Study of Intradermal Injections of RCS-01 in Male and Female Subjects

• ClinicalTrials.gov Identifier: NCT02391935
• Recruitment Status: Completed
• First Posted: March 18, 2015
• Last Update Posted: October 11, 2017
• Sponsor: RepliCel Life Sciences, Inc.
• Collaborators: JensonR+ Limited; PHARMALOG Institut für klinische Forschung GmbH; Syreon Corporation; Innovacell Biotechnologie AG; Datinf GmbH
• Information provided by (Responsible Party): RepliCel Life Sciences, Inc.

Tracking Information

• First Submitted Date: March 12, 2015
• First Posted Date: March 18, 2015
• Last Update Posted Date: October 11, 2017
• Actual Study Start Date: October 10, 2015
• Actual Primary Completion Date: July 12, 2017 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: April 4, 2017)

Incidence of local adverse events [ Time Frame: 52 weeks after first injection ]

Within-patient comparison of the local (reported at treatment evaluation sites) adverse event profile between treatment evaluation sites treated with RCS-01 or placebo. The local adverse event profile is defined by the incidence, relationship to treatment, severity and seriousness of adverse events reported at the treatment evaluation sites.

Original Primary Outcome Measures (submitted: March 17, 2015)

Incidence of local adverse events [ Time Frame: 104 weeks after first injection ]

Within-patient comparison of the local (reported at treatment evaluation sites) adverse event profile between treatment evaluation sites treated with RCS-01 or placebo. The local adverse event profile is defined by the incidence, relationship to treatment, severity and seriousness of adverse events reported at the treatment evaluation sites.

Current Secondary Outcome Measures (submitted: April 4, 2017)

• Incidence of adverse events [ Time Frame: 52 weeks after first injection ]
  Between-subject comparison of the systemic (not reported at treatment evaluation sites) adverse event profile between participants in the RCS-01 Subgroup versus Placebo Subgroup.
• Incidence of abnormalities in skin structure [ Time Frame: 52 weeks after first injection ]
  A within-patient and between patient comparison of histology results from analyses performed on biopsies obtained from treatment evaluation sites.
• Incidence of changes in markers related to skin aging [ Time Frame: 12 weeks after first injection ]
  Markers to be assessed include fibrillar collagen type I (COL 1A1, COL 1A2) and type III (COL 3A1), transforming growth factor beta1, connective tissue growth factor, matrix metalloproteinases 1, 3 and 9, tissue inhibitor of matrix metalloproteinase 1 and lumican . Incidence of changes within- and between-participants.

Original Secondary Outcome Measures (submitted: March 17, 2015)

• Incidence of adverse events [ Time Frame: 104 weeks after first injection ]
  Between-subject comparison of the systemic (not reported at treatment evaluation sites) adverse event profile between participants in the RCS-01 Subgroup versus Placebo Subgroup.
• Incidence of abnormalities in skin structure [ Time Frame: 104 weeks after first injection ]
  A within-patient and between patient comparison of histology results from analyses performed on biopsies obtained from treatment evaluation sites.
• Incidence of changes in markers related to skin aging [ Time Frame: 104 weeks after first injection ]
  Gene expression data of markers will be done according to the 2(-ΔΔC(T)) Method. Incidence of changes within- and between-participants

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Study of Intradermal Injections of RCS-01 in Male and Female Subjects
• Official Title: Randomized, Double-blind, Placebo-controlled, Single-centre, Phase I Safety Study of Intradermal Injections of RCS-01 in Male and Female Subjects (50 to 65 Years Old)

Brief Summary

The primary purpose of this study is to assess the safety profile of RCS-01 injections as compared to placebo injections. This study will also measure the impact these injections will have on skin markers related to aging through evaluation of gene expression profiles.

30 participants will be selected for this study based on their health status, current/past medications, and ability to adhere to protocol-related requirements. At the first visit and after providing informed consent, participants are evaluated against the study inclusion/exclusion criteria and provide blood samples for screening assessments (including virology). If suitable for study participation, participants will provide a biopsy from the scalp from which RCS-01 will be prepared and will have four treatment evaluation sites identified on their buttocks, two on each side.

Study participants will be randomized to one of two treatment subgroups. Participants in the RCS-01 Subgroup (n=24) will receive injections of RCS-01 or placebo or a 'sham' injection (a needle penetration without injection of liquid). Participants in the the Placebo Subgroup (n= 6) will be randomized to receive only injections of placebo or sham injections.

