A Phase 4 interventional study of Doxycycline and Placebo in Epistaxis, sponsored by University of California, Los Angeles. Completed at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2022-07-20.
Sponsored by University of California, Los Angeles · Phase 4, Interventional, and Treatment
The purpose of this research study is to determine whether Doxycycline can be used to control nosebleeds (epistaxis) for patients with Hereditary Hemorrhagic Telangiectasia (HHT). Patients with HHT will be randomized to one of 2 study arms: Doxycycline or Placebo for a period of 2 months followed by a 1-month washout period before switching treatments for a further 2 months period. Observation and evaluation will continue for a period of one month after treatment is completed.
This study will analyze the effect of doxycycline on nosebleeds in hereditary hemorrhagic telangiectasia (HHT), a rare and debilitating medical condition. Patients with HHT have a variety of vascular abnormalities, including changes in their nasal vasculature that result in chronic, severe nosebleeds. These nosebleeds have been shown to significantly reduce the quality of life in HHT and can be severe enough to require emergent blood transfusion. Current therapies to control nosebleeds in HHT, which include both medications and surgical treatments, are inconsistently effective and associated with a variety of problematic side effects and complications.
Doxycycline is a common, FDA-approved antibiotic medication that has been used by physicians for decades. It is a safe medication that can be used on an outpatient basis for extended periods of time with minimal side effects. Recent research has shown that doxycycline is a potent inhibitor of angiogenesis, the process of new blood vessel development. Since the vascular abnormalities in HHT form due to uncontrolled angiogenesis, it follows that doxycycline may have potential in treating HHT. A growing body of research demonstrates the efficacy of doxycycline in treating a variety of vascular conditions, including cancer of the breast, duodenum, liver prostate, and lung. Our trial will be the first to analyze the efficacy of doxycycline in treating HHT epistaxis.
This a randomized, double-blind, crossover study design. Each patient will take both placebo and doxycycline. Since this is a cross-over study, half the participants will start out with taking placebo and half start with doxycyline and then switch halfway through. After a baseline observation period, participants will be given doxycycline at 100 mg twice a day, or an identical-appearing placebo, for a period of three months. Each month they will be seen be a physician, who will take a detailed clinical history and perform a physical examination. Further, each month the patient will have blood testing to assess blood loss, and fill out scientifically validated surveys to assess quality of life and the severity of bleeding. Following this three month treatment period, participants will be observed for one additional month, with one additional blood test and clinical assessment performed after this month.
96 studies on the registry are indexed under Epistaxis; 22 are open to participants now.
This study's enrollment of 26 is below the median of 64 across 81 interventional studies indexed under Epistaxis.
Browse Epistaxis studies →University of California, Los Angeles is the lead sponsor of 1,170 studies on the registry; 195 are open to participants now.
Of its 95 completed or terminated interventional studies of FDA-regulated products, 70 (74%) have results posted.
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Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Doxycycline for a period of 2 months followed by a 1-month washout period, and then placebo for a further 2 months period followed by a 1-month washout period.
Drug: Doxycycline · Other: Placebo
Placebo for a period of 2 months followed by a 1-month washout period, and then Doxycycline for a further 2 months period followed by a 1-month washout period.
Drug: Doxycycline · Other: Placebo
Doxycycline, 100 mg twice a day for 2 months
Matching placebo, twice a day for 2 months
Frequency of Epistaxis
Change in number of bleeding episodes per week by participant self-report via nosebleed diary
Time frame: Baseline, post-washout - approximately 6 months
Duration of Epistaxis
Change in duration of bleeding per week by patient self-report via nosebleed diary
Time frame: Baseline, post-washout - approximately 6 months
Change in Severity of Epistaxis
Epistaxis Severity Scale (ESS) for Hereditary Hemorrhagic Telangiectasia, is based on 6 nosebleed variables such as frequency and duration which are reported by patients.The ESS has a minimum value of 0 and maximum value of 10, with 10 representing more severe epistaxis.
