CClinicalTrials.gg
CompletedNCT01256671Updated Oct 18, 2017Results posted

DHEA Against Vaginal Atrophy - Safety Study of 12 Months

A Phase 3 interventional study of DHEA in Vaginal Atrophy, sponsored by EndoCeutics Inc.. Completed at 41 sites in 2 countries. Open to female participants aged 40 Years to 75 Years. Per ClinicalTrials.gov, last updated 2017-10-18.

Sponsored by EndoCeutics Inc. · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
530
Allocation
Not applicable
Ages
40 Years to 75 Years
Sex
Female
01

Study summary

The purpose of this Phase III trial is to assess the long-term safety of intravaginal dehydroepiandrosterone (DHEA) in non-hysterectomized postmenopausal women with vaginal atrophy aged 40 to 75 years.

02

Conditions studied

  • Vaginal Atrophy

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Keywords

  • Vulvar/Vaginal Atrophy
  • Atrophic Vaginitis
  • Dehydroepiandrosterone
  • DHEA
  • Prasterone
  • Vaginorm
  • Menopause
  • Intrarosa
03

Who can participate

Ages eligible
40 Years to 75 Years
Sexes eligible
Female
Accepts healthy volunteers
No

Eligibility criteria

Main Inclusion Criteria:

  • Postmenopausal women (non-hysterectomized)
  • Women between 40 and 75 years of age.
  • Willing to participate in the study and sign an informed consent.
  • Women who have self-identified symptom(s) of vaginal atrophy.
  • Willing to have endometrial biopsy at screening and end of study (Week 52).

Main Exclusion Criteria:

  • Undiagnosed abnormal genital bleeding.
  • Hypertension equal to or above 140/90 mm Hg.
  • The administration of any investigational drug within 30 days of screening visit.
  • Endometrial hyperplasia, cancer or endometrial histology showing proliferative, secretory or menstrual type characteristics at histologic evaluation of endometrial biopsy performed at screening.
04

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
530 participants (actual)

Study arms

  • Experimental
    DHEA

    0.5% DHEA (intravaginal)

    Drug: DHEA

Interventions

  • DrugDHEA

    Vaginal suppository containing 0.5% (6.5 mg) DHEA; daily dosing with one suppository for 52 weeks.

    Also known as: Prasterone, Dehydroepiandrosterone, Vaginorm

05

What researchers measure

Primary outcomes

  1. Long-term Safety of Intravaginal Prasterone (DHEA): Endometrium

    The long-term safety of intravaginal prasterone has been evaluated on different parameters including the endometrium. For this purpose, endometrial biopsies were performed at screening and at the end of the study (52 weeks) or at discontinuation visit for women who were exposed to intravaginal DHEA (prasterone) for at least 12 weeks. At screening, the endometrium had to be atrophic/inactive for women to be enrolled in the study. Only the end-of-study data are presented.

    Time frame: Baseline and Week 52 (or discontinuation)

  2. Long-term Safety of Intravaginal Prasterone (DHEA): Serum Steroid Levels

    The long-term safety of intravaginal prasterone has been evaluated on different parameters including the serum levels of DHEA and its metabolites. For this purpose, blood samples were collected at Baseline and different post-Baseline timepoints for the determination of serum steroid levels by a central laboratory using validated liquid chromatography tandem mass spectrometry (LC-MS/MS) methods. The serum levels of dehydroepiandrosterone (DHEA), estradiol (E2) and testosterone (TESTO) obtained at Baseline and Week 52 as well as the change from Baseline to Week 52 are presented.

    Time frame: Baseline and Week 52

Secondary outcomes

  1. Change From Baseline to Week 52 of Vaginal Cell Maturation (Percentage of Parabasal Cells).

    The percentage of parabasal cells was determined from the vaginal smears collected during the study. A 100-cell count was performed by a central laboratory to classify cells as parabasal (P) (including basal), intermediate (I), and superficial (S) squamous cell types. Data obtained at Baseline and Week 52 as well as the change from Baseline to Week 52 are presented.

