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Stargardt Disease-1Phase 2NCT04489511

Study of STG-001 in Subjects With Stargardt Disease

A Phase 2 study testing STG-001 in people with Stargardt Disease-1, run by Stargazer Pharmaceuticals, Inc. at 5 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This is an open-label, multicenter study in subjects with Stargardt disease, comparing 2 doses of STG-001 with regard to safety, pharmacokinetics and pharmacodynamics. Read more about this study

Who is it for?
Ages 18 to 55 years
Conditions the study is for
Stargardt Disease-1
What is being tested?
STG-001
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
10 participants
Where
5 sitesUnited States
Study dates
1 Oct 2020to 24 Apr 2021
Phase
Phase 2
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 18 to 55 years
  • All sexes
  • No healthy volunteers
  • Adult

Who may be able to join

  • Understand the study procedures and agree to participate by providing written informed consent.
  • STGD1 caused by autosomal recessive mutation in the ABCA4 gene (i.e. at least 2 mutations), confirmed genotypically by a fully accredited certified central genotyping laboratory.
  • 18 to 55 years of age inclusive.
  • Negative pregnancy testing for women of childbearing potential and highly effective double barrier contraception before, during, and for a period after study treatment as described in the clinical protocol.
  • Primary study eye must have at least one well-demarcated area of significantly reduced autofluorescence as imaged by fundus autofluorescence (FAF)
  • In at least one eye and in the same eye:
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  • ETDRS BCVA of less than or equal to 0.4 logMAR (≤68 letters; 20/50 Snellen equivalent) and greater than or equal to 1.3 logMAR (≥19 letters; 20/400 Snellen equivalent); and
  • Clinical evidence of a macular lesion phenotypically consistent with Stargardt Disease
  • Naïve to investigational treatment for STGD1 with no history of gene therapy, stem cell therapy, surgical implantation of prosthetic retinal chips or intravitreal or sub-retinal injections. History of experimental oral therapy is allowed if it occurred greater than 3 months prior to Screening.
  • Primary study eye must have clear ocular media and adequate pupillary dilation, including no allergy to dilating eyedrops, to permit good quality retinal imaging.
  • In good general health, aside from STGD1, as judged by Investigator.
  • Willing and able to comply with the protocol, including attending assessment visits.

Who cannot join

  • The subject is an employee of the Sponsor or study site or immediate family member (e.g., spouse, parent, child, sibling) of the Sponsor or study site.
  • Use of prescription or nonprescription drugs and dietary supplements within 7 days or 5 half-lives (whichever is longer) prior to Day 1 first dose. Herbal supplements and HRT must be discontinued 7 days prior to Day 1 first dose.
  • Presence of any concurrent ocular disease that would affect study outcomes (e.g., severe cataracts; subjects can be enrolled 3 months after successful cataract surgery).
  • Presence of any meaningful retinopathy/maculopathy/atrophy other than as a result of STGD1 in either eye as determined by the Investigator.
  • Has taken non-approved items (supplement containing vitamin A or beta-carotene, liver-based products, or prescription oral retinoid medications) within 30 days of Screening.
  • Use of medications that may interact with Vitamin A metabolism within 60 days of Screening.
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  • Participation in an oral interventional study of a vitamin A derivative up to 3 months prior to Screening.
  • The subject had a history of vitamin A deficiency as defined based upon serum values less than 20 mcg/dl (0.7 μmol/L) or clinical signs during slit lamp examination (conjunctival or corneal xerosis; Bitot's spots; corneal ulcers or scarring not due to trauma or other secondary causes).
  • Presence of significant cardiovascular or cerebrovascular disease, including stroke, within 12 months of entry.
  • Has a clinically significant abnormal electrocardiogram (ECG), or has a corrected QT interval (QTc) that is 450 ms or greater.
  • Resting heart rate outside specified limits (< 40/minute, > 100/min) upon repeated measurement.
  • History of diabetes, hepatitis, pancreatitis, cirrhosis, liver failure, uncontrolled thyroid disease or hypervitaminosis A.
  • Any intraocular surgery or thermal laser within 3 months of trial entry. Any prior thermal laser in the macular region.
  • Any major surgical procedure within one month of Screening or planned or anticipated surgery during the study period.
  • Women who are pregnant, nursing, or planning to become pregnant during the study period.
  • Uncontrolled blood pressure outside specified limits (90 mm Hg > Systolic > 140 mm Hg and/or 40 mm Hg > Diastolic > 90 mm Hg) upon repeated measurement.
  • Clinically significant abnormal lab results at Screening, including liver function test (aspartate transaminase, alanine transaminase, bilirubin and alkaline phosphatase) greater than 1.5 x the upper limit of normal (ULN).
  • Actively participating in an investigational therapy study or have received any investigational therapy within 60 days of Screening or 5 half-lives, whichever is longer.
  • Known serious allergies to the fluorescein dye used to measure intraocular pressure, ocular dilating drops, topical ocular anesthetic, components of the STG-001 formulation or any history of anaphylaxis reaction.
  • In the Investigator's assessment, any acute or chronic medical condition, psychiatric condition, physical examination finding or laboratory abnormality that might increase the risk associated with study participation or administration of study treatment or interfere with the interpretation of study results.

