Recurrent Respiratory PapillomatosisPhase 1NCT02854761
Safety Study of EF-022 in Adults With Recurrent Respiratory Papillomatosis (RRP)
A Phase 1 study testing EF-022 (Modified Vitamin D Binding Protein Macrophage Activator) in people with Recurrent Respiratory Papillomatosis, run by Efranat Ltd..
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Recurrent Respiratory Papillomatosis
- What is being tested?
- EF-022 (Modified Vitamin D Binding Protein Macrophage Activator)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- No sites listed yet
- Study dates
- Sep 2016to Sep 2018
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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
- 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.
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- 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
Who cannot join
- 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
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- 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.
Trial locations and contacts
No sites are listed yet.
About this study
Official title: Phase I Safety and Tolerability Study of EF-022 in Adult Subjects With Recurrent Respiratory Papillomatosis
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.
Read the 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
- Conditions
- Recurrent Respiratory Papillomatosis
- Being tested
- EF-022 (Modified Vitamin D Binding Protein Macrophage Activator)
- Medical subject headings
- Recurrent respiratory papillomatosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EF-022 (Modified Vitamin D Binding Protein Macrophage Activator)Drug
| Study group | Role | What they receive |
|---|---|---|
| SC administration | Experimental | Subcutaneous (SC) administration of 500 ng of EF-022 (Modified Vitamin D Binding Protein Macrophage Activator) once weekly, for 6 months Receives: Drug: EF-022 (Modified Vitamin D Binding Protein Macrophage Activator) |
| IM administration | Experimental | Intramuscular (IM) adminstration of 500 ng EF-022 (Modified Vitamin D Binding Protein Macrophage Activator) once weekly for 6 months Receives: Drug: EF-022 (Modified Vitamin D Binding Protein Macrophage Activator) |
What is being measured
The primary outcome is the main question the study is designed to answer.
CTCAE v.4.03 criteria will constitute the primary safety endpoints to be monitored throughout this study
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Aug 2016 (estimated)
- Study startSep 2016
- Primary completionSep 2018 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Recurrent Respiratory PapillomatosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Efranat Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02854761
- Sponsor's protocol ID
- CS-102
- Status last verified
- Aug 2016
- First submitted
- 1 Aug 2016
- Last updated
- 3 Aug 2016
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Aug 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Safety and Tolerability Study of EF-022 in Adult Subjects With Recurrent Respiratory Papillomatosis
- ClinicalTrials.gov ID
- NCT02854761
- Acronym / sponsor ID
- CS-102
- Conditions
- Recurrent Respiratory Papillomatosis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- Efranat Ltd.Industry (pharma, biotech, devices)
- Study start
- Sep 2016
- Primary completion
- Sep 2018 (estimated)
- First posted
- 3 Aug 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| SC administration | Experimental | Drug: EF-022 (Modified Vitamin D Binding Protein Macrophage Activator)Subcutaneous (SC) administration of 500 ng of EF-022 (Modified Vitamin D Binding Protein Macrophage Activator) once weekly, for 6 months |
| IM administration | Experimental | Drug: EF-022 (Modified Vitamin D Binding Protein Macrophage Activator)Intramuscular (IM) adminstration of 500 ng EF-022 (Modified Vitamin D Binding Protein Macrophage Activator) once weekly for 6 months |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- 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
Key exclusion criteria (16 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EF-022 (Modified Vitamin D Binding Protein Macrophage Activator) | Drug | IM administration, SC administration |
Primary outcome
CTCAE v.4.03 criteria will constitute the primary safety endpoints to be monitored throughout this study
Time frame: 6 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Aug 2016.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing EF-022 (Modified Vitamin D Binding Protein Macrophage Activator) in people with Recurrent Respiratory Papillomatosis, run by Efranat Ltd..
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 11 inclusion and 16 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: status not confirmed, as of the sponsor's last update on 3 Aug 2016. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this 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.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Myriam Golembo, PhD (972-8-9724972, myriam.golembo@efranat.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
CTCAE v.4.03 criteria will constitute the primary safety endpoints to be monitored throughout this study (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 3 Aug 2016.