Clinical trials: Telangiectasia
Macular Telangiectasia Type 2
Safety Study to Evaluate Bilateral CNTF Implants in Subjects With MacTel Type 2
This is a multi-center, open-label, 6-month study to evaluate the safety of bilateral CNTF implants in participants with Macular Telangiectasia Type 2.
- Trial name
- NCT04729972
- Testing
- NT-501 CNTF implant
- Where
- 10 sites in United States, Australia
- Volunteers
- 33 people
- Sponsor
- Neurotech Pharmaceuticals
Non-Small Cell Lung Cancer
Tuvusertib (M1774) in Combination With Cemiplimab in Participants With Non-Squamous NSCLC (DDRiver NSCLC 322)
This is an Open-label, multicenter clinical study conducted in two Phases to establish the efficacy, safety, tolerability, and pharmacokinetics of the ataxia telangiectasia mutated and Rad3-related protein kinase (ATR) inhibitor…
- Trial name
- NCT05882734
- Testing
- M1774, Cemiplimab
- Where
- 54 sites in United States, Belgium and 6 more
- Volunteers
- 61 people
- Sponsor
- EMD Serono Research & Development Institute, Inc.
Telangiectasia, Hereditary Hemorrhagic
Evaluation of Pazopanib on Bleeding in Subjects With Hereditary Haemorrhagic Telangiectasia
This study will investigate whether pazopanib can reduce epistaxis and improve anaemia in subjects with hereditary haemorrhagic telangiectasia (HHT) at a dose that is well tolerated. The study will have 2 parts.
- Trial name
- NCT02204371
- Testing
- Pazopanib
- Where
- 6 sites in United States, Canada
- Volunteers
- 7 people
- Sponsor
- GlaxoSmithKline
Hereditary Hemorrhagic Telangiectasia
A Study to Evaluate ALN-6400 in Healthy Volunteers and Patients With Hereditary Hemorrhagic Telangiectasia (HHT)
The purpose of this study is to: evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of single ascending doses of ALN-6400 in healthy volunteers evaluate the efficacy, safety, tolerability and PD of…
- Trial name
- InsigHHT (NCT06659640)
- Testing
- ALN-6400
- Where
- 16 sites in United States, Australia and 4 more
- Volunteers
- 89 people, healthy volunteers welcome
- Sponsor
- Alnylam Pharmaceuticals
Advanced Solid Tumors
Study to Test How Well Patients With Advanced Solid Tumors Respond to Treatment With the Elimusertib in Combination With Pembrolizumab, to Find the Optimal Dose for Patients, How the Drug is Tolerated and the Way the Body Absorbs, Distributes and Discharges the Drug
The purpose of the study is to test how well patients with advanced solid tumors respond to treatment with elimusertib (BAY1895344) in combination with pembrolizumab.
- Trial name
- NCT04095273
- Testing
- Elimusertib (BAY1895344), Pembrolizumab (Keytruda®)
- Where
- 16 sites in United States, Germany and 3 more
- Volunteers
- 56 people
- Sponsor
- Bayer
Ataxia Telangiectasia
Open-Label Extension of EryDex Study IEDAT-04-2022
This is an international, multi-center, prospective, open-label, non-comparative study aiming to provide access to treatment with EryDex to ataxia telangiectasia (A-T) patients who completed the IEDAT-04-2022 trial which studied…
- Trial name
- OLE_NEAT (NCT06664853)
- Testing
- Dexamethasone sodium phosphate
- Where
- 21 sites in United States, Denmark and 7 more
- Volunteers
- 101 people
- Sponsor
- Quince Therapeutics S.p.A.
Ataxia Telangiectasia Louis-Bar, Ataxia Telangiectasia in Children
MBM-01 (Tempol) for the Treatment of Ataxia Telangiectasia
Ataxia Telangiectasia (A-T) is an autosomal recessively inherited neurodegenerative disorder that also has dramatic effects on the immune and endocrine systems.
- Conditions
- Ataxia Telangiectasia Louis-Bar, Ataxia Telangiectasia in Children, Ataxia Telangiectasia
- Trial name
- NCT04887311
- Testing
- MBM-01
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Matrix Biomed, Inc.
Hirsutism, Hypertrichosis
Enhanced Safety Aesthetic Laser System
The purpose of this study is to determine whether a new laser system provides better safety and efficacy than existing lasers in popular cosmetic procedures such as hair removal, treatment of cosmetically disturbing vascular…
- Conditions
- Hirsutism, Hypertrichosis, Telangiectases, Rhytides
- Trial name
- NCT00495443
- Testing
- Peterio
- Where
- Israel
- Volunteers
- 25 people
- Sponsor
- Scilex Ltd.
Ataxia Telangiectasia, Genetic Syndrome
Extension Treatment Using EryDex System in Patients With AT Who Participated in the ATTeST-IEDAT-02-2015 Study
Primary Objective To monitor and evaluate the long-term safety and tolerability of EDS-EP in AT patients. Secondary Objective To evaluate the long-term effect of EDS-EP on health-related Quality of Life (QoL; EQ-5D-5L scale).
- Trial name
- OLE-IEDAT (NCT03563053)
- Testing
- EryDex System
- Where
- 17 sites in United States, Australia and 9 more
- Volunteers
- 104 people
- Sponsor
- Quince Therapeutics S.p.A.
Ataxia-Telangiectasia, Ataxia-Telangiectasia (A-T)
A Pivotal Study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T)
A pivotal, randomized, double-blind, placebo-controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of Ataxia-Telangiectasia (A-T).
- Trial name
- NCT06673056
- Testing
- N-Acetyl-L-Leucine
- Where
- 11 sites in United States, Germany and 4 more
- Volunteers
- 60 people
- Sponsor
- IntraBio Inc
Hereditary Hemorrhagic Telangiectasia, Pulmonary Arterial Hypertension
DIAG723 in Adults With Hereditary Hemorrhagic Telangiectasia
This is a Phase 1/2, randomized, double-blind, placebo-controlled, first-in-human study evaluating the safety, tolerability, pharmacokinetics, and preliminary efficacy of subcutaneously administered DIAG723 in adult patients with…
- Trial name
- DIAMOND (NCT07623525)
- Testing
- DIAG723
- Where
- 11 sites in Australia, Canada and 1 more
- Volunteers
- 93 people
- Sponsor
- Diagonal Therapeutics, Inc.
Small-cell Lung Cancer
Berzosertib + Topotecan in Relapsed Platinum-Resistant Small-Cell Lung Cancer (DDRiver SCLC 250)
The main purpose of this study is to assess efficacy, safety, tolerability and pharmacokinetics (PK) of Berzosertib in combination with Topotecan in participants with relapsed, platinum-resistant small-cell lung cancer (SCLC).
- Trial name
- NCT04768296
- Testing
- Berzosertib, Topotecan
- Where
- 51 sites in United States, Belgium and 5 more
- Volunteers
- 76 people
- Sponsor
- EMD Serono Research & Development Institute, Inc.
Ataxia Telangiectasia
N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T)
This is a multinational, multicenter, open-label, rater-blinded prospective Phase II study which will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of Ataxia-Telangiectasia (A-T).
- Trial name
- NCT03759678
- Testing
- IB1001
- Where
- 4 sites in United States, Germany and 2 more
- Volunteers
- 17 people
- Sponsor
- IntraBio Inc
Hereditary Hemorrhagic Telangiectasia (HHT)
A Trial of TER-1754 in Patients With Hereditary Hemorrhagic Telangiectasia
This is a Phase 1a/1b, multicenter study evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and clinical activity of TER-1754 (a novel AKT1 inhibitor) in patients with HHT.
- Trial name
- NCT07255846
- Testing
- TER-1754
- Where
- United States
- Sponsor
- Terremoto Biosciences Inc.
Pain
Evaluation of the Tolerability of Switching Subjects on Chronic ATC Opioid Therapy to Buprenorphine HCl Buccal Film
The primary aim of this study is to determine if chronic pain subjects on around-the-clock opioids who are receiving 100 to 220 mg oral morphine sulfate equivalent (MSE) can be safely transitioned on to buprenorphine…
- Trial name
- NCT01871285
- Testing
- Buprenorphine, Oxycodone, Morphine sulfate
- Where
- 3 sites in United States
- Volunteers
- 39 people
- Sponsor
- BioDelivery Sciences International
Hereditary Hemorrhagic Telangiectasia (HHT), Osler Weber Rendu Disease
Harmony-HHT: ATV-1601 in Participants With Hereditary Hemorrhagic Telangiectasia (HHT)
This is a 2-part study evaluating ATV-1601 in participants with moderate to severe HHT. Part 1 is a randomized, double-blind, placebo-controlled study evaluating 3 dosing regimens of ATV-1601.
- Trial name
- NCT07601425
- Testing
- ATV-1601
- Where
- 7 sites in United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Atavistik Bio, Inc
Hereditary Hemorrhagic Telangiectasia (HHT)
Assess Safety and Efficacy of VAD044 in HHT Patients
Part I: The purpose of this Phase 1b proof of concept study, randomised, placebo controlled, double blind, multicentre study is to asssess safety and efficacy of 2 doses of VAD044 in adult HHT patients.
- Trial name
- NCT05406362
- Testing
- VAD044 Part I, VAD044 Part II
- Where
- 7 sites in United States, Belgium and 4 more
- Volunteers
- 75 people
- Sponsor
- Vaderis Therapeutics AG
Retinal Telangiectasis, Idiopathic Juxtafoveal Retinal Telangiectasia
A Study to Evaluate THR-317 Treatment for Macular Telangiectasia Type 1 (MacTel 1)
This study is conducted to evaluate the safety of THR-317 when administered intravitreally and to assess the compound's efficacy in reducing central subfield thickness (CST) and improving best-corrected visual acuity (BCVA) in…
- Trial name
- NCT03669393
- Testing
- THR-317 8mg
- Where
- 3 sites in France, Switzerland
- Volunteers
- 8 people
- Sponsor
- ThromboGenics
Nervous System Disease, Genetic Syndrome
Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Telangiectasia Patients
Objectives: The objective of study was to evaluate the safety and the efficacy of EryDex (Dexamethasone sodium phosphate encapsulated in autologous erythrocytes, using the EryDex System - EDS) at two dose levels (low dose and…
- Trial name
- ATTeST (NCT02770807)
- Testing
- EryDex Low dose DSP, EryDex High dose DSP
- Where
- 22 sites in United States, Australia and 10 more
- Volunteers
- 176 people
- Sponsor
- Quince Therapeutics S.p.A.
Macular Telangiectasia Type 2 (MacTel)
Phase 4 Study: Long-term Safety and Efficacy of NT-501 in MacTel Type 2, Including Sham Procedure Participants
The purpose of this global, multicenter, open-label, Phase 4 clinical extension study is to evaluate the long-term safety and efficacy of revakinagene taroretcel-lwey (Encelto™; hereinafter referred to as NT-501), in participants…
- Trial name
- NCT06971939
- Testing
- NT-501/revakinagene taroretcel-lwey
- Where
- 26 sites in United States, Australia
- Volunteers
- 234 people
- Sponsor
- Neurotech Pharmaceuticals