Trial locations and contacts

5 sites in 1 country.

5 sites in 1 country

United States5 sites

  • Retina Foundation of Southwest

    Dallas, Texas, 75231

  • Vitreo Retinal Associates, P.A.

    Gainesville, Florida, 32607

  • Associated Retina Consultants

    Phoenix, Arizona, 85020

  • UPMC Eye Center

    Pittsburgh, Pennsylvania, 15213

  • Casey Eye Institute - OHSU

    Portland, Oregon, 97239

Other trials in United States

About this study

Official title: A Phase 2a Study of the Safety, Pharmacokinetics and Pharmacodynamics of STG-001 in Subjects With Stargardt Disease (STGD1) Caused by Autosomal Recessive Mutation in ATP Binding Cassette Subfamily A Member 4 (ABCA4) Gene

This is an open-label, multicenter study in subjects with Stargardt disease, comparing 2 doses of STG-001 with regard to safety, pharmacokinetics and pharmacodynamics.

Read the detailed description

This is an open-label, multicenter, active treatment study in approximately 12 subjects aged 18 to 55 years (inclusive) with STGD1, genotyped with a minimum of two ABCA4 gene mutations. Treatment course of STG-001 at Dose 1 (Cohort 1) or Dose 2 (Cohort 2) testing doses will be administered once daily for 28 days. Cohorts will run in parallel. Subjects will receive their designated dose daily for 28 days and be monitored for safety measures during the dosing period and for an additional 28 days after dosing (through Day 56).

Being tested
STG-001
Medical subject headings
Stargardt Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • STG-001Drug

    Dose 1 or Dose 2 of STG-001 will be administered daily for 28 days to compare safety, pharmacokinetics and pharmacodynamics in Stargardt disease subjects.

Study groups in NCT04489511
Study groupRoleWhat they receive
Dose 1ExperimentalSTG-001 given orally at Dose 1 once a day for 28 days
Receives: Drug: STG-001
Dose 2ExperimentalSTG-001 given orally at Dose 2 once a day for 28 days
Receives: Drug: STG-001

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Safety events and Tolerability of STG-001 from baseline to 28 days: adverse events

    Safety and tolerability of 28 days of daily dosing of 2 doses of STG-001 assessed by Incidence and/or clinically significant changes of a combination of ocular and non-ocular adverse events

    Measured: From baseline to 28 days

Secondary outcomes (3)

  1. Pharmacokinetics (PK) of STG-001 maximum plasma concentration (Cmax) at Day 28

    Cmax of 2 doses of STG-001 after 28 days of daily dosing of STG-001 assessed by a validated STG-001 assay

    Measured: From baseline to 28 days

  2. Pharmacokinetics (PK) of STG-001 area under the curve (AUC) at Day 28

    Exposure to STG-001 as assessed by calculated AUC of 2 doses of STG-001 after 28 days of daily dosing of STG-001

    Measured: From baseline to 28 days

  3. Pharmacodynamics (PD) of STG-001 change in plasma Retinol-Binding Protein 4 (RBP4) at Day 28

    Change in Plasma RBP4 levels from baseline to Day 28 with 2 doses of STG-001 after 28 days of daily dosing of STG-001

    Measured: From baseline to 28 days

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted28 Jul 2020
  2. Study start1 Oct 2020
  3. Primary completion24 Apr 2021
  4. Study completion24 Apr 2021

Resources

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 2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
10 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04489511
Sponsor's protocol ID
STG-001-2a
Status last verified
Apr 2021
First submitted
21 Jul 2020
Last updated
27 Apr 2021
Results posted
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing STG-001 in people with Stargardt Disease-1, run by Stargazer Pharmaceuticals, Inc. at 5 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 12 inclusion and 20 exclusion criteria. 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 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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 events and Tolerability of STG-001 from baseline to 28 days: adverse events (time frame: From baseline to 28 days). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04489511.

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 2021.

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