Tranexamic Acid in Adolescents With Heavy Menstrual Bleeding

This is a study to learn more about a drug called Tranexamic acid (TA), otherwise known as Lysteda, and whether or not this drug can decrease menstrual blood loss in young women and lead to an increase in the quality of life.

Menorrhagia in young women with bleeding disorders is typically treated with a combination of treatments including hormonal contraceptives. However, there are barriers to hormonal contraception use in younger adolescents.

Tranexamic acid is taken orally during the first 5 days of menstrual bleeding.

The purposes of this study include:

To test the safety and efficacy of Lysteda in adolescent females. To learn how well Lysteda works in decreasing menstrual blood loss. To see if parents and children participating in this study think the drug is improving their quality of life.

Lysteda has been approved by the Food and Drug Administration for use in patients > than 18 years of age but not for younger patients.

Study Overview

• Status: Completed
• Conditions: Heavy Menstrual Bleeding
• Intervention / Treatment: Drug: Tranexamic Acid

Detailed Description

This is a multi-center (5 sites) open-label, single-arm, efficacy study of Lysteda in the treatment of adolescent females (10-19 years of age) with heavy menstrual bleeding (HMB). Each subject will receive oral Lysteda 1.3 grams three times daily during the first five days of menses. Subjects will be monitored over the course of four menstrual cycles (1 pre-treatment cycle and 3 treatment cycles). During all cycles, assessments of menstrual bleeding will be performed using the Menorrhagia Impact Questionnaire (MIQ) and Pictorial Blood Assessment Chart (PBAC).

The study will begin as a currently funded pilot study to assess the efficacy of Lysteda in 32 patients followed to study completion.

• Study Type: Interventional
• Enrollment (Actual): 32
• Phase: Phase 4

Contacts and Locations

Study Locations

• United States
  • Indiana
    • Indianapolis, Indiana, United States, 46202
      • Riley Hospital for Children at IU Health
  • Missouri
    • Kansas City, Missouri, United States, 64108
      • Children's Mercy Hospital
  • Ohio
    • Akron, Ohio, United States, 44302
      • Akron Children's Hospital
    • Cleveland, Ohio, United States, 44106
      • Rainbow Babies & Children's Hospital
    • Columbus, Ohio, United States, 43205
      • Nationwide Children's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 10 years to 19 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Female

Description

Inclusion Criteria:

1. Menstruating females 10-19 years of age
2. Non-smoker
3. Physician and patient have agreed to initiate Lysteda
4. Diagnosis of HMB based on the medical judgment of the principal or site investigator
5. Subjects must report menstrual periods occurring within 21-60 days from the start of one period to the start of the next menstrual period
6. Negative pregnancy test
7. Informed consent obtained and signed
8. Informed assent obtained and signed
9. Understanding of study procedures
10. Ability to comply with study procedures for the entire length of the study
11. Subjects should be either sexually inactive (abstinent) or agree to use a barrier method with spermicide in the event of sexual activity throughout the study period

Exclusion Criteria:

1. Active thromboembolic disease, history of thromboembolic disease (including retinal vein or artery occlusion), known inherited thrombophilia, or family history of thrombosis in a first degree relative
2. Subject has a severe medical or psychiatric illness that, in the opinion of the Investigator, would affect subject safety or compliance
3. Clinical evidence of severe bleeding disorder. Patients with mild bleeding disorders such as type 1 von Willebrand disease, mild platelet function defects such as platelet storage pool or release defects, and patients with bleeding due to Ehlers Danlos syndrome WILL be eligible to participate in the study.
4. Pregnancy within the past 6 months and/or breast-feeding
5. Use of hormonal contraception (estrogen and progestin) within 3 months of study entry, or anticipated need to initiate estrogen-containing hormonal contraception during the study period
6. Use of systemic steroids within 1 month of study entry
7. History of subarachnoid hemorrhage
8. History of Hepatitis B, C, or HIV
9. Baseline creatinine >20% above the upper limit of normal for age
10. Severe anemia (hemoglobin <8 g/dL)
11. Systolic blood pressure <85 or diastolic blood pressure <55
12. Heart rate <50 at time of screening
13. Use of intranasal DDAVP during menses will be permitted, but only if the patient has a history of using DDAVP consistently for ≥3 menstrual cycles prior to study enrollment, so that change in menstrual blood loss due to addition of Lysteda can be assessed. Use of one-time DDAVP during a DDAVP/Stimate challenge is also permitted during the study period, as is use of DDAVP in the event of severe epistaxis, trauma, or surgical procedures during the study period.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Tranexamic acid; Subjects will complete a baseline menses (no treatment) followed by 3 menses using tranexamic acid.	Drug: Tranexamic Acid; Subjects will be instructed to take 2 tablets (1300) mg of Lysteda three times daily (3900 mg/daily) for five days during monthly menstruation.; Other Names: Lysteda

