Uterine LeiomyomaPhase 2NCT00332033
Development of a Non-Invasive Treatment for Uterine Leiomyoma (Fibroids)
A Phase 2 study testing Pirfenidone in people with Uterine Leiomyoma and Fibroids, run by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) at 1 site in United States.
Trial summary
Uterine leiomyomas (fibroids) represent a major public health problem with few effective therapies. Currently, the only definitive treatment is hysterectomy and women are demanding alternative therapies to surgery. Read more about this study
- Who is it for?
- Ages 35 to 50 years, women only
- Conditions the study is for
- Uterine Leiomyoma, Fibroids
- What is being tested?
- Pirfenidone
- How many people
- 32 participants
- Where
- 1 siteUnited States
- Study dates
- 25 May 2006to 16 Feb 2007
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 35 to 50 years
- Women only
- No healthy volunteers
- Adult
Who may be able to join
- Parous and non-parous women who have completed child-bearing (age criteria of greater than 35 years-old, and less than 50 years-old).
- Currently practicing or willing to practice contraception throughout the duration of the study.
- Women with at least one fibroid greater than 4 cm diameter on ultrasound examination.
- Women who were planning to undergo hysterectomy for treatment of symptomatic fibroids may also participate if they are willing to defer the operation until the study is completed. The patients will understand that they may choose to have surgery or any other non-study treatment at any time after enrollment and end study participation.
Who cannot join
- Inability to comply with study visits and requirements.
- BMI greater than 33kg/m(2)
- History of a serious adverse reaction to study medication.
- Inability to tolerate MRI and 3D ultrasound procedures (i.e. presence of an intra-uterine device (IUD), pacemakers, aneurismal clips or other metallic devices that are not compatible with magnetic resonance imaging).
- Medical problems including: genetic diseases that cause fibroids, history of thromboembolic (blood clot) events or need for anticoagulation (Coumadin, Heparin, etc.).
- A history of cancer within the past 5 years.
Show all 15Show fewer
- Abnormal liver function tests (typically, will be greater than 20% elevation). Mild elevations will be at the discretion of the investigators, but undiagnosed liver conditions will represent an exclusion criterion.
- Pregnancy or lactation. Pregnancy will be evaluated by (Beta)hCG test every 30 days. Patients will be asked to use non-hormonal contraception methods while on study drug.
- Severe anemia (hct. Less than 30). Mild anemia is common in women with fibroids.
- Recent rapid growth of fibroids (i.e. doubling in size within one-six months).
- Use any of the following medications:
- Oral, injectable, or inhaled steroids or megesterol within the past year
- Estrogen or progesterone-containing compounds (including oral contraceptives, hormone replacement therapy, transdermal/injectable/vaginal/oral preparations, herbal medications with estrogenic or anti-estrogenic effects) within the past 8-12 weeks; or GnRH analogs (Danazol) or other compounds that affect menstrual cyclicity
- Agents like Imidazoles, due to possible interference with metabolism.
- Unevaluated gynecologic abnormalities (unexplained vaginal bleeding, cervical dysplasia, or abnormal adnexal/ovarian mass).
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, 20892
About this study
Uterine leiomyomas (fibroids) represent a major public health problem with few effective therapies. Currently, the only definitive treatment is hysterectomy and women are demanding alternative therapies to surgery. We have developed a new approach to the treatment of uterine fibroids based on collaborative laboratory research into the molecular, ultra-structural, and histopathologic changes that occur with the transformation of normal uterine myocytes into abnormal myocytes comprising uterine fibroids. We have confirmed that excessive, dysregulated collagen production (fibrosis) and abnormal collagen deposition is an underlying etiology in the pathogenesis of leiomyoma. We will test the hypothesis that an anti-tumor drug (Pirfenidone) will decrease the size of clinically relevant leiomyomas by 30%. The specific aim is to compare the effects of pirfenidone with placebo on uterine leiomyoma volume. Thirty-two (32) women will be randomized in a double-blinded treatment design. Inclusion criteria include women that have completed child-bearing, who are candidates for hysterectomy, are using effective contraceptive, and have at least one uterine leiomyoma greater than 4 cm diameter confirmed by ultrasound. Women will be excluded if they have a body mass index greater than 33 kg/m(2), other gynecological diseases, and history of cardiovascular disease or smoking. Response in each treatment group will be assessed by T-2 weighted magnetic resonance imaging (MRI) and 3-D ultrasound imaging studies during the enrollment period. To our knowledge, this will be the first study to document the response of large fibroids to a short-term trial of an anti-tumor drug. The data will be used to further define the role of fibrosis in leiomyoma and establish other clinical trials to thoroughly evaluate such therapeutic approaches for uterine leiomyomas.
