9 completed, 3 terminated, 2 recruiting, 1 unknown. 16 Phase 2, 4 Phase 1, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 16 | Completed 9, Terminated 3, Recruiting 2, Unknown 1, Withdrawn 1 |
| Phase 1 | 4 | Completed 3, Terminated 1 |
| Phase 3 | 1 | Terminated 1 |
Follow Albinism: new trials by email Literature Review of Albinism trials Research Report on Albinism
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 |
|---|---|---|---|---|---|---|
| Comparing Regorafenib Combined With Envafolimab to Physician's Choice in Patients With Metastatic Gastrointestinal Stromal Tumors Harboring KIT Exon 17 Mutations Refractory to Standard Treatment physician-selected treatment, Regorafenib + Envafolimab - GIST - Gastrointestinal Stromal Tumor, KIT Gene Mutation | Phase 2 | Recruiting | Jian Li | 100 | 2025-07-14 | 2026-04-30 |
| Efficacy and Safety of Pirfenidone Treatment in HPS-ILD Pirfenidone - Hermansky Pudlak Syndrome, Interstitial Lung Disease | Phase 2 | Unknown | Jesse Roman | 50 | 2019-12-01 | 2019-12-10 |
| Ponatinib for Patients Whose Advanced Solid Tumor Cancer Has Activating Mutations Involving the Following Genes: FGFR1, FGFR2, FGFR3, FGFR4, RET, KIT. ponatinib hydrochloride, laboratory biomarker analysis - Malignant Neoplasm | Phase 2 | Completed | Sameek Roychowdhury | 22 | 2015-02-24 | 2025-03-24 |
| Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT Hydroxyurea, Alemtuzumab, Fludarabine - Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes | Phase 2 | Recruiting | Paul Szabolcs | 100 | 2014-02-04 | 2025-12-15 |
| Immune Disorder HSCT Protocol Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan - Immune Deficiency Disorders, Severe Combined Immunodeficiency | Phase 2 | Completed | Washington University School of Medicine | 28 | 2013-04-29 | 2026-10-27 |
| Nitisinone for Type 1B Oculocutaneous Albinism Nitisinone - Albinism, Vision Loss | Phase 1 / Phase 2 | Completed | National Eye Institute (NEI) | 5 | 2013-04-16 | 2019-02-26 |
| Vision Response to Dopamine Replacement Levodopa/carbidopa - Albinism, Oculocutaneous Albinism | Phase 2 | Terminated | University of Wisconsin, Madison | 20 | 2012-10-17 | 2019-06-11 |
| Trial of L-DOPA as a Treatment to Improve Vision in Albinism Levodopa, Placebo - Albinism | Phase 2 | Completed | University of Minnesota | 45 | 2010-09-01 | 2018-05-02 |
| Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells Hematopoietic stem cell transplantation - Sickle Cell Disease, Thalassemia | Phase 1 / Phase 2 | Completed | Children's Hospital Los Angeles | 25 | 2008-08-01 | 2016-06-23 |
| Medical Treatment of Colitis in Patients With Hermansky-Pudlak Syndrome Mesalamine, Infliximab, Corticosteroids - Hermanski-Pudlak Syndrome, Colitis | Phase 2 | Withdrawn | National Institute of Allergy and Infectious Diseases (NIAID) | 2007-08-07 | 2017-07-02 | |
| Pilot Study of a Multi-Drug Regimen for Severe Pulmonary Fibrosis in Hermansky-Pudlak Syndrome Losartan, Zileuton, N-Acetylcysteine - Hermansky-Pudlak Syndrome (HPS), Pulmonary Fibrosis | Phase 1 / Phase 2 | Terminated | National Human Genome Research Institute (NHGRI) | 3 | 2007-04-01 | 2013-08-02 |
| Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome Pirfenidone, Placebo - Albinism, Inborn Errors of Metabolism | Phase 2 | Completed | William Gahl, M.D. | 35 | 2005-09-01 | 2017-10-16 |
