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A Double-blind Placebo-controlled Randomized Trial Evaluating the Efficacy and Safety of a Novel HSP90 Inhibitor (RGRN-305) in the Treatment of Moderate to Severe Hidradenitis Supppurativa.

Primary Purpose

Hidradenitis Suppurativa

Status
Completed
Phase
Phase 1
Locations
Denmark
Study Type
Interventional
Intervention
RGRN-305
Placebo
Sponsored by
Aarhus University Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hidradenitis Suppurativa

Eligibility Criteria

18 Years - 99 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Subjects will be eligible to be enrolled in the study if they meet all of the following criteria at the Screening and Baseline (Day 0) Visits, unless specified otherwise:

  1. Men or women aged 18 years or older at the time of consent.
  2. Subject has a history of onset of hidradenitis suppurativa for at least 6 months prior to Baseline (Day 0) Visit.
  3. Subject has hidradenitis suppurativa with at least 5 inflammatory nodules or abscesses (total AN count) in at least 2 distinct anatomic areas, both at Screening and Baseline (Day 0) Visits. Furthermore, one additional inflammatory nodule must be present for collection of skin biopsies.
  4. Subjects (women and men) involved in any sexual intercourse that could lead to pregnancy must agree to use an effective contraceptive method from at least 4 weeks before Baseline (Day 0) until at least 4 weeks after the last study product administration for the duration of the study. Effective contraceptive methods are: systemic hormonal contraceptives (oral contraceptive, patch, vaginal ring, injectables, or implants), intrauterine devices, vasectomy, or barrier methods of contraception in conjunction with spermicide. Hormonal contraceptives must be on a stable dose for at least 4 weeks before Baseline (Day 0).

    Note: Woman of non-childbearing potential are as follows:

    • Women who have had surgical sterilization (hysterectomy, bilateral oophorectomy, bilateral salpingectomy, or bilateral tubal ligation).
    • Women ≥ 60 years of age.
    • Women > 40 and < 60 years of age who have had a cessation of menses for at least 12 months and a follicle-stimulating hormone (FSH) test confirming non-childbearing potential (FSH ≥ 40 mIU/mL) or cessation of menses for at least 24 months without FSH levels confirmed.

    Protocol RGRN-305 - Safety and Efficacy of RGRN-305 in HS Page 13 of 61 Protocol version 3.2: 13 September 2021

  5. Women of childbearing potential must have a negative serum pregnancy test at screening and negative urine pregnancy test at Baseline (Day 0).
  6. Subject must have negative tuberculosis (TB) infection tests. Subjects will be evaluated for latent TB infection with a purified protein derivative (PPD) test, T-spot test or a Quantiferon Gold test, and with a chest x-ray, if one has not been performed in the last 6 months. Subject who demonstrates evidence of latent TB infection (either PPD ≥ 5 mm of induration or positive Quantiferon Gold or T-spot test, irrespective of Bacillus Calmette-Guérin (BCG) vaccination status and negative chest x-rays findings for active TB, or suspicious chest x-ray findings) will not be allowed to participate in the study.
  7. Subject must be willing to participate and must be capable of giving informed consent, and the consent must be obtained prior to any study-related procedures.

Exclusion Criteria:

  1. Female subject who is breastfeeding, pregnant, or who is planning a pregnancy during the study.
  2. Subject has a history of skin disease or presence of a skin condition that, in the opinion of the investigator, would interfere with the study assessments.
  3. Subject is known to have immune deficiency or is immunocompromised.
  4. Subject has a history of cancer or lymphoproliferative disease within 5 years prior to Baseline (Day 0). Subjects with successfully treated non-metastatic cutaneous squamous cell or basal cell carcinoma and/or localized carcinoma in situ of the cervix are not to be excluded.
  5. Subject has had a major surgery within 8 weeks prior to Baseline (Day 0) or has a major surgery planned during the study.
  6. Subject has any clinically significant medical condition including ongoing infections, or physical/laboratory/ECG/vital signs abnormality that would, in the opinion of the investigator, put the subject at undue risk or interfere with interpretation of study results.
  7. Subject has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) values ≥ 2 times the upper limit of normal (ULN) at Screening.
  8. Subject has absolute neutrophil count ≤ 1.5 X 109/L or platelet count ≤ 100 X 109/L at Screening.
  9. Subject has a history of clinically significant anemia or hemoglobin (Hgb) value ≤ 10 g/dL (6.21 mmol/dL) at Screening.
  10. Subject has a creatine clearance ≤ 60 mL/min at Screening (calculated with Cockcroft-Gault formula).
  11. Subject with positive results for hepatitis B surface antigens (HBsAg), anti-hepatitis B core antibodies (anti-HBc), hepatitis C virus (HCV), or human immunodeficiency virus (HIV).
  12. Subject has a known or suspected allergy to RGRN-305 or any component of the investigational product.
  13. Subject has a history of clinically significant drug or alcohol abuse in the last year prior to Baseline (Day 0) Visit.
  14. Subject is currently receiving an investigational product or device or has received one within 4 weeks prior to Baseline (Day 0) Visit.
  15. Subject has received a live attenuated vaccine within 4 weeks prior to Baseline (Day 0) Visit or plan to receive a live attenuated vaccine during the study and up to 1 month after the last study drug administration.
  16. Subject has a history of an allergic reaction or significant sensitivity to lidocaine or other local anesthetics.
  17. Subject has a history of hypertrophic scarring or keloid formation in scars or suture sites.
  18. Known inability or unavailability of a subject to complete required study visits during study participation.
  19. A psychiatric condition (e.g., suicidal ideation), chronic alcohol, or drug abuse problem, determined from the subject's medical history, which, in the opinion of the investigator, may pose a threat to subject compliance.

Sites / Locations

  • Aarhus University Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

RGRN-305

Placebo

Arm Description

1 tablet of 250mg RGRN-305 once daily for 16 weeks

1 tablet of placebo once daily for 16 weeks

Outcomes

Primary Outcome Measures

Percentage of subjects achieving Hidradenitis Suppurativa Clinical Response 50 (HiSCR-50) score

Secondary Outcome Measures

Percentage of subjects with Hidradenitis Suppurativa Physician's Global Assessment (HS-PGA) scores of clear or minimal
Percentage of subjects achieving HiSCR-50, HiSCR-75, and HiSCR-90
Changes from Baseline in the Dermatology Life Quality Index (DLQI) total score
Minimum score: 0 (no effect at all on patient's life) Maximum score: 30 (extremely large effect on patient's life)
Changes from Baseline in the Pain Numerical Ranging score
Minimum score: 0 (No pain) Maximum score: 10 (worst pain)
Change and percent change from Baseline in lesion counts (abscess count, inflammatory nodule count, AN count, and draining tunnel count).
Change from Baseline in skin biomarkers
Change from Baseline in blood biomarkers
Incidence of treatment-emergent adverse events (TEAEs).
Incidence of related TEAEs.

Full Information

First Posted
March 10, 2022
Last Updated
January 30, 2023
Sponsor
Aarhus University Hospital
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1. Study Identification

Unique Protocol Identification Number
NCT05286567
Brief Title
A Double-blind Placebo-controlled Randomized Trial Evaluating the Efficacy and Safety of a Novel HSP90 Inhibitor (RGRN-305) in the Treatment of Moderate to Severe Hidradenitis Supppurativa.
Official Title
An Investigator Initiated, Phase 1b, Randomized, Double-Blind, Placebo-Controlled, Proof-Of-Concept Study to Assess the Safety and Efficacy of a Novel HSP90 Inhibitor (RGRN-305) in the Treatment of Moderate to Severe Hidradenitis Suppurativa
Study Type
Interventional

2. Study Status

Record Verification Date
January 2023
Overall Recruitment Status
Completed
Study Start Date
September 1, 2021 (Actual)
Primary Completion Date
August 29, 2022 (Actual)
Study Completion Date
August 29, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Aarhus University Hospital