    Time frame: Baseline and Week 52

  2. Change From Baseline to Week 52 of Vaginal Cell Maturation (Percentage of Superficial Cells).

    The percentage of superficial cells was determined from the vaginal smears collected during the study. A 100-cell count was performed by a central laboratory to classify cells as parabasal (P) (including basal), intermediate (I), and superficial (S) squamous cell types. Data obtained at Baseline and Week 52 as well as the change from Baseline to Week 52 are presented.

    Time frame: Baseline and Week 52

  3. Change From Baseline to Week 52 of Vaginal pH.

    A pH strip fixed on an Ayre spatula (or equivalent) was applied directly to the lateral wall of the vagina. The change in color of the pH indicator strip was compared to the color chart for pH evaluation. The corresponding pH value (with one decimal) was recorded. Data obtained at Baseline and Week 52 as well as the change from Baseline to Week 52 are presented.

    Time frame: Baseline and Week 52

  4. Change From Baseline to Week 52 of Self-assessment of VVA Symptom Dyspareunia

    The severity of dyspareunia was evaluated by a questionnaire. The severity of dyspareunia recorded as none, mild, moderate or severe was analyzed using the score values of 0, 1, 2 or 3, respectively. Data obtained at Baseline and Week 52 as well as the change from Baseline to Week 52 are presented.

    Time frame: Baseline and Week 52

  5. Change From Baseline to Week 52 of Self-assessment of VVA Symptom Vaginal Dryness

    The severity of vaginal dryness was evaluated by a questionnaire. The severity of vaginal dryness recorded as none, mild, moderate or severe was analyzed using the score values of 0, 1, 2 or 3, respectively. Data obtained at Baseline and Week 52 as well as the change from Baseline to Week 52 are presented.

    Time frame: Baseline and Week 52

  6. Change From Baseline to Week 52 of Self-assessment of VVA Symptom Irritation/Itching

    The severity of irritation/itching was evaluated by a questionnaire. The severity of irritation/itching recorded as none, mild, moderate or severe was analyzed using the score values of 0, 1, 2 or 3, respectively. Data obtained at Baseline and Week 52 as well as the change from Baseline to Week 52 are presented.

    Time frame: Baseline and Week 52

06

Results

Posted Oct 18, 2017

Participant flow

A total of 798 subjects were screened at 41 medical/research sites located in the US (31 centers) and Canada (10 centers) and 530 subjects were randomized. The first subject first visit was on 30-NOV-2010 and the last subject last visit was on 16-JUL-2012.

Participant flow — Overall Study
Milestone0.50% DHEA
Started530
Safety population521
Completed435
Not completed95
Withdrew: Adverse event29
Withdrew: Lost to follow-up16
Withdrew: Withdrawal by subject31
Withdrew: Physician decision3
Withdrew: Lack of efficacy4
Withdrew: Non-compliance/sponsor decision/other12

Outcome measures

PrimaryLong-term Safety of Intravaginal Prasterone (DHEA): Endometrium

The long-term safety of intravaginal prasterone has been evaluated on different parameters including the endometrium. For this purpose, endometrial biopsies were performed at screening and at the end of the study (52 weeks) or at discontinuation visit for women who were exposed to intravaginal DHEA (prasterone) for at least 12 weeks. At screening, the endometrium had to be atrophic/inactive for women to be enrolled in the study. Only the end-of-study data are presented.

Time frame:
Baseline and Week 52 (or discontinuation)
Reported as:
Count of participants · Participants
Long-term Safety of Intravaginal Prasterone (DHEA): Endometrium
Participants0.50% DHEA
Endometrium: Atrophic/Inactive421
Endometrium: No/Insufficient Tissue for Diagnosis36
SecondaryChange From Baseline to Week 52 of Vaginal Cell Maturation (Percentage of Parabasal Cells).

