Bevacizumab In Hereditary Hemorrhagic Telangiectasia
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|ClinicalTrials.gov Identifier: NCT04404881|
Recruitment Status : Recruiting
First Posted : May 28, 2020
Last Update Posted : July 2, 2021
This research study is studying to see whether bevacizumab may treat chronic bleeding and iron deficiency anemia in Hereditary Hemorrhagic Telangiectasia (HHT).
Hereditary Hemorrhagic Telangiectasia (HHT) is a disorder that causes abnormal blood vessel formation. In HHT, there is a mutation in the TGF-β pathway, which results in an increase of vascular endothelial growth factor (VEGF) levels. An increase in VEGF levels can result in poorly formed blood vessels that have a higher rate of bleeding than normal blood vessels. Bevacizumab is designed to block VEGF activity. It is believed that targeting increased VEGF levels may be able to treat HHT.
This research study involves the following study drug:
|Condition or disease||Intervention/treatment||Phase|
|Hereditary Hemorrhagic Telangiectasia||Drug: Bevacizumab||Phase 2|
In this research study, the investigators are studying the study drug, bevacizumab. Researchers would like to see whether bevacizumab effectively treats Hereditary Hemorrhagic Telangiectasia (HHT) and what side effects occur.
- The research study procedures include: screening for eligibility, pretreatment period, study treatment, end-of-study visit, and follow-up visit.
- Participants will be observed for 12 weeks and then receive study treatment for 24 weeks and will be followed for 30 days after ending study treatment.
- This research study involves the following study drug: Bevacizumab
- It is expected that about 20 people will take part in this research study.
This research study is a Phase II clinical trial. Phase II clinical trials test the safety and effectiveness of an investigational drug to learn whether the drug works in treating Hereditary Hemorrhagic Telangiectasia (HHT). "Investigational" means that the drug is being studied.
The U.S. Food and Drug Administration (FDA) has not approved bevacizumab for Hereditary Hemorrhagic Telangiectasia (HHT), but it has been approved for other uses.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||20 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase 2 Study of Bevacizumab for Chronic Bleeding and Iron Deficiency Anemia in Hereditary Hemorrhagic Telangiectasia|
|Actual Study Start Date :||November 23, 2020|
|Estimated Primary Completion Date :||April 1, 2025|
|Estimated Study Completion Date :||February 1, 2026|
- Change in hematologic support score from pretreatment to maintenance [ Time Frame: 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.
- Difference in the individual patient mean hemoglobin [ Time Frame: 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.
- Difference in the individual patient pRBC transfusion requirement [ Time Frame: 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
- Difference in the individual patient intravenous iron infusion requirement [ Time Frame: 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
- Average Maintenance Epistaxis Severity Score [ Time Frame: 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.
- Number of Participants With Treatment-Related Adverse Events as Assessed by CTCAE v5.0. [ Time Frame: 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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04404881
|Contact: Hanny Al-Samkari, MDfirstname.lastname@example.org|
|United States, Massachusetts|
|Massachusetts General Hospital||Recruiting|
|Boston, Massachusetts, United States, 02115|
|Contact: Hanny Al-Samkari, MD 617-643-6214 email@example.com|
|Principal Investigator: Hanny Al-Samkari, MD|
|Principal Investigator:||Hanny Al-Samkari, MD||Massachusetts General Hospital|