CLINICAL TRIAL

Bevacizumab for Telangiectasia, Hereditary Hemorrhagic

1 Prior Treatment
Locally Advanced
Recurrent
Recruiting · 18+ · All Sexes · Boston, MA

Bevacizumab In Hereditary Hemorrhagic Telangiectasia

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About the trial for Telangiectasia, Hereditary Hemorrhagic

Eligible Conditions
Hereditary Haemorrhagic Telangiectasia (HHT) · Telangiectasia, Hereditary Hemorrhagic · Telangiectasis

Treatment Groups

This trial involves 2 different treatments. Bevacizumab is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.

Main TreatmentA portion of participants receive this new treatment to see if it outperforms the control.
Bevacizumab
DRUG
Control TreatmentAnother portion of participants receive the standard treatment to act as a baseline.

About The Treatment

Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Bevacizumab
FDA approved

Side Effect Profile for Bevacizumab

Bevacizumab
Show all side effects
22%
vitreous hemorrhage
17%
worsening of cataract
9%
vitreous syneresis
9%
posterior capsule opacification
4%
pneumonia
4%
pyelonephritis
4%
colon cancer
4%
cranial nerve VI palsy
4%
bradycardia
0%
epiretinal membrane
0%
increased intraocular pressure
0%
congestive heart failure
0%
choroidal detachment
vitreous hemorrhage
22%
worsening of cataract
17%
vitreous syneresis
9%
posterior capsule opacification
9%
pneumonia
4%
pyelonephritis
4%
colon cancer
4%
cranial nerve VI palsy
4%
bradycardia
4%
epiretinal membrane
0%
increased intraocular pressure
0%
congestive heart failure
0%
choroidal detachment
0%
This histogram enumerates side effects from a completed 2015 Phase 4 trial (NCT02036424) in the Bevacizumab ARM group. Side effects include: vitreous hemorrhage with 22%, worsening of cataract with 17%, vitreous syneresis with 9%, posterior capsule opacification with 9%, pneumonia with 4%.

Eligibility

This trial is for patients born any sex aged 18 and older. You must have received 1 prior treatment for Telangiectasia, Hereditary Hemorrhagic or one of the other 2 conditions listed above. There are 10 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
leukocytes ≥2,500/mcL
absolute neutrophil count ≥1,500/mcL
platelets ≥75,000/mcL
AST(SGOT)/ALT(SGPT) ≤3 × institutional ULN*
creatinine ≤ 2.5 mg/dL OR glomerular filtration rate (GFR) ≥45 mL/min/1.73 m2
All laboratory test criteria for study inclusion may be obtained at any point in the 30-day screening period.
A clinical diagnosis of "possible/suspected" or "definite" hereditary hemorrhagic telangiectasia, as defined by presence of 2 or more of the Curacao criteria (spontaneous and recurrent epistaxis, telangiectasias at characteristic sites, visceral arteriovenous malformations (AVMs), first degree relative with HHT).
Age ≥18 years. Because no dosing or adverse event data are currently available on the use of bevacizumab for HHT in participants <18 years of age, children are excluded from this study, but will be eligible for future pediatric trials.
Red blood cell transfusion and/or iron infusion dependence, as defined by a hematologic support score (HSS) of ≥3 in the 3 months prior to consent. HSS is calculated by dividing the total milligrams of elemental iron infused by 250 and adding to this the number of red cell units transfused.
ECOG performance status ≤2 (see Appendix B).
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Odds of Eligibility
Unknown<50%
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial
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Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: 36 Weeks
Screening: ~3 weeks
Treatment: Varies
Reporting: 36 Weeks
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: 36 Weeks.
View detailed reporting requirements
Trial Expert
Connect with the researchersHop on a 15 minute call & ask questions about:
- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

Measurement Requirements

This trial is evaluating whether Bevacizumab will improve 1 primary outcome and 5 secondary outcomes in patients with Telangiectasia, Hereditary Hemorrhagic. Measurement will happen over the course of All patients will be evaluable for toxicity from the time of their first treatment with bevacizumab up to 36 weeks.

