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Showing 1–24 of 419 matching trials from the live ClinicalTrials.gov search.
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Approved For Marketing No phase listed Expanded access

Treatment of Angioedema Attacks in Adolescent and Adult Patients 12 Years and Older With HAE Type I or II With Sebetralstat

NCT06628713
View directory record Official record
Conditions
Hereditary Angioedema
Interventions
Sebetralstat
Drug
Lead sponsor
KalVista Pharmaceuticals, Ltd.
Industry
Eligibility
12 Years and older
U.S. locations
1
States / cities
Scottsdale, Arizona
Source: ClinicalTrials.gov public record
Updated Aug 5, 2025 · Synced Sep 17, 2026, 5:15 AM EDT
Completed Phase 2 Interventional Results available

Reduced Intensity Conditioning for Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (BMT CTN 1204)

NCT01998633
View directory record Official record
Conditions
Hemophagocytic Lymphohistiocytosis, Chronic Active Epstein-Barr Virus Infection, Chronic Granulomatous Disease, HIGM-1, Leukocyte Adhesion Deficiency, IPEX
Interventions
Hematopoietic Stem Cell Transplant
Biological
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
4 Months to 45 Years
Enrollment
47 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
19
States / cities
Birmingham, Alabama • Washington D.C., District of Columbia • Atlanta, Georgia + 16 more
Source: ClinicalTrials.gov public record
Updated Dec 7, 2022 · Synced Sep 17, 2026, 5:15 AM EDT
Completed No phase listed Observational

Patients Treated for Wiskott-Aldrich Syndrome (WAS) Since 1990

NCT02064933
View directory record Official record
Conditions
Wiskott-Aldrich Syndrome
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Male only
Enrollment
305 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
38
States / cities
Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 33 more
Source: ClinicalTrials.gov public record
Updated Aug 9, 2020 · Synced Sep 17, 2026, 5:15 AM EDT
Completed Phase 3 Interventional

An Open-label Extension Trial to Evaluate the Long-term Safety of KVD900 (Sebetralstat) for On-Demand Treatment of Angioedema Attacks in Adolescent and Adult Patients With Hereditary Angioedema (HAE)

NCT05505916
View directory record Official record
Conditions
Hereditary Angioedema
Interventions
KVD900 600 mg, KVD900 300 mg
Drug
Lead sponsor
KalVista Pharmaceuticals, Ltd.
Industry
Eligibility
12 Years and older
Enrollment
145 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
20
States / cities
Scottsdale, Arizona • Little Rock, Arkansas • San Diego, California + 16 more
Source: ClinicalTrials.gov public record
Updated Jun 4, 2026 · Synced Sep 17, 2026, 5:15 AM EDT
Not listed No phase listed Observational

A Single-arm Safety Study of Transplantation Using Umbilical Cord Blood and Human Placental-derived Stem Cells From Partially Matched Related Donors in Persons With Certain Malignant Blood Diseases and Non-malignant Disorders

NCT00596999
View directory record Official record
Conditions
Hematologic Malignancies
Interventions
UCB and HPDSC
Procedure
Lead sponsor
Celgene Corporation
Industry
Eligibility
Up to 55 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2013
U.S. locations
1
States / cities
New Orleans, Louisiana
Source: ClinicalTrials.gov public record
Updated Apr 9, 2012 · Synced Sep 17, 2026, 5:15 AM EDT
Completed Phase 2 Interventional

CMV Retinitis Retreatment Trial

NCT00000766
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Foscarnet sodium, Ganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years and older
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
9
States / cities
La Jolla, California • Los Angeles, California • San Francisco, California + 4 more
Source: ClinicalTrials.gov public record
Updated Feb 28, 2011 · Synced Sep 17, 2026, 5:15 AM EDT
Completed Phase 2 Interventional

Interferon Gamma to Treat Leukocyte Adhesion Deficiency Type I

NCT00001905
View directory record Official record
Conditions
Leukocyte Adhesion Deficiency Syndrome
Interventions
Interferon gamma
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Not listed
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Sep 17, 2026, 5:15 AM EDT
Completed Phase 1 Interventional

A Phase I/II Open-Labelled Trial of Intravitreal Ganciclovir Salvage Therapy for AIDS Patients With Active CMV Retinitis Who Are Intolerant of Systemic Therapy

NCT00000673
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Ganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
13 Years to 65 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Ends 1993
U.S. locations
2
States / cities
Miami, Florida • St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Oct 26, 2021 · Synced Sep 17, 2026, 5:15 AM EDT
Terminated Phase 1 Interventional Results available

Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases

NCT02231710
View directory record Official record
Conditions
Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies, Metabolic Disorders
Interventions
BPX-501 and Rimiducid
Biological
Lead sponsor
Bellicum Pharmaceuticals
Industry
Eligibility
4 Months to 55 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Mar 24, 2024 · Synced Sep 17, 2026, 5:15 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant

NCT03301168
View directory record Official record
Conditions
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes, Primary Immune Deficiency Disorder, Osteopetrosis, Cytopenia, Hemoglobinopathy in Children, Anemia, Aplastic
Interventions
BPX-501 T cells, Rimiducid
Biological · Drug
Lead sponsor
Bellicum Pharmaceuticals
Industry
Eligibility
1 Month to 26 Years
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2034
U.S. locations
10
States / cities
Los Angeles, California • Palo Alto, California • Washington D.C., District of Columbia + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 11, 2022 · Synced Sep 17, 2026, 5:15 AM EDT
Recruiting Phase 2 Interventional

Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures

NCT04232085
View directory record Official record
Conditions
Primary Immune Deficiency Disorder, Immune Deficiency Disease, Bone Marrow Failure, Short Telomere Length, Fanconi Anemia, Non Fanconi DNA-DSB Repair Disorder, Hoyeraal-Hreidarsson Syndrome, Dyskeratosis Congenita, Telomere Biology Disorder, Short Telomere Syndrome
Interventions
Alemtuzumab, Fludarabine, Melphalan, Low Dose Total Body Irradiation, Cyclophosphamide, Tacrolimus, Mycophenolate Mofetil
Drug · Radiation
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Other
Eligibility
4 Months to 50 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2028
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Nov 27, 2025 · Synced Sep 17, 2026, 5:15 AM EDT
Completed Not applicable Interventional

An Open-Label Safety Study of Oral Ganciclovir Maintenance Treatment of CMV Retinitis in People With Limited Venous Access

NCT00002135
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Ganciclovir
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
13 Years and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Sep 17, 2026, 5:15 AM EDT
Completed Phase 1 Interventional

Interleukin-2 Treatment for Wiskott-Aldrich Syndrome

NCT00774358
View directory record Official record
Conditions
Wiskott-Aldrich Syndrome (WAS), X-linked Thrombocytopenia
Interventions
Interleukin-2
Drug
Lead sponsor
Soma Jyonouchi
Other
Eligibility
24 Months and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2016
U.S. locations
3
States / cities
Philadelphia, Pennsylvania • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Aug 15, 2017 · Synced Sep 17, 2026, 5:15 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Natural History of Bronchiectasis

NCT00943514
View directory record Official record
Conditions
Bronchiectasis, Cystic Fibrosis, Autoimmune Disease, Common Variable Immunodeficiency
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
5 Years to 100 Years
Enrollment
900 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2009
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 31, 2026 · Synced Sep 17, 2026, 5:15 AM EDT
Not listed Phase 1Phase 2 Interventional

Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency

NCT03315078
View directory record Official record
Conditions
X-Linked Combined Immunodeficiency Diseases
Interventions
CD34+ HSCs transduced with the lentivirus vector, VSV-G pseudotyped CL20-4i-EF1α-hγc-OPT, Palifermin, Busulfan
Biological · Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
2 Years to 40 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2022
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 3, 2019 · Synced Sep 17, 2026, 5:15 AM EDT
Completed Phase 2 Interventional

Stem Cell Transplantation to Treat Leukocyte Adhesion Deficiency

NCT00031005
View directory record Official record
Conditions
Leukocyte-Adhesion Deficiency Syndrome
Interventions
modified stem cell transplant
Procedure
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Not listed
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Sep 17, 2026, 5:15 AM EDT
Completed Phase 1 Interventional

A Pharmacokinetic and Tolerance Study of Oral Ganciclovir in HIV-Infected Children With Asymptomatic Cytomegalovirus Infection and Low CD4 Cell Counts or Quiescent Cytomegalovirus Disease

NCT00000805
View directory record Official record
Conditions
Cytomegalovirus Infections, HIV Infections
Interventions
Ganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
14 Days to 20 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Ends 1998
U.S. locations
18
States / cities
Los Angeles, California • Oakland, California • San Diego, California + 10 more
Source: ClinicalTrials.gov public record
Updated Nov 2, 2021 · Synced Sep 17, 2026, 5:15 AM EDT
Completed Phase 3 Interventional Results available

Pharmacokinetics, Efficacy, and Safety Study of RI-002 (IGIV) in Subjects With Primary Immunodeficiency Diseases (PIDD)

NCT01814800
View directory record Official record
Conditions
Primary Immune Deficiency Disorder
Interventions
RI-002
Biological
Lead sponsor
ADMA Biologics, Inc.
Industry
Eligibility
2 Years to 75 Years
Enrollment
59 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
9
States / cities
Cenntennial, Colorado • North Palm Beach, Florida • Atlanta, Georgia + 5 more
Source: ClinicalTrials.gov public record
Updated Oct 4, 2016 · Synced Sep 17, 2026, 5:15 AM EDT
Completed Early Phase 1 Interventional

Baclofen Treatment of Ataxia Telangiectasia

NCT00640003
View directory record Official record
Conditions
Ataxia Telangiectasia
Interventions
Baclofen, Placebo
Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
12 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2011
U.S. locations
2
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 19, 2017 · Synced Sep 17, 2026, 5:15 AM EDT
Not listed No phase listed Observational

Study of Genetic and Molecular Defects in Primary Immunodeficiency Disorders

NCT00004341
View directory record Official record
Conditions
X-Linked Agammaglobulinemia, X-Linked Hyper IgM Syndrome, Wiskott-Aldrich Syndrome, Leukocyte Adhesion Deficiency Syndrome
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
0 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1995
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Sep 17, 2026, 5:15 AM EDT
Recruiting Phase 1Phase 2 Interventional

Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning

NCT03311503
View directory record Official record
Conditions
Severe Combined Immunodeficiency, X Linked, Gene Therapy
Interventions
autologous CD34+ cell transduced with G2SCID vector
Biological
Lead sponsor
David Williams
Other
Eligibility
0 Years to 5 Years · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2028
U.S. locations
4
States / cities
Los Angeles, California • Atlanta, Georgia • Boston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Dec 10, 2025 · Synced Sep 17, 2026, 5:15 AM EDT
Completed Phase 4 Interventional

Contribution of Dolutegravir to Obesity and Cardiovascular Disease

NCT04340388
View directory record Official record
Conditions
HIV-1-infection, Antiviral Drug Adverse Reaction, Vascular Diseases, Cardiovascular Abnormalities, Abnormality of Adipose Tissue, Body Weight Changes, Body Fat Disorder, HIV-Associated Lipodystrophy Syndrome
Interventions
Dolutegravir 50 MG, Antiretroviral/Anti HIV
Drug
Lead sponsor
Augusta University
Other
Eligibility
18 Years to 100 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
1
States / cities
Augusta, Georgia
Source: ClinicalTrials.gov public record
Updated Jul 26, 2023 · Synced Sep 17, 2026, 5:15 AM EDT
Enrolling by invitation Phase 1Phase 2 Interventional

A Study of the Safety and Efficacy of Prime Editing (PM359) in Participants With p47phox Autosomal Recessive Chronic Granulomatous Disease (CGD )

NCT06559176
View directory record Official record
Conditions
Chronic Granulomatous Disease, Granulomatous Disease, Chronic
Interventions
PM359
Biological
Lead sponsor
Prime Medicine, Inc.
Industry
Eligibility
6 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
3
States / cities
Los Angeles, California • Bethesda, Maryland • Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Apr 5, 2026 · Synced Sep 17, 2026, 5:15 AM EDT
Completed Phase 3 Interventional Results available

Study of Subcutaneous Immunoglobulin in Patients With PID Requiring IgG Replacement Therapy

NCT00419341
View directory record Official record
Conditions
Primary Immune Deficiency
Interventions
Human Normal Immunoglobulin for Subcutaneous Administration
Biological
Lead sponsor
CSL Behring
Industry
Eligibility
2 Years to 75 Years
Enrollment
49 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2008
U.S. locations
13
States / cities
Los Angeles, California • Centennial, Colorado • North Palm Beach, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Jan 24, 2013 · Synced Sep 17, 2026, 5:15 AM EDT