7 completed, 2 not yet recruiting, 2 terminated, 1 recruiting. 13 Early Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Early Phase 1 | 13 | Completed 7, Not yet recruiting 2, Terminated 2, Recruiting 1, Unknown 1 |
Follow Abnormalities, Multiple: new trials by email Literature Review of Abnormalities, Multiple trials Research Report on Abnormalities, Multiple
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| A Pilot Study to Examine the Efficacy and Tolerability of Sirolimus on Epidermal Nevi Lesions in Patients 6 Years Old and Above. sirolimus topical gel 0.2% - Epidermal Nevi | Early Phase 1 | Not yet recruiting | Northwestern University | 14 | 2027-02-01 | 2026-11-09 |
| GBP-213 in Patients With Autosomal Dominant Polycystic Kidney Disease GBP-213 - Autosomal Dominant Polycystic Kidney | Early Phase 1 | Not yet recruiting | RenJi Hospital | 24 | 2026-11-01 | 2026-10-23 |
| A Clinical Study of CD19/BCMA CAR-T Cells in the Treatment of Refractory POEMS Syndrome, Amyloidosis, Autoimmune Hemolytic Anemia, and Vasculitis CD19/BCMA CAR T-cells - POEMS Syndrome, Amyloidosis | Early Phase 1 | Unknown | Zhejiang University | 75 | 2021-10-08 | 2022-03-03 |
| Functional Studies of Novel Genes Mutated in Primary Ciliary Dyskinesia II: Genotype to Phenotype Albuterol, Technetium99m - Sulfur Colloid (Tc99m-SC) - Primary Ciliary Dyskinesia | Early Phase 1 | Completed | University of North Carolina, Chapel Hill | 27 | 2021-06-10 | 2025-09-05 |
| Safety and Efficacy of CT103A Cells for Relapsed/Refractory Antibody-associated Inflammatory Diseases of the Nervous System CT103A cells, Cyclophosphamide and fludarabine - Autoimmune Diseases, Autoimmune Diseases of the Nervous System | Early Phase 1 | Recruiting | Tongji Hospital | 36 | 2020-09-22 | 2024-10-30 |
| Angelman Syndrome (AS) Biomarker Study Lumbar Puncture, Blood Collection - Angelman Syndrome, Chromosome 15q Duplication (dup15q) Syndrome | Early Phase 1 | Completed | Biogen | 20 | 2019-12-18 | 2022-01-28 |
| Burosumab for CSHS Burosumab - Cutaneous Skeletal Hypophosphatemia Syndrome (CSHS), Epidermal Nevus Syndrome | Early Phase 1 | Completed | Laura Tosi | 1 | 2019-07-01 | 2026-07-30 |
| Assessing a Dose-Response Relationship of Hydralazine and Its Effects on DNA Methyltransferase 1 in Polycystic Kidney Disease Patients Hydralazine - Polycystic Kidney Diseases | Early Phase 1 | Completed | University of Kansas Medical Center | 14 | 2018-04-23 | 2020-02-26 |
| 68Ga-DOTA-TOC PET/CT in Imaging Participants With Neuroendocrine Tumors Computed Tomography, Gallium Ga 68-Edotreotide, Positron Emission Tomography - Meningioma, Metastatic Well Differentiated Neuroendocrine Neoplasm | Early Phase 1 | Terminated | M.D. Anderson Cancer Center | 4 | 2017-05-16 | 2021-02-15 |
| Ixazomib Citrate, Lenalidomide, and Dexamethasone in Treating Patients With POEMS Syndrome Dexamethasone, Ixazomib Citrate, Lenalidomide - Plasmacytoma, POEMS Syndrome | Early Phase 1 | Completed | Mayo Clinic | 21 | 2016-10-31 | 2025-02-12 |
| Enhancing Radioiodine (RAI) Incorporation Into BRAF Mutant, RAI-Refractory Thyroid Cancers With the BRAF Inhibitor Vemurafenib: A Pilot Study Vemurafenib, serial 124I PET/CT, thyrotropin alpha - Thyroid Carcinoma | Early Phase 1 | Completed | Memorial Sloan Kettering Cancer Center | 12 | 2014-05-20 | 2025-06-18 |
| Workshops for Veterans With Vision and Hearing Loss DSL Workshop - Deafness, Blindness | Early Phase 1 | Completed | US Department of Veterans Affairs | 13 | 2011-11-01 | 2015-09-29 |
| Bevacizumab (Avastin) in Unresectable/Recurrent Hemangioblastoma From Von-Hippel-Lindau Disease Avastin - Hemangioblastomas, Von Hippel Lindau Disease | Early Phase 1 | Terminated | Dartmouth-Hitchcock Medical Center | 1 | 2009-12-01 | 2012-05-11 |
POEMS Syndrome (3) · Nevus, Sebaceous of Jadassohn (2) · von Hippel-Lindau Disease (2) · Amyloidosis (1) · Anemia, Hemolytic, Autoimmune (1) · Angelman Syndrome (1) · Autoimmune Diseases (1) · Autoimmune Diseases of the Nervous System (1) · Blindness (1) · Chromosome 15q, trisomy (1) · Ciliary Motility Disorders (1) · Deaf-Blind Disorders (1) · Deafness (1) · Hemangioblastoma (1) · Heterotaxy Syndrome (1)
Congenital Abnormalities (13) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (13) · Nervous System Diseases (9) · Neoplasms (7) · Genetic Diseases, Inborn (6) · Ciliopathies (5) · Neoplasms by Histologic Type (5) · Cardiovascular Diseases (4) · Hemic and Lymphatic Diseases (4) · Neurocutaneous Syndromes (4) · Blood Protein Disorders (3) · Hematologic Diseases (3) · Immune System Diseases (3) · Immunoproliferative Disorders (3)
Albuterol (1) · Bevacizumab (1) · Blood Specimen Collection (1) · Calcium Dobesilate (1) · Cyclophosphamide (1) · Dexamethasone (1) · Hydralazine (1) · Lenalidomide (1) · Magnetic Resonance Spectroscopy (1) · Nebulizers and Vaporizers (1) · Spinal Puncture (1) · Thyrotropin Alfa (1) · Vemurafenib (1) · apaflurane (1) · auricularum (1)
Biogen (1) · Dartmouth-Hitchcock Medical Center (1) · Laura Tosi (1) · M.D. Anderson Cancer Center (1) · Mayo Clinic (1) · Memorial Sloan Kettering Cancer Center (1) · Northwestern University (1) · RenJi Hospital (1) · Tongji Hospital (1) · US Department of Veterans Affairs (1) · University of Kansas Medical Center (1) · University of North Carolina, Chapel Hill (1) · Zhejiang University (1)
United States (10) · China (3)
Alan L. Ho, MD, PhD (1) · Amy Paller, MD (1) · Angela Dispenzieri, M.D. (1) · He Huang, PhD (1) · Homer A Macapinlac, MD (1) · J Marc Pipas, MD (1) · Kerri McGreal, MD (1) · Laura Tosi, MD (1) · Lawrence Ostrowski, PhD (1) · Leyi Gu (1) · Mitchel Turbin, PhD (1) · Wei Wang, MD (1)
PMID 30256977, PMID 37658205, PMID 40425008, PMID 41101309
R01HL117836 (1), UL1TR002366 (1)