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Stem Cell Therapy

Stem Cell Therapy for Age-Related Macular Degeneration

Phase 1

Recruiting

Research Sponsored by Astellas Institute for Regenerative Medicine

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Participant must be willing to take tacrolimus and willing to discontinue any medications that have a known strong interaction with tacrolimus.

Participant is able and willing to undertake all scheduled visits and assessments up to the week 52 visit.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, weeks 1, 4, 6, 8, 12, 16, 26 and 52/end of study (eos)

Awards & highlights

No Placebo-Only Group

Summary

This trial is for adults 50+ who are losing their vision due to dry age-related macular degeneration. A new treatment, ASP7317, which is a specially created type of cell derived from human stem cells, will be injected into the macula of the eye in an effort to slow or reverse atrophy.

Who is the study for?

Adults over 50 with dry age-related macular degeneration (dry AMD) and geographic atrophy, resulting in central vision loss. Participants must be willing to discontinue certain supplements and medications, agree to use contraception if of childbearing potential, and not have a history of certain eye treatments or conditions that could affect the study's outcome.

What is being tested?

The safety and effectiveness of ASP7317, a new cell-based therapy for dry AMD are being tested. Patients will receive one of three different doses via injection into the eye under anesthesia. They'll also take tacrolimus orally to prevent rejection of these cells. The trial includes frequent clinic visits for monitoring.

What are the potential side effects?

Potential side effects may include reactions related to the immune system due to stem cell transplantation and immunosuppression from tacrolimus such as increased infection risk, possible organ inflammation, or other complications associated with surgery under anesthesia.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am willing to take tacrolimus and stop any medications that strongly interact with it.

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I can attend all appointments and assessments for up to 52 weeks.

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I will not donate eggs for one year after the last treatment.

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I am willing to take tacrolimus and stop any medications that strongly interact with it.

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I have advanced age-related vision loss in both eyes.

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My vision ranges from light perception to reading a few letters for Cohort 1, or is very poor but not complete blindness for Cohorts 2 and 3.

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I am on long-term medication for an infection.

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I have had a solid organ or bone marrow transplant.

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I cannot use systemic immunosuppressants like tacrolimus.

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I had a heart attack in the last year and my heart condition is unstable or causes symptoms.

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My kidney function, measured by eGFR, is 45 mL/min or lower.

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I have a serious blood clotting disorder.

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I have a history of familial adenomatous polyposis or inflammatory bowel disease.

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I have never participated in gene or cell transplant therapy trials.

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I am willing to stop taking certain medications while on tacrolimus.

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My vision loss is not due to age-related macular degeneration.

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I have had eye surgery for AMD or similar conditions.

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I have had treatments like photodynamic therapy or radiation for my eyes.

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I have had laser treatment for serious eye conditions.

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I have an eye condition that affects the thickness or structure of my macula.

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I have a history of specific serious eye conditions.

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I have or had eye inflammation issues like uveitis.

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I have a scar from eye toxoplasmosis.

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I have a high-risk eye mole or lesion.

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I have severe nearsightedness or related eye conditions.

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I have glaucoma that is not controlled, use more than 2 medications for it, or had surgery for it.

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I have had a corneal transplant.

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I can only see with one eye or I have lost an eye.

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I have an eye condition that affects vision tests.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, weeks 1, 4, 6, 8, 12, 16, 26 and 52/end of study (eos)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, weeks 1, 4, 6, 8, 12, 16, 26 and 52/end of study (eos)

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, weeks 1, 4, 6, 8, 12, 16, 26 and 52/end of study (eos) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Incidence of cellular graft failure or rejection

Number of participants with cellular graft failure or rejection

Safety as assessed by incidence, frequency and severity of Serious Adverse Events (SAEs)

+2 more

Secondary study objectives

Mean change from baseline in PWS in junctional zone

Mean change from baseline in PWS in remote zone

Mean change from baseline in area of Geographic Atrophy (GA) (mm^2) in study eye and fellow eye

+8 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: ASP7317 Dose Escalation/ Expansion (Group 2: Moderate Vision Loss)Experimental Treatment5 Interventions

Successive cohorts of participants (3 participants each) will be given escalating doses (cohort 4: low cells/dose; cohort 5: medium cells/dose; cohort 6: high cells/dose). Expansion cohorts 5b and 6b (6 participants each) will be opened after cohorts 5 and 6 have been filled. Dose levels for the expansion cohorts will align with dose levels in escalation cohorts. Cohort 4 (low cells/dose) dosing may begin after the DSMB recommendation to begin dosing in Group 1 cohort 2 (medium cells/dose). Cohort 5 (medium cells/dose) dosing may begin after DSMB review of the 4 week safety data of the first participant in Group 1 cohort 2 (medium cells/dose). Cohort 6 (high cells/dose) dosing may begin after DSMB review of 4 week safety data of the first participant in Group 1 cohort 3 (high cells/dose). Dosing in cohort 5 and 6 can only begin after the DSMB review and the completion of the preceding cohort. Participants will receive tacrolimus and other medicines to stop infection.

Group II: ASP7317 Dose Escalation/ Expansion (Group 1: Severe Vision Loss)Experimental Treatment5 Interventions

Successive cohorts of participants (3 participants each) will be given escalating doses (cohort 1: low cells/dose; cohort 2: medium cells/dose; cohort 3: high cells/dose). Expansion cohorts (6 participants each) 3b will be opened after cohort 3 has been filled and 2b will be opened only if necessary. Dose levels for the expansion cohort will align with dose levels in escalation cohorts. Sentinel dosing will be required for each dose level. After the first participant in Group 1 dose cohort is dosed and followed for 4 weeks, the Data Safety Monitoring Board (DSMB) will review the 4 week safety data and recommend if the second and third participants in Group 1 dose cohort may be treated. The DSMB recommendation to progress to the next dosing cohort will be based on 4 week follow-up safety review of the second and third participants in the preceding dose cohort. Participants will receive tacrolimus and other medicines to stop infection.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

trimethoprim-sulfamethoxazole

2020

Completed Phase 4

~2920

Acyclovir

2005

Completed Phase 4

~5230