Time frame: Baseline, post-washout - approximately 6-months
Change in Quality of Life
The 12-item Short Form (SF-12) is a survey designed for use with patients with multiple chronic conditions. This 12-item scale can be used to assess the physical and mental health of respondents. 10 of the 12 questions are answered on a 5-point Likert scale and 2 are answered on a 3-point Likert scale. The questions are then scored and weighted into 2 subscales, physical health and mental health. Respondents can have a score that ranges from 0-100 with 100 being the best score and indicating high physical or mental health.
Time frame: Baseline, post-washout - approximately 6 months
Change in Ferritin Level
Time frame: Baseline, post-washout - approximately 6 months
Change in Hemoglobin Level
Time frame: Baseline, post-washout (approximately 6 months)
Change in Monthly Units of Packed Red Blood Cells (PRBCs) Transfused
Time frame: Baseline, post-washout (approximately 6 months)
Incidence of Treatment Failure
Treatment failure, defined as need for nasal surgery or other epistaxis treatments
Time frame: Baseline, post-washout (approximately 6 months)
| Milestone | Doxycycline, Then Placebo | Placebo, Then Doxycycline |
|---|---|---|
| Started | 13 | 13 |
| Day 1 | 12 | 12 |
| Completed | 11 | 12 |
| Not completed | 2 | 1 |
| Withdrew: Withdrawal by subject | 2 | 1 |
| Milestone | Doxycycline, Then Placebo | Placebo, Then Doxycycline |
|---|---|---|
| Started | 11 | 12 |
| Completed | 11 | 12 |
| Not completed | 0 | 0 |
| Milestone | Doxycycline, Then Placebo | Placebo, Then Doxycycline |
|---|---|---|
| Started | 11 | 12 |
| Completed | 10 | 12 |
| Not completed | 1 | 0 |
| Withdrew: Withdrawal by subject | 1 | 0 |
| Milestone | Doxycycline, Then Placebo | Placebo, Then Doxycycline |
|---|---|---|
| Started | 10 | 12 |
| Completed | 10 | 12 |
| Not completed | 0 | 0 |
| Milestone | Doxycycline, Then Placebo | Placebo, Then Doxycycline |
|---|---|---|
| Started | 10 | 12 |
| Completed | 10 | 12 |
| Not completed | 0 | 0 |
Change in number of bleeding episodes per week by participant self-report via nosebleed diary
| Nosebleeds per week | Doxycycline | Placebo |
|---|---|---|
| Frequency of Epistaxis | -1.68 ± 6.55 | 1.14 ± 4.44 |
Change in duration of bleeding per week by patient self-report via nosebleed diary
| Minutes per week | Doxycycline | Placebo |
|---|---|---|
| Duration of Epistaxis | -41.17 ± 159.26 | 24.36 ± 58.03 |
Epistaxis Severity Scale (ESS) for Hereditary Hemorrhagic Telangiectasia, is based on 6 nosebleed variables such as frequency and duration which are reported by patients.The ESS has a minimum value of 0 and maximum value of 10, with 10 representing more severe epistaxis.
| score on a scale | Doxycycline | Placebo |
|---|---|---|
| Change in Severity of Epistaxis | -.095 ± 1.55 | -0.32 ± 1.3 |
The 12-item Short Form (SF-12) is a survey designed for use with patients with multiple chronic conditions. This 12-item scale can be used to assess the physical and mental health of respondents. 10 of the 12 questions are answered on a 5-point Likert scale and 2 are answered on a 3-point Likert scale. The questions are then scored and weighted into 2 subscales, physical health and mental health. Respondents can have a score that ranges from 0-100 with 100 being the best score and indicating high physical or mental health.
