Topical Fibrinogen-Depleted Human Platelet Lysate in Patients With Dry Eye Secondary to Graft vs. Host Disease

NCT03414645 | Completed Phase 1 FDA Drug

• 1 other identifier: CAM-101
• Study Type: interventional
• Target: 64
• Locations: 1 country
• Sites: 6

Brief Summary

The purpose of this Phase 1/2 study is to compare the safety and tolerability of four times a day (QID) dosing of a non-preserved topical ocular drop formulation of 10 vol/vol % and 30 vol/vol % of FD hPL to vehicle control eye drops in patients with Dry Eye Disease (DED) secondary to Graft vs. Host Disease (GvHD).

Conditions

Dry Eye; Graft-versus-host-disease; Ocular Discomfort

Outcome Measures

Primary Outcomes (3)

• Number of participants with ocular treatment-related adverse events as assessed by CTCAE v4.0

  To evaluate the safety and tolerability of two concentrations of CAM-101 (FD hPL 10 vol/vol % and 30 vol/vol %) topical ophthalmic solution in patients with dry eye disease (DED) secondary to graft versus host disease (GvHD) after 6 weeks (42 days) of treatment. The primary outcome measure: Percentage of patients in each dose group with ocular adverse events at Day 42

  42 Days

• Number of participants with systemic treatment-related adverse events as assessed by CTCAE v4.0

  Number of participants with ocular treatment-related adverse events as assessed by CTCAE v4.0 To evaluate the safety and tolerability of two concentrations of CAM-101 (FD hPL 10 vol/vol % and 30 vol/vol %) topical ophthalmic solution in patients with dry eye disease (DED) secondary to graft versus host disease (GvHD) after 6 weeks (42 days) of treatment. The primary outcome measure: Percentage of patients in each dose group with systemic adverse event at Day 42

  42 Days

• Number of participants with treatment-related adverse events as assessed by CTCAE v4.0

  To evaluate the safety and tolerability of two concentrations of CAM-101 (FD hPL 10 vol/vol % and 30 vol/vol %) topical ophthalmic solution in patients with dry eye disease (DED) secondary to graft versus host disease (GvHD) after 6 weeks (42 days) of treatment. The primary outcome measure: The percentage of patients in each dose group that show a change from Normal to Abnormal with clinical significance in any ocular examination assessment at Day 42

  42 Days

Secondary Outcomes (3)

• Efficacy as measured by change in corneal staining

  42 Days

• Efficacy as measured by ocular surface disease index (OSDI)

  42 Days

• Efficacy as measured by ocular discomfort using the 100 point visual analogue scale (VAS) scores

  42 Days

Study Arms (3)

CAM-101 10%

EXPERIMENTAL

FD hPL 10 vol/vol %

Biological: CAM-101 10%

CAM-101 30%

EXPERIMENTAL

FD hPL 30 vol/vol %

Biological: CAM-101 30%

Vehicle Control

PLACEBO COMPARATOR

PlasmaLyte-A, vehicle control, a preservative-free ophthalmic drop

Biological: Vehicle Control

Interventions

CAM-101 10% BIOLOGICAL

fibrinogen-depleted human platelet lysate

Also known as: FD hPL 10% vol/vol

CAM-101 10%

Vehicle Control BIOLOGICAL

PlasmaLyte-A, vehicle control, a preservative-free ophthalmic drop

Vehicle Control

CAM-101 30% BIOLOGICAL

fibrinogen-depleted human platelet lysate

Also known as: FD hPL 30% vol/vol

CAM-101 30%

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female patients, of age 18 years (inclusive) or older at the time of signing the ICF;
• Diagnosis of DED secondary to GvHD following allogeneic hematopoietic stem cell transplantation as determined by medical history
• For females:
• Be of non-child-bearing potential. Surgically sterilized (e.g., hysterectomy or bilateral oophorectomy) for at least 6 months prior to screening or postmenopausal (postmenopausal women must have no menstrual bleeding for at least 1 year prior to screening) and menopause will be confirmed by a plasma FSH level of \>40 IU/L) or
• Women of childbearing potential must be non-lactating and agree to use a highly effective acceptable form of birth control (e.g., established hormonal birth control plus a barrier method, double barrier method: intrauterine device plus condom or spermicidal gel plus condom) from 21 days prior to dosing until 7 days after dosing, and
• Women with a negative pregnancy test (β-hCG assay) in urine at screening and Day 1 predose;
• Schirmer tear test with anesthesia \<7 mm/5 min in at least one eye during screening;
• Willingness and and ability to undergo, and return for, all scheduled study-related visits through Follow-up;
• Willingness and and ability to provide written Informed Consent consistent with privacy language as per national regulations (e.g., HIPAA authorization) and which signature may be obtained from the patient or legally authorized representative prior to any study-related procedures (including withdrawal of prohibited medication);
• Willingness to communicate with the Investigator and site staff and comply with all study procedures and requirements;
• Agreement not to participate in another interventional study while participating in this study.

You may not qualify if:

• Any abnormal lid anatomy or blinking function in either eye;
• Any history of other ocular disease requiring topical ocular treatment other than artificial tears and/or Restasis® (cyclosporine ophthalmic emulsion; Allergan Irvine, CA) or Xiidra® (lifitegrast, Shire, Lexington, MA). Patients currently using Restasis® or Xiidra® for conditions other than DED (e.g., allergies);
• Previous intraocular or ocular laser surgery within the past 3 months or any refractive surgery procedure within the past 6 months of the screening visit in either eye;
• Any relevant ocular anomaly interfering with the ocular surface, including active ocular herpes simplex infection, recurrent corneal erosion, symptomatic epithelial basement membrane dystrophy, mucus fishing syndrome, giant papillary conjunctivitis, post-radiation keratitis, Stevens-Johnson syndrome, corneal ulcer, abnormalities of the nasolachrymal drainage system, chemical injury, destruction of the conjunctival goblet cells or scaring, diagnosed significant anterior blepharitis and/or progressive pterygium, or any other additional condition(s) associated with or causing dry eye;
• Presence or history of any ocular disorder or condition, including ocular surgery (including palpebral and cataract surgery, trauma), infection (viral, bacterial, fungal), disease or inflammation not associated with dry eye unless disorder or disease is:
• Stable for at least 3 months before the Screening Visit; and
• As determined by the Investigator not likely to impact or possibly interfere with the interpretation of study results.
• History of ocular allergy (including seasonal conjunctivitis) or chronic conjunctivitis other than dry eye;
• Known hypersensitivity to one of the components of the study or procedural medications (e.g., fluorescein, lissamine green, Refresh Plus®);
• Inability to refrain from contact lens wear during the study, including the 2 week Run-in period;
• Anticipated temporary or permanent need for punctal plugs during the study, except if punctal plugs have been in place for at least 2 weeks prior to Screening, in which case the plugs are allowed to remain in place during the study; if plugs should fall out during the course of the study, the instance will be recorded and the plug(s) can be replaced;
• Ocular or clinically significant systemic disease (e.g., diabetes with glycemia out of range, thyroid malfunction, uncontrolled autoimmune disease) or condition(s) not stabilized within 1 month (30 days) before Screening or a condition judged by the Investigator to be incompatible or interferes with the study results;
• Inability or unwillingness to discontinue use of autologous or platelet rich plasma eye drops during the 2 week washout period and the duration of the study;
• Anticipated change in the use or dose of Restasis®, Xiidra®, or artificial tears within 14 days before Screening or during the study. If currently taking Restasis®, Xiidra®, or artificial tears, treatment(s) for DED can continue throughout the study without a change in dose. If currently using Restasis® or Xiidra® for conditions other than DED, then patient will be excluded from study. If not currently taking Restasis®, Xiidra® or artificial tears, these drugs cannot begin during the study;
• Anticipated change in the patient's use of or dosage of systemic medications that could affect tear function (e.g., antihistamines, tricyclic antidepressants, anxiolytics, antimuscarinics, beta-blocking agents, phenothiazines, tacrolimus, sirolimus, etc.) during the study. If currently taking any of these drugs, treatment(s) can continue throughout the study without a change in dose;

Sponsors & Collaborators

• Cambium Bio Limited: lead
• Massachusetts Eye and Ear Infirmary: collaborator
• Oregon Health and Science University: collaborator
• University of Michigan: collaborator
• Duke University: collaborator
• Stanford University: collaborator
• University of Minnesota: collaborator

Study Sites (6)

Byers Eye Institute of Stanford University

Palo Alto, California, 94303, United States

Location

Massachusetts Eye and Ear Infirmary

Boston, Massachusetts, 02114, United States

Location

University of Michigan - Kellogg Clinical Research Center

Ann Arbor, Michigan, 48105, United States

Location

University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

Duke University Eye Center

Durham, North Carolina, 27710, United States

Location

Oregon Health Sciences University - Casey Eye Institute

Portland, Oregon, 97239, United States

Location

MeSH Terms

Conditions

Dry Eye Syndromes; Graft vs Host Disease

Condition Hierarchy (Ancestors)

Lacrimal Apparatus Diseases; Eye Diseases; Immune System Diseases

Study Officials

• Edmund K Waller, MD PhD

  Cambium Bio Limited

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 8, 2018
• First Posted: January 30, 2018
• Study Start: May 1, 2018
• Primary Completion: December 12, 2019
• Study Completion: February 20, 2020
• Last Updated: July 27, 2021

Record last verified: 2021-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (6)