| A Study of Imatinib With Reinduction Chemotherapy Using Mitoxantrone, Etoposide and Cytarabine in Patients With Relapsed/Refractory C-kit Positive (AML) Acute Myeloid Leukemia Imatinib (Gleevec) - Leukemia | Phase 1 / Phase 2 | Completed | University Health Network, Toronto | 35 | 2004-07-01 | 2015-06-24 |
| Stem Cell Transplant for Immunologic or Histiocytic Disorders Stem Cell Transplant, Fludarabine, Melphalan - Hemophagocytic Lymphohistiocytosis, X-Linked Lymphoproliferative Disorders | Phase 2 | Completed | Masonic Cancer Center, University of Minnesota | 19 | 2002-08-01 | 2017-12-28 |
| T-Cell Depletion and Stem Cell Transplant for Immune Deficiencies and Histiocytic Disorders Stem Cell Transplant, Myeloablative conditioning regimen - Hemophagocytic Lymphohistiocytosis, X-Linked Lymphoproliferative Disorders | Phase 2 / Phase 3 | Terminated | Masonic Cancer Center, University of Minnesota | 22 | 2000-09-01 | 2018-01-23 |
| A Study of the Efficacy and Safety of Imatinib Mesylate in Patients With Unresectable or Metastatic Gastrointestinal Stromal Tumors Expressing C-kit Gene Imatinib mesylate - Unresectable or Metastatic Malignant Gastrointestinal Stromal Tumor (GIST) | Phase 2 | Completed | Novartis Pharmaceuticals | 148 | 2000-06-01 | 2014-08-19 |
Chediak-Higashi Syndrome (5) · Hermanski-Pudlak Syndrome (4) · Lymphohistiocytosis, Hemophagocytic (4) · Lymphoproliferative Disorders (4) · Piebaldism (4) · Albinism, Oculocutaneous (3) · Granulomatous Disease, Chronic (3) · Wiskott-Aldrich Syndrome (3) · Anemia, Sickle Cell (2) · Autoimmune Lymphoproliferative Syndrome (2) · Congenital amegakaryocytic thrombocytopenia (2) · Gastrointestinal Stromal Tumors (2) · Histiocytosis, Langerhans-Cell (2) · Hyper-IgM Immunodeficiency Syndrome (2)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (16) · Eye Diseases, Hereditary (16) · Genetic Diseases, Inborn (15) · Nutritional and Metabolic Diseases (14) · Metabolic Diseases (13) · Skin and Connective Tissue Diseases (13) · Eye Diseases (12) · Metabolism, Inborn Errors (12) · Skin Diseases (12) · Skin Diseases, Genetic (12) · Amino Acid Metabolism, Inborn Errors (11) · Hypopigmentation (11) · Pigmentation Disorders (11) · Hemic and Lymphatic Diseases (10)
Melphalan (3) · fludarabine (3) · Alemtuzumab (2) · Imatinib Mesylate (2) · Stem Cell Transplantation (2) · Thiotepa (2) · fludarabine phosphate (2) · pirfenidone (2) · Acetylcysteine (1) · Adalimumab (1) · Adrenal Cortex Hormones (1) · Antilymphocyte Serum (1) · Busulfan (1) · Cyclophosphamide (1) · Cyclosporine (1)
Masonic Cancer Center, University of Minnesota (2) · Children's Hospital Los Angeles (1) · Jesse Roman (1) · Jian Li (1) · National Eye Institute (NEI) (1) · National Human Genome Research Institute (NHGRI) (1) · National Institute of Allergy and Infectious Diseases (NIAID) (1) · Novartis Pharmaceuticals (1) · Paul Szabolcs (1) · Sameek Roychowdhury (1) · University Health Network, Toronto (1) · University of Minnesota (1) · University of Wisconsin, Madison (1) · Washington University School of Medicine (1) · William Gahl, M.D. (1)
United States (14) · Australia (1) · Canada (1) · China (1) · Finland (1) · Puerto Rico (1)
Angela Smith, MD (2) · Brian P Brooks, M.D. (1) · Gail Summers, M.D. (1) · Hisham Abdel-Azim, MD (1) · Jesse Roman, MD (1) · Jian Li, MD (1) · Michael C Struck, MD (1) · Paul Szabolcs, MD (1) · Sameek Roychowdhury, MD, PhD (1) · Thomas Markello, M.D. (1)