- Conditions
- AMPD3, OMIM*102772, AMP Deaminase Deficiency, AK1, OMIM *103000, Adenylate Kinase Deficiency, AMPD1, OMIM *102770, Myopathy Due to Myoadenylate Deaminase Deficiency, TPMT, OMIM *187680, Thoipurines, Poor Metabolism of, IMPDH1, OMIM *146690, Retinitis Pigmentosa Type 10, Leber Congenital Amauriosis Type 11, APRT, OMIM *102600, Adenine Phosphoribosyltransferase Deficiency, HPRT1, OMIM *308000 Lesch-Nyhan Disease, XDH, OMIM *607633, Xanthinuria Type 1, SLC2A9, OMIM *606142 Hypouricemia, SLC22A12, OMIM *607096 Hypouricemia, PRPS1 Def, OMIM *311850, Arts Syndrome; Charcot-Marie-Tooth Disease, PRPS1 SA, OMIM *311850 Gout, PRPS-related Phosphoribosylpyrophosphate Synthetase Superactivity, AMPD2, OMIM *102771, Spastic Paraplegia 63; Pontocerebellar Hypoplasia, ITPA, OMIM *147520, Inosine Triphosphatase Deficiency; Developmental and Epileptic Encephalopathy 35, ADSL, OMIM *608222, Adenylosuccinate Lyase Deficiency, PNP, OMIM *164050, Nucleoside Phosphorylase Deficiency, ADA2, OMIM *607575,Sneddon Syndrome; VAIHS, CAD, *1140120, Developmental and Epileptic Encephalopathy, UPB1, OMIM *606673, Beta-ureidopropionase Deficiency, DPYS, OMIM *613326, Dihydropyrimidinase Deficiency, DPYD, OMIM *274270, Dihydropyrimidine Dehydrogenase Deficiency, DHODH, OMIM *126064, Miller Syndrome (Postaxial Acrofacial Dysostosis), UMPS, OMIM *613891, Orotic Aciduria, NT5C3A<TAB>, OMIM *606224, Anemia, Hemolytic, Due to UMPH1 Deficiency, UNG, OMIM *191525, Hyper-IgM Syndrome 5, AICDA, OMIM *605257, Immunodeficiency With Hyper-IgM, Type 2; HIGM2, Purine-Pyrimidine Metabolism, Metabolic Disease
- Interventions
- Not listed
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 1 Month to 100 Years
- Enrollment
- 999 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2099
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
Search Results
Search by objective public record fields.
Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results are retrieved from ClinicalTrials.gov and synchronized into the directory. Search pages remain noindex by default.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Showing 1–7
of 7
matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
- Conditions
- Chronic Obstructive Pulmonary Disease, Alpha1-antitrypsin Deficiency
- Interventions
- ARALAST NP 60 mg/kg, ARALAST NP 120 mg/kg, GLASSIA 60 mg/kg, GLASSIA 120 mg/kg, Human Albumin 2%
- Biological
- Lead sponsor
- Baxalta now part of Shire
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2018
- U.S. locations
- 14
- States / cities
- Peoria, Arizona • Newport Beach, California • Kissimmee, Florida + 11 more
- Conditions
- Adenosine Deaminase Deficiency, Autosomal Recessive Disorder, Immune System Disorder, Purine-Nucleoside Phosphorylase Deficiency, Severe Combined Immunodeficiency, Severe Combined Immunodeficiency With Absence of T and B Cells, X-Linked Severe Combined Immunodeficiency
- Interventions
- Allogeneic Bone Marrow Transplantation, Cyclosporine, Laboratory Biomarker Analysis, Mycophenolate Mofetil, Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation, Total-Body Irradiation
- Procedure · Drug · Other + 1 more
- Lead sponsor
- Fred Hutchinson Cancer Center
- Other
- Eligibility
- Not listed
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1997 – 2018
- U.S. locations
- 1
- States / cities
- Seattle, Washington
- Conditions
- Diabetes Mellitus, Type 1
- Interventions
- Aralast NP, Placebo
- Drug
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- 8 Years to 35 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2014
- U.S. locations
- 15
- States / cities
- La Jolla, California • Aurora, Colorado • New Haven, Connecticut + 10 more
- Conditions
- HIV Infections
- Interventions
- sD-NP-GT8 DNA, IL-12 DNA, Trimer 4571, 3M-052-AF, Alum
- Biological · Drug
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 46 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2023 – 2025
- U.S. locations
- 4
- States / cities
- Birmingham, Alabama • San Francisco, California • Boston, Massachusetts
- Conditions
- Diabetes Mellitus, Type 1
- Interventions
- Aralast NP 45 mg dose, Aralast NP 90 mg dose, Aralast NP 180 mg dose
- Biological
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- 8 Years to 35 Years
- Enrollment
- 17 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2013
- U.S. locations
- 15
- States / cities
- San Diego, California • Aurora, Colorado • New Haven, Connecticut + 10 more
- Conditions
- MLD, Krabbe Disease, ALD, MPS I, MPS II, MPS III, Vanishing White Matter Disease, GM3 Gangliosidosis, PKAN, Tay-Sachs Disease, NP Deficiency, Osteopetrosis, Alpha-Mannosidosis, Sandhoff Disease, Niemann-Pick Diseases, MPS IV, Gaucher Disease, GAN, GM1 Gangliosidoses, Morquio Disease, S-Adenosylhomocysteine Hydrolase Deficiency, Batten Disease, Pelizaeus-Merzbacher Disease, Leukodystrophy, Lysosomal Storage Diseases, Purine Nucleoside Phosphorylase Deficiency, Multiple Sulfatase Deficiency Disease
- Interventions
- Palliative Care, Hematopoetic Stem Cell Transplantation
- Other · Biological
- Lead sponsor
- University of Pittsburgh
- Other
- Eligibility
- Not listed
- Enrollment
- 1,500 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2035
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania