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Ocular Graft vs Host DiseasePhase 1/2NCT02702518

rhDNase Eye Drops in Patients With Ocular Graft-Vs.-Host Disease

A Phase 1/2 study testing rhDNase I in people with Ocular Graft vs Host Disease and Dry Eye, run by University of Illinois Chicago at 2 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

The purpose of this study is to evaluate the tolerability and preliminary efficacy of rhDNase I eye drops in patients with ocular Graft-vs.-Host disease (oGVHD). Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Ocular Graft vs Host Disease, Dry Eye
What is being tested?
rhDNase I
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
58 participants
Where
2 sitesUnited States
Study dates
Apr 2016to 8 Apr 2020
Phase
Phase 1/2
Are there results?
Yes, posted in the registrySummary in the doctors' view

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 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • The PI and other members of International Chronic Ocular GVHD Consensus Group have established the consensus diagnostic criteria and classification for chronic ocular GVHD.
  • Table 1: Severity scale in chronic ocular GVHD
  • Severity scores (points) Schirmer's test (mm) CFS (points) OSDI (points) Conj (points) 0 >15 0 <13 None
  • 11-15 <2 13-22 Mild/Moderate
  • 6-10 2-3 23-32 Severe
  • ≤5 ≥4 ≥33
Show all 15Show fewer
  • CFS; corneal fluorescein staining, OSDI; Ocular Surface Disease Index. Conj; conjunctival injection. Severity classification; Total score (points); (Schirmer's test score+ CFS score+ OSDI score+ Conj injection score) = None;0-4, Mild/Moderate; 5-8, Severe, 9-11.
  • Table 2: Diagnosis of chronic ocular GVHD None (points) Probable GVHD (points) Definite GVHD (points) Systemic GVHD(-) 0-5 6-7 ≥8 Systemic GVHD(+) 0-3 4-5 ≥6
  • Based on these criteria (Tables 1 and 2),40 patients with definite ocular GVHD will be enrolled. Additionally, all of the following criteria should be met to be eligible for the study:
  • Aged 18 years or older.
  • Capable of giving informed consent and does provide informed consent.
  • Schirmer I <10
  • Corneal/ conjunctival (Rose Bengal) staining ≥1
  • Ocular symptoms must be considered as annoying or activity limiting (OSDI ≥13; mild).
  • Women must be post-menopausal ≥ 1 year, or surgically sterilized. If not, a negative urine pregnancy test is required within 14 days of receiving her first dose of test medication (placebo/ study drug) along with definite evidence of contraceptive use during the duration of the study. Women of reproductive age should use a method of birth control that is acceptable to the subject and the study doctor. This may include oral contraceptive pills, birth control implants, barrier methods or abstinence. If a subject mentions she suspects she may be pregnant after being enrolled, another pregnancy test will be administered. If the test is positive, she will be discontinued from the study immediately.

Who cannot join

  • Subjects will not be eligible for the study if any of the following criteria are met:
  • Allergic to rhDNase I or any similar products, or recipient of rhDNase I eye drops 0.1%.
  • Receiving or have received within 30 days any experimental systemic medication.
  • Active ocular infection or ocular allergies.
  • Any history of eyelid surgery or ocular surgery within the past 3 months.
  • Corneal epithelial defect larger than 1 mm2 in either eye.
  • Have active drug/alcohol dependence or abuse history.
  • Vulnerable populations, such as neonates, pregnant women, children, prisoners, institutionalized individuals, or others who may be considered vulnerable populations.

Trial locations and contacts

2 sites in 1 country.

United States2 sites

  • Translational Clinic of Corneal Neurobiology laboratory, Illinois Eye and Ear Infirmary, University of Illinois at Chicago

    Chicago, Illinois, 60612

  • University of Illinois Eye and Ear Infirmary

    Chicago, Illinois, 60612

Other trials in United States

About this study

Official title: A Phase I/II Randomized Placebo-Controlled, Double-Blind, Single-Center, Tolerability and Preliminary Efficacy Clinical Trial of Recombinant Human Deoxyribonuclease (rhDNase) Eye Drops in Patients With Ocular Graft-Vs.-Host Disease

The purpose of this study is to evaluate the tolerability and preliminary efficacy of rhDNase I eye drops in patients with ocular Graft-vs.-Host disease (oGVHD).

