A clinical research study to evaluate the safety and tolerability of a topically applied investigational drug product in adults with vitiligo and to evaluate if it helps in restoring the pigment/color to the affected skin
| ISRCTN | ISRCTN78313275 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN78313275 |
| Secondary identifying numbers | VIM-004 Gel-01/ 2324CMPH078 |
- Submission date
- 15/10/2024
- Registration date
- 30/10/2024
- Last edited
- 30/10/2024
- Recruitment status
- Recruiting
- Overall study status
- Ongoing
- Condition category
- Skin and Connective Tissue Diseases
Plain English summary of protocol
Background and study aims
Vitiligo, a skin disorder characterized by the loss of pigment, can significantly impact the affected individual's mental well-being, social interactions, and quality of life due to its visible nature and associated stigma. This study involves a new topical skin treatment called VIM-004 Gel, which combines afamelanotide, a known medication that increases the pigmentation of the skin with STAR particles, a medical device used for creating micropores across the top layers of the skin to allow the delivery of afamelanotide, that otherwise would not penetrate the skin. VIM-004 Gel aims to help repigment the skin areas affected by vitiligo.
Who can participate?
Adult patients aged between 18-70 years old with vitiligo
What does the study involve?
Participants must pass a clinical evaluation including lab tests and a physical examination to confirm eligibility to participate. Two skin areas affected by vitiligo will be selected for each participant. Except for the areas located in the face, these areas should include between 10-20% of pigmented skin. Participants will receive NB-UVB 3 times/week for 30 days during a pre-treatment phase. The investigational product (VIM-004 Gel) will then be applied to one area 5 times/week for 12 weeks, while a placebo (which is the gel with STAR particles, but without afamelanotide) will be applied to the other. Participants will continue to receive NB-UVB treatment 3 times/week during the treatment phase. Please note that not all areas affected by vitiligo will receive treatment. Participants will be regularly evaluated for safety and tolerability of the drug product, blood samples will be collected periodically and skin images will be collected throughout the treatment period to evaluate response to treatment. Skin biopsies will be performed in a subset of participants.
What are the possible benefits and risks of participating?
Participants may see repigmentation of the treated skin lesion. However, it is not known if taking part in this study will help your condition stay the same or worsen. As part of the study, your health will be closely monitored by qualified doctors. Nevertheless, even if there is no guarantee that you will have a personal medical benefit from taking part in this clinical study, the information obtained may help to better treat people with your condition.
Afamelanotide administered as a subcutaneous implant is associated with plasma exposure and side effects such as nausea, headache, fatigue and hypersensitivity reactions. In addition, systemic exposure to afamelanotide has been shown to induce skin hyperpigmentation of non-lesional skin in vitiligo patients. In this study, VIM-004 Gel will be applied on restricted areas of the skin, so the amount of the active ingredient expected to reach the bloodstream is anticipated to be minimal, and the blood levels at which these side effects occur are unlikely to be reached. You may experience an increase in pigmentation in the application area. In addition, when applied on the skin, there is a possible risk of local skin reactions, including erythema, oedema, and/or bleeding, associated with the mechanical mode of action of the STAR particles. When VIM-004 Gel is applied on the face there is a potential risk of translocation of STAR particles to the eyes and the mouth. There may be side effects from the investigational product that are not known at this time.
Where is the study run from?
The study is being conducted by Centre International de Développement Pharmaceutique, Mauritius
When is the study starting and how long is it expected to run for?
August 2024 to May 2026
Who is funding the study?
Vimela Therapeutics, Ltd
Who is the main contact?
