Back to resultsCC-11050 Trial in Nepalese Patients With Erythema Nodosum Leprosum
Primary Purpose
Erythema Nodosum Leprosum, Leprosy
Status
Recruiting
Phase
Phase 2
Locations
Nepal
Study Type
Interventional
Intervention
CC-11050
Sponsored by
About this trial
This is an interventional treatment trial for Erythema Nodosum Leprosum
Eligibility Criteria
Inclusion Criteria:
Study participants must satisfy the following criteria to be enrolled in the study:
- Must be 18 -65 years old, weight > 35kg for women and >40kg for men at the time of signing the informed consent.
- Understand and voluntarily sign an informed consent document prior to any study related assessments/procedures are conducted.
- Have signs or symptoms of new or new episode ENL. In Step one, participants must be male. In Step 2, participants can be either male or female.
- Able to adhere to the study schedule and other protocol requirements.
Exclusion Criteria:
The presence of any of the following will exclude a participant from enrollment:
- Any significant medical condition, laboratory abnormality, or psychiatric illness that would prevent the participants from participating in the study.
- Patients on continuous rifampicin will be excluded, however, If rifampicin is given only once a month as part of a multi-drug regimen, a minimum of 2-week washout period is required prior to administration of CC-11050
- Any condition including the presence of laboratory abnormalities, which places the participant at unacceptable risk if he were to participate in the study.
- Any condition that confounds the ability to interpret data from the study (ie, HIV, chronic HepB, chronic HepC or TB patients under active treatment). Resolved TB patients will not be excluded from the study.
- Stable and well managed patients with Diabetes and hypertension will not be excluded from the study.
- Use of systemic corticosteroid or immunosuppressant therapy within 7 days of study medication initiation
- Pregnant or nursing females.
Sites / Locations
- The Leprosy Mission Nepal
- Anandaban HospitalRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
CC-11050 treatment
Arm Description
200mg CC-11050 will be administered twice daily as a pill taken with food (at breakfast and evening meal) for participants with moderate to severe ENL for 10 days, then up to 28 days (during Step 1) and then up to 1 year (during Step 2).
Outcomes
Primary Outcome Measures
Number of participants with moderate to severe ENL with severity changes as assessed by the ENL Severity Scale.
Changes in ENL Severity Scale Score (Walker et al., PLoS Negl Trop Dis 11(7): e0005716, https://doi.org/10.1371/journal.pntd.0005716) as compared to baseline and any previous time points. Ten clinical categories contribute 0-3 points toward the total score of 0-30. An ENL patient with a total scale score of less than 9 is considered to have mild ENL, while scores >9 are considered moderate to severe ENL. A minimum score of 9 is required to indicate sufficient ENL symptoms from which post-intervention reduction in score(s) can be determined meaningful improvement.
Secondary Outcome Measures
Number of participants with moderate to severe ENL with decreased duration of episode(s) as assessed by standard ENL episode definition.
Changes in ENL episode duration(s) as compared to baseline and any previous time points. By definition, if ENL symptoms are separated by time and no need of treatment for >28 days, they are considered separate episodes. Recurrence is anytime another episode occurs after >28 days of no prior treatment or symptoms.
Number of participants with moderate to severe ENL with decreased bacterial index (BI) as assessed by slit skin smear microscopy.
Changes in slit skin smear microscopy BI as compared to baseline. BI can range from 0-6.
Number of participants with moderate to severe ENL with decreased bacterial index (BI) as assessed skin histopathology.
Changes in skin histopathology bacterial index (BI) as compared to baseline. BI can range from 0-6.
Number of patients with moderate to severe ENL with improved peripheral neuropathy as assessed by standardized monofilament and voluntary muscle tests.
Changes in scores for peripheral neuropathy as indicated by monofilament and voluntary muscle tests compared from baseline and any previous time points. To check neuropathy, 7 motor function nerves and 6 sensory function nerves on each side of the body are assessed for a total 26 nerves. Each are assessed on 3 sites using a standard set of Semmes-Weinstein monofilaments. The 200mg filament and the 2g filament represent threshold for the hand and foot, respectively. The 0-5 Medical Research Council (MRC) scale is used for motor function assessment. Sensory scores >3 and motor nerve scoring (MRC) < 5 are regarded impaired (indicating neuropathy). More method specifics can be found here: van Veen N.H., et al., Corticosteroids for treating nerve damage in leprosy. Cochrane Database Syst Rev, 2007(2): p. CD005491.
Number of participants with moderate to severe ENL with improved quality of life as assessed by the World Health Organization Quality of Life tool.
Changes in scores for the World Health Organization Quality of Life assessment as compared to baseline and any previous time points. There are 26 questions in this questionnaire and each can be scored from 1 to 5 points. The highest possible score is 130 and lowest possible score is 26. A higher score denotes better quality of life.
Number of participants with moderate to severe ENL as assessed by standard blood panel and basic metabolic testing.
Abnormalities or change in values in comparison to baseline and any previous time points.
