333. ハッチンソン・ギルフォード症候群 Hutchinson-Gilford syndrome Clinical trials / Disease details
臨床試験数 : 8 / 薬物数 : 8 - (DrugBank : 3) / 標的遺伝子数 : 4 - 標的パスウェイ数 : 6
No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
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1 | ChiCTR2100050052 | 2021-09-01 | 2021-08-16 | Observational study of Lonafarnib in Chinese patients with progeria and progeroid laminopathy | Observational study of Lonafarnib in Chinese patients with progeria and progeroid laminopathy | Hutchinson-Gilford Progeria Syndrome and Progeroid Laminopathies | Experimental group:Treatment with Lonafarnib; | The Children's Hospital Zhejiang University School of Medicine | NULL | Pending | 1 | 99 | Both | Experimental group:25; | N/A | China |
2 | NCT04512963 (ClinicalTrials.gov) | August 24, 2020 | 6/8/2020 | Phase I Study of Progerinin in Healthy Volunteers | A Phase I, Randomized, Double-Blind, Placebo-Controlled, Single Ascending Dose (SAD) Study Including a Food Interaction Study, Followed by a Multiple Ascending Dose (MAD) Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamic Profile of Progerinin in Healthy Volunteers | Hutchinson-Gilford Progeria Syndrome;Werner Syndrome | Drug: Progerinin;Drug: Placebo | PRG Science & Technology Co., Ltd. | Amarex Clinical Research | Completed | 18 Years | 45 Years | All | 64 | Phase 1 | United States |
3 | NCT03895528 (ClinicalTrials.gov) | June 8, 2020 | 27/3/2019 | Lonafarnib for Patients With Hutchinson-Gilford Progeria Syndrome or Progeroid Laminopathy | A Treatment IND (Investigational New Drug) Protocol for EAP (Expanded Access Program) for the Use of Lonafarnib in Patients With Hutchinson-Gilford Progeria Syndrome (HGPS) or Progeroid Laminopathy | Progeria;HGPS | Drug: Lonafarnib | Eiger BioPharmaceuticals | NULL | Approved for marketing | 12 Months | N/A | All | NULL | ||
4 | NCT03871972 (ClinicalTrials.gov) | March 5, 2019 | 7/3/2019 | Umbilical Cord Blood Transfusion in Progeria Syndrome | Safety and Efficacy of Umbilical Cord Blood Transfusion in Patients With Hutchinson-Gilford Progeria Syndrome | Hutchinson-Gilford Progeria Syndrome | Drug: Umbilical Cord Blood Unit | Bundang CHA Hospital | NULL | Completed | N/A | N/A | All | 2 | Phase 1/Phase 2 | Korea, Republic of |
5 | NCT00916747 (ClinicalTrials.gov) | August 2009 | 5/6/2009 | Study of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Progeria | An Open Label Phase II Trial of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Hutchinson-Gilford Progeria Syndrome(HGPS) and Progeroid Laminopathies | Progeria | Drug: Lonafarnib, Zoledronic Acid, and Pravastatin | Boston Children's Hospital | Schering-Plough;Merck Sharp & Dohme LLC;Eiger BioPharmaceuticals | Active, not recruiting | N/A | N/A | All | 85 | Phase 2 | United States |
6 | NCT00879034 (ClinicalTrials.gov) | March 2009 | 8/4/2009 | A Study of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Progeria | A Phase II Pilot Study of Zoledronic Acid, Pravastatin, and Lonafarnib (SCH66336) for Patients With Hutchinson-Gilford Progeria Syndrome (HGPS) and Progeroid Laminopathies | Progeria;Hutchinson-Gilford Syndrome | Drug: Lonafarnib;Drug: Zoledronic Acid;Drug: Pravastatin | Boston Children's Hospital | Dana-Farber Cancer Institute;Brigham and Women's Hospital;Schering-Plough | Completed | N/A | N/A | All | 5 | Phase 2 | United States |
7 | NCT00731016 (ClinicalTrials.gov) | October 2008 | 1/8/2008 | Treatment of the Hutchinson-Gilford Progeria Syndrome With a Combination of Pravastatin and Zoledronic Acid | Treatment of the Hutchinson-Gilford Progeria Syndrome With a Combination of Pravastatin and Zoledronic Acid | Hutchinson-Gilford Progeria Syndrome | Drug: Zoledronic acid, pravastatin | Assistance Publique Hopitaux De Marseille | NULL | Completed | 3 Years | N/A | Both | 15 | Phase 2 | France |
8 | NCT00425607 (ClinicalTrials.gov) | May 2007 | 22/1/2007 | Phase II Trial of Lonafarnib (a Farnesyltransferase Inhibitor) for Progeria | An Open Label Dose Adjusted Phase II Trial of the Oral Farnesyltransferase Inhibitor (FTI) Lonafarnib (SCH66336) for Patients With Hutchinson-Gilford Progeria Syndrome (HGPS) and Progeroid Laminopathies | Progeria;Hutchinson-Gilford Syndrome | Drug: Lonafarnib | Monica E. Kleinman | Schering-Plough | Completed | 1 Year | N/A | All | 29 | Phase 2 | United States |