A Treatment Study for Premenstrual Syndrome (PMS)

NCT ID: NCT00001259

Last Updated: 2023-03-02

Study Results

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

60 participants

Study Classification

INTERVENTIONAL

Study Start Date

1992-08-11

Study Completion Date

2020-02-06

Brief Summary

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This study examines the effects of estrogen and progesterone on mood, the stress response, and brain function and behavior in women with premenstrual syndrome.

Previously this study has demonstrated leuprolide acetate (Lupron (Registered Trademark)) to be an effective treatment for PMS. The current purpose of this study is to evaluate how low levels of estrogen and progesterone (that occur during treatment with leuprolide acetate) compare to menstrual cycle levels of estrogen and progesterone (given during individual months of hormone add-back) on a variety of physiologic measures (brain imaging, stress testing, etc.) in women with PMS.

PMS is a condition characterized by changes in mood and behavior that occur during the second phase of the normal menstrual cycle (luteal phase). This study will investigate possible hormonal causes of PMS by temporarily stopping the menstrual cycle with leuprolide acetate and then giving, in sequence, the menstrual cycle hormones progesterone and estrogen. The results of these hormonal studies will be compared between women with PMS and healthy volunteers without PMS (see also protocol 92-M-0174).

At study entry, participants will undergo a physical examination. Blood, urine, and pregnancy tests will be performed. Cognitive functioning and stress response will be evaluated during the study along with brain imaging and genetic studies.

Detailed Description

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This protocol is designed to accompany Clinical Protocol # 81-M-0126, The Phenomenology and Biophysiology of Menstrually-Related Mood and Behavioral Disorders. Its original purposes were as follows: 1) to evaluate the efficacy of the gonadotropin releasing hormone (GnRH) agonist depot leuprolide acetate (Lupron) in the treatment of menstrually-related mood disorders (MRMD) by determining whether mood and behavioral symptoms are eliminated when the cyclic secretion of both gonadotropic hormones and gonadal steroids is suppressed, and 2) to determine the possible relevance of gonadal steroids to affective state by sequentially replacing estradiol and progesterone during continued GnRH suppression in those patients whose premenstrual symptoms remit following administration of the GnRH agonist. We observed that GnRH agonist induced ovarian suppression was an effective treatment compared to placebo in women with MRMD. Additionally, women with MRMD but not asymptomatic controls (participating in companion protocol 92-M-0174) experienced a recurrence of mood and behavioral symptoms when either estradiol or progesterone (but not placebo) was added back. These data suggest that women with MRMD have a differential sensitivity to the mood destabilizing effects of gonadal steroids.

Having established that women with MRMD show a differential behavioral response to estrogen and progesterone, we now hope to identify the underlying mechanisms and physiologic concomitants of the differential behavioral sensitivity by performing studies (described in companion protocols) under the three hormonal conditions created by this protocol, and comparing results obtained with those seen in normal controls (Protocol #92-M-0174). Planned studies include the following: cognitive testing, brain imaging \[(3D-positron emission tomography (PET), functional magnetic resonance imaging (FMRI), magnetic resonance spectroscopy (MRS)\] and genetic studies including induced pluripotent cells.

Conditions

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Premenstrual Syndrome Menstruation Disturbances

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Investigators Outcome Assessors

Study Groups

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Study 1, Phase 1, Assignment 1 - Placebo

As part of double Blind randomized trial, participants in Assignment 1 were randomized to 8 weeks of placebo (P) injections (1 injection per month). These participants then continued on to Study 2 after completion of 8 weeks of placebo injections.

Group Type PLACEBO_COMPARATOR

Placebo injection

Intervention Type DRUG

Placebo given intramuscularly monthly

Study 1, Phase 1, Assignment 2 - GnRH agonist injections (Lupron-L only)

As part of double Blind randomized trial, participants in Assignment 2 were randomized to 8 weeks of GnRH agonist treatment 3.75 mg given intramuscularly monthly. Those who exhibited a remission of symptoms after 8 weeks continued on to receive one more month of GnRH agonist treatment (12 weeks total) and then entered Study 1, Phase 2.

