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Safety Study of EF-022 in Adults With Recurrent Respiratory Papillomatosis (RRP)

Primary Purpose

Recurrent Respiratory Papillomatosis

Status

Unknown status

Phase

Phase 1

Locations

Study Type

Interventional

Intervention

EF-022 (Modified Vitamin D Binding Protein Macrophage Activator)

About this trial

This is an interventional treatment trial for Recurrent Respiratory Papillomatosis

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Patients with documented diagnosis of recurrent respiratory papillomatosis
Patients with measurable disease
Patients presenting at least one of the following severity criteria: Derkay score ≥ 8 , Voice handicap index (VHI) ≥11
Adult male and female subjects, age of 18 and above (≥18 yrs)
Patients with documentation on number of debulking procedures done during past 12 months
Estimated expectancy time for next debulking procedure must be at least 3 months.
Must be at least 4 weeks (>4 weeks) since last treatment including cytotoxic therapy, biological or immunotherapy, anti-viral therapy, molecularly targeted therapy or steroid treatment.
Must be at least 2 weeks (>2weeks) since last NSAID treatment.
Must have adequately recovered (to at least Grade 1 level) from adverse effects of prior therapies.
Female of child bearing potential must have a negative pregnancy test and must be practicing an effective method of birth control
Patients or their guardians must sign an informed consent indicating that they are aware of the investigational nature of this study

Exclusion Criteria:

Patient on concurrent steroids or anti-inflammatory non steroid treatment.
Active autoimmune disease
Known major immunodeficiency
Known to be Positive for HIV, Hepatitis B surface antigen, Hepatitis C antibody
Have absolute neutrophil counts below 1.5X 10^9/L
Hemoglobin below 10.0 g/dL
White blood cell counts below 3.5X10^9/L.
Granulocytes below 1.5X10^9/L.
Have Platelets below 100 X 10^9/L
Abnormal renal function with creatinine above 1.5X the upper limit of normal (ULN)
Abnormal liver function tests with bilirubin above 1.5X the ULN, AST and ALT above 2.5 X ULN.
Patients with active cardiovascular disease under continuous treatment
Patients with associated malignancy currently receiving chemotherapy and/or radiation.
Female adult patients that are pregnant, nursing or plan to nurse during and up to 12 months of treatment period.
Subjects receiving another investigational drug.
Patients with concurrent or history of malignancy within 5 years.

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Arm Label

SC administration

IM administration

Arm Description

Subcutaneous (SC) administration of 500 ng of EF-022 (Modified Vitamin D Binding Protein Macrophage Activator) once weekly, for 6 months

Intramuscular (IM) adminstration of 500 ng EF-022 (Modified Vitamin D Binding Protein Macrophage Activator) once weekly for 6 months

Outcomes

Primary Outcome Measures

CTCAE v.4.03 criteria will constitute the primary safety endpoints to be monitored throughout this study

Secondary Outcome Measures

Full Information

NCT ID

NCT02854761

First Posted

August 1, 2016

Last Updated

August 2, 2016

Sponsor

Efranat Ltd.

1. Study Identification

Unique Protocol Identification Number

NCT02854761

Brief Title

Safety Study of EF-022 in Adults With Recurrent Respiratory Papillomatosis (RRP)

Official Title

Phase I Safety and Tolerability Study of EF-022 in Adult Subjects With Recurrent Respiratory Papillomatosis

Study Type

Interventional

2. Study Status

Record Verification Date

August 2016

Overall Recruitment Status

Unknown status

Study Start Date

September 2016 (undefined)

Primary Completion Date

September 2018 (Anticipated)

Study Completion Date

undefined (undefined)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Efranat Ltd.

4. Oversight

5. Study Description

Brief Summary

This study evaluates the safety and tolerability of the investigational drug EF-022 in the treatment of adult patients with Recurrent Respiratory Papillomatosis (RRP). Patients will be administered EF-022, either intramuscular or subcutaneous, for a period of 6 months. Preliminary effect of the drug on the disease will be evaluated by following the number and severity of the lesions in the respiratory tract and the effect on voice changes.

