NCT03227263: BABH

BABH Study: Efficacy and Safety of Bevacizumab on Severe Bleedings Associated With Hemorrhagic Hereditary Telangiectasia (HHT).

Completed Phase 3 Last updated 6 August 2025

What this trial tests

Phase 3 trial testing Bevacizumab in Rendu Osler Disease in 24 participants. Completed in 15 May 2020.

Timeline

28 September 2017

Primary endpoint
15 May 2020

15 May 2020

Quick facts

Lead sponsor	Hospices Civils de Lyon
Phase	Phase 3
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	triple
Primary purpose	treatment
Enrollment	24
Start date	28 September 2017
Primary completion	15 May 2020
Estimated completion	15 May 2020
Sites	4 locations across France

Drugs / interventions tested

• Bevacizumab (Bevacizumab-Bvzr) — full drug profile →
• sodium chloride 0.9%

Conditions studied

• Rendu Osler Disease — all drugs for Rendu Osler Disease →
• Telangiectasia, Hereditary Hemorrhagic — all drugs for Telangiectasia, Hereditary Hemorrhagic →

Sponsor

Hospices Civils de Lyon — full company profile →

Who can join

18 and older, any sex, with Rendu Osler Disease or Telangiectasia, Hereditary Hemorrhagic. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

The recognized manifestations of HHT are all due to abnormalities of vascular structure. Epistaxis and digestive arteriovenous malformations may be responsible for severe hemorrhages in 5% of HHT patients, requiring repeated blood transfusions and are associated with high morbidity. There is currently no standard and efficient management of this severe symptom. It is also well known that HHT-associated hemorrhages have the greatest negative impact on quality of life among HHT patients, and is responsible for anemia, blood transfusions, hospitalizations, depressive syndrome and a high psycho-social impact.

Since 2006, it has been suggested by animal models and then by clinical reports that anti-VEGF therapy may be useful to treat HHT. 4 case reports have been published on efficacy of intravenous bevacizumab, a humanized monoclonal antibody in HHT on severe hemorrhages.

Intravenous bevacizumab has been used in a previous clinical trial to measure efficacy and tolerance of this drug in HHT patients with severe liver involvement. Furthermore, a reduction was observed in the duration of the nosebleeds after treatment and was encouraging to treat bleeding. We completed this study by a pharmacokinetic-pharmacodynamic (PK-PD) model in order to assess the individual concentration-effect relationship of bevacizumab.

However, no randomized prospective study has been performed and published to evaluate the efficacy in this indication. A total of 24 patients will be randomized versus placebo in a multicenter phase III trial. The Avastin or placebo will be infused at 5mg/kg every 14 days with a total of 6 cures with a 3 months following period.

Publications & conference data

5 peer-reviewed publications reference this trial (live from Europe PMC):

1. Pathophysiology in Brain Arteriovenous Malformations: Focus on Endothelial Dysfunctions and Endothelial-to-Mesenchymal Transition.
   Jeong JY, Bafor AE, Freeman BH, Chen PR, et al · · 2024 · cited 6× · PMID 39200259 · DOI 10.3390/biomedicines12081795
2. Genetic Insights Into Hemorrhagic Stroke and Vascular Malformations: Pathogenesis and Emerging Therapeutic Strategies.
   Kalailingam P, Rannikmae K, Hausman-Kedem M, Musolino PL, et al · · 2025 · cited 4× · PMID 40084704 · DOI 10.1161/strokeaha.124.045182
3. Bevacizumab for refractory gastrointestinal angiodysplasia: a case report and literature review.
   Furumaya A, Goltstein LCMJ, Tushuizen ME, Klemt-Kropp M. · · 2025 · cited 1× · PMID 40929654 · DOI 10.1097/meg.0000000000003059
4. Perspectives on Brain Arteriovenous Malformations From the Surgical Battlefield.
   Rahmani R, Scherschinski L, Mozaffari K, Eberle AT, et al · · 2024 · PMID 41586286 · DOI 10.1161/svin.124.001054
5. Epistaxis Prevention, Treatment, and Future Perspectives for Hereditary Hemorrhagic Telangiectasia.
   Ficany A, Del Alamo M, Bernabeu C, Shovlin CL, et al · · 2025 · PMID 41227119 · DOI 10.3390/jcm14217724