10 terminated. 10 Phase 1, 7 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 10 | Terminated 10 |
| Phase 2 | 7 | Terminated 7 |
Follow Skin Abnormalities: new trials by email Literature Review of Skin Abnormalities trials Research Report on Skin Abnormalities
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 |
|---|---|---|---|---|---|---|
| Neoadjuvant ADI-PEG 20 + Ifosfamide + Radiotherapy in Soft Tissue Sarcoma ADI PEG20, Ifosfamide, Radiotherapy - Soft Tissue Sarcoma, Sts | Phase 1 | Terminated | Washington University School of Medicine | 6 | 2024-03-14 | 2026-09-03 |
| A Study to Explore Safety, Pharmacokinetics, and Early Clinical Signal of Efficacy of DS-2325a in Patients With Netherton Syndrome DS-2325a, Placebo - Netherton Syndrome | Phase 1 / Phase 2 | Terminated | Daiichi Sankyo | 9 | 2023-09-28 | 2026-05-14 |
| The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency INZ-701 - Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets | Phase 1 | Terminated | Inozyme Pharma | 9 | 2023-06-25 | 2026-10-15 |
| FOCUS: A Phase I/II First in Human Study to Evaluate the Safety and Efficacy of GT005 Administered in Subjects With Dry AMD GT005, GT005 / Device: Orbit™ Subretinal Delivery System - Dry Age-related Macular Degeneration, Macular Degeneration | Phase 1 / Phase 2 | Terminated | Gyroscope Therapeutics Limited | 56 | 2018-12-17 | 2026-01-28 |
| A Double-blind, Randomized, Intra-subject Placebo-controlled, Multicenter, Multiple Dose Study, Evaluating Safety, Proof of Mechanism, Preliminary Efficacy and Systemic Exposure in Subjects With Confirmed DDEB or RDEB Diagnosis With One or More Pathogenic Mutations in Exon 73 in the COL7A1 Gene QR-313, Placebo - Epidermolysis Bullosa Dystrophica, Recessive, Epidermolysis Bullosa Dystrophica, Dominant | Phase 1 / Phase 2 | Terminated | Phoenicis Therapeutics | 2 | 2018-07-02 | 2021-08-25 |
| Clinical Trial to Assess Safety and Efficacy of Autologous Cultured Epidermal Grafts Containing Epidermal Stem Cells Genetically Modified in Patients With JEB (HOLOGENE17) Transplantation surgery of genetically corrected cultured epidermal autograft (ATMP) - Junctional Epidermolysis Bullosa | Phase 1 / Phase 2 | Terminated | Holostem s.r.l. | 1 | 2018-03-19 | 2022-03-07 |
| Clinical Trial to Assess Safety and Efficacy of Autologous Cultured Epidermal Grafts Containing Epidermal Stem Cells Genetically Modified in Patients With RDEB. Genetically corrected cultured epidermal autograft (ATMP) - Recessive Dystrophic Epidermolysis Bullosa | Phase 1 / Phase 2 | Terminated | Holostem s.r.l. | 3 | 2017-01-30 | 2022-02-16 |
| A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa (RDEB) FCX-007 - Epidermolysis Bullosa Dystrophica, Recessive | Phase 1 / Phase 2 | Terminated | Castle Creek Biosciences, LLC. | 6 | 2016-07-01 | 2023-02-08 |
| Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases BPX-501 and Rimiducid - Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis | Phase 1 | Terminated | Bellicum Pharmaceuticals | 1 | 2015-02-01 | 2024-03-25 |
| Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita danazol - Fanconi Anemia, Dyskeratosis Congenita | Phase 1 / Phase 2 | Terminated | Boston Children's Hospital | 5 | 2009-11-01 | 2019-02-19 |
Epidermolysis Bullosa Dystrophica (3) · Dyskeratosis Congenita (2) · Fanconi Anemia (2) · Anemia, Diamond-Blackfan (1) · Anemia, Sickle Cell (1) · Anetoderma (1) · Arterial calcification of infancy (1) · Congenital Bone Marrow Failure Syndromes (1) · Epidermolysis Bullosa, Junctional (1) · Eye Diseases (1) · Geographic Atrophy (1) · Granulomatous Disease, Chronic (1) · Hemoglobinopathies (1) · Hypophosphatemic Rickets, Autosomal Recessive, 1 (1) · Ichthyosis, X-Linked (1)
Skin Diseases (10) · Skin and Connective Tissue Diseases (10) · Congenital Abnormalities (9) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (9) · Genetic Diseases, Inborn (9) · Skin Diseases, Genetic (9) · Connective Tissue Diseases (6) · Epidermolysis Bullosa (4) · Skin Diseases, Vesiculobullous (4) · Collagen Diseases (3) · Genetic Diseases, X-Linked (3) · Hematologic Diseases (3) · Hemic and Lymphatic Diseases (3) · Infant, Newborn, Diseases (3)
cyclic adenosine-5'-trimetaphosphate (2) · ADI PEG20 (1) · Danazol (1) · Ifosfamide (1) · Mesna (1) · Radiotherapy (1)
Holostem s.r.l. (2) · Bellicum Pharmaceuticals (1) · Boston Children's Hospital (1) · Castle Creek Biosciences, LLC. (1) · Daiichi Sankyo (1) · Gyroscope Therapeutics Limited (1) · Inozyme Pharma (1) · Phoenicis Therapeutics (1) · Washington University School of Medicine (1)
United States (7) · Austria (2) · France (2) · Spain (2) · United Kingdom (2)
Johann W. Bauer, MD (2) · Michele De Luca, MD/Professor (2) · Colin A Sieff, MB.BCh (1) · Mia Weiss, M.D. (1)
2017 (1)