HemophiliaEarly Phase 1NCT03272568
Extended Half Life Factor (EHF) Products For Heavy Menstrual Bleeding in Hemophilia Carriers
A Early Phase 1 study testing Recombinant FVIII Fc fusion product Eloctate, Recombinant FIX Fc fusion product Alprolix and Patient-operated diagnostic device for anemia AnemoCheck. in people with Hemophilia and Menstrual Flow Excessive, run by Emory University at 1 site in United States.
Trial summary
The purpose of this feasibility study is to find out if two clotting factor products, Eloctate [hemophilia A] and Alprolix [hemophilia B], can reduce the amount of menstrual bleeding in female hemophilia A and B carriers (14 years of age or older) who have severe or heavy bleeding. Read more about this study
- Who is it for?
- Ages 14 to 55 years, women only
- Conditions the study is for
- Hemophilia, Menstrual Flow Excessive
- What is being tested?
- Recombinant FVIII Fc fusion product Eloctate, Recombinant FIX Fc fusion product Alprolix and Patient-operated diagnostic device for anemia AnemoCheck.
- Type of treatment
- Injection or infusion, Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteUnited States
- Study dates
- 14 Feb 2018to 31 Dec 2019
- Phase
- Early Phase 1
- 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 14 to 55 years
- Women only
- No healthy volunteers
- Child, Adult
Who may be able to join
- Females of reproductive age who experience monthly menstrual bleedings
- Female Hemophilia A or B carrier (heterozygote, lyonized or Turner's Syndrome)
- FVIII or FIX activity ≤60% at time of the study
- Baseline Pictorial Bleeding Assessment Chart >150 mean at time of recruitment
- Negative pregnancy test at time of enrollment
- Both female and her male partner have agreed to use an acceptable barrier method of birth control (e.g., diaphragm, cervical cap, male condom, female condom, and spermicidal foam, sponges, and film) throughout the duration of this study (for sexually active participants)
Who cannot join
- Has not reached menarche
- Menopause: natural or induced by surgical/medical treatment
- Pregnant or breasfeeding
- Female or her male partner refuses to use barrier method of birth control (for sexually active)
- Current use of any of the following contraceptives (copper IUD, oral combined, Progestin-only including but not limited to: Provera, Aygestin, Nexplanon, Implanon, Depo Provera, Mirena IUD)
- VWF:Ag or VWF:RCo <40%
Show all 12Show fewer
- Diagnosis of a qualitative platelet disorder
- Personal history of thrombosis or superficial thrombosis
- First degree relative with a history of thrombosis
- Personal history of concomitant bleeding or clotting disorder
- Cigarette smoker
- Willing to avoid taking an anti-fibrinolytic agent (amicar, transexamic acid, lysteda) during the trial
Trial locations and contacts
1 site in 1 country.
United States1 site
Emory University Hospital Clinical Research Network
Atlanta, Georgia, 30322
About this study
Official title: A Feasibility Trial Using Extended Half Life Factor Products in the Reduction of Menstrual Bleeding in Symptomatic Hemophilia A and B Carriers
The purpose of this feasibility study is to find out if two clotting factor products, Eloctate [hemophilia A] and Alprolix [hemophilia B], can reduce the amount of menstrual bleeding in female hemophilia A and B carriers (14 years of age or older) who have severe or heavy bleeding. These products are FDA-approved for use in males with hemophilia A and B to prevent and treat bleeding. They are not approved specifically to reduce menstrual bleeding, but may be useful for this purpose. Both products have an "extended half life" which means they circulate in the body longer than other FVIII or FIX products. The study team will gather additional information about the safety of these drugs and how well they work. The results of this feasibility study will provide information for an upcoming larger study.
Read the detailed description
Hemophilia A or B is caused by defects in the factor VIII or IX gene, respectively, of which is located on the X chromosome. This disorder exhibits X-linked inheritance, in which primarily males, with a single X chromosome, are affected and females, with two X chromosomes, are heterozygotes, or carriers. Hemophilia carriers show a wide distribution of factor VIII or IX levels with a mean of 50%, which overlaps the distribution of non-carrier women.
Heavy menstrual bleeding is defined as menstrual bleeding that lasts more than 7 days or more specifically as the loss of more than 80cc of blood per cycle. Management is critical as it can lead to iron deficiency anemia, lead to school absence and affects the general quality of life. There are multiple options to control or reduce menstrual bleeding in hemophilia A and B carriers, they include but not limited to the following options: Antifibrinolytics (Aminocaproic acid, Tranexamic acid), Synthetic desmopressin (DDAVP, SQ or IN) or hormonal therapies (Combined oral contraceptives, Progestin only options, IUD, etc).