Read the detailed description
Uterine leiomyomas (fibroids) represent a major public health problem with few effective therapies. Currently, the only definitive treatment is hysterectomy and women are demanding alternative therapies to surgery. We have developed a new approach to the treatment of uterine fibroids based on collaborative laboratory research into the molecular, ultra-structural, and histopathologic changes that occur with the transformation of normal uterine myocytes into abnormal myocytes comprising uterine fibroids. We have confirmed that excessive, dysregulated collagen production (fibrosis) and abnormal collagen deposition is an underlying etiology in the pathogenesis of leiomyoma. We will test the hypothesis that an anti-tumor drug (Pirfenidone) will decrease the size of clinically relevant leiomyomas by 30%. The specific aim is to compare the effects of pirfenidone with placebo on uterine leiomyoma volume. Thirty-two (32) women will be randomized in a double-blinded treatment design. Inclusion criteria include women that have completed child-bearing, who are candidates for hysterectomy, are using effective contraceptive, and have at least one uterine leiomyoma greater than 4 cm diameter confirmed by ultrasound. Women will be excluded if they have a body mass index greater than 33 kg/m(2), other gynecological diseases, and history of cardiovascular disease or smoking. Response in each treatment group will be assessed by T-2 weighted magnetic resonance imaging (MRI) and 3-D ultrasound imaging studies during the enrollment period. To our knowledge, this will be the first study to document the response of large fibroids to a short-term trial of an anti-tumor drug. The data will be used to further define the role of fibrosis in leiomyoma and establish other clinical trials to thoroughly evaluate such therapeutic approaches for uterine leiomyomas.
- Conditions
- Uterine Leiomyoma, Fibroids
- Being tested
- Pirfenidone
- Keywords
- Fibroids, Leiomyoma, Pirfenidone, Misoprostol, Non-Surgical, Collagen, Uterus, Uterine Fibroids, Uterine Leiomyoma
- Medical subject headings
- Myofibroma, Leiomyoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PirfenidoneDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jun 2006 (estimated)
- Study start25 May 2006
- Study completion16 Feb 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Uterine LeiomyomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Enrolment
- 32 participants
Sponsor and investigators
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- Sponsor type
- US National Institutes of Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00332033
- Sponsor's protocol ID
- 060029
- Other IDs
- 06-CH-0029
- Status last verified
- 16 Feb 2007
- First submitted
- 31 May 2006
- Last updated
- 2 Jul 2017
- Results posted
- No
Publications
Marshall LM, Spiegelman D, Barbieri RL, Goldman MB, Manson JE, Colditz GA, Willett WC, Hunter DJ. Variation in the incidence of uterine leiomyoma among premenopausal women by age and race. Obstet Gynecol. 1997 Dec;90(6):967-73. doi: 10.1016/s0029-7844(97)00534-6.
PubMed 9397113
Study at a glance
From the registry record, last updated by the sponsor on 2 Jul 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Development of a Non-Invasive Treatment for Uterine Leiomyoma (Fibroids)
- ClinicalTrials.gov ID
- NCT00332033
- Acronym / sponsor ID
- 060029
- Conditions
- Uterine Leiomyoma, Fibroids
- Keywords
- Fibroids, Leiomyoma, Pirfenidone, Misoprostol, Non-Surgical, Collagen, Uterus, Uterine Fibroids
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 32 participants
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)US National Institutes of Health
- Study start
- 25 May 2006
- Study completion
- 16 Feb 2007
- First posted
- 1 Jun 2006 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Uterine leiomyomas (fibroids) represent a major public health problem with few effective therapies. Currently, the only definitive treatment is hysterectomy and women are demanding alternative therapies to surgery. We have developed a new approach to the treatment of uterine fibroids based on collaborative laboratory research into the molecular, ultra-structural, and histopathologic changes that occur with the transformation of normal uterine myocytes into abnormal myocytes comprising uterine fibroids. We have confirmed that excessive, dysregulated collagen production (fibrosis) and abnormal collagen deposition is an underlying etiology in the pathogenesis of leiomyoma. We will test the hypothesis that an anti-tumor drug (Pirfenidone) will decrease the size of clinically relevant leiomyomas by 30%. The specific aim is to compare the effects of pirfenidone with placebo on uterine leiomyoma volume. Thirty-two (32) women will be randomized in a double-blinded treatment design. Inclusion criteria include women that have completed child-bearing, who are candidates for hysterectomy, are using effective contraceptive, and have at least one uterine leiomyoma greater than 4 cm diameter confirmed by ultrasound. Women will be excluded if they have a body mass index greater than 33 kg/m(2), other gynecological diseases, and history of cardiovascular disease or smoking. Response in each treatment group will be assessed by T-2 weighted magnetic resonance imaging (MRI) and 3-D ultrasound imaging studies during the enrollment period. To our knowledge, this will be the first study to document the response of large fibroids to a short-term trial of an anti-tumor drug. The data will be used to further define the role of fibrosis in leiomyoma and establish other clinical trials to thoroughly evaluate such therapeutic approaches for uterine leiomyomas.
