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Showing 1–24 of 71 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Hemoglobinuria Clear all
Completed No phase listed Observational Accepts healthy volunteers

Microvascular and Cardiac Dysfunction in Paroxysmal Nocturnal Hemoglobinuria and Sickle Cell Disease

NCT01294891
View directory record Official record
Conditions
Rheologic Disease, Sickle Cell Disease, Paroxysmal Nocturnal Hemoglobinuria
Interventions
Imaging
Other
Lead sponsor
Oregon Health and Science University
Other
Eligibility
19 Years and older
Enrollment
6 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2014
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Oct 27, 2014 · Synced Jul 23, 2026, 1:31 AM EDT
Completed Phase 3 Interventional Results available

Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 Who Switch to Iptacopan.

NCT05630001
View directory record Official record
Conditions
Paroxysmal Nocturnal Hemoglobinuria
Interventions
Iptacopan
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years to 100 Years
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
9
States / cities
Duarte, California • Los Angeles, California • Miami Lakes, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Jan 12, 2026 · Synced Jul 23, 2026, 1:31 AM EDT
Completed Phase 3 Interventional Results available

ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab

NCT03056040
View directory record Official record
Conditions
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Interventions
Ravulizumab, Eculizumab
Biological
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
202 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
5
States / cities
Duarte, California • Los Angeles, California • Baltimore, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Mar 11, 2025 · Synced Jul 23, 2026, 1:31 AM EDT
Completed Phase 3 Interventional

Haploidentical Stem Cell Transplantation for Patients With Hematologic Malignancies

NCT00186823
View directory record Official record
Conditions
Leukemia, Acute Lymphocytic (ALL), Leukemia, Myeloid, Acute(AML), Leukemia, Myeloid, Chronic(CML), Juvenile Myelomonocytic Leukemia(JMML), Hemoglobinuria, Paroxysmal Nocturnal (PNH), Lymphoma, Non-Hodgkin (NHL), Myelodysplastic Syndrome (MDS)
Interventions
Miltenyi Biotec CliniMACS, Stem Cell Transplantation, TBI, systemic chemotherapy and antibodies as follows:
Device · Procedure · Drug
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
2 Years to 21 Years
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2009
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jan 28, 2009 · Synced Jul 23, 2026, 1:31 AM EDT
Terminated Not applicable Interventional

Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

NCT02179359
View directory record Official record
Conditions
Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia, Diamond Blackfan Anemia, Paroxysmal Nocturnal Hemoglobinuria, Glanzmann Thrombasthenia, Severe Congenital Neutropenia, Shwachman-Diamond Syndrome, Non-Malignant Hematologic Disorders
Interventions
Reduced Toxicity Ablative Regimen, Reduced Intensity Preparative Regimen, Myeloablative Preparative Regimen
Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 55 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2024
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Mar 2, 2025 · Synced Jul 23, 2026, 1:31 AM EDT
Completed Phase 1 Interventional

Chemotherapy Plus Sargramostim in Treating Patients With Refractory Myeloid Cancer

NCT00012376
View directory record Official record
Conditions
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Blastic Phase Chronic Myelogenous Leukemia, Chronic Myelomonocytic Leukemia, Chronic Phase Chronic Myelogenous Leukemia, Paroxysmal Nocturnal Hemoglobinuria, Previously Treated Myelodysplastic Syndromes, Recurrent Adult Acute Myeloid Leukemia, Refractory Anemia, Refractory Anemia With Ringed Sideroblasts, Relapsing Chronic Myelogenous Leukemia, Thrombocytopenia, Untreated Adult Acute Myeloid Leukemia
Interventions
bryostatin 1, sargramostim, laboratory biomarker analysis, pharmacological study
Drug · Biological · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2001
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 8, 2013 · Synced Jul 23, 2026, 1:31 AM EDT
Recruiting No phase listed Observational

Global PNH Patient Registry

NCT05755867
View directory record Official record
Conditions
Paroxysmal Nocturnal Hemoglobinuria
Interventions
Not listed
Lead sponsor
Aplastic Anemia and MDS International Foundation
Other
Eligibility
Not listed
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2031
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Feb 19, 2025 · Synced Jul 23, 2026, 1:31 AM EDT
Completed Phase 3 Interventional

Stem Cell Transplantation for Patients With Hematologic Malignancies

NCT00152139
View directory record Official record
Conditions
Acute Lymphoblastic Leukemias, Acute Myelocytic Leukemia, Chronic Myeloid Leukemia, Juvenile Myelomonocytic Leukemia, Myelodysplastic Syndrome, Hemoglobinuria, Paroxysmal, Non-Hodgkin Lymphoma
Interventions
Allogeneic Stem Cell Transplantation, Chemotherapy and antibodies
Procedure · Drug
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
2 Years to 21 Years
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2009
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jan 28, 2009 · Synced Jul 23, 2026, 1:31 AM EDT
Completed Phase 2 Interventional Results available

Basiliximab #2: In-Vivo Activated T-Cell Depletion to Prevent Graft-Versus_Host Disease (GVHD) After Nonmyeloablative Allotransplantation for the Treatment of Blood Cancer

NCT00975975
View directory record Official record
Conditions
Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia, Chronic Myelogenous Leukemia, Chronic Lymphocytic Leukemia, Myelodysplasia, Non-Hodgkin's Lymphoma, Hodgkin's Disease, Multiple Myeloma, Myelofibrosis, Anemia, Aplastic, Hemoglobinuria, Paroxysmal
Interventions
Basiliximab
Drug
Lead sponsor
Indiana University
Other
Eligibility
18 Years and older
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2013
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Feb 25, 2016 · Synced Jul 23, 2026, 1:31 AM EDT
Recruiting Phase 3 Interventional

Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients

NCT06934967
View directory record Official record
Conditions
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Interventions
LNP023
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 18 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2031
U.S. locations
3
States / cities
New Brunswick, New Jersey • Philadelphia, Pennsylvania • Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Apr 15, 2026 · Synced Jul 23, 2026, 1:31 AM EDT
Completed Phase 2 Interventional Results available

Trial of Allogeneic Stem Cell Transplants From HLA Compatible, Related and Unrelated Donors After a Myeloablative Preparative Regimen With Hyperfractionated TBI, Thiotepa and Fludarabine For Adult Patients With Lymphohematopoietic Disorders

NCT00587054
View directory record Official record
Conditions
Allogeneic Stem Cell Transplant, Leukemia, Non-Hodgkins, Lymphoblastic Lymphoma, Myelodysplastic Syndrome, Paroxysmal Nocturnal Hemoglobinuria (PNH)
Interventions
cytoreductive regimen followed by a CD34+E- selected allogeneic stem cell transplant
Drug
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
18 Years to 55 Years
Enrollment
129 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2011
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 9, 2017 · Synced Jul 23, 2026, 1:31 AM EDT
Terminated Phase 2 Interventional Results available

Extension Study of RA101495 for Patients With PNH Who Have Completed a Zilucoplan (RA101495) Clinical Study

NCT03225287
View directory record Official record
Conditions
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Interventions
Zilucoplan (RA101495)
Drug
Lead sponsor
Ra Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
2
States / cities
Los Angeles, California • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Sep 27, 2023 · Synced Jul 23, 2026, 1:31 AM EDT
Active, not recruiting Phase 3 Interventional

Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria

NCT04747613
View directory record Official record
Conditions
Paroxysmal Nocturnal Hemoglobinuria
Interventions
Iptacopan
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years to 100 Years
Enrollment
208 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
11
States / cities
Duarte, California • Los Angeles, California • Orange, California + 5 more
Source: ClinicalTrials.gov public record
Updated Jul 9, 2026 · Synced Jul 23, 2026, 1:31 AM EDT
Recruiting Phase 2 Interventional

A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

NCT04901936
View directory record Official record
Conditions
Paroxysmal Nocturnal Hemoglobinuria (PNH), Paroxysmal Hemoglobinuria
Interventions
Pegcetacoplan
Drug
Lead sponsor
Apellis Pharmaceuticals, Inc.
Industry
Eligibility
12 Years to 17 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2028
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Jun 25, 2026 · Synced Jul 23, 2026, 1:31 AM EDT
Terminated Phase 1 Interventional

Safety and Pharmacokinetics of TT30 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)

NCT01335165
View directory record Official record
Conditions
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Interventions
ALXN1102, ALXN1103
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated May 21, 2014 · Synced Jul 23, 2026, 1:31 AM EDT
Completed Phase 2 Interventional Results available

Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients Who Have an Inadequate Response to Eculizumab

NCT03030183
View directory record Official record
Conditions
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Interventions
Zilucoplan (RA101495)
Drug
Lead sponsor
Ra Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
6
States / cities
Duarte, California • Los Angeles, California • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Jul 26, 2022 · Synced Jul 23, 2026, 1:31 AM EDT
Completed Phase 3 Interventional Results available

Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab

NCT03748823
View directory record Official record
Conditions
Paroxysmal Nocturnal Hemoglobinuria
Interventions
Ravulizumab OBDS, Ravulizumab
Combination Product · Biological
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
139 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Sep 18, 2024 · Synced Jul 23, 2026, 1:31 AM EDT
Completed Phase 2 Interventional Results available

Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes

NCT00004143
View directory record Official record
Conditions
Sickle Cell Anemia, Severe Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria (PNH), Pure Red Cell Aplasia
Interventions
Campath, Chemo and/or TBI Allo SCT
Drug
Lead sponsor
David Rizzieri, MD
Other
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2009
U.S. locations
2
States / cities
Orlando, Florida • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Dec 4, 2014 · Synced Jul 23, 2026, 1:31 AM EDT
Completed Phase 3 Interventional Results available

Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody Treatment

NCT04558918
View directory record Official record
Conditions
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Interventions
LNP023, Eculizumab, Ravulizumab
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
97 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
5
States / cities
Duarte, California • Orange, California • Augusta, Georgia + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 8, 2024 · Synced Jul 23, 2026, 1:31 AM EDT
Completed Not applicable Interventional

Nonmyeloablative Allogeneic Transplant

NCT01272817
View directory record Official record
Conditions
Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria, Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia, Myelodysplastic Syndrome, Chronic Myelogenous Leukemia, Chronic Lymphocytic Leukemia, Hodgkin's Lymphoma, Non-Hodgkin's Lymphoma, Mantle Cell Lymphoma, Multiple Myeloma, Waldenstrom Macroglobulinemia, Breast Cancer, Renal Cell Carcinoma, Melanoma, Sarcoma, Ovarian Cancer, Thymoma
Interventions
Nonmyeloablative Allogeneic Transplant
Procedure
Lead sponsor
Scripps Health
Other
Eligibility
18 Years to 72 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2015
U.S. locations
1
States / cities
La Jolla, California
Source: ClinicalTrials.gov public record
Updated Nov 22, 2022 · Synced Jul 23, 2026, 1:31 AM EDT
Completed No phase listed Observational

Transfer of GPI-Linked Proteins to Transfused Patients With Paroxysmal Nocturnal Hemoglobinuria

NCT00039923
View directory record Official record
Conditions
Paroxysmal Hemoglobinuria
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
Not listed
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Jul 23, 2026, 1:31 AM EDT
Recruiting No phase listed Observational

The International PNH Interest Group PNH Registry

NCT06524726
View directory record Official record
Conditions
Paroxysmal Nocturnal Hemoglobinuria
Interventions
Not listed
Lead sponsor
International PNH Interest Group
Other
Eligibility
Not listed
Enrollment
2,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
1
States / cities
Altamonte Springs, Florida
Source: ClinicalTrials.gov public record
Updated Jul 28, 2024 · Synced Jul 23, 2026, 1:31 AM EDT
Completed Phase 2 Interventional Results available

Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies

NCT01529827
View directory record Official record
Conditions
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Adult Grade III Lymphomatoid Granulomatosis, Adult Nasal Type Extranodal NK/T-cell Lymphoma, Anaplastic Large Cell Lymphoma, Angioimmunoblastic T-cell Lymphoma, Aplastic Anemia, Burkitt Lymphoma, Childhood Acute Lymphoblastic Leukemia in Remission, Childhood Acute Myeloid Leukemia in Remission, Childhood Chronic Myelogenous Leukemia, Childhood Diffuse Large Cell Lymphoma, Childhood Grade III Lymphomatoid Granulomatosis, Childhood Immunoblastic Large Cell Lymphoma, Childhood Myelodysplastic Syndromes, Childhood Nasal Type Extranodal NK/T-cell Lymphoma, Chronic Myelomonocytic Leukemia, Chronic Phase Chronic Myelogenous Leukemia, Congenital Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue, Hepatosplenic T-cell Lymphoma, Juvenile Myelomonocytic Leukemia, Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable, Nodal Marginal Zone B-cell Lymphoma, Paroxysmal Nocturnal Hemoglobinuria, Peripheral T-cell Lymphoma, Polycythemia Vera, Post-transplant Lymphoproliferative Disorder, Previously Treated Myelodysplastic Syndromes, Primary Myelofibrosis, Recurrent Adult Acute Lymphoblastic Leukemia, Recurrent Adult Acute Myeloid Leukemia, Recurrent Adult Burkitt Lymphoma, Recurrent Adult Diffuse Large Cell Lymphoma, Recurrent Adult Diffuse Mixed Cell Lymphoma, Recurrent Adult Diffuse Small Cleaved Cell Lymphoma, Recurrent Adult Grade III Lymphomatoid Granulomatosis, Recurrent Adult Hodgkin Lymphoma, Recurrent Adult Immunoblastic Large Cell Lymphoma, Recurrent Adult Lymphoblastic Lymphoma, Recurrent Adult T-cell Leukemia/Lymphoma, Recurrent Childhood Acute Lymphoblastic Leukemia, Recurrent Childhood Acute Myeloid Leukemia, Recurrent Childhood Anaplastic Large Cell Lymphoma, Recurrent Childhood Grade III Lymphomatoid Granulomatosis, Recurrent Childhood Large Cell Lymphoma, Recurrent Childhood Lymphoblastic Lymphoma, Recurrent Childhood Small Noncleaved Cell Lymphoma, Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma, Recurrent Grade 1 Follicular Lymphoma, Recurrent Grade 2 Follicular Lymphoma, Recurrent Grade 3 Follicular Lymphoma, Recurrent Mantle Cell Lymphoma, Recurrent Marginal Zone Lymphoma, Recurrent Mycosis Fungoides/Sezary Syndrome, Recurrent Small Lymphocytic Lymphoma, Recurrent/Refractory Childhood Hodgkin Lymphoma, Refractory Chronic Lymphocytic Leukemia, Refractory Hairy Cell Leukemia, Refractory Multiple Myeloma, Secondary Acute Myeloid Leukemia, Secondary Myelodysplastic Syndromes, Secondary Myelofibrosis, Severe Combined Immunodeficiency, Severe Congenital Neutropenia, Shwachman-Diamond Syndrome, Splenic Marginal Zone Lymphoma, T-cell Large Granular Lymphocyte Leukemia, Waldenstrom Macroglobulinemia, Wiskott-Aldrich Syndrome
Interventions
fludarabine phosphate, melphalan, total-body irradiation, tacrolimus, mycophenolate mofetil, methotrexate, laboratory biomarker analysis, allogeneic hematopoietic stem cell transplantation, peripheral blood stem cell transplantation
Drug · Radiation · Other + 1 more
Lead sponsor
Roswell Park Cancer Institute
Other
Eligibility
3 Years to 75 Years
Enrollment
94 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2019
U.S. locations
1
States / cities
Buffalo, New York
Source: ClinicalTrials.gov public record
Updated Sep 23, 2019 · Synced Jul 23, 2026, 1:31 AM EDT