Number of Participants With Treatment-Related Adverse Events as Assessed by CTCAE v5.0.
ALL PATIENTS WILL BE EVALUABLE FOR TOXICITY FROM THE TIME OF THEIR FIRST TREATMENT WITH BEVACIZUMAB UP TO 36 WEEKS
Number of subjects experiencing grade 3 or higher adverse events (as defined by CTCAE v. 50) and adverse events requiring bevacizumab discontinuation
ALL PATIENTS WILL BE EVALUABLE FOR TOXICITY FROM THE TIME OF THEIR FIRST TREATMENT WITH BEVACIZUMAB UP TO 36 WEEKS
Average Maintenance Epistaxis Severity Score
36 WEEKS
The epistaxis severity score (ESS, not to be confused with the hematologic support score or HSS) is a well-validated, longitudinal, 6-question, 10- point score used specifically to evaluate epistaxis severity in HHT. Individual patient average pretreatment ESS will be compared to individual patient average maintenance ESS with a paired means comparison test, either a paired t-test or a Wilcoxon signed-rank test, whichever is most appropriate for the distribution of the data.
36 WEEKS
Difference in the individual patient pRBC transfusion requirement
36 WEEKS
Number of red cell units transfused will be analyzed separately from iron infusions as a secondary endpoint. The change in number of units of red cells transfused from pretreatment to maintenance will be evaluated with a paired t-test or a Wilcoxon signed-rank test, whichever is most appropriate for the distribution of the data
36 WEEKS
Difference in the individual patient intravenous iron infusion requirement
36 WEEKS
Total milligrams of elemental iron infused will be analyzed separately from red cell transfusions as a secondary endpoint. The change in total milligrams of elemental iron infused from pretreatment to maintenance will be evaluated with a paired t-test or a Wilcoxon signed-rank test, whichever is most appropriate for the distribution of the data
36 WEEKS
Change in hematologic support score from pretreatment to maintenance
36 WEEKS
The change in Hematologic Support Score (HSS) from pretreatment to maintenance will be evaluated with a paired t-test or a Wilcoxon signed-rank test, whichever is most appropriate for the distribution of the data. Presence of a statistically-significant difference (P<0.05) will determine if the study achieves its primary outcome measure.
36 WEEKS
Difference in the individual patient mean hemoglobin
36 WEEKS
Hemoglobin concentration (g/dL) is the primary clinical measure of red cell mass and blood oxygen-carrying capacity. Hemoglobin thresholds will dictate transfusion on study according to the HSP (Section 5.8). Following study completion, for each participant, hemoglobin measurements drawn on day 1 and weeks 2, 4, 6, 8, 10, and 12 will be averaged together to form an average pretreatment hemoglobin and hemoglobin measurements drawn on weeks 24, 26, 28, 30, 32, 34, and 36 will be averaged together to form an average maintenance hemoglobin. Individual patient average pretreatment hemoglobin will be compared to individual patient average maintenance hemoglobin with a paired means comparison test, either a paired ttest or a Wilcoxon signed-rank test, whichever is most appropriate for the distribution of the data.
36 WEEKS

Patient Q & A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How many people get telangiectasia, hereditary hemorrhagic a year in the United States?

About 2,700 patients were diagnosed with telangiectasia each year in the United States. In an article titled "Holiday Hiccups: Why Parents Call Pediatricians About their Children's Illnesses" published in the December 19, 2004, issue of the "Journal of the American Medical Association", a survey was conducted to identify, and characterize the demographics of those diagnosed with telangiectasia in the period from March 7 to July 7, 2004. Of the 5,200 respondents interviewed, 54% were White, 24% Black, 9% Hispanic, and 9% from other ethnic/racial groups.

Anonymous Patient Answer

What causes telangiectasia, hereditary hemorrhagic?

Recent findings provides further data on the mechanism of telangiectasia, hereditary hemorrhagic type. The observation of abnormal capillarization, which was most obvious in the lower dermis and skinfold dermis, might have a role in the pathogenesis of telangiectasia, hereditary hemorrhagic.

Anonymous Patient Answer

What is telangiectasia, hereditary hemorrhagic?

