Clinical trials: Small Vessel Disease
Pulmonary Hypertension, Chronic Obstructive Pulmonary Disease
Safety and Tolerability of Inhaled Treprostinil in Adult PH Due to COPD
This is a multicenter, single-arm trial to evaluate the safety and efficacy of inhaled treprostinil in subjects with pre-capillary pulmonary hypertension (PH) associated with Chronic Obstructive Pulmonary Disease (COPD).
- Trial name
- NCT03012646
- Testing
- Inhaled Treprostinil
- Sponsor
- United Therapeutics
Fabry Disease
Open-Label Phase 3 Long-Term Safety Study of Migalastat
This was a long-term, open-label study of migalastat (123 milligrams [mg] of migalastat [equivalent to 150 mg of migalastat hydrochloride]) (migalastat) in participants with Fabry disease who completed treatment in a previous…
- Trial name
- AT1001-041 (NCT01458119)
- Testing
- migalastat hydrochloride
- Where
- 25 sites in United States, Argentina and 11 more
- Volunteers
- 85 people
- Sponsor
- Amicus Therapeutics
Fabry Disease
Post Trial Access to Lucerastat for Patients With Fabry Disease Who Participated in Study ID-069A302
This is a Post Trial Access program for patients with Fabry Disease who previously participated in the ID-069A302 (MODIFY OLE) trial and who were still enrolled in the study at the time the Sponsor decided to discontinue…
- Trial name
- NCT07762638
- Testing
- Lucerastat
- Sponsor
- Idorsia Pharmaceuticals Ltd.
Fabry Disease
An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease
This is a prospective multicenter, open-label, dose-escalation trial to assess the safety, tolerability, and pharmacodynamics of 4D-310 following a single IV administration.
- Trial name
- NCT04519749
- Testing
- 4D-310
- Where
- 4 sites in United States
- Volunteers
- 18 people
- Sponsor
- 4D Molecular Therapeutics
Coronary Artery Disease, Myocardial Ischemia
BIOFLOW-III All-comers Orsiro Safety and Performance Registry
This registry is a clinical post-market evaluation of the Orsiro LESS in subjects requiring coronary revascularization with Drug Eluting Stents (DES).
- Trial name
- BIOFLOW-III (NCT01553526)
- Where
- 43 sites in Austria, Chile and 11 more
- Volunteers
- 1,356 people
- Sponsor
- Biotronik AG
Mature B-cell Malignancies
Study of Zanubrutinib in Japanese Participants With B-Cell Malignancies
This is a Phase 1/2 study of zanubrutinib in Japanese participants with mature B-cell malignancies. This study intends to assess the use of zanubrutinib as an investigational agent to develop new treatment options for Japanese…
- Trial name
- NCT04172246
- Testing
- Zanubrutinib
- Where
- 15 sites in Japan
- Volunteers
- 55 people
- Sponsor
- BeiGene
Fabry Disease
Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease
This study will evaluate safety and clinical outcomes of treatment with Replagal in adult participants with Fabry disease who have completed Study TKT028 (NCT00864851).
- Trial name
- NCT01124643
- Testing
- Replagal
- Where
- 9 sites in United States, Australia and 5 more
- Volunteers
- 35 people
- Sponsor
- Shire
Hereditary Cystatin C Amyloid Angiopathy (HCCAA), Hemorrhage Brain
Safety, Tolerability and Efficacy of NPI-001 in Patients With Hereditary Cystatin C Amyloid Angiopathy (HCCAA)
The study aims to measure safety, tolerability and biomarker-based efficacy of NPI-001 (AT-001) in Subjects with Hereditary Cystatin C Amyloid Angiopathy (HCCAA).
- Conditions
- Hereditary Cystatin C Amyloid Angiopathy (HCCAA), Hemorrhage Brain, Hemorrhage Cerebral
- Trial name
- HCCAA (NCT07716865)
- Testing
- NACA
- Where
- Iceland
- Volunteers
- 15 people
- Sponsor
- Arctic Therapeutics
Non-Small Cell Lung Cancer
A Prospective, Multi-Center Evaluation of a Powered Vascular Stapler in Video-Assisted Thoracoscopic Lobectomies
The primary objective of this trial is to demonstrate the effectiveness of the powered vascular stapler for transection of the pulmonary artery (PA) and pulmonary vein (PV) during Video-Assisted Thoracoscopic Lobectomy.
- Trial name
- NCT03056300
- Testing
- Video-Assisted Thoracoscopic Lobectomy with powered vascular stapler
- Where
- China
- Volunteers
- 50 people
- Sponsor
- Ethicon, Inc.
Fabry Disease
Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry Disease
The objective was to determine the effects of a single dose of migalastat hydrochloride (HCl) (migalastat) 150 and 450 milligrams (mg) on the safety and plasma pharmacokinetics (PK) of agalsidase and the effects of agalsidase on…
- Trial name
- NCT01196871
- Testing
- Migalastat HCl, Agalsidase Beta, Agalsidase Alfa
- Where
- 10 sites in United States, Australia and 3 more
- Volunteers
- 20 people
- Sponsor
- Amicus Therapeutics
Mitochondrial Diseases, Mitochondrial Myopathies
The KHENERGYZE Study
Mitochondrial diseases, estimated prevalence 1 in 4,300 adults, is caused by pathogenic mutations in genes finally encoding for mitochondrial proteins of the various enzyme complexes of the OXPHOS.
- Conditions
- Mitochondrial Diseases, Mitochondrial Myopathies, Mitochondrial Encephalomyopathies, MELAS Syndrome
- Trial name
- NCT04165239
- Testing
- KH176
- Where
- 4 sites in Denmark, Germany and 2 more
- Volunteers
- 27 people
- Sponsor
- Khondrion BV
MELAS
Phase 2a Study of IW-6463 in Adults Diagnosed With Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes (MELAS)
This is a single-arm study to evaluate safety and tolerability of oral IW-6463 in adults diagnosed with MELAS.
- Trial name
- NCT04475549
- Testing
- IW-6463 Tablets
- Where
- 5 sites in United States
- Volunteers
- 8 people
- Sponsor
- Tisento Therapeutics
Fabry Disease
Pharmacokinetics, Pharmacodynamics, and Safety of Moss-aGalactosidase A in Patients With Fabry Disease
Six patients with Fabry disease will be recruited. Patients will receive a single dose of 0.2 mg/kg recombinant human alpha-galactosidase A produced in moss (moss-aGal) as intravenous infusion.
- Trial name
- NCT02995993
- Testing
- Moss-aGal (recombinant human alpha-galactosidase A produced in moss)
- Where
- 2 sites in Germany
- Volunteers
- 6 people
- Sponsor
- Greenovation Biotech GmbH
Extensive-stage Small-cell Lung Cancer, Small-cell Lung Cancer
Safety, Preliminary Effectiveness of BNT327, an Investigational Therapy for Patients With Small-cell Lung Cancer in Combination With Chemotherapy
This is a Phase II, multi-site, open-label, parallel group study in participants with untreated extended-stage small-cell lung cancer (ES-SCLC) (Cohort 1) or small-cell lung cancer (SCLC) which has progressed on first- or…
- Trial name
- NCT06449209
- Testing
- BNT327 Dose Level 1 (DL1), BNT327 Dose Level 2 (DL2), Etoposide
- Where
- 64 sites in United States, Australia and 3 more
- Volunteers
- 110 people
- Sponsor
- BioNTech SE
Neurologic Disorder, Paralysis
COMMAND Early Feasibility Study: Implantable BCI to Control a Digital Device for People With Paralysis
The Synchron motor neuroprosthesis (MNP) is intended to be used in subjects with severe motor impairment, unresponsive to medical or rehabilitative therapy and a persistent functioning motor cortex.
- Conditions
- Neurologic Disorder, Paralysis, Paralysis; Stroke, Amyotrophic Lateral Sclerosis
- Trial name
- NCT05035823
- Testing
- Motor Neuroprosthesis (MNP)
- Where
- 3 sites in United States
- Volunteers
- 6 people
- Sponsor
- Synchron Medical, Inc.
MELAS, LHON
A Dose-escalating Clinical Trial With KH176
Mitochondrial Diseases are rare progressive, multi-system, often early fatal disorders affecting both children and adults. KH176 is a novel chemical entity currently under development for the treatment of inherited mitochondrial…
- Conditions
- MELAS, LHON, Leigh Syndrome, Mitochondrial Disease
- Trial name
- NCT02544217
- Testing
- KH176
- Where
- Belgium
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Khondrion BV
Pulmonary Hypertension Associated With HFpEF
Open-label Extension of Oral Treprostinil in Subjects With PH Associated With HFpEF
This was an open-label study to evaluate the safety of continued therapy with oral treprostinil in subjects who completed Study TDE-HF-301.
- Trial name
- NCT03043651
- Testing
- Oral treprostinil
- Where
- 82 sites in United States
- Volunteers
- 48 people
- Sponsor
- United Therapeutics
Non-Small Cell Lung Cancer, Multiple Myeloma
Common Safety Follow-up Trial of Tecemotide (L-BLP25)
This is an open-label, common follow-up trial. Subjects who were enrolled in a Merck KGaA, EMD Serono or Merck Serono Japan sponsored trial with tecemotide (L-BLP25) were enrolled in this follow-up trial to continue their…
- Trial name
- NCT01423760
- Testing
- Tecemotide, No intervention
- Where
- Germany
- Volunteers
- 27 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Non-Hodgkin Lymphoma (NHL), Multiple Myeloma (MM)
Study Evaluating Venetoclax in Subjects With Hematological Malignancies
This study is evaluating the safety, pharmacokinetic profile and efficacy of venetoclax under a once daily dosing schedule in Japanese participants with hematological malignancies.
- Conditions
- Non-Hodgkin Lymphoma (NHL), Multiple Myeloma (MM), Chronic Lymphocytic Leukemia (CLL), Small Lymphocytic Lymphoma (SLL)
- Trial name
- NCT02265731
- Testing
- azacitadine, venetoclax, rituximab / IDEC-C2B8
- Where
- 14 sites in Japan
- Volunteers
- 38 people
- Sponsor
- AbbVie
Follicular Lymphoma (FL), CLL
ACP-196 (Acalabrutinib) in Combination With Pembrolizumab, for Treatment of Hematologic Malignancies
This study is evaluating the safety, pharmacodynamics (PD), and efficacy of acalabrutinib and pembrolizumab in hematologic malignancies.
- Conditions
- Follicular Lymphoma (FL), CLL, Small Lymphocytic Lymphoma (SLL), Richter's Syndrome
- Trial name
- KEYNOTE145 (NCT02362035)
- Testing
- Acalabrutinib, Pembrolizumab
- Where
- 19 sites in United States
- Volunteers
- 161 people
- Sponsor
- Acerta Pharma BV