Evaluate the Effects and Safety of ALK4290 in Patients With Newly Diagnosed Wet Age-Related Macular Degeneration

• Sponsor: Alkahest, Inc. (Industry)
• Overall Status: Completed
• CT.gov ID: NCT03558061
• Collaborator: (none)
• Enrollment: 30
• Locations: 5
• Arm: 1
• Actual Duration (Months): 7.5
• Patients Per Site: 6
• Patients Per Site Per Month: 0.8

Study Details

Study Description

Brief Summary

This study will evaluate the therapeutic effects and safety of 800 mg ALK4290 administered daily over a 6-week dosing period in newly diagnosed patients with wet age-related macular degeneration (wAMD).

Condition or Disease	Intervention/Treatment	Phase
Wet Age-related Macular Degeneration	Drug: ALK4290	Phase 2

Detailed Description

This study is designed to evaluate the therapeutic effects and safety of oral ALK4290 administered at 800 mg daily over a 6-week dosing period in newly diagnosed (treatment naïve) patients with wAMD. The study agent will be orally self-administered. All subjects will receive the study agent, ALK4290. This study does not contain a placebo.

Study Design

• Study Type: Interventional
• Actual Enrollment: 30 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Single Arm Open-Label Study to Evaluate the Therapeutic Effects and Safety of a 6-Week Treatment Regimen of ALK4290 in Patients With Newly Diagnosed Wet Age-Related Macular Degeneration (wAMD)
• Actual Study Start Date: Apr 4, 2018
• Actual Primary Completion Date: Nov 18, 2018
• Actual Study Completion Date: Nov 18, 2018

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Active ALK4290 800 mg daily	Drug: ALK4290; ALK4290 400 mg tablet twice a day; Other Names: AKST4290 BI 144807

Outcome Measures

Primary Outcome Measures

1. Best Corrected Visual Acuity (BCVA) [Baseline to 6 weeks]

   Mean change in BCVA letter score as measured by the Early Treatment Diabetic Retinopathy Study (ETDRS) method. The ETDRS chart assesses changes in vision based on the number of letters correctly read by the subject.

Secondary Outcome Measures

1. Incidence of Treatment-emergent Adverse Events (Safety) [Baseline to 10 weeks]

   Treatment-emergent adverse events identified by the Common Terminology Criteria for Adverse Events (CTCAE v4.03)

Eligibility Criteria

Criteria

• Ages Eligible for Study: 50 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Men and women with newly diagnosed active CNV secondary to AMD, diagnosed by a retinal specialist with all the following characteristics and ophthalmic inclusion criteria applied to the study eye:

• No prior treatment for wAMD in the study eye and no current or planned concomitant intravitreal anti-VEGF treatment in the fellow eye

• Central subfield retinal thickness ≥ 250 microns on SD-OCT (exclusive of subretinal pigment epithelial fluid, inclusive of SRF)

• Presence of SRF and/or IRF on SD-OCT

• Any active CNV with subfoveal leakage as determined by FA

• Total lesion size not greater than 12 disc areas on FA

• If present, subretinal hemorrhage must comprise < 50% of the total lesion area on FA

• No subfoveal fibrosis or atrophy on FA

• BCVA letter score, as measured by ETDRS in the study eye, between 70 and 24 letters, inclusive, at screening

• Patients 50 years of age or older at screening visit 1

• Body mass index (BMI) between18 and ≤ 40 at screening visit 1

• Female subjects must not be pregnant or breastfeeding. Women of childbearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry.

• Signed informed consent consistent with ICH-GCP guidelines and local legislation prior to participation in the trial, which includes medication washout and restrictions

Exclusion Criteria:

• Previous participation in any studies of investigational drugs within 1 month preceding screening visit

• Any form of macular degeneration that is not age-related (e.g., Best's disease, Stargardt's disease, Sorsby's disease, etc.)