The percentage of parabasal cells was determined from the vaginal smears collected during the study. A 100-cell count was performed by a central laboratory to classify cells as parabasal (P) (including basal), intermediate (I), and superficial (S) squamous cell types. Data obtained at Baseline and Week 52 as well as the change from Baseline to Week 52 are presented.

Time frame:
Baseline and Week 52
Reported as:
Mean · percentage of parabasal cells
Change From Baseline to Week 52 of Vaginal Cell Maturation (Percentage of Parabasal Cells).
percentage of parabasal cells0.50% DHEA
Baseline: Subgroup ALL55.49 ± 2.03
Week 52: Subgroup ALL12.81 ± 0.97
Change from Baseline: Subgroup ALL-42.67 ± 1.84
Baseline: Subgroup VVA63.95 ± 2.41
Week 52: Subgroup VVA14.80 ± 1.27
Change from Baseline: Subgroup VVA-49.14 ± 2.22
SecondaryChange From Baseline to Week 52 of Vaginal Cell Maturation (Percentage of Superficial Cells).

The percentage of superficial cells was determined from the vaginal smears collected during the study. A 100-cell count was performed by a central laboratory to classify cells as parabasal (P) (including basal), intermediate (I), and superficial (S) squamous cell types. Data obtained at Baseline and Week 52 as well as the change from Baseline to Week 52 are presented.

Time frame:
Baseline and Week 52
Reported as:
Mean · percentage of superficial cells
Change From Baseline to Week 52 of Vaginal Cell Maturation (Percentage of Superficial Cells).
percentage of superficial cells0.50% DHEA
Baseline: Subgroup ALL2.02 ± 0.19
Week 52: Subgroup ALL9.42 ± 0.36
Change from Baseline: Subgroup ALL7.41 ± 0.38
Baseline: Subgroup VVA0.96 ± 0.08
Week 52: Subgroup VVA8.81 ± 0.41
Change from Baseline: Subgroup VVA7.85 ± 0.42
SecondaryChange From Baseline to Week 52 of Vaginal pH.

A pH strip fixed on an Ayre spatula (or equivalent) was applied directly to the lateral wall of the vagina. The change in color of the pH indicator strip was compared to the color chart for pH evaluation. The corresponding pH value (with one decimal) was recorded. Data obtained at Baseline and Week 52 as well as the change from Baseline to Week 52 are presented.

Time frame:
Baseline and Week 52
Reported as:
Mean · pH
Change From Baseline to Week 52 of Vaginal pH.
pH0.50% DHEA
Baseline: Subgroup ALL6.23 ± 0.04
Week 52: Subgroup ALL5.09 ± 0.04
Change from Baseline: Subgroup ALL-1.14 ± 0.04
Baseline: Subgroup VVA6.40 ± 0.04
Week 52: Subgroup VVA5.13 ± 0.05
Change from Baseline: Subgroup VVA-1.27 ± 0.05
SecondaryChange From Baseline to Week 52 of Self-assessment of VVA Symptom Dyspareunia

The severity of dyspareunia was evaluated by a questionnaire. The severity of dyspareunia recorded as none, mild, moderate or severe was analyzed using the score values of 0, 1, 2 or 3, respectively. Data obtained at Baseline and Week 52 as well as the change from Baseline to Week 52 are presented.

Time frame:
Baseline and Week 52
Reported as:
Mean · units on a scale
Change From Baseline to Week 52 of Self-assessment of VVA Symptom Dyspareunia
units on a scale0.50% DHEA
Baseline: Subgroup MS2.53 ± 0.03
Week 52: Subgroup MS0.85 ± 0.06
Change from Baseline: Subgroup MS-1.68 ± 0.06
Baseline: Subgroup MBS/MS2.57 ± 0.04
Week 52: Subgroup MBS/MS0.87 ± 0.07
Change from Baseline: Subgroup MBS/MS-1.69 ± 0.07
SecondaryChange From Baseline to Week 52 of Self-assessment of VVA Symptom Vaginal Dryness

The severity of vaginal dryness was evaluated by a questionnaire. The severity of vaginal dryness recorded as none, mild, moderate or severe was analyzed using the score values of 0, 1, 2 or 3, respectively. Data obtained at Baseline and Week 52 as well as the change from Baseline to Week 52 are presented.

Time frame:
Baseline and Week 52
Reported as:
Mean · units on a scale
Change From Baseline to Week 52 of Self-assessment of VVA Symptom Vaginal Dryness
units on a scale0.50% DHEA
Baseline: Subgroup MS2.22 ± 0.03
Week 52: Subgroup MS0.59 ± 0.05
Change from Baseline: Subgroup MS-1.63 ± 0.05
Baseline: Subgroup MBS/MS2.19 ± 0.04
Week 52: Subgroup MBS/MS0.67 ± 0.09
Change from Baseline: Subgroup MBS/MS-1.52 ± 0.09
SecondaryChange From Baseline to Week 52 of Self-assessment of VVA Symptom Irritation/Itching

The severity of irritation/itching was evaluated by a questionnaire. The severity of irritation/itching recorded as none, mild, moderate or severe was analyzed using the score values of 0, 1, 2 or 3, respectively. Data obtained at Baseline and Week 52 as well as the change from Baseline to Week 52 are presented.

Time frame:
Baseline and Week 52
Reported as:
Mean · units on a scale
Change From Baseline to Week 52 of Self-assessment of VVA Symptom Irritation/Itching
units on a scale0.50% DHEA
Baseline: Subgroup MS2.10 ± 0.03
Week 52: Subgroup MS0.60 ± 0.08
Change from Baseline: Subgroup MS-1.50 ± 0.09
Baseline: Subgroup MBS/MS2.13 ± 0.07
Week 52: Subgroup MBS/MS0.74 ± 0.14
Change from Baseline: Subgroup MBS/MS-1.39 ± 0.16
PrimaryLong-term Safety of Intravaginal Prasterone (DHEA): Serum Steroid Levels

The long-term safety of intravaginal prasterone has been evaluated on different parameters including the serum levels of DHEA and its metabolites. For this purpose, blood samples were collected at Baseline and different post-Baseline timepoints for the determination of serum steroid levels by a central laboratory using validated liquid chromatography tandem mass spectrometry (LC-MS/MS) methods. The serum levels of dehydroepiandrosterone (DHEA), estradiol (E2) and testosterone (TESTO) obtained at Baseline and Week 52 as well as the change from Baseline to Week 52 are presented.

Time frame:
Baseline and Week 52
Reported as:
Mean · pg/mL
Long-term Safety of Intravaginal Prasterone (DHEA): Serum Steroid Levels
pg/mL0.50% DHEA
DHEA: Baseline2071.61 ± 65.70
DHEA: Week 522997.25 ± 85.23
DHEA: Change from Baseline925.65 ± 74.35
E2: Baseline6.05 ± 1.11
E2: Week 524.46 ± 0.32
E2: Change from Baseline-1.59 ± 1.13
TESTO: Baseline161.28 ± 6.93
TESTO: Week 52189.44 ± 4.79
TESTO: Change from Baseline28.17 ± 6.55

Adverse events

Collected over From Baseline to Week 52 (+ 30-day follow-up period after last dose). Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
0.50% DHEA—18/521 (3.5%)177/521 (34%)
Most frequent serious events
Showing 10 of 23
Most frequent serious events
Event0.50% DHEA
OsteoarthritisMusculoskeletal and connective tissue disorders2/521
Colitis ischaemicGastrointestinal disorders1/521
Duodenal stenosisGastrointestinal disorders1/521
DyspepsiaGastrointestinal disorders1/521
PancreatitisGastrointestinal disorders1/521
Infectious peritonitisInfections and infestations1/521
Staphylococcal infectionInfections and infestations1/521
Femur fractureInjury, poisoning and procedural complications1/521
LacerationInjury, poisoning and procedural complications1/521
Muscle ruptureInjury, poisoning and procedural complications1/521
Most frequent other events
Most frequent other events
Event0.50% DHEA
Application site dischargeGeneral disorders73/521
Urinary tract infectionInfections and infestations53/521
NasopharyngitisInfections and infestations51/521