| score on a scale | Doxycycline | Placebo |
|---|---|---|
| SF-12 Physical | 2.21 ± 6.94 | -0.83 ± 6.72 |
| SF-12 Mental | -0.63 ± 9.97 | -0.63 ± 9.74 |
| ng/ml | Doxycycline | Placebo |
|---|---|---|
| Change in Ferritin Level | 14.9 ± 72.82 | -47.4 ± 214.83 |
| g/dl | Doxycycline | Placebo |
|---|---|---|
| Change in Hemoglobin Level | 0.26 ± 1.09 | -0.12 ± 1.19 |
| count of PBRC units | Doxycycline | Placebo |
|---|---|---|
| Change in Monthly Units of Packed Red Blood Cells (PRBCs) Transfused | 0 ± 0.31 | 0 ± 0 |
Treatment failure, defined as need for nasal surgery or other epistaxis treatments
| Participants | Doxycycline | Placebo |
|---|---|---|
| Incidence of Treatment Failure | 0 | 1 |
Collected over Baseline, post-washout (approximately 6 months). Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Doxycycline | 0/23 (0%) | 0/23 (0%) | 6/23 (26.1%) |
| Placebo | 0/22 (0%) | 0/22 (0%) | 6/22 (27.3%) |
| Event | Doxycycline | Placebo |
|---|---|---|
| GI bleedingGastrointestinal disorders | 0/23 | 1/22 |
| Flu-like symptomsInfections and infestations | 0/23 | 1/22 |
| Prolonged nosebleed requiring cauterizationRespiratory, thoracic and mediastinal disorders | 0/23 | 1/22 |
| Pneumonia hospitalizationInfections and infestations | 0/23 | 1/22 |
| Allergy-like symptomsRespiratory, thoracic and mediastinal disorders | 0/23 | 1/22 |
| Throat infectionInfections and infestations | 0/23 | 1/22 |
| Metastatic lung cancer dxNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 1/23 | 0/22 |
| Sinus infectionInfections and infestations | 1/23 | 0/22 |
| Foot infectionInfections and infestations | 1/23 | 0/22 |
| Blurred visionEye disorders | 1/23 | 0/22 |
| Age, Categorical(Participants) | Doxycycline, Then Placebo | Placebo, Then Doxycycline | Total |
|---|---|---|---|
| <=18 years | 0 | 0 | 0 |
| Between 18 and 65 years | 8 | 7 | 15 |
| >=65 years | 5 | 6 | 11 |
| Age, Continuous(years) | Doxycycline, Then Placebo | Placebo, Then Doxycycline | Total |
|---|---|---|---|
| Mean | 51.3 ± 12.9 | 63.6 ± 13.8 | 57.4 ± 14.5 |
| Sex: Female, Male(Participants) | Doxycycline, Then Placebo | Placebo, Then Doxycycline | Total |
|---|---|---|---|
| Female | 6 | 7 | 13 |
| Male | 7 | 6 | 13 |
| Ethnicity (NIH/OMB)(Participants) | Doxycycline, Then Placebo | Placebo, Then Doxycycline | Total |
|---|---|---|---|
| Hispanic or Latino | 3 | 3 | 6 |
| Not Hispanic or Latino | 8 | 9 | 17 |
| Unknown or Not Reported | 2 | 1 | 3 |
| Race (NIH/OMB)(Participants) | Doxycycline, Then Placebo | Placebo, Then Doxycycline | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 1 | 1 | 2 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 0 |
| White | 6 | 9 | 15 |
| More than one race | 3 | 2 | 5 |
| Unknown or Not Reported | 3 | 1 | 4 |
| Region of Enrollment(participants) | Doxycycline, Then Placebo | Placebo, Then Doxycycline | Total |
|---|---|---|---|
| United States | 13 | 13 | 26 |
| Epistaxis Severity Scale (ESS) score(Score on a scale) | Doxycycline, Then Placebo | Placebo, Then Doxycycline | Total |
|---|---|---|---|
| Mean | 6.3 ± 1.2 | 5.7 ± 1.5 | 6.0 ± 1.4 |
| Quality of Life(Score on a scale) | Doxycycline, Then Placebo | Placebo, Then Doxycycline | Total |
|---|---|---|---|
| SF-12 Physical | 43.6 ± 10.7 | 40.2 ± 9.7 | 41.9 ± 10.2 |
| SF-12 Mental | 47.0 ± 12.5 | 54.4 ± 6.4 | 50.7 ± 10.4 |
4 further baseline measures are reported on the registry.
PDFs hosted by the registry, ClinicalTrials.gov.
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Studies of the drug tested here: Doxycycline
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