• Additional eye disease in the study eye that could compromise BCVA (i.e., uncontrolled glaucoma (intraocular pressure > 24) with visual field loss, clinically significant diabetic maculopathy, history of ischemic optic neuropathy or retinal vascular occlusion, vitreomacular traction, monocular vision, or genetic disorders such as retinitis pigmentosa; high myopia > 8 diopters)

• The presence of polypoidal choroidal vasculopathy (PCV) or retinal angiomatous proliferation (RAP) in the study eye

• Anterior segment and vitreous abnormalities in the study eye that would preclude adequate observation with fundus photography/FA or SD-OCT

• Intraocular surgery in the study eye within 3 months prior to screening

• Aphakia or total absence of the posterior capsule (yttrium aluminum garnet (YAG) laser capsulotomy permitted, a minimum of 1 month prior to enrollment) in the study eye

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Jahn Ferenc South-Pest Hospital and Clinic	Budapest		Hungary
2	Borsod-Abauj-Zemplen County Hospital and Teaching Hospital	Miskolc		Hungary
3	Szabolcs-Szatmar-Bereg County Hospital and University Hospital	Nyíregyháza		Hungary
4	University of Szeged Faculty of Medicine	Szeged		Hungary
5	Markusovszky University Teaching Hospital	Szombathely		Hungary

Sponsors and Collaborators

• Alkahest, Inc.

Investigators

• Study Director: Alkahest Medical Monitor, Alkahest, Inc.

Study Documents (Full-Text)

• Study Protocol - May 15, 2018
• Statistical Analysis Plan - Aug 20, 2018

More Information

Publications

None provided.

• Responsible Party: Alkahest, Inc.
• ClinicalTrials.gov Identifier: NCT03558061
• Other Study ID Numbers: ALK4290-201
• First Posted: Jun 15, 2018
• Last Update Posted: Dec 16, 2020
• Last Verified: Dec 1, 2018

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Product Manufactured in and Exported from the U.S.: Yes
• Additional relevant MeSH terms: Macular Degeneration

Study Results

Participant Flow

Arm/Group Title	Active
Arm/Group Description	ALK4290 800 mg daily
Period Title: Overall Study
STARTED	30
COMPLETED	29
NOT COMPLETED	1

Baseline Characteristics

Arm/Group Title	Active
Arm/Group Description	ALK4290 800 mg daily
Overall Participants	30
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	73.1 (8.78)
Sex: Female, Male (Count of Participants)
Female	18 60%
Male	12 40%
Ethnicity (NIH/OMB) (Count of Participants)
Hispanic or Latino	0 0%
Not Hispanic or Latino	30 100%
Unknown or Not Reported	0 0%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	0 0%
Asian	0 0%
Native Hawaiian or Other Pacific Islander	0 0%
Black or African American	0 0%
White	30 100%
More than one race	0 0%
Unknown or Not Reported	0 0%
Region of Enrollment (participants) [Number]
Hungary	8 26.7%
Poland	22 73.3%

Outcome Measures

1. Primary Outcome

Title	Best Corrected Visual Acuity (BCVA)
Description	Mean change in BCVA letter score as measured by the Early Treatment Diabetic Retinopathy Study (ETDRS) method. The ETDRS chart assesses changes in vision based on the number of letters correctly read by the subject.
Time Frame	Baseline to 6 weeks

Outcome Measure Data

Analysis Population Description

Evaluable Set

Arm/Group Title	Active
Arm/Group Description	ALK4290 800 mg daily
Measure Participants	29
Mean (Standard Deviation) [letters]	7.0 (12.51)

2. Secondary Outcome

Title	Incidence of Treatment-emergent Adverse Events (Safety)
Description	Treatment-emergent adverse events identified by the Common Terminology Criteria for Adverse Events (CTCAE v4.03)
Time Frame	Baseline to 10 weeks

Outcome Measure Data

Analysis Population Description

Safety Set

Arm/Group Title	Active
Arm/Group Description	ALK4290 800 mg daily
Measure Participants	30
Count of Participants [Participants]	14 46.7%

Adverse Events

Time Frame	11 weeks
Arm/Group Title	Active
Arm/Group Description	ALK4290 800 mg daily
All Cause Mortality
	Active
	Affected / at Risk (%)	# Events
Total	0/30 (0%)
Serious Adverse Events
	Active
	Affected / at Risk (%)	# Events
Total	0/30 (0%)
Other (Not Including Serious) Adverse Events
	Active
	Affected / at Risk (%)	# Events
Total	14/30 (46.7%)
Eye disorders
Visual acuity reduced	2/30 (6.7%)	2
Infections and infestations
Cystitis	2/30 (6.7%)	2
Musculoskeletal and connective tissue disorders
Arthralgia	5/30 (16.7%)	7
Back pain	3/30 (10%)	3
Nervous system disorders
Headache	2/30 (6.7%)	3

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.