The use of recombinant factor replacement has been poorly studied and limited by a relatively short half-life in relation to the typical length of a menstrual period. The purpose of this feasibility study is to find out if two clotting factor products, Eloctate [hemophilia A] and Alprolix [hemophilia B], can reduce the amount of menstrual bleeding in female hemophilia A and B carriers (14 years of age or older) who have severe or heavy bleeding. These products are FDA-approved for use in males with hemophilia A and B to prevent and treat bleeding. They are not approved specifically to reduce menstrual bleeding, but may be useful for this purpose. Both products have an "extended half life" which means they circulate in the body longer than other FVIII or FIX products. The study team will gather additional information about the safety of these drugs and how well they work. Five out of the 16 anticipated enrolled patients will be approach to request participation in the exploratory aim in which the subject is taught to perform a hemoglobin check (CBC) every other day using a novel point of care device (Anemocheck) during 2 consecutive menstrual cycles. In addition, they will have CBCs drawn within 4-6 hours for comparison and correlation. The study team will also correlate symptoms of heavy menstrual bleeding with a 2grams drop in the hemoglobin. All the results of this study will provide information for an upcoming larger study.
- Conditions
- Hemophilia, Menstrual Flow Excessive
- Being tested
- Recombinant FVIII Fc fusion product Eloctate, Recombinant FIX Fc fusion product Alprolix, Patient-operated diagnostic device for anemia AnemoCheck.
- Keywords
- Extended Life Plasma Derived Factor Products, Female hemophilia A and B carriers, Menorrhagia
- Medical subject headings
- Hemophilia A, Menorrhagia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Recombinant FVIII Fc fusion product EloctateDrug
Also called Eloctate
Subjects will get FVIII activity corrected from the baseline level to 100-150% with Eloctate on the first day of menstruation at the beginning of the study interventional phase. The first dose of the drug will be given in the clinics via slow injection into a vein (infusion). A short assessment of pharmacokinetics (PK) (4 time-points blood draw: 15 min, 2 hours, 4 hours, 20 hours after the infusion) will be performed to assist with subsequent dosing. 2 total doses of Eloctate likely to be given on day 1 and 3 of menstrual period. The subject will then receive three additional PK-adjusted monthly doses of Eloctate (with no less than 21 days interval).
Recombinant FIX Fc fusion product AlprolixDrug
Also called Alprolix
Subjects will get FIX activity corrected from the baseline level to 100-150% with Alprolix on the first day of menstruation at the beginning of the study interventional phase. The first dose of the drug will be given in the clinics via slow injection into a vein (infusion). A short assessment of pharmacokinetics (PK) (4 time-points blood draw: 15 min, 4 hours, 48 hours, 72 hours after the infusion) will be performed (by home nursing or local lab) to assist with subsequent dosing. Single dose of Alprolix likely to be given on day 1 of menstrual period. The subject will then receive three additional PK-adjusted monthly doses of Alprolix (with no less than 21 days interval).
Patient-operated diagnostic device for anemia AnemoCheck.Device
Also called AnemoCheck
Each AnemoCheck test kit enables patients to check hemoglobin levels by pricking their finger to draw 5 μL of blood into a capillary tube, inserting that tube into a larger tube with a pre-filled chemical solution, mixing, waiting one minute, and assessing the color change of the solution using a reference color scale card included with the kit. The test is able to subjectively differentiate low, medium and high levels of hemoglobin to diagnose anemia at home. Study participants are taught to perform a hemoglobin check every other day using Anemocheck during 2 consecutive menstrual cycles. In addition they will have hemoglobin level obtained locally (LabCorp) drawn within 4-6 hours for comparison and correlation.
| Study group | Role | What they receive |
|---|---|---|
| Hemophilia A symptomatic female carriers | Experimental | Hemophilia A symptomatic female carriers with a baseline FVIII activity of ≤60% receive recombinant FVIII Fc fusion product Eloctate.
Some of participants will be taught to perform a hemoglobin check using a patient-operated diagnostic device for anemia AnemoCheck every other day during 2 consecutive menstrual cycles. Receives: Drug: Recombinant FVIII Fc fusion product Eloctate; Device: Patient-operated diagnostic device for anemia AnemoCheck. |
| Hemophilia B symptomatic female carriers | Experimental | Hemophilia B symptomatic female carriers with a baseline FIX activity of ≤60% receive recombinant FIX Fc fusion product Alprolix.