Detailed Description

The etiology of RRP is associated with local immune suppression at disease-involved sites. EF-022, a Vitamin D-Binding protein Macrophage Activating Factor, modulates the innate arm of the immune system to alleviate immune suppression. The investigators hypothesize that EF-022 may modulate the immune response in a manner that might slow down or even prevent disease progression. Adult subjects will be treated weekly with EF-022 for a period of 6 months by either intramuscular or subcutaneous administration. The primary objective of the study is to evaluate the safety and tolerability of EF-022 administered to adult subjects with recurrent respiratory papillomatosis either IM or SC. The main response assessment will be performed over the 6 months period by: Evaluating lesion size and/or number of disease-involved sites by Coltera-Derkay score Assessment of degree of voice disorder using the VHI-10 score Symptomatic assessment of dyspnea/stridor

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Recurrent Respiratory Papillomatosis

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

6 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

SC administration

Arm Type

Experimental

Arm Description

Subcutaneous (SC) administration of 500 ng of EF-022 (Modified Vitamin D Binding Protein Macrophage Activator) once weekly, for 6 months

Arm Title

IM administration

Arm Type

Experimental

Arm Description

Intramuscular (IM) adminstration of 500 ng EF-022 (Modified Vitamin D Binding Protein Macrophage Activator) once weekly for 6 months

Intervention Type

Drug

Intervention Name(s)

EF-022 (Modified Vitamin D Binding Protein Macrophage Activator)

Primary Outcome Measure Information:

Title

CTCAE v.4.03 criteria will constitute the primary safety endpoints to be monitored throughout this study

Time Frame

6 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Patients with documented diagnosis of recurrent respiratory papillomatosis Patients with measurable disease Patients presenting at least one of the following severity criteria: Derkay score ≥ 8 , Voice handicap index (VHI) ≥11 Adult male and female subjects, age of 18 and above (≥18 yrs) Patients with documentation on number of debulking procedures done during past 12 months Estimated expectancy time for next debulking procedure must be at least 3 months. Must be at least 4 weeks (>4 weeks) since last treatment including cytotoxic therapy, biological or immunotherapy, anti-viral therapy, molecularly targeted therapy or steroid treatment. Must be at least 2 weeks (>2weeks) since last NSAID treatment. Must have adequately recovered (to at least Grade 1 level) from adverse effects of prior therapies. Female of child bearing potential must have a negative pregnancy test and must be practicing an effective method of birth control Patients or their guardians must sign an informed consent indicating that they are aware of the investigational nature of this study Exclusion Criteria: Patient on concurrent steroids or anti-inflammatory non steroid treatment. Active autoimmune disease Known major immunodeficiency Known to be Positive for HIV, Hepatitis B surface antigen, Hepatitis C antibody Have absolute neutrophil counts below 1.5X 10^9/L Hemoglobin below 10.0 g/dL White blood cell counts below 3.5X10^9/L. Granulocytes below 1.5X10^9/L. Have Platelets below 100 X 10^9/L Abnormal renal function with creatinine above 1.5X the upper limit of normal (ULN) Abnormal liver function tests with bilirubin above 1.5X the ULN, AST and ALT above 2.5 X ULN. Patients with active cardiovascular disease under continuous treatment Patients with associated malignancy currently receiving chemotherapy and/or radiation. Female adult patients that are pregnant, nursing or plan to nurse during and up to 12 months of treatment period. Subjects receiving another investigational drug. Patients with concurrent or history of malignancy within 5 years.

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Myriam Golembo, PhD

Phone

972-8-9724972

myriam.golembo@efranat.com

First Name & Middle Initial & Last Name or Official Title & Degree

Uri Yogev

Phone

972-8-9724972

uri.yogev@efranat.com

12. IPD Sharing Statement

Learn more about this trial

Safety Study of EF-022 in Adults With Recurrent Respiratory Papillomatosis (RRP)

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