Group Type EXPERIMENTAL

Leuprolide

Intervention Type DRUG

Eight to 12 weeks of GnRH agonist, Leuprolide Acetate 3.75 mg given intramuscularly monthly

Study 1, Phase 2, Arm 1 - Estradiol, then progesterone

12 weeks of GnRH agonist treatment 3.75 mg given intramuscularly monthly. Additionally, 4 weeks of transdermal Estradiol (100mcg/day by skin patch) and placebo suppositories. Week 5 involves 100mcg/day transdermal Estradiol and active Progesterone suppositories (200mg vaginally twice/day). Followed by 1-2 weeks (weeks 6-7) washout period. Then crossover to 5 weeks (week 8-12) of Progesterone suppositories (200mg vaginally twice/day) and placebo patches.

Group Type EXPERIMENTAL

Leuprolide

Intervention Type DRUG

Eight to 12 weeks of GnRH agonist, Leuprolide Acetate 3.75 mg given intramuscularly monthly

Estradiol Patches

Intervention Type DRUG

Transdermal Estradiol, 100mcg/day by skin patch

Progesterone

Intervention Type DRUG

Progesterone suppository, 200mg vaginally twice/day

Placebo patch

Intervention Type DRUG

Placebo by skin patch

Placebo suppository

Intervention Type DRUG

Placebo vaginal suppository

Study 1, Phase 2, Arm 2 - Progesterone, then estradiol

12 weeks of GnRH agonist treatment 3.75 mg given intramuscularly monthly. Additionally, 5 weeks of Progesterone suppositories (200mg vaginally twice/day) and placebo patches. Followed by 1-2 weeks (weeks 6-7) washout period. Then crossover to 4 weeks (weeks 8-11) of transdermal Estradiol (100mcg/day by skin patch) and placebo suppositories. Week 12 involves 100mcg/day transdermal Estradiol and active Progesterone suppositories (200mg vaginally twice/day).

Group Type EXPERIMENTAL

Leuprolide

Intervention Type DRUG

Eight to 12 weeks of GnRH agonist, Leuprolide Acetate 3.75 mg given intramuscularly monthly

Estradiol Patches

Intervention Type DRUG

Transdermal Estradiol, 100mcg/day by skin patch

Progesterone

Intervention Type DRUG

Progesterone suppository, 200mg vaginally twice/day

Placebo patch

Intervention Type DRUG

Placebo by skin patch

Placebo suppository

Intervention Type DRUG

Placebo vaginal suppository

Study 2, Phase 1 - GnRH agonist injections (Lupron-L only)

Eight to 12 weeks of GnRH agonist treatment 3.75 mg given intramuscularly monthly.

Group Type EXPERIMENTAL

Leuprolide

Intervention Type DRUG

Eight to 12 weeks of GnRH agonist, Leuprolide Acetate 3.75 mg given intramuscularly monthly

Study 2, Phase 2, Arm 1 - Estradiol, then progesterone

12 weeks of GnRH agonist treatment 3.75 mg given intramuscularly monthly. Additionally, 4 weeks of transdermal Estradiol (100mcg/day by skin patch) and placebo suppositories. Week 5 involves 100mcg/day transdermal Estradiol and active Progesterone suppositories (200mg vaginally twice/day). Followed by 1-2 weeks (weeks 6-7) washout period. Then crossover to 5 weeks (week 8-12) of Progesterone suppositories (200mg vaginally twice/day) and placebo patches.

Group Type EXPERIMENTAL

Leuprolide

Intervention Type DRUG

Eight to 12 weeks of GnRH agonist, Leuprolide Acetate 3.75 mg given intramuscularly monthly

Estradiol Patches

Intervention Type DRUG

Transdermal Estradiol, 100mcg/day by skin patch

Progesterone

Intervention Type DRUG

Progesterone suppository, 200mg vaginally twice/day

Placebo patch

Intervention Type DRUG

Placebo by skin patch

Placebo suppository

Intervention Type DRUG

Placebo vaginal suppository

Study 2, Phase 2, Arm 2 - Progesterone, then estradiol

12 weeks of GnRH agonist treatment 3.75 mg given intramuscularly monthly. Additionally, 5 weeks of Progesterone suppositories (200mg vaginally twice/day) and placebo patches. Followed by 1-2 weeks (weeks 6-7) washout period. Then crossover to 4 weeks (weeks 8-11) of transdermal Estradiol (100mcg/day by skin patch) and placebo suppositories. Week 12 involves 100mcg/day transdermal Estradiol and active Progesterone suppositories (200mg vaginally twice/day).

Group Type EXPERIMENTAL

Leuprolide

Intervention Type DRUG

Eight to 12 weeks of GnRH agonist, Leuprolide Acetate 3.75 mg given intramuscularly monthly

Estradiol Patches

Intervention Type DRUG

Transdermal Estradiol, 100mcg/day by skin patch

Progesterone

Intervention Type DRUG

Progesterone suppository, 200mg vaginally twice/day

Placebo patch

Intervention Type DRUG

Placebo by skin patch

Placebo suppository

Intervention Type DRUG

Placebo vaginal suppository

Interventions

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Leuprolide

Eight to 12 weeks of GnRH agonist, Leuprolide Acetate 3.75 mg given intramuscularly monthly

Intervention Type DRUG

Estradiol Patches

Transdermal Estradiol, 100mcg/day by skin patch

Intervention Type DRUG

Progesterone

Progesterone suppository, 200mg vaginally twice/day

Intervention Type DRUG

Placebo patch

Placebo by skin patch

Intervention Type DRUG

Placebo injection

Placebo given intramuscularly monthly

Intervention Type DRUG

Placebo suppository

Placebo vaginal suppository

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

The subjects of this study will be women who meet the criteria for MRMD as described in Protocol No. 81-M-0126, 'The Phenomenology and Biophysiology of Menstrually-related Mood and Behavioral Disorders.' In brief, these criteria include:

1. history within the last two years of at least six months with menstrually-related mood or behavioral disturbances of at least moderate severity--i.e., disturbances that are distinct in appearance and associated with a notable degree of subjective distress;
2. symptoms should have a sudden onset and offset;
3. age 18-50;
4. not pregnant and in good medical health;
5. medication free.

All patients participating in this protocol will have already participated in Protocol No. 81-M-0126 and will have a prospectively confirmed and predictable relationship between their mood disorder and the premenstrual phase of the menstrual cycle, i.e., a 30% change in severity of symptom self rating scales, relative to the range of the scale employed, during the seven days premenstrually compared with the seven days post-menstrually in two out of three months of study.

The Schedule for Affective Disorders and Schizophrenia will be administered to all patients prior to study entry. Any patient with a current axis I psychiatric diagnosis will be excluded from participating in this protocol.

Prior to treatment, a complete physical and neurological examination will have been performed and the following routine laboratory data obtained:

A. Blood

Complete blood count; thyroid function tests; cortisol; renal function tests, such as blood urea nitrogen (BUN) and creatinine; electrolytes; glucose; liver function tests.

B. Urine

Routine urinalysis; urine pregnancy test.

GnRH agonist will not be administered to any subject with significant clinical or laboratory abnormalities. The blood tests and urinalysis will be repeated 2 weeks after GnRH agonist administration to rule out any evidence of acute renal, hepatic or hematologic toxicity.

Results of Pap smear performed within one year of the onset of treatment will be obtained.

Exclusion Criteria

The following conditions will constitute contraindications to treatment with hormonal therapy and will preclude a subject's participation in this protocol:
* current Axis I psychiatric diagnosis
* history consistent with endometriosis,
* diagnosis of ill-defined, obscure pelvic lesions, particularly, undiagnosed ovarian enlargement,
* hepatic disease as manifested by abnormal liver function tests,
* history of mammary carcinoma,
* history of pulmonary embolism or phlebothrombosis
* undiagnosed vaginal bleeding
* porphyria
* diabetes mellitus
* history of malignant melanoma
* cholecystitis or pancreatitis,
* cardiovascular or renal disease
* pregnancy
* Any woman meeting the Stages of Reproductive Aging Workshop Criteria (STRAW) for the perimenopause. Specifically, we will exclude any woman with an elevated plasma follicle stimulating hormone (FSH) level (\>= 14 IU/L) and with menstrual cycle variability of \> 7 days different from their normal cycle length.
* Subjects taking birth control pills will be excluded from the study.
* Subjects taking diuretics, prostaglandin inhibitors, or pyridoxine (putative treatments for MRMD) will similarly be excluded from the study
* Patients taking psychotropic agents (e.g., lithium carbonate, tricyclic antidepressants).
* All subjects will be required to use non-hormonal forms of birth control (e.g., barrier methods) to avoid pregnancy during this study.
Minimum Eligible Age

18 Years

Maximum Eligible Age

45 Years

Eligible Sex

FEMALE

Accepts Healthy Volunteers

No

Sponsors

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National Institute of Mental Health (NIMH)

NIH

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Peter J Schmidt, M.D.

