| CTRI Number |
CTRI/2025/06/089293 [Registered on: 23/06/2025] Trial Registered Prospectively |
| Last Modified On: |
14/06/2025 |
| Post Graduate Thesis |
No |
| Type of Trial |
Interventional |
|
Type of Study
|
Drug |
| Study Design |
Randomized, Parallel Group Trial |
|
Public Title of Study
|
A study to assess the efficacy and safety of Diacerein ointment in patients with bullous skin disease (EBS) |
|
Scientific Title of Study
|
An International, Multicenter, Randomized, Double-Blind, Parallel Group, Vehicle-Controlled, Phase 2/3 Study with Open-Label Extension Evaluating the Efficacy and Safety of Diacerein 1 percent Ointment for the Treatment of Generalized Epidermolysis Bullosa Simplex (EBS) [EBShield Study] |
| Trial Acronym |
NIL |
|
Secondary IDs if Any
|
| Secondary ID |
Identifier |
| AC-203-EBS-007 V 3 dated 16 Jun 2024 |
Protocol Number |
|
|
Details of Principal Investigator or overall Trial Coordinator (multi-center study)
|
| Name |
Dr Rakesh Patel |
| Designation |
Head - Clinical Operations |
| Affiliation |
Veeda Clinical research Limited |
| Address |
Veeda Clinical Research Ltd., Shivalik Plaza, Near I.I.M.,Ambawadi, Ahmadabad
Ahmadabad
GUJARAT
380015
India |
| Phone |
8308843660 |
| Fax |
|
| Email |
Rakesh.Patel@veedalifesciences.com |
|
Details of Contact Person
Scientific Query
|
| Name |
Dr Ravi Alamchandani |
| Designation |
General Manager |
| Affiliation |
Veeda Clinical research Limited |
| Address |
Veeda Clinical Research Ltd., Shivalik Plaza, Near I.I.M.,Ambawadi, Ahmadabad
Ahmadabad
GUJARAT
380015
India |
| Phone |
9687306158 |
| Fax |
|
| Email |
Ravi.A1950@veedalifesciences.com |
|
Details of Contact Person
Public Query
|
| Name |
Dr Ravi Alamchandani |
| Designation |
General Manager |
| Affiliation |
Veeda Clinical research Limited |
| Address |
Veeda Clinical Research Ltd., Shivalik Plaza, Near I.I.M.,Ambawadi, Ahmadabad
GUJARAT
380015
India |
| Phone |
9687306158 |
| Fax |
|
| Email |
Ravi.A1950@veedalifesciences.com |
|
|
Source of Monetary or Material Support
|
| TWi Biotechnology, Inc.
8F, No. 41, Lane 221, Gangqian Rd.,
Nei hu Dist. Taipei 114723,
Taiwan
|
|
|
Primary Sponsor
|
| Name |
TWi Biotechnology, Inc. |
| Address |
8F, No. 41, Lane 221, Gangqian Rd.,
Nei hu Dist. Taipei 114723,
Taiwan
|
| Type of Sponsor |
Pharmaceutical industry-Global |
|
|
Details of Secondary Sponsor
|
| Name |
Address |
| Veeda Clinical Research Ltd |
Veeda Clinical Research Ltd.,
Shivalik Plaza, Near I.I.M., Ambawadi Ahmedabad –380015,India |
|
|
Countries of Recruitment
|
India
Australia
Austria
Belgium
France
Greece
Ireland
Israel
Italy
Malaysia
Philippines
Poland
Republic of Korea
Spain
Taiwan
United Arab Emirates
United Kingdom
United States of America |
|
Sites of Study
|
| No of Sites = 2 |
| Name of Principal
Investigator |
Name of Site |
Site Address |
Phone/Fax/Email |
| Dr Rahul Mahajan |
Post Graduate Institute of Medical Education and Research |
Department of Dermatology, Venereology and Leprology, Post Graduate Institute of Medical Education and Research (PGIMER), Sector-12, UT, Chandigarh-160012, India
Chandigarh
CHANDIGARH |
9878920348
drrahulpgi@yahoo.com |
| Dr Manoj Srinivasa |
Sri Sathya Sai Sarla Memorial Hospital |
Department of Clinical Research, Sathya Sai Grama, Muddenahalli, Chikkaballapur Taluk, Post - 562101
Kolar
KARNATAKA |
08156275801
drmanojsrinivas@gmail.com |
|
|
Details of Ethics Committee
|
| No of Ethics Committees= 2 |
| Name of Committee |
Approval Status |
| Ethics Committee,MS Ramaiah Medical College n Hosp |
Submittted/Under Review |
| Institutional Ethics Committee Post Graduate Institute of Medical Education and Research |
Approved |
|
|
Regulatory Clearance Status from DCGI
|
|
|
Health Condition / Problems Studied
|
| Health Type |