Some of participants will be taught to perform a hemoglobin check using a patient-operated diagnostic device for anemia AnemoCheck every other day during 2 consecutive menstrual cycles. Receives: Drug: Recombinant FIX Fc fusion product Alprolix; Device: Patient-operated diagnostic device for anemia AnemoCheck. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Reduction in menstrual bleeding measured by the Pictorial Bleeding Assessment Chart (PBAC).
Menstrual loss is tracked via paper and electronic PBAC, calculated via visual interpretation centrally and correlated to the ability of a participant to correctly assign pad or tampon saturation. The PBAC will be estimated (mean) over the three-month intervention period to assess if 30% reduction in menstrual bleeding was achieved. PBAC is a semi-quantitative, self-administered pictorial tool. The total score is calculated by adding up the sum of all scores for the tampons or pads used, with a score greater than 150 having an 86% sensitivity and 89% specificity for heavy menstrual bleeding (HMB). Possible range is dependent on quantitative and qualitative amount of bleeding during menstruation:
- For tampons: 0.5-1 for lightly stained, 1-4 for moderately soiled and 4-12 for completely saturated tampons.
- For pads: 1 for lightly stained, 2-6 for moderately soiled and 4-15 for completely saturated pads.
- Clots were given a score of 1 for small and 5 for large clots.
Measured: Monthly throughout study duration for 7 months
Secondary outcomes (7)
Change in Patient-Reported Outcomes Measurement Information Systems (PROMIS-43) score.
To determine whether the use of the study drugs leads to a significant improvement in Quality of Life (QOL) the study establishes a baseline QOL and measures change monthly and 6-month change. The PROMIS 43 profile is a QOL assessment tool of 43 questions from 9 health domains and 10 questions focused on sexual health. The PROMIS assesses patient reported outcomes in three major categories: physical health, mental health, and social health. Each item has five response options except for the Pain Intensity item which has eleven options.A raw score is created from each short form. To find the total raw score the values of the response to each question are summed up within each domain. The applicable score conversion table translates the total raw score into standardized T-score that is reported as the final score.
Measured: Monthly, prior to the beginning of each menstrual cycle either by phone or in person for the study duration of 7 months.
Change in Short Form 36 (SF-36) score.
To determine whether the study treatment leads to a clinically significant improvement in Quality of Life (QOL) the study team establishes a baseline quality of life and measures change monthly as well as 6-month change in SF-36. SF-36 is a a 36-item patient reported survey of health consisting of 8 scaled scores with a lower score indicating disability in the one of the 8 sections or domains.The scoring system for the SF-36 is relatively complex and generates subscale scores for physical functioning, role limitations due to physical problems, bodily pain, general health perceptions, vitality, social functioning, role-limitations due to emotional problems, and mental health. Two summary scores can also be derived from the SF-36: the physical component summary and the mental component summary.
Measured: Monthly, prior to the beginning of each menstrual cycle either by phone or in person for the study duration of 7 months
Change in Menorrhagia Impact Questionnaire (MIQ) score.
The MIQ consists of six individual measures or items. The first 4 items are evaluated by using 4-point (item 1) or 5-point (items 2-4) response scales. No summative scale is derived for item 5 as it serves as a descriptive tool to characterize limitations of specific activities and is viewed as supporting information for items 2-4. Item 6 represents a global assessment (impact, degree, and meaningfulness) of the change in menstrual bleeding lost (MBL) when compared to the previous period. Impact response options included a 0 ('almost the same'), 1 ('better'), or 2 ('worse'). If a change is reported, then a 7-point rating scale is used to specify the degree of improvement (item 6a) or worsening (item 6b). Finally, item 6c (meaningfulness of change in MBL) provides an overall assessment of the subjective perception of the change in MBL and may be considered an important component of the MIQ.
Measured: Monthly, prior to the beginning of each menstrual cycle either by phone or in person for the study duration of 7 months
Number of hypersensitivity reactions.
Since Eloctate and Alprolix are biological products, hypersensitivity reactions, including anaphylaxis and anaphylactic reactions, may occur. Nurses and patients will be trained to recognize the signs and symptoms associated with potential hypersensitivity, anaphylactic, and anaphylactoid reactions, and instructed to provide/seek immediate care. Patients/caregivers will receive two alert cards with this information and instructions. For patients with a previous history of a clinically significant hypersensitivity reaction, the site will call the patient 24 hours after each dose to assess the status of the patient. Additional precautions following each of these doses may also be considered including having an extended observation period or IV access prior to dosing.
Measured: Througout the interventional phase of the study (visit 4-7 of the study or months 4-7)
Accuracy of hemoglobin levels detection by Anemocheck.
Exploratory arm participants will self-perform hemoglobin check using Anemocheck every other day during 2 consecutive menstrual cycles. To determine the feasibility and utility of Anemocheck in the monitoring of hemoglobin during a menstruation, the study team will assess correlation of symptoms of heavy menstrual bleeding with a 2g drop in the hemoglobin detected by Anemocheck, and correlation of hemoglobin level obtained by local Lab (within 4-6 hours).
Measured: Every other day during 2 consecutive menstrual cycles during non-interventional study phase (up to Day 60 of the study).
Change in FVIII level after the first dose of Eloctate.
An assessment of FVIII level via blood draw will be performed after the first dose of the drug at four time points.
Measured: First visit of interventional study phase (visit 4, day 90 of the study) : 15-30 min, 2 hours, 4 hours, 18-24 hour after infusion.
Change in FIX level after the first dose of Alpolix.
An assessment of FIX level via blood draw will be performed after the first dose of the drug at four time points.
Measured: First visit of interventional study phase (visit 4, day 90 of the study) : 15-30 min,4 hours,48 hours, and 72 hours after infusion.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Sep 2017
- Study start14 Feb 2018
- Primary completion31 Dec 2019
- Study completion31 Dec 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HemophiliaStudies of the same condition, open and under way.
- More trials run by Emory UniversityThe sponsor's other registered studies.
- 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Model description
- During interventional portion of the study, participants will be infused with either Eloctate (hemophilia A carriers) or Alprolix (hemophilia B carriers) to maintain FVIII or FIX \>48hours. Five out of the 16 enrolled patients will self-perform a hemoglobin check every other day using a novel device Anemocheck during 2 consecutive menstrual cycles.
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- Emory University
- Sponsor type
- University, hospital or other
- Collaborators
- Bioverativ Therapeutics Inc.
- Responsible party
- Principal investigator, Robert Sidonio, Emory University
- Investigators
- Robert Sidonio, MD, MSc, Principal investigator, Emory University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03272568
- Sponsor's protocol ID
- IRB00095067
- Status last verified
- May 2020
- First submitted
- 29 Aug 2017
- Last updated
- 18 May 2020
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 18 May 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Feasibility Trial Using Extended Half Life Factor Products in the Reduction of Menstrual Bleeding in Symptomatic Hemophilia A and B Carriers
- ClinicalTrials.gov ID
- NCT03272568
- Acronym / sponsor ID
- IRB00095067
- Conditions
- Hemophilia, Menstrual Flow Excessive
- Keywords
- Extended Life Plasma Derived Factor Products, Female hemophilia A and B carriers, Menorrhagia
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Emory UniversityUniversity, hospital or other
- Collaborators
- Bioverativ Therapeutics Inc.
- Study start
- 14 Feb 2018 (actual)
- Primary completion
- 31 Dec 2019 (actual)
- Study completion
- 31 Dec 2019 (actual)
- First posted
- 5 Sep 2017 (actual)
- 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.
The purpose of this feasibility study is to find out if two clotting factor products, Eloctate [hemophilia A] and Alprolix [hemophilia B], can reduce the amount of menstrual bleeding in female hemophilia A and B carriers (14 years of age or older) who have severe or heavy bleeding. These products are FDA-approved for use in males with hemophilia A and B to prevent and treat bleeding. They are not approved specifically to reduce menstrual bleeding, but may be useful for this purpose. Both products have an "extended half life" which means they circulate in the body longer than other FVIII or FIX products. The study team will gather additional information about the safety of these drugs and how well they work. The results of this feasibility study will provide information for an upcoming larger study.
Detailed description
Hemophilia A or B is caused by defects in the factor VIII or IX gene, respectively, of which is located on the X chromosome. This disorder exhibits X-linked inheritance, in which primarily males, with a single X chromosome, are affected and females, with two X chromosomes, are heterozygotes, or carriers. Hemophilia carriers show a wide distribution of factor VIII or IX levels with a mean of 50%, which overlaps the distribution of non-carrier women.