1. Dr Juliana Gordilho, juliana.gordilho@aldenatherapeutics.com
2. Dr Claire Bouix-Peter, claire.bouix-peter@aldenatherapeutics.com
3. Dr Ounisha Mungur, o.mungur@cidp-cro.com
Contact information
Scientific
Aldena Therapeutics
Place de La Gare 12
Lausanne
CH-1003
Switzerland
 ORCID ID |
0000-0003-3447-5808 |
|---|---|
| Phone | +41 76 389 0554 |
| juliana.gordilho@aldenatherapeutics.com |
Public
Aldena Therapeutics
Place de La Gare 12
Lausanne
CH-1003
Switzerland
| Phone | +41 76 389 0554 |
|---|---|
| claire.bouix-peter@aldenatherapeutics.com |
Principal Investigator
BioPark Mauritius, Socota Phoenicia, Sayed Hossen Road
Phoenix
73408
Mauritius
| Phone | +230 401 2600 |
|---|---|
| g.petkar@cidp-cro.com |
Study information
| Study design | Single-center double-blind placebo-controlled intraindividual randomized study |
|---|---|
| Primary study design | Interventional |
| Secondary study design | Randomised controlled trial |
| Study setting(s) | Pharmaceutical testing facility |
| Study type | Safety, Efficacy |
| Participant information sheet | No participant information sheet available |
| Scientific title | A phase Ib/IIa, proof of concept, double-blind, placebo-controlled, intraindividual randomized study to evaluate the safety, tolerability, and preliminary efficacy of VIM-004 gel in participants with vitiligo |
| Study objectives | 1. VIM-004 Gel is safe and well tolerated. 2. Topical application of VIM-004 Gel has minimal to no systemic exposure. 3. VIM-004 Gel + NB-UVB treatment repigments vitiligo lesions more than NB-UVB treatment + VIM-004 Placebo Gel. |
| Ethics approval(s) |
Submitted 30/10/2024, Clinical Research Regulatory Council (2nd Floor, Bacha Building Right Wing, Cathedral Square, Port Louis, -, Mauritius; -; not@provided.com), ref: 2324CMPH078 |
| Health condition(s) or problem(s) studied | Vitiligo |
| Intervention | All eligible participants will be randomized using the randomization list provided by an unblinded statistician. The randomization list will define the treatment (VIM-004 Gel or VIM-004 Placebo Gel) to be allocated to each treatment area (Area 1 and Area 2) per participant (intra-individually). The pre-defined list will ensure that treatment assignment is unbiased, and this will be concealed from participants and blinded site staff. The randomization schedule will be produced by the unblinded staff using SAS 9.4. The unblinded statistician will not take part in any of the study-related processes. Two application areas between 9-25 cm2 will be defined. Participants will start with a narrow-band (NB)-UVB pretreatment period for 30 days in all application areas. These areas will be randomised in a 1:1 ratio on Day 1 to VIM-004 Gel and VIM-004 Placebo Gel, with NB-UVB treatment. VIM-004 Gel or VIM-004 Placebo Gel will be applied topically once a day on the 2 application areas. The application will be repeated for a total of 5 consecutive days per week for 12 weeks. During the treatment period, all application areas will also receive NB-UVB treatment 3 times per week. There will be a 4-week follow-up period. |
| Intervention type | Drug |
| Pharmaceutical study type(s) | Pharmacokinetic |
| Phase | Phase I/II |
| Drug / device / biological / vaccine name(s) | VIM-004 Gel |
| Primary outcome measure | Safety and tolerability will be assessed by: 1. Local tolerability assessment measured using a dermal response scale, sensation scale, and pain Numeric Rating Scale (NRS) and evaluating adverse events during all study visits during the treatment period 2. Recording vital signs and performing physical examinations weekly throughout the 12-week treatment period and at 4 weeks post-treatment 3. Evaluating clinical laboratory results at week 6 during the treatment period and at 4 weeks post-treatment. |
| Secondary outcome measures | 1. Plasma concentration of VIM-004 measured using pharmacokinetic methods on Day 1, Day 8 and Week 12 Exploratory outcome measures: 1. Repigmentation measured using planimetry and image analysis weekly 2. Repigmentation percentage for target lesions measured using the Vitiligo Extent Score for a Target Area (VESTA) weekly |
| Overall study start date | 12/08/2024 |
| Completion date | 30/04/2026 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Mixed |
| Lower age limit | 18 Years |
| Upper age limit | 70 Years |
| Sex | Both |
| Target number of participants | 25 |