Role: PRINCIPAL_INVESTIGATOR

National Institute of Mental Health (NIMH)

Locations

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National Institutes of Health Clinical Center, 9000 Rockville Pike

Bethesda, Maryland, United States

Site Status

Countries

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United States

References

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Rubinow DR, Hoban MC, Grover GN, Galloway DS, Roy-Byrne P, Andersen R, Merriam GR. Changes in plasma hormones across the menstrual cycle in patients with menstrually related mood disorder and in control subjects. Am J Obstet Gynecol. 1988 Jan;158(1):5-11. doi: 10.1016/0002-9378(88)90765-x.

Reference Type BACKGROUND
PMID: 2962499 (View on PubMed)

Schmidt PJ, Nieman LK, Grover GN, Muller KL, Merriam GR, Rubinow DR. Lack of effect of induced menses on symptoms in women with premenstrual syndrome. N Engl J Med. 1991 Apr 25;324(17):1174-9. doi: 10.1056/NEJM199104253241705.

Reference Type BACKGROUND
PMID: 2011161 (View on PubMed)

Schmidt PJ, Nieman LK, Danaceau MA, Adams LF, Rubinow DR. Differential behavioral effects of gonadal steroids in women with and in those without premenstrual syndrome. N Engl J Med. 1998 Jan 22;338(4):209-16. doi: 10.1056/NEJM199801223380401.

Reference Type BACKGROUND
PMID: 9435325 (View on PubMed)

Li HJ, Goff A, Rudzinskas SA, Jung Y, Dubey N, Hoffman J, Hipolito D, Mazzu M, Rubinow DR, Schmidt PJ, Goldman D. Altered estradiol-dependent cellular Ca2+ homeostasis and endoplasmic reticulum stress response in Premenstrual Dysphoric Disorder. Mol Psychiatry. 2021 Nov;26(11):6963-6974. doi: 10.1038/s41380-021-01144-8. Epub 2021 May 25.

Reference Type DERIVED
PMID: 34035477 (View on PubMed)

Di Florio A, Alexander D, Schmidt PJ, Rubinow DR. Progesterone and plasma metabolites in women with and in those without premenstrual dysphoric disorder. Depress Anxiety. 2018 Dec;35(12):1168-1177. doi: 10.1002/da.22827. Epub 2018 Sep 5.

Reference Type DERIVED
PMID: 30184299 (View on PubMed)

Nguyen TV, Reuter JM, Gaikwad NW, Rotroff DM, Kucera HR, Motsinger-Reif A, Smith CP, Nieman LK, Rubinow DR, Kaddurah-Daouk R, Schmidt PJ. The steroid metabolome in women with premenstrual dysphoric disorder during GnRH agonist-induced ovarian suppression: effects of estradiol and progesterone addback. Transl Psychiatry. 2017 Aug 8;7(8):e1193. doi: 10.1038/tp.2017.146.

Reference Type DERIVED
PMID: 28786978 (View on PubMed)

Dubey N, Hoffman JF, Schuebel K, Yuan Q, Martinez PE, Nieman LK, Rubinow DR, Schmidt PJ, Goldman D. The ESC/E(Z) complex, an effector of response to ovarian steroids, manifests an intrinsic difference in cells from women with premenstrual dysphoric disorder. Mol Psychiatry. 2017 Aug;22(8):1172-1184. doi: 10.1038/mp.2016.229. Epub 2017 Jan 3.

Reference Type DERIVED
PMID: 28044059 (View on PubMed)

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Related Links

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Other Identifiers

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90-M-0088

Identifier Type: -

Identifier Source: secondary_id

900088

Identifier Type: -

Identifier Source: org_study_id

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