Estriol Treatment in Multiple Sclerosis (MS): Effect on Cognition

NCT ID: NCT01466114

Last Updated: 2019-11-06

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE2

Total Enrollment

64 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-10-31

Study Completion Date

2022-04-30

Brief Summary

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Approximately 50% of people diagnosed with Multiple Sclerosis (MS) will develop problems with cognition. Currently, there are no FDA-approved treatments targeting cognitive function in Multiple Sclerosis. This trial will ascertain whether treatment with an estrogen pill, used in combination with standard MS anti-inflammatory drugs, can improve cognitive testing as compared to treatment with a placebo pill in combination with standard anti-inflammatory drugs in women with MS.

Detailed Description

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Approximately 50% of people diagnosed with Multiple Sclerosis (MS) will develop problems with cognition. Currently, there are no FDA-approved treatments for cognitive function in Multiple Sclerosis. Multiple sclerosis relapses are known to be significantly decreased by approximately 80% during late pregnancy. This disease improvement may be due to estriol, an estrogen unique to pregnancy. Estriol blood levels go from undetectable levels prior to pregnancy, increase during pregnancy and reach highest levels during late pregnancy. Further, estrogen treatment has been shown to have favorable effects on cognition in animal models of other neurological diseases. This proposal will establish whether oral treatment with estriol, induces an improvement in cognitive functioning in subjects with multiple sclerosis when used in combination with the major FDA-approved standard treatments for MS, (Betaseron® (or Extavia®), Rebif®, Avonex®, Copaxone®, Gilenya®, Aubagio®, Tecfidera®, or Ocrevus®).

The combination of standard MS treatment plus estriol pill (8 mg per day) will be compared to standard MS treatment plus placebo in a double-blinded fashion. The duration of treatment will be one year and the primary outcome measure will be cognitive testing processing speed ability.

Secondary outcomes will be improvement in other cognitive tests, brain MRIs, cognitive evoked potentials, as well as relapse rates and disability measures (EDSS, 25 foot walk, 9 hole peg test, low contrast visual acuity, MS Quality of Life, Modified Fatigue Impact Scale, Beck Depression Inventory, Level of Activity using accelerometry). Safety measures (blood tests and gynecologic evaluations) will also be followed. The overall goal of this study will be the development of an oral treatment, estriol, to improve cognitive function in MS.

Conditions

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Relapsing-remitting Multiple Sclerosis Secondary-progressive Multiple Sclerosis Primary-progressive Multiple Sclerosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Investigators Outcome Assessors

Study Groups

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Group A: Estriol

Standard MS Treatment + Estriol

Group Type EXPERIMENTAL

estriol

Intervention Type DRUG

4 capsules of 2 mg (total of 8 mg) PO QD

Norethindrone

Intervention Type DRUG

Starting at month 6, and at Months 9 and 12: subjects who are on estriol (Group A) take 0.7 mg PO QD for 2 weeks.

Group B: Placebo

Standard MS Treatment + Placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type OTHER

4 capsules PO QD

Progestin Placebo

Intervention Type OTHER

Starting at Month 6 and at Months 9 and 12: subjects who are on placebo (Group B) take a second progestin placebo pill PO QD for 2 weeks.

Interventions

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estriol

4 capsules of 2 mg (total of 8 mg) PO QD

Intervention Type DRUG

Placebo

4 capsules PO QD

Intervention Type OTHER

Norethindrone

Starting at month 6, and at Months 9 and 12: subjects who are on estriol (Group A) take 0.7 mg PO QD for 2 weeks.

Intervention Type DRUG

Progestin Placebo

Starting at Month 6 and at Months 9 and 12: subjects who are on placebo (Group B) take a second progestin placebo pill PO QD for 2 weeks.

Intervention Type OTHER

Other Intervention Names

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Synapause Progestin

Eligibility Criteria

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Inclusion Criteria

* Diagnosis of clinically definite or MacDonald criteria relapsing-remitting multiple sclerosis, secondary-progressive multiple sclerosis or primary-progressive multiple sclerosis.
* No relapse within 30 days before day of trial enrollment (month 0 visit). If steroids given for relapse, then the month 0 visit must be 30 days after last steroid dose.
* Females age 18 to 55, inclusive.
* Expanded Disability Status Score (EDSS) = 0.0 to 6.0.
* Screening PASAT (3-second) score 25-50, inclusive.
* Must be mentally competent enough to comply with study guidelines and give informed consent.
* Must be willing and able to travel to the study center at frequencies in the protocol for a total period of 12 months.
* Patients must be on no treatment or be on a stable dose of one of the following agents for a minimum of 3 months duration prior to the month 0 visit: Copaxone®, Betaseron® (or Extavia®), Rebif®, Avonex®, PLEGRITY®, Ocrelizumab, Rituximab, Gilenya®, Aubagio®, or Tecfidera®. The time spent in the screening period may serve as part of this 3-month period.
* Patients who are currently being treated with ACTH, corticosteroids, intravenous immunoglobulins (IVIG), plasma exchange, Lipitor® or minocycline may be included.
* If patients plan to start treatment with Copaxone® or an interferon \[Betaseron® (or Extavia®), Rebif®, Avonex®, PLEGRITY®\], Ocrelizumab, Rituximabor an oral agent \[Gilenya®, Aubagio® or Tecfidera®\] and then they must be on for at least 3 months prior to month 0 (as above).

Exclusion Criteria

* Males
* Subjects on oral contraceptives (OCP), hormone replacement therapy (HRT) other sex hormones during screening and during the 12-month study period (Mirena® IUD is permitted).
* Females who are pregnant or who plan to become pregnant during the 12 months of enrollment, who wish to become pregnant within 3 months following completion of the study, or who will be within 6 months post partum at the day of first enrollment visit (month 0).
* Females who plan to breastfeed after first enrollment visit (month 0).
* Fertile sexually active women who are unwilling to practice reliable barrier methods of contraception other than oral contraceptives (i.e. condom, diaphragm, IUDs Note: Hormonal IUD \[Mirena®\] is permitted).
* Patients with surgical ovariectomy with no hormone replacement for 1 year or more.
* Menopause with no hormone replacement for 3 years or more prior to the first enrollment visit.
* Patients who smoke at any time during screening or during the 12 month study period.
* Patients who have serious pulmonary, renal, gastrointestinal, hepatic, immunologic, infectious, neoplastic, major psychiatric disease (major depression, schizophrenia), endocrine disease (including major diabetes, thyroid disease), or gynecologic disease, including but not limited to those with: Thrombophlebitis or thromboembolic disorders, a past history of deep vein thrombophlebitis or thromboembolic disorders, cerebral vascular or coronary artery disease, migraine with focal aura, known or suspected carcinoma of the breast, carcinoma of the endometrium or other known or suspected estrogen-dependent neoplasia, undiagnosed abnormal genital bleeding, polycystic ovary disease, amenorrhea of unknown etiology, cholestatic jaundice of pregnancy or jaundice with prior birth control pill use, acute or chronic hepatocellular disease with abnormal liver function, hepatic adenomas or carcinomas, known or suspected pregnancy, known hypersensitivity to birth control pill Copaxone or Betaseron use.
* B12 level \< 200.
* Drug abuse within the past five years.
* Conditions that would interfere with assessing neurologic functions such as deforming arthritis or a major amputation.
* Have at any time been treated with total lymphoid irradiation, monoclonal antibody, T cell vaccination, cladribine, bone marrow transplantation, azathioprine, cyclophosphamide, methotrexate, mitoxantrone, or cyclosporine.
* Have been treated with natalizumab (Tysabri®) in the 6 months prior to screening.
* Positive titers to HIV in the past.
* Previous serious adverse effects with estrogen treatment.
* Patients who participated in the previous multi-center estriol trial for RRMS ("A Combination Trial of Copaxone plus Estriol in RRMS").
Minimum Eligible Age

18 Years

Maximum Eligible Age

55 Years

Eligible Sex

FEMALE

Accepts Healthy Volunteers

No

Sponsors

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University of California, Los Angeles

OTHER

Sponsor Role lead

Responsible Party

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Rhonda Voskuhl

Professor, Department of Neurology; Director Multiple Sclerosis Program

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Rhonda Voskuhl, M.D.

Role: PRINCIPAL_INVESTIGATOR

University of California, Los Angeles

Locations

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University of California Los Angeles

Los Angeles, California, United States

Site Status RECRUITING

The University of Colorado Denver

Aurora, Colorado, United States

Site Status COMPLETED

The University of New Mexico

Albuquerque, New Mexico, United States

Site Status COMPLETED

The University of Pennsylvania

Philadelphia, Pennsylvania, United States

Site Status COMPLETED

Countries

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United States

Central Contacts

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Mike Montag, M.S.

Role: CONTACT

(310)206-2176

Facility Contacts

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Mike Montag, M.S.

Role: primary

310-206-2176

References

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Ziehn MO, Avedisian AA, Dervin SM, O'Dell TJ, Voskuhl RR. Estriol preserves synaptic transmission in the hippocampus during autoimmune demyelinating disease. Lab Invest. 2012 Aug;92(8):1234-45. doi: 10.1038/labinvest.2012.76. Epub 2012 Apr 23.