Heavy menstrual bleeding is defined as menstrual bleeding that lasts more than 7 days or more specifically as the loss of more than 80cc of blood per cycle. Management is critical as it can lead to iron deficiency anemia, lead to school absence and affects the general quality of life. There are multiple options to control or reduce menstrual bleeding in hemophilia A and B carriers, they include but not limited to the following options: Antifibrinolytics (Aminocaproic acid, Tranexamic acid), Synthetic desmopressin (DDAVP, SQ or IN) or hormonal therapies (Combined oral contraceptives, Progestin only options, IUD, etc).
The use of recombinant factor replacement has been poorly studied and limited by a relatively short half-life in relation to the typical length of a menstrual period. The purpose of this feasibility study is to find out if two clotting factor products, Eloctate [hemophilia A] and Alprolix [hemophilia B], can reduce the amount of menstrual bleeding in female hemophilia A and B carriers (14 years of age or older) who have severe or heavy bleeding. These products are FDA-approved for use in males with hemophilia A and B to prevent and treat bleeding. They are not approved specifically to reduce menstrual bleeding, but may be useful for this purpose. Both products have an "extended half life" which means they circulate in the body longer than other FVIII or FIX products. The study team will gather additional information about the safety of these drugs and how well they work. Five out of the 16 anticipated enrolled patients will be approach to request participation in the exploratory aim in which the subject is taught to perform a hemoglobin check (CBC) every other day using a novel point of care device (Anemocheck) during 2 consecutive menstrual cycles. In addition, they will have CBCs drawn within 4-6 hours for comparison and correlation. The study team will also correlate symptoms of heavy menstrual bleeding with a 2grams drop in the hemoglobin. All the results of this study will provide information for an upcoming larger study.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- During interventional portion of the study, participants will be infused with either Eloctate (hemophilia A carriers) or Alprolix (hemophilia B carriers) to maintain FVIII or FIX >48hours. Five out of the 16 enrolled patients will self-perform a hemoglobin check every other day using a novel device Anemocheck during 2 consecutive menstrual cycles.
| Arm | Type | Receives |
|---|---|---|
| Hemophilia A symptomatic female carriers | Experimental | Drug: Recombinant FVIII Fc fusion product Eloctate, Device: Patient-operated diagnostic device for anemia AnemoCheck.Hemophilia A symptomatic female carriers with a baseline FVIII activity of ≤60% receive recombinant FVIII Fc fusion product Eloctate. Some of participants will be taught to perform a hemoglobin check using a patient-operated diagnostic device for anemia AnemoCheck every other day during 2 consecutive menstrual cycles. |
| Hemophilia B symptomatic female carriers | Experimental | Drug: Recombinant FIX Fc fusion product Alprolix, Device: Patient-operated diagnostic device for anemia AnemoCheck.Hemophilia B symptomatic female carriers with a baseline FIX activity of ≤60% receive recombinant FIX Fc fusion product Alprolix. Some of participants will be taught to perform a hemoglobin check using a patient-operated diagnostic device for anemia AnemoCheck every other day during 2 consecutive menstrual cycles. |
Who may qualify
- Ages
- 14 to 55 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- Females of reproductive age who experience monthly menstrual bleedings
- Female Hemophilia A or B carrier (heterozygote, lyonized or Turner's Syndrome)
- FVIII or FIX activity ≤60% at time of the study
- Baseline Pictorial Bleeding Assessment Chart >150 mean at time of recruitment
- Negative pregnancy test at time of enrollment
Key exclusion criteria (12 listed)
- Has not reached menarche
- Menopause: natural or induced by surgical/medical treatment
- Pregnant or breasfeeding
- Female or her male partner refuses to use barrier method of birth control (for sexually active)
- Current use of any of the following contraceptives (copper IUD, oral combined, Progestin-only including but not limited to: Provera, Aygestin, Nexplanon, Implanon, Depo Provera, Mirena IUD)
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 |
|---|---|---|
| Recombinant FVIII Fc fusion product EloctateAlso: EloctateSubjects will get FVIII activity corrected from the baseline level to 100-150% with Eloctate on the first day of menstruation at the beginning of the study interventional phase. The first dose of the drug will be given in the clinics via slow injection into a vein (infusion). A short assessment of pharmacokinetics (PK) (4 time-points blood draw: 15 min, 2 hours, 4 hours, 20 hours after the infusion) will be performed to assist with subsequent dosing. 2 total doses of Eloctate likely to be given on day 1 and 3 of menstrual period. The subject will then receive three additional PK-adjusted monthly doses of Eloctate (with no less than 21 days interval). | Drug | Hemophilia A symptomatic female carriers |