| Key inclusion criteria | 1. Aged 18 to 70 years 2. Clinical diagnosis of stable nonsegmental vitiligo lesions 3. Fitzpatrick's skin phototype of >=4 4. Has nonsegmental vitiligo lesions that can accommodate 2 application areas between 9-25 cm2 of comparable VESTA score in easy-to-treat areas 5. Has nonsegmental vitiligo lesions with a maximum of 10% white hairs in the 2 selected application areas 6. Females of childbearing potential must have a negative serum pregnancy test at screening and a negative urine pregnancy test on Day 1 7. Involved in sexual intercourse that could lead to pregnancy: must agree to use a highly effective contraceptive method from >= 4 weeks prior to the pretreatment period and until >=2 weeks after the last study product application 7. Willing to participate and is capable of giving informed consent 8. Willing and able to comply with all study procedures and must be available for the duration of the study |
| Key exclusion criteria | 1. Has active form(s) of inflammatory skin disease or evidence of skin condition that would interfere with evaluation of vitiligo or response to treatment 2. Has active acute or chronic skin infection requiring treatment with systemic antibiotics, antivirals, antiparasitics, antiprotozoals or antifungals within 2 weeks prior to Day 1 or superficial skin infections within 1 week prior to Day 1 3. Has the presence of any tattoos, scratches, open sores, excessive hair, or skin damage in the application areas 4. Has active forms of other hypopigmentation 5. Is a female who is breastfeeding, pregnant, or who is planning to become pregnant during the time of the study 6. Is known to have immune deficiency or is immunocompromised. 7. Has a history of cancer or lymphoproliferative disease within 5 years prior to Day 1 8. Had a major surgery within 8 weeks prior to Day 1 or had a major surgery planned during the study 9. Has any clinically significant medical condition or physical/laboratory/vital signs abnormality that would put the participant at undue risk or interfere with the interpretation of study results 10. Has a positive result for hepatitis B virus, hepatitis C virus, or human immunodeficiency virus 11. Has used permanent depigmentation treatment for vitiligo and/or other types of pigmentation disorders at any time 12. Has used systemic treatments that could affect vitiligo within 8 weeks prior to the pretreatment period or within 5 half-lives, whichever is longer 13. Has used topical medicated treatment that could affect vitiligo 14. Has used herbal medications with unknown properties or known beneficial effects for vitiligo within 1 week of the pretreatment period 15. Has received any marketed or investigational biological agent within 12 weeks or 5 half-lives (whichever is longer) prior to the pretreatment period 16. Is currently receiving a nonbiological investigational product or device or has received one within 4 weeks prior to the pretreatment period 17. Has received any ultraviolet (UV)-B phototherapy, has had psoralen-UV-A (PUVA) treatment, or excimer laser within 4 weeks prior to the pretreatment period 18. Use of any prior and concomitant therapy not listed above that may interfere with the objective of the study as per the discretion of the investigator, including drugs that cause photosensitivity in the UVB range or skin pigmentation (eg, antibiotics such as tetracyclines, antifungals) within 8 weeks of screening 19. Has had excessive sun exposure, is planning outdoor activities with long-hours exposure to the sun, has used tanning booths within 4 weeks prior to the pretreatment period or is not willing to minimize natural and artificial sunlight exposure during the study 20. Has a known history of clinically significant drug or alcohol abuse in the last year prior to the pretreatment period 21. Has a known or suspected allergy to the active ingredient (afamelanotide) in VIM-004 Gel or any other component of the investigational medicinal product. 22. Is institutionalized because of legal or regulatory order 23. Has a history of an allergic reaction or significant sensitivity to lidocaine or other local anesthetics and latex |
| Date of first enrolment | 15/01/2025 |
| Date of final enrolment | 15/08/2025 |
Locations
Countries of recruitment
- Mauritius
Study participating centre
Phoenix
73408
Mauritius
Sponsor information
Industry
The Cambridge Partnership, The Dorothy Hodgkin Building
Babraham Research Campus
Cambridge
CB22 3FH
England
United Kingdom
| Phone | +41 76 389 05 54 |
|---|---|
| claire.bouix-peter@aldenatherapeutics.com |
Funders
Funder type
Industry
No information available
Results and Publications
| Intention to publish date | 30/04/2027 |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Data sharing statement to be made available at a later date |
| Publication and dissemination plan | Planned publication in a peer-reviewed journal |
| IPD sharing plan | The data-sharing plans for the current study are unknown and will be made available at a later date. |
Editorial Notes
30/10/2024: Study's existence confirmed by the Clinical Research Regulatory Council, Mauritius.
