Phase 1 Safety Study of ALK-001 in Healthy Volunteers

NCT02230228 | Completed Phase 1 FDA Drug

• 1 other identifier: ALK001-P1001
• Study Type: interventional
• Target: 40
• Locations: 0 countries
• Sites: N/A

Brief Summary

This protocol is a phase 1 clinical study to assess the safety and pharmacokinetics of ALK-001 in healthy volunteers. Please contact trials@alkeus.com for any questions.

Conditions

Stargardt Disease; Age-related Macular Degeneration; Other Retinal Dystrophies

Outcome Measures

Primary Outcomes (1)

• Safety of 4-week daily dosing of ALK-001 in healthy adults.

  Safety evaluations combine: adverse events (AE), laboratory testing (hematology and biochemistry panels), 12-lead electrocardiograms (ECGs), vital signs, physical examination, and visual function (visual acuity and a self-reported questionnaire).

  4 weeks

Study Arms (1)

ALK-001 capsules

EXPERIMENTAL

Drug: ALK-001 (No generic name)

Interventions

ALK-001 (No generic name) DRUG

Daily, oral administration of ALK-001 capsules.

Also known as: C20-D3-Retinyl Acetate, C20 Deuterated vitamin A

ALK-001 capsules

Eligibility Criteria

• Age: 21 Years - 70 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Adult between 21 and 70 years old (inclusive)
• Healthy subject, as judged by investigator
• Subject is able and willing to comply with study requirements (study medication compliance, schedule of follow-up visits)
• Subject has provided informed consent to participate
• If female, subject uses a medically accepted birth control method and agrees to use such a method for entire clinical trial period

You may not qualify if:

• Subject has taken disallowed items during the past 30 days
• Female with a positive urine pregnancy test at screening
• Lactating woman
• Subject has participated in any clinical study involving an investigational drug, biologic or device, during the past 30 days
• History or current evidence of gastrointestinal malabsorption
• Subject has any other medical condition, which in the opinion of the investigator, is likely to prevent compliance with protocol and/or interfere with successful collection of study
• Subject has, in the opinion of investigator, clinically significant laboratory result(s), positive drug or alcohol screening, or ECG, which makes subject unsuitable for participation.

Sponsors & Collaborators

• Alkeus Pharmaceuticals, Inc.: lead

MeSH Terms

Conditions

Stargardt Disease; Macular Degeneration

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: BASIC SCIENCE
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 27, 2014
• First Posted: September 3, 2014
• Study Start: April 1, 2014
• Primary Completion: September 1, 2014
• Study Completion: February 1, 2015
• Last Updated: April 27, 2025

Record last verified: 2025-04