Reference Type BACKGROUND
PMID: 22525427 (View on PubMed)

Voskuhl RR, Wang H, Wu TC, Sicotte NL, Nakamura K, Kurth F, Itoh N, Bardens J, Bernard JT, Corboy JR, Cross AH, Dhib-Jalbut S, Ford CC, Frohman EM, Giesser B, Jacobs D, Kasper LH, Lynch S, Parry G, Racke MK, Reder AT, Rose J, Wingerchuk DM, MacKenzie-Graham AJ, Arnold DL, Tseng CH, Elashoff R. Estriol combined with glatiramer acetate for women with relapsing-remitting multiple sclerosis: a randomised, placebo-controlled, phase 2 trial. Lancet Neurol. 2016 Jan;15(1):35-46. doi: 10.1016/S1474-4422(15)00322-1. Epub 2015 Nov 29.

Reference Type BACKGROUND
PMID: 26621682 (View on PubMed)

Voskuhl R, Wang H, Elashoff RM. Why use sex hormones in relapsing-remitting multiple sclerosis? - Authors' reply. Lancet Neurol. 2016 Jul;15(8):790-791. doi: 10.1016/S1474-4422(16)00129-0. No abstract available.

Reference Type BACKGROUND
PMID: 27302354 (View on PubMed)

Voskuhl R, Patti F. Hormone replacement in menopausal women with multiple sclerosis: Looking back, thinking forward. Neurology. 2016 Oct 4;87(14):1430-1431. doi: 10.1212/WNL.0000000000003189. Epub 2016 Sep 7. No abstract available.

Reference Type BACKGROUND
PMID: 27605172 (View on PubMed)

Voskuhl R. Rebound Relapses After Ceasing Another Disease-Modifying Treatment in Patients With Multiple Sclerosis: Are There Lessons to Be Learned? JAMA Neurol. 2016 Jul 1;73(7):775-6. doi: 10.1001/jamaneurol.2016.0934. No abstract available.

Reference Type BACKGROUND
PMID: 27136400 (View on PubMed)

Itoh N, Kim R, Peng M, DiFilippo E, Johnsonbaugh H, MacKenzie-Graham A, Voskuhl RR. Bedside to bench to bedside research: Estrogen receptor beta ligand as a candidate neuroprotective treatment for multiple sclerosis. J Neuroimmunol. 2017 Mar 15;304:63-71. doi: 10.1016/j.jneuroim.2016.09.017. Epub 2016 Oct 3.

Reference Type BACKGROUND
PMID: 27771018 (View on PubMed)

Kim RY, Mangu D, Hoffman AS, Kavosh R, Jung E, Itoh N, Voskuhl R. Oestrogen receptor &beta; ligand acts on CD11c&plus; cells to mediate protection in experimental autoimmune encephalomyelitis. Brain. 2018 Jan 1;141(1):132-147. doi: 10.1093/brain/awx315.

Reference Type BACKGROUND
PMID: 29228214 (View on PubMed)

MacKenzie-Graham A, Brook J, Kurth F, Itoh Y, Meyer C, Montag MJ, Wang HJ, Elashoff R, Voskuhl RR. Estriol-mediated neuroprotection in multiple sclerosis localized by voxel-based morphometry. Brain Behav. 2018 Sep;8(9):e01086. doi: 10.1002/brb3.1086. Epub 2018 Aug 24.

Reference Type BACKGROUND
PMID: 30144306 (View on PubMed)

Voskuhl RR, Itoh N, Tassoni A, Matsukawa MA, Ren E, Tse V, Jang E, Suen TT, Itoh Y. Gene expression in oligodendrocytes during remyelination reveals cholesterol homeostasis as a therapeutic target in multiple sclerosis. Proc Natl Acad Sci U S A. 2019 May 14;116(20):10130-10139. doi: 10.1073/pnas.1821306116. Epub 2019 Apr 30.

Reference Type BACKGROUND
PMID: 31040210 (View on PubMed)

Voskuhl R. It is time to conduct phase 3 clinical trials of sex hormones in MS - Yes. Mult Scler. 2018 Oct;24(11):1413-1415. doi: 10.1177/1352458518768764. Epub 2018 Jul 30. No abstract available.

Reference Type BACKGROUND
PMID: 30058469 (View on PubMed)

Voskuhl R, Momtazee C. Pregnancy: Effect on Multiple Sclerosis, Treatment Considerations, and Breastfeeding. Neurotherapeutics. 2017 Oct;14(4):974-984. doi: 10.1007/s13311-017-0562-7.

Reference Type BACKGROUND
PMID: 28766273 (View on PubMed)

Other Identifiers

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11-002055

Identifier Type: -

Identifier Source: org_study_id

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