Baseline characteristics

Baseline Analysis Population corresponds to the Safety Population which includes all subjects who received any amount of DHEA, and who have any safety information available.

Age, Continuous
Age, Continuous(years)0.50% DHEA
Mean57.95 ± 5.65
Sex/Gender, Customized
Sex/Gender, Customized(Participants)0.50% DHEA
Female521
Race/Ethnicity, Customized
Race/Ethnicity, Customized(Participants)0.50% DHEA
White Caucasian478
Black or African American31
Asian3
American Indian or Alaska Native3
Native Hawaiian or Other Pacific Islander2
Other4
07

Study locations

41 sites
  • EndoCeutics site # 39
    Montgomery, Alabama 36117, United States
  • EndoCeutics site # 14
    Tucson, Arizona 85712, United States
  • EndoCeutics site # 21
    Sacramento, California 95821, United States
  • EndoCeutics site # 30
    San Diego, California 92108, United States
  • EndoCeutics site # 17
    San Diego, California 92120, United States
  • EndoCeutics site # 36
    Denver, Colorado 80218, United States
  • EndoCeutics site # 42
    Milford, Connecticut 06460, United States
  • EndoCeutics site # 07
    Washington, D.C., District of Columbia 20036, United States
  • EndoCeutics site # 45
    Boynton Beach, Florida 33472, United States
  • EndoCeutics site # 26
    Jacksonville, Florida 32207, United States
  • EndoCeutics site # 41
    West Palm Beach, Florida 33401, United States
  • EndoCeutics site # 23
    Sandy Springs, Georgia 30328, United States
  • EndoCeutics site # 10
    Meridian, Idaho 83642, United States
  • EndoCeutics site # 27
    Baltimore, Maryland 21285-6815, United States
  • EndoCeutics site # 22
    Kalamazoo, Michigan 49009, United States
  • EndoCeutics site # 25
    Lincoln, Nebraska 68510, United States
  • EndoCeutics site # 24
    Omaha, Nebraska 68131, United States
  • EndoCeutics site # 28
    Moorestown, New Jersey 08057, United States
  • EndoCeutics site # 50
    Neptune City, New Jersey 07753, United States
  • EndoCeutics site # 44
    New Brunswick, New Jersey 08901, United States
  • EndoCeutics site # 19
    New York, New York 10016, United States
  • EndoCeutics site # 16
    Durham, North Carolina 27713, United States
  • EndoCeutics site # 33
    Beachwood, Ohio 44122, United States
  • EndoCeutics site # 05
    Cleveland, Ohio 44122, United States
  • EndoCeutics site # 47
    Cleveland, Ohio 44124, United States
  • EndoCeutics site # 15
    Columbus, Ohio 43213, United States
  • EndoCeutics site # 35
    Pittsburgh, Pennsylvania 15206, United States
  • EndoCeutics site # 09
    West Jordan, Utah 84088, United States
  • EndoCeutics site # 31
    Charlottesville, Virginia 22903, United States
  • EndoCeutics site # 03
    Norfolk, Virginia 23507, United States
  • EndoCeutics site # 38
    Renton, Washington 98055, United States
  • EndoCeutics site # 13
    Calgary, Alberta T2N 4L7, Canada
  • EndoCeutics site # 06
    Bathurst, New Brunswick E2A 4X7, Canada
  • EndoCeutics site # 04
    Drummondville, Quebec J2B 7T1, Canada
  • EndoCeutics site # 34
    Montreal, Quebec H2X 3J4, Canada
  • EndoCeutics site # 12
    Montreal, Quebec H4N 3C5, Canada
  • EndoCeutics site # 02
    Quebec City, Quebec G1S 2L6, Canada
  • EndoCeutics site # 01
    Quebec City, Quebec G1V 2L9, Canada
  • EndoCeutics site # 08
    Shawinigan, Quebec G9N 2H6, Canada
  • EndoCeutics site # 11
    Sherbrooke, Quebec J1H 1Z1, Canada
  • EndoCeutics site # 18
    St-Romuald, Quebec G6W 5M6, Canada
08

