NIH Clinical Center: Search the Studies

Search the Studies

50 Studies found from all possible combinations of your search terms. Select a protocol that you wish to review.

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Protocol Number	Title	Protocol Status	Min-Max Age	Institute	Keywords
000686-CH	Intrathecal 2-Hydroxypropyl-Beta-Cyclodextrin for Neurological Decline in Patients with Niemann-Pick Disease Type C1	Participants currently recruited/enrolled	18-125 Years	NICHD	Autosomal Recessive
18-I-0022	Use of Ustekinumab (Anti-IL-12/23p40 Monoclonal Antibody) in Patients with Leukocyte Adhesion Deficiency Type 1 (LAD1) who have Inflammatory Pathology	Participants currently recruited/enrolled	12-65 Years	NIAID	Autosomal Recessive
18-HG-0064	A Natural History Study of Patients with Generalized Arterial Calcification of Infancy (GACI) or Autosomal Recessive Hypophosphatemic Rickets Type 2 (ARHR2)	Completed Study; data analyses ongoing	0-125 Years	NHGRI	Autosomal Recessive
15-H-0155	Hereditary Parkinson Disease Natural History Protocol	Completed Study; data analyses ongoing	18-80 Years	NHLBI	Autosomal Recessive
00-HG-0153	Investigations into Chediak-Higashi Syndrome and Related Disorders	Participants currently recruited/enrolled	0-70 Years	NHGRI	Autosomal Recessive
98-CH-0081	Clinical and Basic Investigations into Smith-Lemli-Opitz Syndrome	Completed Study; data analyses ongoing	0-125 Years	NICHD	Autosomal Recessive
001109-C	Defining the Natural History of Squamous Cell Carcinoma in Fanconi Anemia	Participants currently recruited/enrolled	8-125 Years	NCI	Anemia
001061-H	Observational Study Investigating the Mechanistic Effects of Mitapivat in Subjects with Sickle Cell Disease	Enrolling by Invitation	16-125 Years	NHLBI	Anemia
000760-H	Extension Study (extended access) of Syk-inhibition Using Fostamatinib to Treat Post-Transplant Immune-mediated Cytopenias	Enrolling by Invitation	18-75 Years	NHLBI	Anemia
000758-H	A Phase II Study Using Fostamatinib to Treat Post-Hematopoietic Stem Cell Transplant Immune-Mediated Cytopenias	Participants currently recruited/enrolled	18-75 Years	NHLBI	Anemia
000696-C	A Phase III, Multicenter, International Study with a Parallel, Randomized, Double-blind, Placebo-controlled, 2 Arm Design to Assess the Efficacy and Safety of Selumetinib in Adult Participants with NF1 who have Symptomatic, Inoperable Plexiform Neurofibr	Participants currently recruited/enrolled	18-120 Years	NCI	Autosomal
000539-H	Addition of JSP191 (c-kit antibody) to Non-myeloablative Hematopoietic Cell Transplantation For Sickle Cell Disease and Beta-Thalassemia	Participants currently recruited/enrolled	4-100 Years	NHLBI	Anemia
000520-C	Phase I/II Evaluation of a Cancer Lysate Vaccine and Montanide(R) ISA-51 VG with or without the IL-15 Super-Agonist N-803 as Adjuvant Therapy for PD-L1 Negative Non-Small Cell Lung Cancer	Recruitment has not started	18-125 Years	NCI	Autosomal
000464-H	Evaluation of Clonal Hematopoiesis in Patients with Sickle Cell Disease Receiving Curative Therapies	Enrolling by Invitation	4-125 Years	NHLBI	Anemia
000423-C	Feasibility and Preliminary Efficacy of Acceptance and Commitment Therapy (ACT) for Sleep Disturbances in Adults with Sickle Cell Disease (SCD): A Pilot Randomized Controlled Trial	Participants currently recruited/enrolled	18-55 Years	NCI	Anemia
000413-H	Natural History study of CADASIL	Participants currently recruited/enrolled	18-100 Years	NHLBI	Autosomal
000387-H	The Natural History of Acquired and Inherited Bone Marrow Failure Syndromes.	Participants currently recruited/enrolled	2-125 Years	NHLBI	Anemia
000049-H	Evaluation of the Safety,Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects with Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat	No longer recruiting/follow-up only	18-70 Years	NHLBI	Anemia
20-H-0080	Red Cell Half Life Determination in Patients with and without Sickle Cell Disease	Completed Study; data analyses ongoing	18-100 Years	NHLBI	Anemia
20-H-0033	Early Initiation of Oral Therapy with Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA)	Participants currently recruited/enrolled	3-125 Years	NHLBI	Anemia
20-H-0021	Treatment of Refractory Diamond-Blackfan Anemia with Eltrombopag	No longer recruiting/follow-up only	2-125 Years	NHLBI	Anemia
20-DC-0047	Natural History of Autosomal Dominant Hearing Loss	Participants currently recruited/enrolled	3-99 Years	NIDCD	Autosomal
20-D-0122	A Phase IIb, Open-label Dose-ranging Study Evaluating the Safety, Tolerability, Pharmacodynamics and Pharmacokinetics, and Efficacy of CLTX-305 (encaleret) in Autosomal Dominant Hypocalcemia Type 1 (ADH1)	No longer recruiting/follow-up only	16-125 Years	NIDCR	Autosomal
18-H-0090	Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH using Peripheral Blood Stem Cells and post-transplant Cyclophosphamide for GVHD prophylaxis	Participants currently recruited/enrolled	4-75 Years	NHLBI	Anemia
18-H-0004	Low Dose Danazol for the Treatment of Telomere Related Diseases	Participants currently recruited/enrolled	3-125 Years	NHLBI	Anemia
17-H-0121	Eltrombopag for Patients with Fanconi Anemia	Participants currently recruited/enrolled	2-125 Years	NHLBI	Anemia
17-H-0091	Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS using CordIn, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, to Expedite Engraftment and Improve Transplant Outcome	Participants currently recruited/enrolled	4-75 Years	NHLBI	Anemia
17-H-0019	A Randomized Trial of Sirolimus (Rapamune(R)) for Relapse Prevention in Patients with Severe Aplastic Anemia Responsive to Immunosuppressive Therapy	Participants currently recruited/enrolled	2-125 Years	NHLBI	Anemia
16-I-0065	Screening for Alpha Globin Deletions	Participants currently recruited/enrolled	18-39 Years	NIAID	Anemia
16-H-0132	CADASIL Disease Discovery	Completed Study; data analyses ongoing	18-100 Years	NHLBI	Autosomal
13-H-0133	Extended Dosing with Eltrombopag in Refractory Severe Aplastic Anemia	Completed Study; data analyses ongoing	2-125 Years	NHLBI	Anemia
13-H-0116	A Multicenter Safety Study of Unlicensed, Investigational Cryopreserved Cord Blood Units (CBUs) Manufactured by the National Cord Blood Program (NCBP) and Provided for Unrelated Hematopoietic Stem Cell Transplantation of Pediatric and Adult Patients	Participants currently recruited/enrolled	4-125 Years	NHLBI	Anemia
12-H-0150	Eltrombopag Added to Standard Immunosuppression in Treatment-Naive Severe Aplastic Anemia	Participants currently recruited/enrolled	2-125 Years	NHLBI	Anemia
12-H-0064	A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs) for Transplantation in Pediatric and Adult Patients with Hematologic Malignancies and Other Indications	Participants currently recruited/enrolled	4-75 Years	NHLBI	Anemia
11-H-0209	Male Hormones for Telomere Related Diseases	Completed Study; data analyses ongoing	2-125 Years	NHLBI	Anemia
11-H-0134	A Pilot Study of a Thrombopoietin-Receptor Agonist (TPO-R Agonist), Eltrombopag, in Moderate Aplastic Anemia Patients	No longer recruiting/follow-up only	2-125 Years	NHLBI	Anemia
10-H-0154	Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused with a Reduced Dose of Non-Mobilized Donor T-Cells	Participants currently recruited/enrolled	4-80 Years	NHLBI	Anemia
09-H-0225	Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Severe Congenital Anemias Including Sickle Cell Disease and Beta-Thalassemia	No longer recruiting/follow-up only	2-125 Years	NHLBI	Anemia
09-H-0199	A Pilot Study of a Thrombopoietin-Receptor Agonist (TPO-R agonist), Eltrombopag, in Patients with Low to Int-2 Risk Myelodysplastic Syndrome (MDS)	Completed Study; data analyses ongoing	18-125 Years	NHLBI	Anemia
08-H-0046	Co-Infusion of Umbilical Cord Blood and Haploidentical CD34+ Cells Following Nonmyeloablative Conditioning as Treatment for Severe Aplastic Anemia and MDS Associated with Severe Neutropenia Refractory to Immunosuppressive Therapy	Completed Study; data analyses ongoing	4-75 Years	NHLBI	Anemia
08-DK-0004	High Sensitivity Screening of Compound Libraries to Discover a Drug for the Treatment of Sickle Cell Disease	Participants currently recruited/enrolled	18-100 Years	NIDDK	Anemia
08-C-0130	A Phase II Trial of Peginterferon alfa-2b (PEG-Intron) for Neurofibromatosis Type 1 Related Unresectable, Symptomatic or Life-Threatening Plexiform Neurofibromas	Completed Study; data analyses ongoing	0-21 Years	NCI	Autosomal
07-H-0113	Procurement and Analysis of Blood, Bone Marrow, and Buccal Mucosa Samples from Healthy Volunteers to Support Clinical and Translational Research Projects in the NHLBI	Participants currently recruited/enrolled	8-125 Years	NHLBI	Anemia
06-H-0190	Treatment of T-Large Granular Lymphocyte (T-LGL) Lymphoproliferative Disorders with Alemtuzumab (Campath)	Completed Study; data analyses ongoing	18-85 Years	NHLBI	Anemia
05-H-0242	A Pilot Study of Alemtuzumab (Campath) in Patients with Relapsed or Refractory Severe Aplastic Anemia	Completed Study; data analyses ongoing	2-110 Years	NHLBI	Anemia
03-H-0170	Nonmyeloablative Allogeneic Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation For Severe Congenital Anemias Including Sickle Cell Disease (SCD) and Beta-Thalassemia	Participants currently recruited/enrolled	2-125 Years	NHLBI	Anemia
02-C-0052	Etiologic Investigation of Cancer Susceptibility in Inherited Bone Marrow Failure Syndromes: A Natural History Study	Participants currently recruited/enrolled	0-125 Years	NCI	Anemia
01-H-0088	Determining the Prevalence and Prognosis of Secondary Pulmonary Hypertension in Adult Patients with Sickle Cell Anemia	Completed Study; data analyses ongoing	18-125 Years	NHLBI	Anemia
00-I-0159	Natural History, Management, and Genetics of the Hyperimmunoglobulin E Recurrent Infection Syndrome (HIES)	Participants currently recruited/enrolled	0-125 Years	NIAID	Autosomal
97-H-0041	Screening Protocol for Subjects Being Evaluated for National Heart, Lung and Blood Institute (NHLBI) Protocols	Enrolling by Invitation	2-125 Years	NHLBI	Anemia