Number of participants with moderate to severe ENL as assessed by urinalysis.
Detection of albumin (qualitative) as compared to baseline and any previous time points. Urine albumin is related to ENL severity and kidney function test.
Number of participants with moderate to severe ENL as assessed by serological indicators associated with ENL.
Detection of rheumatoid arthritis factor (positive) and/or C-reactive protein (positive) as compared to baseline and any previous time points.
Full Information
NCT ID
NCT03807362
First Posted
September 14, 2018
Last Updated
July 6, 2020
Sponsor
The Leprosy Mission Nepal
1. Study Identification
Unique Protocol Identification Number
NCT03807362
Brief Title
CC-11050 Trial in Nepalese Patients With Erythema Nodosum Leprosum
Official Title
A Single Center, Open Label Pilot Study to Evaluate the Safety and Efficacy of CC-11050 in Nepalese Patients With Erythema Nodosum Leprosum
Study Type
Interventional
2. Study Status
Record Verification Date
July 2020
Overall Recruitment Status
Recruiting
Study Start Date
January 7, 2018 (Actual)
Primary Completion Date
March 30, 2021 (Anticipated)
Study Completion Date
December 31, 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
The Leprosy Mission Nepal
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
This study will be a single center, Phase 2, open-label trial to evaluate the safety and efficacy of 200mg CC-11050 administered twice daily taken with food for patients with moderate to severe ENL. The study will be performed in two steps: 1) to evaluate immediate effect in safety and efficacy of drug in 10 males with new or new recurrent episode ENL and, if found to be safe and effective by the DSMB and 2) if allowed by the DSMB, and approved by relevant study stakeholders, an additional 40 ENL patients will be enrolled for up to 52 weeks of treatment. A safety analysis will be conducted on all patients who have received at least one dose of study drug, and will include the frequency of all adverse events and laboratory abnormalities as well as frequency of dose interruptions, dose reductions and treatment discontinuation.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Erythema Nodosum Leprosum, Leprosy
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Model Description
A single center, open label pilot study
Masking
None (Open Label)
Allocation
N/A
Enrollment
50 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
CC-11050 treatment
Arm Type
Experimental
Arm Description
200mg CC-11050 will be administered twice daily as a pill taken with food (at breakfast and evening meal) for participants with moderate to severe ENL for 10 days, then up to 28 days (during Step 1) and then up to 1 year (during Step 2).
Intervention Type
Drug
Intervention Name(s)
CC-11050
Intervention Description
CC-11050 is a novel anti-inflammatory compound with potential to treat a variety of chronic inflammatory conditions.
Primary Outcome Measure Information:
Title
Number of participants with moderate to severe ENL with severity changes as assessed by the ENL Severity Scale.
Description
Changes in ENL Severity Scale Score (Walker et al., PLoS Negl Trop Dis 11(7): e0005716, https://doi.org/10.1371/journal.pntd.0005716) as compared to baseline and any previous time points. Ten clinical categories contribute 0-3 points toward the total score of 0-30. An ENL patient with a total scale score of less than 9 is considered to have mild ENL, while scores >9 are considered moderate to severe ENL. A minimum score of 9 is required to indicate sufficient ENL symptoms from which post-intervention reduction in score(s) can be determined meaningful improvement.
Time Frame
10 days, 28 days and monthly up to 1 year of treatment
Secondary Outcome Measure Information:
Title
Number of participants with moderate to severe ENL with decreased duration of episode(s) as assessed by standard ENL episode definition.
Description
Changes in ENL episode duration(s) as compared to baseline and any previous time points. By definition, if ENL symptoms are separated by time and no need of treatment for >28 days, they are considered separate episodes. Recurrence is anytime another episode occurs after >28 days of no prior treatment or symptoms.
Time Frame
10 days, 28 days, monthly up to 1 year of treatment and up to 1 year followup after treatment cessation.
Title
Number of participants with moderate to severe ENL with decreased bacterial index (BI) as assessed by slit skin smear microscopy.
Description
Changes in slit skin smear microscopy BI as compared to baseline. BI can range from 0-6.
Time Frame
Enrolment and after 1 year treatment.
Title
Number of participants with moderate to severe ENL with decreased bacterial index (BI) as assessed skin histopathology.
Description
Changes in skin histopathology bacterial index (BI) as compared to baseline. BI can range from 0-6.
Time Frame
Enrolment and after 1 year treatment.
Title
Number of patients with moderate to severe ENL with improved peripheral neuropathy as assessed by standardized monofilament and voluntary muscle tests.
Description
Changes in scores for peripheral neuropathy as indicated by monofilament and voluntary muscle tests compared from baseline and any previous time points. To check neuropathy, 7 motor function nerves and 6 sensory function nerves on each side of the body are assessed for a total 26 nerves. Each are assessed on 3 sites using a standard set of Semmes-Weinstein monofilaments. The 200mg filament and the 2g filament represent threshold for the hand and foot, respectively. The 0-5 Medical Research Council (MRC) scale is used for motor function assessment. Sensory scores >3 and motor nerve scoring (MRC) < 5 are regarded impaired (indicating neuropathy). More method specifics can be found here: van Veen N.H., et al., Corticosteroids for treating nerve damage in leprosy. Cochrane Database Syst Rev, 2007(2): p. CD005491.
