Evaluating the Safety and Tolerability of Orally Administered DF-003 in ROSAH Syndrome Patients

Conditions

• ROSAH

Eligibility Criteria

Sex

ALL

Age

18 Years - 65 Years

Healthy Volunteers

Not accepted

Interventions

• DF-003 — DRUG
  140 mg on Days 1, 2, and 3 followed by a maintenance dose of 45 mg 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.

Study Details

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.

Key Dates

Start date

May 27, 2025

Status verified

Aug 2025

Primary completion

Jul 31, 2026

Completion

Nov 30, 2026

Study Design

Enrollment

12 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: DF-003
  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.

Primary Outcome Measure

Frequency and Severity of Treatment-Emergent Adverse Events (TEAEs) as Assessed by CTCAE v5.0 [ Time Frame: Baseline to Day 78 (±2) ]

Central Contacts

• Kenneth Truitt, MD
  732-221-7104
  [email protected]

Locations (3)

Find similar trials in Bethesda, MD

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