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This study adopts a randomized, double-blind, parallel placebo-controlled dose-escalation design, consisting of two parts: Part 1 includes a single ascending dose (SAD) study plus a food effect (FE) study, and Part 2 is a multiple ascending dose (MAD) study.
Part 1: SAD and FE Studies The SAD and FE studies are conducted concurrently, with a total of 5 dose groups: 2 mg, 5 mg, 10 mg, 20 mg, and 40 mg. A total of 46 healthy adult subjects are planned for enrollment. Except for the 5 mg group, each dose group will include 8 subjects, who will be randomly assigned to receive DC6001 tablets (6 subjects) or DC6001 placebo (2 subjects). The 5 mg group will be combined with the FE study, with 14 planned subjects randomly allocated to DC6001 tablets (12 subjects) or placebo (2 subjects). In the first cycle, a single dose will be administered under fasting conditions. After blood sample collection and safety assessment on Day 9 (D9), the second cycle will be conducted on Day 10 (D10) with administration under high-fat postprandial conditions, followed by blood sample collection and discharge safety examinations.
Part 2: MAD Study The MAD study is tentatively designed with 3 dose groups: 2 mg, 5 mg, and 10 mg (to be adjusted based on SAD study results). A total of 30 healthy adult subjects are planned for enrollment, with 10 subjects per dose group randomly assigned to receive DC6001 tablets (8 subjects) or DC6001 placebo (2 subjects). The tentative administration regimen is once-daily fasting administration for 14 consecutive days (to be adjusted based on SAD study results).
The SAD and MAD study will proceed sequentially from the lowest dose group. After subjects in a given dose group complete discharge safety examinations, the Safety Review Committee (SRC) will assess whether the dose escalation termination criteria are met. If not, the dose will be escalated to the next group until the highest dose group is completed.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| SAD: DC6001 | Experimental | Participants received single dose of DC6001 orally. Dose levels are 2 mg, 5 mg, 10 mg, 20 mg and 40 mg. |
|
| SAD: Placebo | Placebo Comparator | Participants received single dose of placebo orally. |
|
| MAD: DC6001 | Experimental | Participant received DC6001 orally once daily for 14 days. Dose levels are 2 mg, 5 mg and 10 mg. |
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| MAD: Placebo | Placebo Comparator | Participant received DC6001 placebo matching DC6001 orally once daily for 14 days. |
|
| FE: DC6001 | Experimental | Participants received single dose of DC6001 5 mg orally under fed conditions. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| DC6001 | Drug | DC6001 tablet |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Adverse Events | Incidence and severity of adverse events as assessed by CTCAE Version 6.0 | Up to 28 days |
| Measure | Description | Time Frame |
|---|---|---|
| The maximum plasma concentration of the drug (Cmax) | From time zero up to 192 hours post-dose | |
| The time at which the peak plasma concentration is reached (Tmax) | From time zero up to 192 hours post-dose |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Kang Ren | Contact | +86-13269683867 | renkang@dcpc.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Beijing Tongren Hospital | Recruiting | Beijing | Beijing Municipality | 100730 | China |
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| ID | Term |
|---|---|
| D000080362 | Stargardt Disease |
| ID | Term |
|---|---|
| D015785 | Eye Diseases, Hereditary |
| D005128 | Eye Diseases |
| D008268 | Macular Degeneration |
| D012162 | Retinal Degeneration |
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| Placebo |
| Drug |
DC6001 placebo tablet |
|
| The area under the plasma concentration-time curve from time zero to the last measurable concentration (AUC0-t) | From time zero up to 192 hours post-dose |
| The area under the plasma concentration-time curve from time zero extrapolated to infinity (AUC0-∞) | Time Frame: From time zero up to 192 hours post-dose |
| The time required for the plasma concentration to decrease by half (T1/2) | From time zero up to 192 hours post-dose |
| Change from baseline in plasma RBP4 levels | From time zero up to 336 hours post-dose |
| D012164 |
| Retinal Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |