Stargardt DiseasePhase 1NCT02230228
Phase 1 Safety Study of ALK-001 in Healthy Volunteers
A Phase 1 study testing ALK-001 (No generic name) in people with Stargardt Disease and Age-related Macular Degeneration, run by Alkeus Pharmaceuticals, Inc..
Trial 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. Read more about this study
- Who is it for?
- Ages 21 to 70 yearsHealthy volunteers can take part
- Conditions the study is for
- Stargardt Disease, Age-related Macular Degeneration, Other Retinal Dystrophies
- What is being tested?
- ALK-001 (No generic name)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- No sites listed yet
- Study dates
- Apr 2014to Feb 2015
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 21 to 70 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Main Inclusion Criteria
- 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
Main Exclusion Criteria:
- 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.
About this study
Official title: A Phase 1, Open Label, Repeat Dose Study to Investigate the Safety and Pharmacokinetics of 4-week Daily Dosing of ALK-001 in Healthy Volunteers
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.
- Being tested
- ALK-001 (No generic name)
- Medical subject headings
- Stargardt Disease, Macular Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ALK-001 (No generic name)Drug
Also called C20-D3-Retinyl Acetate, C20 Deuterated vitamin A
Daily, oral administration of ALK-001 capsules.
| Study group | Role | What they receive |
|---|---|---|
| ALK-001 capsules | Experimental | Not described Receives: Drug: ALK-001 (No generic name) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 4 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Sep 2014 (estimated)
- Study startApr 2014
- Primary completionSep 2014
- Study completionFeb 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Stargardt DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alkeus Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02230228
- Sponsor's protocol ID
- ALK001-P1001
- Status last verified
- Apr 2025
- First submitted
- 27 Aug 2014
- Last updated
- 27 Apr 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Open Label, Repeat Dose Study to Investigate the Safety and Pharmacokinetics of 4-week Daily Dosing of ALK-001 in Healthy Volunteers
- ClinicalTrials.gov ID
- NCT02230228
- Acronym / sponsor ID
- ALK001-P1001
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 40 participants (actual)
- Lead sponsor
- Alkeus Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- Apr 2014
- Primary completion
- Sep 2014 (actual)
- Study completion
- Feb 2015 (actual)
- First posted
- 3 Sep 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ALK-001 capsules | Experimental | Drug: ALK-001 (No generic name) |
Who may qualify
- Ages
- 21 to 70 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ALK-001 (No generic name)Also: C20-D3-Retinyl Acetate, C20 Deuterated vitamin ADaily, oral administration of ALK-001 capsules. | Drug | ALK-001 capsules |
Primary outcome
Safety of 4-week daily dosing of ALK-001 in healthy adults.
Time frame: 4 weeks
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).
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Apr 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ALK-001 (No generic name) in people with Stargardt Disease and Age-related Macular Degeneration, run by Alkeus Pharmaceuticals, Inc..
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 27 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety of 4-week daily dosing of ALK-001 in healthy adults. (time frame: 4 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Apr 2025.