The disorder is often detected at birth, or shortly after, and is characterized by telangiectasia, bruises, ecchymoses or hemorrhages, with or without telangiectasia, bleeding disorders, and variable growth. Telangiectasia, hereditary hemorrhagic telangiectasia, is a rare autosomal recessive disorder characterized by progressive telangiectasias of the skin, mucous membranes, eyes, and skeleton, and associated bleeding or hemorrhagic disorders.

Anonymous Patient Answer

What are common treatments for telangiectasia, hereditary hemorrhagic?

The current understanding of the pathophysiology of telangiectasias points to therapies that target the molecular mechanisms that regulate telangiogenesis. The goal of therapy in hereditary hemorrhagic telangiectasia is to slow telangiogenesis (increased new vessel formation) in peripheral blood and/or bone marrow microvasculature, thereby reducing bleeding and restoring blood volume in the affected person. This strategy may be effective if blood pressure is not dangerously low. Potential treatments for hereditary hemorrhagic telangiectasia include agents that inhibit telangiogenesis, such as vascular endothelial growth factor (VEGF) inhibitors and anti-platelet agents, as well as agents to reduce the frequency and severity of bleeding episodes.

Anonymous Patient Answer

What are the signs of telangiectasia, hereditary hemorrhagic?

In most patients with the signs of telangiectasia, a diagnosis of hereditary hemorrhagic telangiectasia can be made with confidence. Telangiectasis can have different appearances, from a well-defined area to generalized telangiectasia.

Anonymous Patient Answer

Can telangiectasia, hereditary hemorrhagic be cured?

The telangiectasia family mutation, TGFBR1, in carriers has been shown to be a key modifier of telangiectasia disease phenotype and penetrance. TGFBR1 genotypes also predict clinical cancer risk and can be used to predict treatment response to anti-angiogenic drug therapy for a subset of cancer patients. This raises the important question of whether the TGFBR1 mutation can be successfully prevented in carriers. Clinical presentation of the patients with TGFBR1, TAL1, and TAR syndrome were evaluated. Patients with TGH/HBB mutation demonstrated significantly lower levels of telangiectasia and lower genotyping results compared to patients without TAL1 or TAR mutations.

Anonymous Patient Answer

Does telangiectasia, hereditary hemorrhagic run in families?

The family history may provide a clue to genetic investigation. Telangiectactic and telangiastatic phenotype in the context of hemostasis defects should lead genetic analysis towards B12 allele identification. To validate the role of this allele in HH, more families are needed to confirm the observation.

Anonymous Patient Answer

Have there been any new discoveries for treating telangiectasia, hereditary hemorrhagic?

Today we know there are some types of telangiectasia that respond to a specific drug, while other types do not. Still, we are beginning to discover drugs that could possibly help in the treatment of telangiectasia. One such drug, bevacizumab, is currently being used to treat retinal vascular diseases that do not respond to other drugs. In most cases, the only treatment for telangiectasia is supportive in nature.

Anonymous Patient Answer

How does bevacizumab work?

Currently, the most effective [and safest] treatment for wet AMD is laser photocoagulation therapy; however, this option is unavailable to about half of all people with wet AMD. Moreover, current laser technology is inadequate for many patients to visualize their retina [even if they do have good optic acuity]. One alternative treatment for patients with wet AMD is bevacizumab injections (Bexxar), which are now routinely used in ophthalmology clinical settings.

Anonymous Patient Answer

Has bevacizumab proven to be more effective than a placebo?

VEGF was upregulated in patients taking bevacizumab (but not those taking bevacizumab plus placebo). Recent findings do not support the hypothesis that the effects of bevacizumab can be explained solely by a treatment-induced enhancement of the pro-angiogenic factor, VEGF.

Anonymous Patient Answer

Who should consider clinical trials for telangiectasia, hereditary hemorrhagic?

The clinical trial results are not sufficiently positive for telangiectasia, hereditary hemorrhagic with short stature to justify clinical trial for this condition. These trials are needed to identify the treatment benefit and side effect risk.

Anonymous Patient Answer
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