Being tested
rhDNase I, Vehicle
Keywords
rhDNase I, Pulmozyme, oGVHD, Dry Eye
Medical subject headings
Dry Eye Syndromes

What participants receive

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

  • rhDNase IDrug

    Also called Pulmozyme

    rhDNase I, 0.1% eye drops 4 times a day for 8 weeks

  • VehicleDrug

    Also called Placebo

    Drug vehicle 4 times a day for 8 weeks

Study groups in NCT02702518
Study groupRoleWhat they receive
rhDNase IActive comparatorrhDNase I 0.1% eye drops 4 times a day for 8 weeks
Receives: Drug: rhDNase I
VehiclePlacebo comparatorDrug vehicle eye drops 4 times a day for 8 weeks
Receives: Drug: Vehicle

What is being measured

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

  1. The Change in the Corneal Surface Staining as Measured by Dye Staining Within Groups.

    Corneal staining score as measured by dye staining using National Eye Institute (NEI) grading scale. Dye was applied to each eye and a slit lamp was used to observe corneal staining. NEI scale relies on a chart that divides the cornea into 5 sections and assigns a value from 0 (absent) to 3 (severe) to each section, based on the density of punctate staining, final staining score being the sum of individual section scores with a range of 0 (minimum) -15 (maximum) points. Complete corneal staining clearance with dye defined as a score of 0 indicating the best outcome.

    Measured: Between baseline and at 8 weeks of treatment

Secondary outcomes (1)

  1. The Change in Ocular Surface Disease Index (OSDI) Score Within Groups

    Ocular Surface Disease Index (OSDI), a 12-item questionnaire, assesses symptom of ocular irritation in dry eye disease (DED) and how it affects functioning related to vision in the past week. It has 3 subscales: ocular symptoms, vision-related function, and environmental triggers. Patients rate their responses on 0 to 4 scale with 0 being "none of the time" and 4 being "all of the time." OSDI score range from 0-100 with score 0-12 being normal, 13-22 being mild DED, 23-32 being moderate DED, and >33 being severe DED. OSDI=[(sum of scores for questions answered)×100]/[(total questions answered)×4]

    Measured: Between baseline and at 8 weeks of treatment

Timeline

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

  1. First posted8 Mar 2016 (estimated)
  2. Study startApr 2016
  3. Primary completion8 Apr 2020
  4. Study completion8 Apr 2020

Why it stopped: The DSMB committee recommended closing the trial because of the COVID-19 risk in this "at-risk" oGVHD population if they came for study visits.

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 1/2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
58 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02702518
Sponsor's protocol ID
2014-0372
Other IDs
R01EY024966-01A1
Status last verified
Aug 2021
First submitted
3 Mar 2016
Last updated
5 Sep 2021
Data monitoring committee
Yes
Individual data sharing
No

Publications

  • Tibrewal S, Sarkar J, Jassim SH, Gandhi S, Sonawane S, Chaudhary S, Byun YS, Ivanir Y, Hallak J, Horner JH, Newcomb M, Jain S. Tear fluid extracellular DNA: diagnostic and therapeutic implications in dry eye disease. Invest Ophthalmol Vis Sci. 2013 Dec 11;54(13):8051-61. doi: 10.1167/iovs.13-12844.

    PubMed 24255046

  • Sonawane S, Khanolkar V, Namavari A, Chaudhary S, Gandhi S, Tibrewal S, Jassim SH, Shaheen B, Hallak J, Horner JH, Newcomb M, Sarkar J, Jain S. Ocular surface extracellular DNA and nuclease activity imbalance: a new paradigm for inflammation in dry eye disease. Invest Ophthalmol Vis Sci. 2012 Dec 17;53(13):8253-63. doi: 10.1167/iovs.12-10430.

    PubMed 23169882

  • Mun CS, Surenkhuu B, Chen YF, Atassi N, Mun J, Kim C, Sheth T, Sarwar MA, Pradeep A, Jain S. Recombinant Deoxyribonuclease I Eye Drops for Ocular Graft Versus Host Disease: Results of a Randomized Clinical Trial. Eye Contact Lens. 2024 May 1;50(5):233-240. doi: 10.1097/ICL.0000000000001078. Epub 2024 Feb 23.

    PubMed 38407974

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing rhDNase I in people with Ocular Graft vs Host Disease and Dry Eye, run by University of Illinois Chicago at 2 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 15 inclusion and 8 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: stopped early, as of the sponsor's last update on 5 Sep 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined 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.

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?

Yes. Results are posted in the registry (first posted 10 Aug 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

The Change in the Corneal Surface Staining as Measured by Dye Staining Within Groups. (time frame: Between baseline and at 8 weeks of treatment). 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 NCT02702518, which also lists 3 linked publications.

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 5 Sep 2021.

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