Clinical trial NCT01846507: Tranexamic Acid in Adolescents With Heavy Menstrual Bleeding
Official title: Tranexamic Acid in Adolescents With Heavy Menstrual Bleeding
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Heavy Menstrual Bleeding
Keywords: Heavy Menstrual Bleeding
HMB
Lysteda
Lead sponsor: Nationwide Children's Hospital
Sponsor class: OTHER
First posted: 2013-05-03
Enrollment: 32
Sex: FEMALE
Minimum age (years): 10
Maximum age (years): 19
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Change From Baseline Menses for Participant Perceived Blood Loss
timeFrame: Baseline menses (no treatment) and 3 menstrual cycles treated with tranexamic acid
Secondary outcomes:
measure: Change From Baseline Menses for Participant Perceived Limitation of School Attendance
timeFrame: Baseline menses (no treatment) and 3 menstrual cycles treated with tranexamic acid
measure: Change From Baseline Menses for Participant Perceived Limitation in Physical Activities
timeFrame: Baseline menses (no treatment) and 3 menstrual cycles treated with tranexamic acid
measure: Change From Baseline Menses for Participant Perceived Limitation of Social or Leisure Activities
timeFrame: Baseline menses (no treatment) and 3 menstrual cycles treated with tranexamic acid
measure: Change From Baseline Menses for Menstrual Blood Loss as Measured by Pictorial Blood Assessment Chart (PBAC) Scores
timeFrame: Baseline menses (no treatment) and 3 menstrual cycles treated with tranexamic acid
measure: Change From Baseline Menses for Hemoglobin Lab Value
timeFrame: Baseline menses (no treatment) and 3 menstrual cycles treated with tranexamic acid
measure: Change From Baseline Menses for Ferritin Lab Value
timeFrame: Baseline menses (no treatment) and 3 menstrual cycles treated with tranexamic acid
Other outcomes:
Locations:
city: Indianapolis
country: United States
facility: Riley Hospital for Children at IU Health
state: Indiana
city: Kansas City
country: United States
facility: Children's Mercy Hospital
state: Missouri
city: Akron
country: United States
facility: Akron Children's Hospital
state: Ohio
city: Cleveland
country: United States
facility: Rainbow Babies & Children's Hospital
state: Ohio
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
Study officials:
affiliation: Nationwide Children's Hospital
name: Sarah O'Brien, MD, MSc
role: PRINCIPAL_INVESTIGATOR
References: