet this eligibility criteria, the eye with worse BCVA will be selected as the study eye and the other eye will be designated as the fellow eye;

• Medically healthy (in the opinion of the Investigator), as determined by pre-study medical history, and without clinically significant abnormalities including:

∙ Physical examination without any clinically relevant findings;

‣ Systolic BP in the range of 90 to 160mmHg and diastolic BP in the range of 50 to 95 mmHg after 5 minutes in supine of semi-supine position;

‣ Heart rate in the range of 45 to 100 bpm after 5 minutes rest in supine or semi-supine position;

‣ Body temperature (tympanic), between 35.5°C and 37.7°C;

‣ ECG without clinically significant abnormalities including QT interval corrected for Fredericia (QTcF) \< 450 msec for male subjects and \< 470 msec for female subjects;

‣ No clinically significant findings in clinical chemistry, hematology, coagulation and urinalysis tests at screening.

• Female volunteers must be of non-child-bearing potential, i.e., surgically sterilized (hysterectomy, bilateral salpingectomy, bilateral oophorectomy at least 6 weeks before the screening visit) or postmenopausal (where postmenopausal is defined as no menses for 12 months without an alternative medical cause and a follicle stimulating hormone \[FSH\] level consistent with postmenopausal status, per local laboratory guidelines). Females receiving hormone replacement therapy (HRT) may be considered for inclusion if the need for HRT is for no other medical reason than to treat symptoms associated with menopause. If female participants are of child-bearing potential, they must:

∙ Have a negative pregnancy test at the screening visit, on study Day -1 and study Day 1 prior to dosing;

‣ Agree not to attempt to become pregnant or donate ova from signing the consent form until at least 130 days after IVT administration of PYC-001 and at least 30 days after the final dose of ANX776;

‣ Agree to use adequate contraception (defined as use of a condom by the male partner combined with use of a highly effective method of contraception from one month prior to screening until at least 30 days after the final dose of ANX776 (study Day 336), if not exclusively in a same-sex relationship or abstinent as a committed lifestyle.

• Male volunteers must:

∙ Agree not to donate sperm from signing the consent form until at least 190 days after IVT administration of PYC-001 and at least 90 days after the final dose of ANX776;

‣ If engaging in sexual intercourse with a female partner who could become pregnant, agree to use adequate contraception (defined as use of a condom combined with use of a highly effective method of contraception) from signing the consent form until at least 190 days after IVT administration of PYC-001 and at least 90 days after the final dose of ANX776;

‣ If engaging in sexual intercourse with a female partner who is not of child-bearing potential or a same-sex partner, agree to use a condom from signing the consent form until at least 190 days after IVT administration of PYC-001 and at least 90 days after the final dose of ANX776;

⁃ Have suitable venous access for blood sampling

⁃ Willing and able to comply with all study assessments and adhere to the protocol schedule and restrictions.

Locations
Other Locations
Australia
Center for Eye Research Australia (CERA)
RECRUITING
East Melbourne
Save Sight Institute - Sydney Eye Hospital
RECRUITING
Sydney
Contact Information
Primary
Sreenivasu Mudumba
adoa@pyctx.com
510-423-2680
Time Frame
Start Date: 2024-10-31
Estimated Completion Date: 2025-08
Participants
Target number of participants: 18
Treatments
Experimental: Single arm dose escalation study of PYC-001
Drug: PYC-001~Phase 1a Open-Label, Single Ascending Dose Study to Evaluate the Safety and Tolerability of Intravitreally Administered PYC-001 in participants with Confirmed OPA1 Mutation-Associated Autosomal Dominant Optic Atrophy
Related Therapeutic Areas
Sponsors
Leads: PYC Therapeutics

This content was sourced from clinicaltrials.gov