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 is a 16-week treatment, randomized, double-blind, proof-of-concept study designed to assess the safety and efficacy of RGRN-305 compared to placebo for use in future efficacy Phase 2 trials. Male or female subjects aged 18 years or older with moderate to severe hidradenitis suppurativa will be included in this study. Objectives are to determine the efficacy and safety of RGRN-305 in patients with moderate to severe hidradenitis supppurativa.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hidradenitis Suppurativa

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
15 (Actual)

8. Arms, Groups, and Interventions

Arm Title
RGRN-305
Arm Type
Experimental
Arm Description
1 tablet of 250mg RGRN-305 once daily for 16 weeks
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
1 tablet of placebo once daily for 16 weeks
Intervention Type
Drug
Intervention Name(s)
RGRN-305
Intervention Description
Heat shock protein 90 inhibitor
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo (blinded)
Primary Outcome Measure Information:
Title
Percentage of subjects achieving Hidradenitis Suppurativa Clinical Response 50 (HiSCR-50) score
Time Frame
Week 16
Secondary Outcome Measure Information:
Title
Percentage of subjects with Hidradenitis Suppurativa Physician's Global Assessment (HS-PGA) scores of clear or minimal
Time Frame
Week 2, 4, 8, 12, 14, 16, and 20.
Title
Percentage of subjects achieving HiSCR-50, HiSCR-75, and HiSCR-90
Time Frame
Week 2, 4, 8, 12, 14, 16, and 20.
Title
Changes from Baseline in the Dermatology Life Quality Index (DLQI) total score
Description
Minimum score: 0 (no effect at all on patient's life) Maximum score: 30 (extremely large effect on patient's life)
Time Frame
Week 4, 8, 12, 16, and 20.
Title
Changes from Baseline in the Pain Numerical Ranging score
Description
Minimum score: 0 (No pain) Maximum score: 10 (worst pain)
Time Frame
Weeks 4, 8, 12, 16, and 20
Title
Change and percent change from Baseline in lesion counts (abscess count, inflammatory nodule count, AN count, and draining tunnel count).
Time Frame
Week 2, 4, 8, 12, 14, 16, and 20.
Title
Change from Baseline in skin biomarkers
Time Frame
Week 16.
Title
Change from Baseline in blood biomarkers
Time Frame
Week 4, 8, 12, and 16.
Title
Incidence of treatment-emergent adverse events (TEAEs).
Time Frame
Week 20
Title
Incidence of related TEAEs.
Time Frame
Week 20

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
99 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Subjects will be eligible to be enrolled in the study if they meet all of the following criteria at the Screening and Baseline (Day 0) Visits, unless specified otherwise: Men or women aged 18 years or older at the time of consent. Subject has a history of onset of hidradenitis suppurativa for at least 6 months prior to Baseline (Day 0) Visit. Subject has hidradenitis suppurativa with at least 5 inflammatory nodules or abscesses (total AN count) in at least 2 distinct anatomic areas, both at Screening and Baseline (Day 0) Visits. Furthermore, one additional inflammatory nodule must be present for collection of skin biopsies. Subjects (women and men) involved in any sexual intercourse that could lead to pregnancy must agree to use an effective contraceptive method from at least 4 weeks before Baseline (Day 0) until at least 4 weeks after the last study product administration for the duration of the study. Effective contraceptive methods are: systemic hormonal contraceptives (oral contraceptive, patch, vaginal ring, injectables, or implants), intrauterine devices, vasectomy, or barrier methods of contraception in conjunction with spermicide. Hormonal contraceptives must be on a stable dose for at least 4 weeks before Baseline (Day 0). Note: Woman of non-childbearing potential are as follows: Women who have had surgical sterilization (hysterectomy, bilateral oophorectomy, bilateral salpingectomy, or bilateral tubal ligation). Women ≥ 60 years of age. Women > 40 and < 60 years of age who have had a cessation of menses for at least 12 months and a follicle-stimulating hormone (FSH) test confirming non-childbearing potential (FSH ≥ 40 mIU/mL) or cessation of menses for at least 24 months without FSH levels confirmed. Protocol RGRN-305 - Safety and Efficacy of RGRN-305 in HS Page 13 of 61 Protocol version 3.2: 13 September 2021 Women of childbearing potential must have a negative serum pregnancy test at screening and negative urine pregnancy test at Baseline (Day 0). Subject must have negative tuberculosis (TB) infection tests. Subjects will be evaluated for latent TB infection with a purified protein derivative (PPD) test, T-spot test or a Quantiferon Gold test, and with a chest x-ray, if one has not been performed in the last 6 months. Subject who demonstrates evidence of latent TB infection (either PPD ≥ 5 mm of induration or positive Quantiferon Gold or T-spot test, irrespective of Bacillus Calmette-Guérin (BCG) vaccination status and negative chest x-rays findings for active TB, or suspicious chest x-ray findings) will not be allowed to participate in the study. Subject must be willing to participate and must be capable of giving informed consent, and the consent must be obtained prior to any study-related procedures. Exclusion Criteria: Female subject who is breastfeeding, pregnant, or who is planning a pregnancy during the study. Subject has a history of skin disease or presence of a skin condition that, in the opinion of the investigator, would interfere with the study assessments. Subject is known to have immune deficiency or is immunocompromised. Subject has a history of cancer or lymphoproliferative disease within 5 years prior to Baseline (Day 0). Subjects with successfully treated non-metastatic cutaneous squamous cell or basal cell carcinoma and/or localized carcinoma in situ of the cervix are not to be excluded. Subject has had a major surgery within 8 weeks prior to Baseline (Day 0) or has a major surgery planned during the study. Subject has any clinically significant medical condition including ongoing infections, or physical/laboratory/ECG/vital signs abnormality that would, in the opinion of the investigator, put the subject at undue risk or interfere with interpretation of study results. Subject has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) values ≥ 2 times the upper limit of normal (ULN) at Screening. Subject has absolute neutrophil count ≤ 1.5 X 109/L or platelet count ≤ 100 X 109/L at Screening. Subject has a history of clinically significant anemia or hemoglobin (Hgb) value ≤ 10 g/dL (6.21 mmol/dL) at Screening. Subject has a creatine clearance ≤ 60 mL/min at Screening (calculated with Cockcroft-Gault formula). Subject with positive results for hepatitis B surface antigens (HBsAg), anti-hepatitis B core antibodies (anti-HBc), hepatitis C virus (HCV), or human immunodeficiency virus (HIV). Subject has a known or suspected allergy to RGRN-305 or any component of the investigational product. Subject has a history of clinically significant drug or alcohol abuse in the last year prior to Baseline (Day 0) Visit. Subject is currently receiving an investigational product or device or has received one within 4 weeks prior to Baseline (Day 0) Visit. Subject has received a live attenuated vaccine within 4 weeks prior to Baseline (Day 0) Visit or plan to receive a live attenuated vaccine during the study and up to 1 month after the last study drug administration. Subject has a history of an allergic reaction or significant sensitivity to lidocaine or other local anesthetics. Subject has a history of hypertrophic scarring or keloid formation in scars or suture sites. Known inability or unavailability of a subject to complete required study visits during study participation. A psychiatric condition (e.g., suicidal ideation), chronic alcohol, or drug abuse problem, determined from the subject's medical history, which, in the opinion of the investigator, may pose a threat to subject compliance.
Facility Information:
Facility Name
Aarhus University Hospital
City
Aarhus
ZIP/Postal Code
8200
Country
Denmark

12. IPD Sharing Statement

Plan to Share IPD
Undecided

Learn more about this trial

A Double-blind Placebo-controlled Randomized Trial Evaluating the Efficacy and Safety of a Novel HSP90 Inhibitor (RGRN-305) in the Treatment of Moderate to Severe Hidradenitis Supppurativa.

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