Time Frame
enrolment, 10 days, 28 days, biweekly (as needed), up to 1 year treatment, up to 1 year followup after treatment cessation
Title
Number of participants with moderate to severe ENL with improved quality of life as assessed by the World Health Organization Quality of Life tool.
Description
Changes in scores for the World Health Organization Quality of Life assessment as compared to baseline and any previous time points. There are 26 questions in this questionnaire and each can be scored from 1 to 5 points. The highest possible score is 130 and lowest possible score is 26. A higher score denotes better quality of life.
Time Frame
Enrolment, 28 days, monthly up to 1 year of treatment and up to 1 year followup after treatment cessation.
Title
Number of participants with moderate to severe ENL as assessed by standard blood panel and basic metabolic testing.
Description
Abnormalities or change in values in comparison to baseline and any previous time points.
Time Frame
Enrolment, day 10, day 17, day 28, monthly up to 1 year of treatment, and 1 month post-cessation of treatment.
Title
Number of participants with moderate to severe ENL as assessed by urinalysis.
Description
Detection of albumin (qualitative) as compared to baseline and any previous time points. Urine albumin is related to ENL severity and kidney function test.
Time Frame
Enrolment, day 10, day 17, day 28, monthly up to 1 year of treatment, and 1 month post-cessation of treatment.
Title
Number of participants with moderate to severe ENL as assessed by serological indicators associated with ENL.
Description
Detection of rheumatoid arthritis factor (positive) and/or C-reactive protein (positive) as compared to baseline and any previous time points.
Time Frame
Enrolment, day 10, day 17, day 28, monthly up to 1 year of treatment, and 1 month post-cessation of treatment.
10. Eligibility
Sex
All
Gender Based
Yes
Gender Eligibility Description
Males only for Step 1 (first 10 patients for testing up to 28 days of treatment). If successful, then both males and females can enroll in the second part of the study (up to 40 patients testing up to 1 year of therapy).
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Study participants must satisfy the following criteria to be enrolled in the study:
Must be 18 -65 years old, weight > 35kg for women and >40kg for men at the time of signing the informed consent.
Understand and voluntarily sign an informed consent document prior to any study related assessments/procedures are conducted.
Have signs or symptoms of new or new episode ENL. In Step one, participants must be male. In Step 2, participants can be either male or female.
Able to adhere to the study schedule and other protocol requirements.
Exclusion Criteria:
The presence of any of the following will exclude a participant from enrollment:
Any significant medical condition, laboratory abnormality, or psychiatric illness that would prevent the participants from participating in the study.
Patients on continuous rifampicin will be excluded, however, If rifampicin is given only once a month as part of a multi-drug regimen, a minimum of 2-week washout period is required prior to administration of CC-11050
Any condition including the presence of laboratory abnormalities, which places the participant at unacceptable risk if he were to participate in the study.
Any condition that confounds the ability to interpret data from the study (ie, HIV, chronic HepB, chronic HepC or TB patients under active treatment). Resolved TB patients will not be excluded from the study.
Stable and well managed patients with Diabetes and hypertension will not be excluded from the study.
Use of systemic corticosteroid or immunosuppressant therapy within 7 days of study medication initiation
Pregnant or nursing females.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Mahesh Shah, MD
Phone
+9779841203417
Email
drmahesh_shah@yahoo.com
First Name & Middle Initial & Last Name or Official Title & Degree
Indra B Napit, MD
Email
indranapit@yahoo.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Mahesh Shah, MD
Organizational Affiliation
The Leprosy Mission Nepal
Official's Role
Principal Investigator
Facility Information:
Facility Name
The Leprosy Mission Nepal
City
Kathmandu
State/Province
Bagmati
ZIP/Postal Code
44700
Country
Nepal
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Mahesh Shah, MD
Email
drmahesh_shah@yahoo.com
First Name & Middle Initial & Last Name & Degree
iIndra B Napit, MD
Email
indran@tlmnepal.org
Facility Name
Anandaban Hospital
City
Lalitpur
State/Province
Bagmati
ZIP/Postal Code
44700
Country
Nepal
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Indra B Napit, MD
Phone
+9779851136027
Email
indran@tlmnepal.org
First Name & Middle Initial & Last Name & Degree
Mahesh Shah, MD
Phone
+9779841203417
Email
drmahesh_shah@yahoo.com
First Name & Middle Initial & Last Name & Degree
Deanna A Hagge, PhD
12. IPD Sharing Statement
Plan to Share IPD
Undecided
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CC-11050 Trial in Nepalese Patients With Erythema Nodosum Leprosum
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