Baseline evaluations of subjects' overall health and skin condition at treatment sites on their buttocks will be performed before receipt of injections at Day 0. In addition to injections delivered at Day 0, the pre-selected treatment evaluation sites will receive intradermal injections of RCS-01 or placebo (cryomedium) or a sham injection four (4) and eight (8) weeks after Day 0 according to a randomization schedule for a total of three (3) injections per treatment site.

All participants will return to the clinic for at least 9 visits during the 52-week follow-up period to monitor long-term safety. At the 12-week time point, 18 randomly-selected participants from the RCS-01 Subgroup will provide biopsies from all injection sites for analysis of skin markers related to aging. At the 26-week time point, the remaining participants will provide biopsies of all injection sites for histopathological analysis.

• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 1
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Triple (Participant, Investigator, Outcomes Assessor); Primary Purpose: Treatment

Condition

• Intrinsic Aging of Skin
• Skin Wrinkling
• Solar Degeneration of Skin

Intervention

• Drug: RCS-01
  Cultured, autologous hair follicle cells suspended in cryomedium
• Drug: Placebo
  Cryomedium
• Device: Sham injection
  skin penetration of the needle without injection of liquid

Study Arms

• Experimental: RCS-01
  Cultured, autologous hair follicle cells suspended in cryomedium
  Interventions:

  • Drug: RCS-01
  • Drug: Placebo
  • Device: Sham injection
• Placebo Comparator: Placebo
  cryomedium
  Interventions:

  • Drug: Placebo
  • Device: Sham injection

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: April 6, 2016): 17
• Original Estimated Enrollment (submitted: March 17, 2015): 30
• Actual Study Completion Date: July 12, 2017
• Actual Primary Completion Date: July 12, 2017 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

1. Male or female Caucasian in good health between the age of 50 and 65 years.
2. Female subjects must be post-menopausal for at least one year or surgically sterile.
3. Signed and dated informed consent and willingness to attend all study visits and complete all procedures required by this protocol.
4. The treatment evaluation sites are of uniform skin color without erythema, dark pigmentation or scars that may confound study results.

Exclusion Criteria:

1. Any transient skin disorder or infections (e.g. pyoderma, tinea corporis, contact or irritative dermatitis, scleroderma etc.) within 20 cm of the treatment evaluation sites or the presence of tattoos near the treatment sites.
2. Subjects diagnosed with psoriasis, lichen planus, vitiligo, systemic scleroderma, or lupus erythematosus.
3. Any condition that, in the investigator's opinion, would impact subject's safety and/or a subject's ability to complete all study related procedures.
4. History of infection with or positive serology results for human immunodeficiency virus (HIV), hepatitis B or C, or syphilis.
5. Presence/history of hypertrophic scars and/or keloids.
6. Subjects diagnosed with cancer with or without chemotherapy treatment within 3 years prior enrollment.
7. Women who are pregnant or nursing.
8. Current use of any medications not permitted in the study
9. Ongoing or recent participation in a cosmetic and/or clinical research study prior to enrolment.
10. Subject not agreeing to avoid UV exposure (sun, solarium) on the nude buttocks during the study period.
11. Known allergy or hypersensitivity to human serum albumin, DMSO, gentamicin, material of bovine origin and/or local anesthetics.
12. Close affiliation with the investigational site (e.g., a close relative of the investigator or a possibly dependent person (e.g., employee or student of the investigational site)).
13. Subjects diagnosed with coagulopathies or significant thrombocytopenia

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 50 Years to 65 Years (Adult, Older Adult)
• Accepts Healthy Volunteers: Yes
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Germany

Administrative Information

• NCT Number: NCT02391935
• Other Study ID Numbers: RCS-01-001-2014
• Has Data Monitoring Committee: No
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Responsible Party: RepliCel Life Sciences, Inc.
• Study Sponsor: RepliCel Life Sciences, Inc.

Collaborators

• JensonR+ Limited
• PHARMALOG Institut für klinische Forschung GmbH
• Syreon Corporation
• Innovacell Biotechnologie AG
• Datinf GmbH

Investigators

• Principal Investigator: Alessandra Marini, MD; IUF Leibniz-Institut für umweltmedizinische Forschung gGmbH

• PRS Account: RepliCel Life Sciences, Inc.
• Verification Date: October 2017