| Recombinant FIX Fc fusion product AlprolixAlso: AlprolixSubjects will get FIX activity corrected from the baseline level to 100-150% with Alprolix on the first day of menstruation at the beginning of the study interventional phase. The first dose of the drug will be given in the clinics via slow injection into a vein (infusion). A short assessment of pharmacokinetics (PK) (4 time-points blood draw: 15 min, 4 hours, 48 hours, 72 hours after the infusion) will be performed (by home nursing or local lab) to assist with subsequent dosing. Single dose of Alprolix likely to be given on day 1 of menstrual period. The subject will then receive three additional PK-adjusted monthly doses of Alprolix (with no less than 21 days interval). | Drug | Hemophilia B symptomatic female carriers |
| Patient-operated diagnostic device for anemia AnemoCheck.Also: AnemoCheckEach AnemoCheck test kit enables patients to check hemoglobin levels by pricking their finger to draw 5 μL of blood into a capillary tube, inserting that tube into a larger tube with a pre-filled chemical solution, mixing, waiting one minute, and assessing the color change of the solution using a reference color scale card included with the kit. The test is able to subjectively differentiate low, medium and high levels of hemoglobin to diagnose anemia at home. Study participants are taught to perform a hemoglobin check every other day using Anemocheck during 2 consecutive menstrual cycles. In addition they will have hemoglobin level obtained locally (LabCorp) drawn within 4-6 hours for comparison and correlation. | Device | Hemophilia A symptomatic female carriers, Hemophilia B symptomatic female carriers |
Primary outcome
Reduction in menstrual bleeding measured by the Pictorial Bleeding Assessment Chart (PBAC).
Time frame: Monthly throughout study duration for 7 months
Menstrual loss is tracked via paper and electronic PBAC, calculated via visual interpretation centrally and correlated to the ability of a participant to correctly assign pad or tampon saturation. The PBAC will be estimated (mean) over the three-month intervention period to assess if 30% reduction in menstrual bleeding was achieved. PBAC is a semi-quantitative, self-administered pictorial tool. The total score is calculated by adding up the sum of all scores for the tampons or pads used, with a score greater than 150 having an 86% sensitivity and 89% specificity for heavy menstrual bleeding (HMB). Possible range is dependent on quantitative and qualitative amount of bleeding during menstruation: * For tampons: 0.5-1 for lightly stained, 1-4 for moderately soiled and 4-12 for completely saturated tampons. * For pads: 1 for lightly stained, 2-6 for moderately soiled and 4-15 for completely saturated pads. * Clots were given a score of 1 for small and 5 for large clots.
7 secondary outcomes
Change in Patient-Reported Outcomes Measurement Information Systems (PROMIS-43) score.
Time frame: Monthly, prior to the beginning of each menstrual cycle either by phone or in person for the study duration of 7 months.
Change in Short Form 36 (SF-36) score.
Time frame: Monthly, prior to the beginning of each menstrual cycle either by phone or in person for the study duration of 7 months
Change in Menorrhagia Impact Questionnaire (MIQ) score.
Time frame: Monthly, prior to the beginning of each menstrual cycle either by phone or in person for the study duration of 7 months
Number of hypersensitivity reactions.
Time frame: Througout the interventional phase of the study (visit 4-7 of the study or months 4-7)
Accuracy of hemoglobin levels detection by Anemocheck.
Time frame: Every other day during 2 consecutive menstrual cycles during non-interventional study phase (up to Day 60 of the study).
Change in FVIII level after the first dose of Eloctate.
Time frame: First visit of interventional study phase (visit 4, day 90 of the study) : 15-30 min, 2 hours, 4 hours, 18-24 hour after infusion.
Change in FIX level after the first dose of Alpolix.
Time frame: First visit of interventional study phase (visit 4, day 90 of the study) : 15-30 min,4 hours,48 hours, and 72 hours after infusion.
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 18 May 2020.
- Individual participant dataSharing planned: Undecided
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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.
Robert Sidonio, MD, MSc principal investigator
Emory University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Recombinant FVIII Fc fusion product Eloctate, Recombinant FIX Fc fusion product Alprolix and Patient-operated diagnostic device for anemia AnemoCheck. in people with Hemophilia and Menstrual Flow Excessive, run by Emory University 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 6 inclusion and 12 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 18 May 2020. 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
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.
What does the primary outcome measure?
Reduction in menstrual bleeding measured by the Pictorial Bleeding Assessment Chart (PBAC). (time frame: Monthly throughout study duration for 7 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 18 May 2020.