References and documents

Publications

  • Labrie F, Archer DF, Bouchard C, Girard G, Ayotte N, Gallagher JC, Cusan L, Baron M, Blouin F, Waldbaum AS, Koltun W, Portman DJ, Cote I, Lavoie L, Beauregard A, Labrie C, Martel C, Balser J, Moyneur E; Members of the VVA Prasterone Group. Prasterone has parallel beneficial effects on the main symptoms of vulvovaginal atrophy: 52-week open-label study. Maturitas. 2015 May;81(1):46-56. doi: 10.1016/j.maturitas.2015.02.005. Epub 2015 Feb 16. PubMed 25771041 ↗
  • Bouchard C, Labrie F, Derogatis L, Girard G, Ayotte N, Gallagher J, Cusan L, Archer DF, Portman D, Lavoie L, Beauregard A, Cote I, Martel C, Vaillancourt M, Balser J, Moyneur E; VVA Prasterone Group. Effect of intravaginal dehydroepiandrosterone (DHEA) on the female sexual function in postmenopausal women: ERC-230 open-label study. Horm Mol Biol Clin Investig. 2016 Mar;25(3):181-90. doi: 10.1515/hmbci-2015-0044. PubMed 26725467 ↗
  • Ke Y, Gonthier R, Simard JN, Archer D, Lavoie L, Martel C, Vaillancourt M, Labrie F. Serum steroids remain within the same normal postmenopausal values during 12-month intravaginal 0.50% DHEA. Horm Mol Biol Clin Investig. 2015 Dec;24(3):117-29. doi: 10.1515/hmbci-2015-0035. PubMed 26509785 ↗
  • Portman DJ, Labrie F, Archer DF, Bouchard C, Cusan L, Girard G, Ayotte N, Koltun W, Blouin F, Young D, Wade A, Martel C, Dube R; other participating members of VVA Prasterone Group. Lack of effect of intravaginal dehydroepiandrosterone (DHEA, prasterone) on the endometrium in postmenopausal women. Menopause. 2015 Dec;22(12):1289-95. doi: 10.1097/GME.0000000000000470. PubMed 25968836 ↗
  • Martel C, Labrie F, Archer DF, Ke Y, Gonthier R, Simard JN, Lavoie L, Vaillancourt M, Montesino M, Balser J, Moyneur E; other participating members of the Prasterone Clinical Research Group. Serum steroid concentrations remain within normal postmenopausal values in women receiving daily 6.5mg intravaginal prasterone for 12 weeks. J Steroid Biochem Mol Biol. 2016 May;159:142-53. doi: 10.1016/j.jsbmb.2016.03.016. Epub 2016 Mar 10. PubMed 26972555 ↗
09

Registry details

Key details

Study ID
NCT01256671
Lead sponsor
EndoCeutics Inc.
Responsible party
Sponsor
First posted
Dec 8, 2010
Start date
Dec 2010
Primary completion
Jul 2012
Completion
Dec 2012
Results posted
Oct 18, 2017
Last update
Oct 18, 2017

Study contacts

David F Archer, MD
principal investigator · Clinical Research Center, Eastern Virginia Medical School, Norfolk, VA

Oversight

Data monitoring committee
Yes
View the source record on ClinicalTrials.gov ↗

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