DF-003 for ROSAH Syndrome

Recruiting at 3 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to explore the safety and tolerability of the experimental drug DF-003 for individuals with ROSAH syndrome, a rare condition affecting the eyes, spleen, sweat glands, and causing headaches. Participants will take DF-003 orally each day for 28 days. The trial includes those diagnosed with ROSAH syndrome through genetic testing and experiencing symptoms such as eye inflammation or swelling. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new drug.

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.

Is there any evidence suggesting that DF-003 is likely to be safe for humans?

A previous study tested DF-003 on 48 healthy volunteers. The researchers conducted a randomized, placebo-controlled study, meaning participants were randomly assigned to different groups, with some receiving a non-active pill for comparison. The study found that DF-003 was generally well-tolerated. Most side effects were mild, such as headaches or nausea, and no serious side effects were reported.

DF-003 is now being tested specifically for ROSAH syndrome, which affects the eyes, nerves, and other parts of the body. As this trial is in an early phase, it focuses on ensuring the treatment's safety and understanding how the body processes it. While some safety information is available, more is being learned as the trial continues. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for ROSAH Syndrome, which mainly focus on symptom relief and may include medications like steroids or immunosuppressants, DF-003 stands out with its novel approach. DF-003 is administered orally, which makes it easier to take compared to more invasive treatments. Researchers are particularly excited because DF-003 offers a unique dosing regimen, initiating with a higher dose for quick impact, followed by a lower maintenance dose that could provide sustained benefits throughout the month. This could potentially lead to better management of symptoms with fewer side effects.

What evidence suggests that DF-003 might be an effective treatment for ROSAH syndrome?

Research has shown that DF-003, the treatment under study in this trial, can effectively target a specific protein linked to ROSAH syndrome. In studies with mice, DF-003 reduced the activity of a harmful protein that causes inflammation. This suggests it might alleviate symptoms like vision problems and headaches in people with ROSAH syndrome. While most human studies focus on safety, this approach shows promise for addressing the main issues of the condition. ^{1 3 5 6 7}

Who Is on the 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

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Citations

1. nature.com nature.com/articles/s41467-025-63731-5

Discovery of a selective alpha-kinase 1 inhibitor for the ... These results demonstrate the ability of DF-003 to inhibit ALPK1[T237M]-induced cytokine expression in this mouse model of ROSAH syndrome. In ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT06395285

Study Details | NCT06395285 | Evaluating the Safety and ... To evaluate the effects of DF-003 on headache in ROSAH syndrome patients. Test scores range from 36 to 78, with larger scores reflecting greater impact ...

3. firstwordpharma.com firstwordpharma.com/story/5967277

Drug Farm Reports Data on Safety and Pharmacokinetics ... This randomized, placebo-controlled, double-blinded study evaluated the safety, tolerability and pharmacokinetics of DF-003 in forty-eight healthy volunteers.

4. clinicaltrialsarena.com clinicaltrialsarena.com/news/fda-drug-farm-trial/

FDA grants IND clearance for Drug Farm's ROSAH ... The trial will evaluate the safety, pharmacokinetics, and efficacy of DF-003 in individuals affected by the ROSAH condition.

5. clinrol.com clinrol.com/study/NCT06395285

DF-003 Safety in ROSAH Syndrome Patients 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 ...

6. businesswire.com businesswire.com/news/home/20250529149642/en/Drug-Farm-Reports-Data-on-Safety-and-Pharmacokinetics-from-Phase-1-First-in-Human-Trial-of-DF-003-in-Healthy-Volunteers

Drug Farm Reports Data on Safety and Pharmacokinetics ... This randomized, placebo-controlled, double-blinded study evaluated the safety, tolerability and pharmacokinetics of DF-003 in forty-eight healthy volunteers.

7. trialx.com trialx.com/clinical-trials/listings/296759/evaluating-the-safety-and-tolerability-of-orally-administered-df-003-in-rosah-syndrome-patients/

Clinical Research Trial Listing ( ROSAH ) ( NCT06395285 ) The purpose of this study is to evaluate the safety and tolerability of DF-003 in retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis ...