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66 Studies found from all possible combinations of your search terms. Select a protocol that you wish to review.
Protocol Number
Title
Protocol Status
Min-Max Age
Institute
Keywords
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 Dominant
000413-H
Natural History study of CADASIL
Participants currently recruited/enrolled
18-100 Years
NHLBI
Autosomal Dominant
20-DC-0047
Natural History of Autosomal Dominant Hearing Loss
Participants currently recruited/enrolled
3-99 Years
NIDCD
Autosomal Dominant
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 Dominant
16-H-0132
CADASIL Disease Discovery
Completed Study; data analyses ongoing
18-100 Years
NHLBI
Autosomal Dominant
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 Dominant
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 Dominant
000861-I
A Phase 2 Open-Label Study to Evaluate the Efficacy and Safety of Ruxolitinib on Hair Regrowth in Patients with Autoimmune Polyendocrinopathy Candidiasis Ectodermal Dystrophy (APECED)-Associated Alopecia Areata
Participants currently recruited/enrolled
12-65 Years
NIAID
Hair
000827-AR
Natural History and Pathogenesis of Alopecia in Children and Adults
Participants currently recruited/enrolled
2-45 Years
NIAMS
Hair
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
000678-C
Rare Tumors and Cancer Predisposition in Individuals and Families
Participants currently recruited/enrolled
0-125 Years
NCI
Hair
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
000509-C
An Open Label, Multi-Center Roll-Over Study to Assess Long-Term Safety in Patients Who are Ongoing or Have Completed a Prior Global Novartis or GSK Sponsored Tafinlar (Dabrafenib) and/or Mekinist (Trametinib) Study and are Judged by the Investigator to B
Enrolling by Invitation
18-125 Years
NCI
Hair
000387-H
The Natural History of Acquired and Inherited Bone Marrow Failure Syndromes.
Participants currently recruited/enrolled
2-125 Years
NHLBI
Hair
000261-AA
Mechanisms Underlying Individual Variations of Taste and Smell in Obesity
Participants currently recruited/enrolled
18-65 Years
NIAAA
Hair
000176-C
A Phase I/II Trial of Lurbinectedin with Berzosertib, an ATR Kinase Inhibitor in Small Cell Cancers and High Grade Neuroendocrine Cancers
Participants currently recruited/enrolled
18-125 Years
NCI
Hair
000152-E
Natural History of The Human Biological Response to Environmental Exposure and Injury
Participants currently recruited/enrolled
18-125 Years
NIEHS
Hair
000144-C
A Phase I/II Study of Sacituzumab Govitecan plus Berzosertib in Small Cell Lung Cancer, Extra-Pulmonary Small Cell Neuroendocrine Cancer and Homologous Recombination-Deficient Cancers Resistant to PARP Inhibitors
Participants currently recruited/enrolled
18-125 Years
NCI
Hair
21-C-0019
Phase I Study of Anti-CD22 Chimeric Receptor T Cells in Patients with Relapsed/Refractory Hairy Cell Leukemia and Variant
Participants currently recruited/enrolled
18-125 Years
NCI
Hair
20-H-0158
Natural History Study: Gene First Approach to Connective Tissue Disease
Enrolling by Invitation
2-100 Years
NHLBI
Hair
20-C-0076
Phase 2 Trial of Encorafenib plus Binimetinib for Patients with BRAF V600 Mutated Relapsed/Refractory HCL
Participants currently recruited/enrolled
18-125 Years
NCI
Hair
20-C-0075
Phase 2 Trial for Binimetinib for Patients with Relapsed/Refractory BRAF Wild Type Hairy Cell Leukemia and Variant
Participants currently recruited/enrolled
18-125 Years
NCI
Hair
20-C-0013
Phase I/II Trial of PLX038 (PEGylated SN38) and Rucaparib in Solid Tumors and Small Cell Cancers
No longer recruiting/follow-up only
18-125 Years
NCI
Hair
19-N-0036
Exploring the Relationship Between Brain Asymmetry and Attention
Completed Study; data analyses ongoing
18-35 Years
NINDS
Dominant
19-E-0110
Reproductive Axis Maturation in the Early Post-Menarchal Years: A Pilot Study
Participants currently recruited/enrolled
8-34 Years
NIEHS
Hair
19-C-0042
A Phase I Study of Moxetumomab Pasudotox-tdfk (Lumoxiti (TM)) and either Rituximab (Rituxan (R)) or Ruxience for Relapsed Hairy Cell Leukemia
No longer recruiting/follow-up only
18-125 Years
NCI
Hair
19-C-0033
Phase II Study of Nivolumab (anti-PD1), Tadalafil and Oral Vancomycin in Patients with Refractory Primary Hepatocellular Carcinoma or Liver Dominant Metastatic Cancer from Colorectal or Pancreatic Cancers
No longer recruiting/follow-up only
18-125 Years
NCI
Dominant
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
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
18-CH-0031
Clinical and Molecular Characteristics of Primary Aldosteronism in Blacks
Participants currently recruited/enrolled
7-70 Years
NICHD
Hair
18-CC-0052
Near-infrared Spectroscopy and Electroencephalography to Assess Cortical Activation During Motor Tasks in Infants and Toddlers with or at high-risk for Cerebral Palsy and Autism Spectrum Disorder Compared to Infants and Toddlers with Typical Development
Participants currently recruited/enrolled
0-5 Years
CC
Hair
17-CH-0020
Observational Study of Males with Creatine Transporter Deficiency (CTD)
No longer recruiting/follow-up only
2-40 Years
NICHD
Hair
17-C-0117
A Phase 1/2a, Open-Label, Multicenter Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Anti-Tumor Activity of PEN-866 in Advanced Solid Malignancies
Completed Study; data analyses ongoing
18-125 Years
NCI
Hair
17-C-0012
A Phase I Study of a Combination of MM-398 and Veliparib in solid tumors
Completed Study; data analyses ongoing
18-125 Years
NCI
Hair
16-EI-0134
Studies of the Natural History, Pathogenesis and Outcome of Ocular Disorders
Participants currently recruited/enrolled
0-125 Years
NEI
Hair
16-EI-0046
Uveitis/Intraocular Inflammatory Disease Biobank (iBank)
No longer recruiting/follow-up only
8-125 Years
NEI
Hair
16-C-0107
A Phase I/II Trial of EP0057, a Nanoparticle Camptothecin with Olaparib in Patients with Relapsed/Refractory Small Cell Lung, Bladder and Prostate Cancers
Participants currently recruited/enrolled
18-125 Years
NCI
Hair
15-H-0155
Hereditary Parkinson Disease Natural History Protocol
Completed Study; data analyses ongoing
18-80 Years
NHLBI
Autosomal
15-EI-0128
The Genetics of Inherited Eye Disease
Participants currently recruited/enrolled
0-125 Years
NEI
Hair
15-C-0150
A Phase I/II Trial of Topotecan with VX-970 (M6620), an ATR Kinase Inhibitor in Small Cell Cancers
No longer recruiting/follow-up only
18-125 Years
NCI
Hair
14-I-0206
Novel Genetic Disorders of the Immune System
Participants currently recruited/enrolled
0-99 Years
NIAID
Hair
14-HG-0125
Insights Into Microbiome and Environmental Contributions to Sickle Cell Disease and Leg Ulcers
Participants currently recruited/enrolled
18-125 Years
NHGRI
Hair
14-C-0131
A Phase II, Open-Label Study in Patients With BRAF V600E-Mutated Rare Cancers With Several Histologies to Investigate the Clinical Efficacy and Safety of the Combination Therapy of Dabrafenib and Trametinib
Completed Study; data analyses ongoing
18-125 Years
NCI
Hair
14-C-0004
A Multicenter Phase 2 Study of the Bruton's Tyrosine Kinase Inhibitor PCI-32765 (Ibrutinib) for Treatment of Relapsed Hairy Cell Leukemia
No longer recruiting/follow-up only
18-125 Years
NCI
Hair
12-EI-0042
NEI Intramural Biorepository for Retinal Diseases
Participants currently recruited/enrolled
2-125 Years
NEI
Hair
11-EI-0245
Generation of Induced Pluripotent Stem (iPS) Cell Lines from Somatic Cells of Participants with Eye Diseases and from Somatic Cells of Matched Controls
Participants currently recruited/enrolled
1-125 Years
NEI
Hair
10-E-0063
Sample Collection Registry for Quality Control of Biological and Environmental Specimens and Assay Development and Testing
Completed Study; data analyses ongoing
18-125 Years
NIEHS
Hair
10-C-0066
A Study of Hairy Cell and other Leukemias with a Focus on Recombinant Immunotoxins for Cancer Treatment
Participants currently recruited/enrolled
18-125 Years
NCI
Hair
10-C-0056
A Phase I Study of Indenoisoquinolines LMP400 and LMP776 in Adults With Relapsed Solid Tumors and Lymphomas
Completed Study; data analyses ongoing
18-125 Years
NCI
Hair
10-C-0025
Randomized Phase II Trial of Rituximab with Either Pentostatin or Bendamustine for Multiply Relapsed or Refractory Hairy Cell Leukemia
Participants currently recruited/enrolled
18-125 Years
NCI
Hair
09-I-0086
Screening Protocol for Genetic Diseases of Allergic Inflammation
Participants currently recruited/enrolled
0-99 Years
NIAID
Hair
09-C-0005
Randomized Trial of Cladribine (CdA) with Simultaneous or Delayed Rituximab to Eliminate Hairy Cell Leukemia Minimal Residual Disease
Participants currently recruited/enrolled
18-125 Years
NCI
Hair
06-H-0217
Genetic Disorders of Mucociliary Clearance
Completed Study; data analyses ongoing
2-65 Years
NHLBI
Hair
06-DA-N415
Screening Protocol for the Evaluation of Potential Research Subjects
Participants currently recruited/enrolled
18-125 Years
NIDA
Hair
06-CH-0011
Natural History Study of Patients with Excess Androgen
Participants currently recruited/enrolled
0-99 Years
NICHD
Hair
06-C-0213
Tissue Procurement Protocol for the Developmental Therapeutics Clinic, National Cancer Institute (NCI)
Participants currently recruited/enrolled
0-125 Years
NCI
Hair
06-C-0150
A Phase II Clinical Trial of Anti-Tac(Fv)-PE38 (LMB-2) Immunotoxin for CD25 Positive Hairy Cell Leukemia
No longer recruiting/follow-up only
18-125 Years
NCI
Hair
02-CH-0311
Investigations into Inborn Errors of Cholesterol Synthesis and Related Disorders
Participants currently recruited/enrolled
0-125 Years
NICHD
Dominant
02-C-0313
Cancer Risk in Xeroderma Pigmentosum Heterozygotes
Participants currently recruited/enrolled
0-99 Years
NCI
Hair
02-C-0210
Clinical, Laboratory, and Epidemiologic Characterization of Individuals and Families at High Risk of Blood and Lymph Node Malignancy
Participants currently recruited/enrolled
0-125 Years
NCI
Hair
02-C-0159
Birt-Hogg-Dub(SqrRoot)(Copyright) Syndrome: Characterization of the FLCN Disease Gene and Predisposition to Renal Cancer, Cutaneous Fibrofolliculoma and Pulmonary Cysts
Participants currently recruited/enrolled
2-125 Years
NCI
Hair
01-DC-0229
Genetic Analysis of Hereditary Disorders of Hearing and Balance
Completed Study; data analyses ongoing
0-99 Years
NIDCD
Hair
00-HG-0153
Investigations into Chediak-Higashi Syndrome and Related Disorders
Participants currently recruited/enrolled
0-70 Years
NHGRI
Autosomal
99-C-0099
Examination of Clinical and Laboratory Abnormalities in Patients with Defective DNA Repair: Xeroderma Pigmentosum, Cockayne Syndrome, or Trichothiodystrophy
Participants currently recruited/enrolled
0-125 Years
NCI
Hair
98-CH-0081
Clinical and Basic Investigations into Smith-Lemli-Opitz Syndrome
Completed Study; data analyses ongoing
0-125 Years
NICHD
Autosomal
94-C-0074
Treatment and Natural History Study of Lymphomatoid Granulomatosis
Participants currently recruited/enrolled
12-125 Years
NCI
Hair