Condition |
| Patients |
(1) ICD-10 Condition: Q810||Epidermolysis bullosa simplex, |
|
|
Intervention / Comparator Agent
|
| Type |
Name |
Details |
| Intervention |
AC-203 diacerein 1 percent ointment |
Dose Formulation : Ointment
Dose Strength : 1 percent
Route of Administration :Topical
Dosage Level : To be applied topically once daily to all EBS lesions and at least approximately 2 cm of the surrounding uninvolved skin for a treatment Period of 8 weeks. |
| Comparator Agent |
Vehicle Ointment |
Dose Formulation : Ointment Dose Strength : NA Route of Administration :Topical
Dosage Level : To be applied topically once daily to all EBS lesions and at least approximately 2 cm of the surrounding uninvolved skin for a treatment Period of 8 weeks. |
|
|
Inclusion Criteria
|
| Age From |
6.00 Month(s) |
| Age To |
99.00 Year(s) |
| Gender |
Both |
| Details |
1) Patient is at least 6 months of age at Visit 2 that is Day 1 or Baseline A.
2) Patient has been clinically diagnosed with severe EBS or intermediate EBS, confirmed by documented genetic diagnosis to have autosomal dominant mutations in KRT5 or KRT14 gene.
3) Patient with more than or equal to 5 percent BSA of EBS lesions excluding palms and soles at Visit 2.
4) EBS lesions of patient within the treatment area have an IGA score of more than or equal to 3 at Visit 2.
5) Patient or caregiver agrees to follow study medication application instructions.
6) Patient and caregiver or legal guardian agrees to report use of all prescription and over-the-counter medications, including topical therapies applied to the body.
7) Patient and caregiver or legal guardian is willing and able to comply with all study visits and all the protocol requirements, including completing questionnaires.
8) Patient and caregiver or legal guardian is able to provide written informed consent or assent based on age.
9) Female patient of childbearing potential must have a negative pregnancy test prior to randomization.
10) Female patient of childbearing potential is willing to practice highly effective contraception from screening throughout the end of the study. |
|
| ExclusionCriteria |
| Details |
1) Patient has a clinically significant skin disease other than EBS or a vascular disorder associated with cutaneous erosions or ulcerations, that may confound assessments of efficacy or safety.
2) Patient has a clinically significant underlying medical condition, psychiatric condition or requires concomitant medication that, based on the Investigator’s judgment, may impair evaluation of the Treatment Area or exposes the patient to an unacceptable risk by study participation.
3) Patient has used any diacerein-containing product within 6 months prior to Visit 2.
4) Patient has had a cutaneous infection in the Treatment Area or use systemic antibiotics within 7 days prior to Visit 2.
5) Patient has uncontrolled diabetes mellitus , hepatic enzyme abnormalities or renal abnormalities during the Screening period.
6) Patient has a current malignancy or a history of treatment for a malignancy within 5 years with the exception of treated non-melanoma cutaneous malignancies prior to Visit 2.
7) Patient is treated with protocol-excluded topical therapies other than steroids within 2 weeks prior to Visit 2 that might influence the assessment of the Treatment Area throughout the study period.
8) Patient has been treated with topical steroids on the EBS lesions within 2 weeks or systemic steroids within 4 weeks. prior to Visit 2.
9) Patient has been treated with: (a) an approved biologic anti-inflammatory therapy (such as monoclonal antibodies that target to modulate the immune responses) and (b) other immunosuppressive or immunomodulatory therapies or chemotherapy within 8 weeks prior to Visit 2.
10) Patient has been treated with any investigational drug or device within 30 days or 5 half-lives, whichever is longer, prior to Visit 2.
11) Patient has a history of allergy or hypersensitivity to any component of study medications, including diacerein or rhein.
12) Patient is pregnant or breastfeeding/lactating.
13) Patient has a planned or anticipated major surgical procedure or other activity that would interfere with their ability to comply with protocol requirements. |
|
|
Method of Generating Random Sequence
|
Computer generated randomization |
|
Method of Concealment
|
Centralized |
|
Blinding/Masking
|
Participant, Investigator, Outcome Assessor and Date-entry Operator Blinded |
|
Primary Outcome
|
| Outcome |
TimePoints |
| To evaluate the efficacy of study drug after 8 weeks of treatment in adult and pediatric patients (aged 6 months and older) with generalized EBS. |
Proportion of patients achieving treatment success on the IGA of the Treatment Area, in which treatment success is defined as a score of 0 or 1 with at least a 2-point reduction from Baseline A (Visit 2 that is Day 1) to Week 8 (Visit 5 that is EOT). |
|
|
Secondary Outcome
|
| Outcome |
TimePoints |
| To evaluate the safety & efficacy of study drug in adult & pediatric patients (aged 6 months & older) after 8 weeks of treatment with generalized EBS |
Efficacy endpoints: Change in percentage BSA of EBS lesions in the Treatment Area, pain intensity, pruritus intensity, EBDASI & QOLEB score from Baseline A Visit 2,3,4 & 5-EOT.
Safety endpoints: Incidence & proportion of patients with AEs, SAEs & relationship to the study medication & change in clinical laboratory results in hematology, biochemistry, urinalysis, vital signs, Physical examination & ECG parameters from Baseline A (Visit 2 that is Day 1).
|
|
|
Target Sample Size
|
Total Sample Size="100"
Sample Size from India="10"
Final Enrollment numbers achieved (Total)= "Applicable only for Completed/Terminated trials"
Final Enrollment numbers achieved (India)="Applicable only for Completed/Terminated trials" |
|
Phase of Trial
|
Phase 2/ Phase 3 |
|
Date of First Enrollment (India)
|
15/07/2025 |
| Date of Study Completion (India) |
Applicable only for Completed/Terminated trials |
| Date of First Enrollment (Global) |
04/04/2024 |
| Date of Study Completion (Global) |
Applicable only for Completed/Terminated trials |
|
Estimated Duration of Trial
|
Years="1"
Months="0"
Days="0" |
|
Recruitment Status of Trial (Global)
|
Open to Recruitment |
| Recruitment Status of Trial (India) |
Not Yet Recruiting |
|
Publication Details
|
N/A |
|
Individual Participant Data (IPD) Sharing Statement
|
Will individual participant data (IPD) be shared publicly (including data dictionaries)?
Response - NO
|
|
Brief Summary
|
This is a double-blind, randomized, multicenter trial with vehicle-controlled phase (Part A) of the Phase 2 and 3 study , designed to demonstrate the efficacy and safety of AC-203 diacerein 1 percent after 8 weeks of treatment in patients with generalized EBS. After completing the Part A of the study, eligible patients in both groups will continue in the open-label extension phase (Part B) to collect long-term safety data Part A 7 visits and 1 video phone call, 16 weeks Visit 1 : Week -10 to Day -1: screening, eligibility assessment Visit 2 : Day 1: Baseline for Part A (Baseline A), enrollment, randomization, first study medication application Visit 3 : Week 2: treatment period follow-up Visit 4 : Week 4: treatment period follow-up Week 6 : treatment period follow-up via Video phone call Visit 5 : Week 8: end of treatment for Part A (EOT) Visit 6 : Week 12: no treatment follow-up Visit 7 : Week 16: end of no treatment follow-up for Part A (EOF), Baseline for Part B (Baseline B)
Part B Visit 7 : Week 16: Baseline for Part B (Baseline B) Visit 8 : Week 24: open-label extension treatment period visit Visit 9 : Week 32: open-label extension treatment period visit Visit 10 : Week 40: end of study visit (EOS) |