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change From Baseline Menses for Participant Perceived Blood Loss	Menorrhagia Impact Questionnaire (MIQ): Question 1 - participant perceived blood loss during most recent menses. Scale ranges from a score of 1 to 4 (with 1 being light blood loss and 4 being very heavy blood loss). Lower values indicate a better outcome (less blood loss during menses). Unit of measure is scores on a scale.	Baseline menses (no treatment) and 3 menstrual cycles treated with tranexamic acid

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change From Baseline Menses for Participant Perceived Limitation of School Attendance	Menorrhagia Impact Questionnaire (MIQ): Question 2 - participant perceived limitation of school attendance during most recent menses. Scale ranges from a score of 1 to 5 (with 1 being school attendance not at all limited and 5 being school attendance limited extremely). Lower values indicate a better outcome (less limitation of school attendance). Unit of measure is scores on a scale.	Baseline menses (no treatment) and 3 menstrual cycles treated with tranexamic acid
Change From Baseline Menses for Participant Perceived Limitation in Physical Activities	Menorrhagia Impact Questionnaire (MIQ): Question 3 - participant perceived limitation in physical activities during most recent menses. Scale ranges from a score of 1 to 5 (with 1 being physical activities not at all limited and 5 being physical activities extremely limited). Lower values indicate a better outcome (less limitation of physical activities). Unit of measure is scores on a scale.	Baseline menses (no treatment) and 3 menstrual cycles treated with tranexamic acid
Change From Baseline Menses for Participant Perceived Limitation of Social or Leisure Activities	Menorrhagia Impact Questionnaire (MIQ): Question 4 - participant perceived limitation of social or leisure during most recent menses. Scale ranges from a score of 1 to 5 (with 1 being social or leisure activities not at all limited and 5 being social or leisure activities limited extremely). Lower values indicate a better outcome (less limitation of social or leisure activities). Unit of measure is scores on a scale.	Baseline menses (no treatment) and 3 menstrual cycles treated with tranexamic acid
Change From Baseline Menses for Menstrual Blood Loss as Measured by Pictorial Blood Assessment Chart (PBAC) Scores	Pictorial Blood Assessment Chart (PBAC) scores - participant assessment of menstrual blood loss during menses using a pictorial chart to score menstrual blood loss. Pictorial scores range from 1 point for mild soaking of a pad/tampon, 5 points for moderate soaking of a pad/tampon, 10 points for severe soaking of a pad/tampon, and 5 points for each episode of flooding and for each blood clot larger than a quarter in size. Lower values indicate a better outcome (less blood loss). Unit of measure is a total computed score (all points during the menses from the pictorial chart added together).	Baseline menses (no treatment) and 3 menstrual cycles treated with tranexamic acid
Change From Baseline Menses for Hemoglobin Lab Value	Hemoglobin lab value normal range is 12 g/dL to 16 g/dL. Values in the range of 12 g/dL to 16 g/dL would be considered normal Hemoglobin values. A score lower than the normal range (below 12 g/dL) would indicate a worse outcome. Unit of measure is g/dL.	Baseline menses (no treatment) and 3 menstrual cycles treated with tranexamic acid
Change From Baseline Menses for Ferritin Lab Value	Ferritin lab value normal range is 7 ng/mL to 142 ng/mL. Values in the range of 7ng/mL to 142 ng/mL would be considered normal Ferritin values. A score lower than the normal range (below 7 ng/mL) would indicate a worse outcome. Unit of measure is ng/mL.	Baseline menses (no treatment) and 3 menstrual cycles treated with tranexamic acid

Collaborators and Investigators

• Sponsor: Nationwide Children's Hospital
• Investigators: Principal Investigator: Sarah O'Brien, MD, MSc, Nationwide Children's Hospital

Study record dates

Study Major Dates

• Study Start: April 1, 2013
• Primary Completion (Actual): November 1, 2016
• Study Completion (Actual): November 1, 2016

Study Registration Dates

• First Submitted: May 1, 2013
• First Submitted That Met QC Criteria: May 1, 2013
• First Posted (Estimate): May 3, 2013

Study Record Updates

• Last Update Posted (Actual): October 24, 2018
• Last Update Submitted That Met QC Criteria: September 28, 2018
• Last Verified: September 1, 2018

More Information

Terms related to this study

• Keywords: HMB; Heavy Menstrual Bleeding; Lysteda
• Additional Relevant MeSH Terms: Pathologic Processes; Uterine Diseases; Menstruation Disturbances; Uterine Hemorrhage; Hemorrhage; Menorrhagia; Molecular Mechanisms of Pharmacological Action; Fibrin Modulating Agents; Antifibrinolytic Agents; Hemostatics; Coagulants; Tranexamic Acid
• Other Study ID Numbers: NCH12-00822