Detailed description
Uterine leiomyomas (fibroids) represent a major public health problem with few effective therapies. Currently, the only definitive treatment is hysterectomy and women are demanding alternative therapies to surgery. We have developed a new approach to the treatment of uterine fibroids based on collaborative laboratory research into the molecular, ultra-structural, and histopathologic changes that occur with the transformation of normal uterine myocytes into abnormal myocytes comprising uterine fibroids. We have confirmed that excessive, dysregulated collagen production (fibrosis) and abnormal collagen deposition is an underlying etiology in the pathogenesis of leiomyoma. We will test the hypothesis that an anti-tumor drug (Pirfenidone) will decrease the size of clinically relevant leiomyomas by 30%. The specific aim is to compare the effects of pirfenidone with placebo on uterine leiomyoma volume. Thirty-two (32) women will be randomized in a double-blinded treatment design. Inclusion criteria include women that have completed child-bearing, who are candidates for hysterectomy, are using effective contraceptive, and have at least one uterine leiomyoma greater than 4 cm diameter confirmed by ultrasound. Women will be excluded if they have a body mass index greater than 33 kg/m(2), other gynecological diseases, and history of cardiovascular disease or smoking. Response in each treatment group will be assessed by T-2 weighted magnetic resonance imaging (MRI) and 3-D ultrasound imaging studies during the enrollment period. To our knowledge, this will be the first study to document the response of large fibroids to a short-term trial of an anti-tumor drug. The data will be used to further define the role of fibrosis in leiomyoma and establish other clinical trials to thoroughly evaluate such therapeutic approaches for uterine leiomyomas.
Study design
- Primary purpose
- Treatment
Who may qualify
- Ages
- 35 to 50 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (4 listed)
- Parous and non-parous women who have completed child-bearing (age criteria of greater than 35 years-old, and less than 50 years-old).
- Currently practicing or willing to practice contraception throughout the duration of the study.
- Women with at least one fibroid greater than 4 cm diameter on ultrasound examination.
- Women who were planning to undergo hysterectomy for treatment of symptomatic fibroids may also participate if they are willing to defer the operation until the study is completed. The patients will understand that they may choose to have surgery or any other non-study treatment at any time after enrollment and end study participation.
Key exclusion criteria (15 listed)
- Inability to comply with study visits and requirements.
- BMI greater than 33kg/m(2)
- History of a serious adverse reaction to study medication.
- Inability to tolerate MRI and 3D ultrasound procedures (i.e. presence of an intra-uterine device (IUD), pacemakers, aneurismal clips or other metallic devices that are not compatible with magnetic resonance imaging).
- Medical problems including: genetic diseases that cause fibroids, history of thromboembolic (blood clot) events or need for anticoagulation (Coumadin, Heparin, etc.).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Pirfenidone | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Jul 2017.
- Background: PubMed 9397113Marshall LM, Spiegelman D, Barbieri RL, Goldman MB, Manson JE, Colditz GA, Willett WC, Hunter DJ. Variation in the incidence of uterine leiomyoma among premenopausal women by age and race. Obstet Gynecol. 1997 Dec;90(6):967-73. doi: 10.1016/s0029-7844(97)00534-6.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Pirfenidone in people with Uterine Leiomyoma and Fibroids, run by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 2 Jul 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00332033, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Jul 2017.