DF-003 for ROSAH Syndrome

Recruiting at 4 trial locations

Overseen By Neil Solomons, MD

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Shanghai Yao Yuan Biotechnology Ltd. (also known as Drug Farm)

Must not be taking: CYP3A4 inhibitors, CYP3A4 inducers

Disqualifiers: Pregnancy, Hepatic impairment, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 29

Duration: 4 weeks

12 Participants Needed

Trial Summary

Do I need to stop my current medications for the trial?

Yes, you may need to stop certain medications. The trial prohibits the use of specific drugs, including some anti-inflammatory agents, certain antibiotics, and medications affecting liver enzymes. Check with the trial team to see if your current medications are on the prohibited list.

What is the purpose of this trial?

The purpose of this study is to evaluate the safety and tolerability of DF-003 in retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis, and migraine headache (ROSAH) syndrome patients.

Research Team

Kathleen Digre

Principal Investigator

University of Utah (Moran Eye Center)

Oleg Alekseev

Principal Investigator

Duke University (Duke Eye Center)

John Grigg

Principal Investigator

University of Sydney (Save Sight Institute)

Simon Chatfield

Principal Investigator

Melbourne Health

Eligibility Criteria

This trial is for individuals with ROSAH syndrome, a condition that affects the skin. Participants must meet certain health criteria to join, but specific inclusion and exclusion details are not provided.

Inclusion Criteria

I understand and can follow the study's requirements.

All women of childbearing potential must have negative pregnancy tests

I agree to use effective birth control methods.

Exclusion Criteria

I am not currently on any treatments that are not allowed in the study.

I have unusual vital signs or physical exam results.

I have a history of heart rhythm problems or significant heart disease.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive oral doses of DF-003 once daily for 28 days, with loading doses on Days 1-3 followed by maintenance doses from Day 4 to Day 28

4 weeks

Daily visits for dosing

Follow-up

Participants are monitored for safety and effectiveness after treatment

8 weeks

Visits on Day 29 and Day 78

Treatment Details

Interventions

• DF-003

Trial Overview The study tests different doses of an oral medication called DF-003 to see how safe it is and how the body responds to it in patients with ROSAH syndrome.

Participant Groups

Group I: DF-003 Experimental Treatment 1 Intervention

Oral (PO) doses of 140 mg DF-003 on Days 1, 2, and 3 followed by a maintenance dose of 45 mg DF-003 once daily (QD) starting on Day 4 through Day 28. DF-003 will be administered PO with approximately 240 mL of water in the morning once daily for 28 consecutive days.

Who Is Running the Clinical Trial?

Shanghai Yao Yuan Biotechnology Ltd. (also known as Drug Farm)

Lead Sponsor

Trials

2

Recruited

110+