Expanded Access Use of Itacitinib to Treat a Single Patient With STAT1 Gain of Function (GOF) Disease
NCT05561985 · Status: AVAILABLE · Type: EXPANDED_ACCESS
Last updated 2025-06-08
Summary
Expanded access use of Itacitinib to treat a single patient with aplastic anemia.
Conditions
- STAT1 Gain-of-Function Disease
Interventions
- DRUG
-
Itacitinib
Sponsors & Collaborators
- lead INDUSTRY
Principal Investigators
-
Lisa Forbes Satter, MD · Baylor College of Medicine - Texas Children's Hospital
Eligibility
- Sex
- Healthy Volunteers
- No
Timeline & Regulatory
More Related Trials
-
An Open-label Trial to Determine Increased Red Blood Cell Production in Subjects With Anemia of Chronic Disease
NCT01846689 ·Status: UNKNOWN ·Phase: PHASE3
-
Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan Anemia
NCT01464164 ·Status: TERMINATED ·Phase: PHASE1/PHASE2
-
A Study of YTS109 Cell in Subjects With Relapsed/Refractory Autoimmune Hemolytic Anemia
NCT07075484 ·Status: COMPLETED ·Phase: PHASE1
-
A Study of Recombinant Human Erythropoietin in Anemic Cancer Patients Undergoing Chemotherapy
NCT00144495 ·Status: COMPLETED ·Phase: PHASE3
-
Erythropoietin Alfa in Elderly Subjects With Unexplained Anemia
NCT00954486 ·Status: WITHDRAWN ·Phase: NA
-
Treatment of Chronic Anemia With Epoetin Alfa in Elderly
NCT00337441 ·Status: UNKNOWN ·Phase: PHASE2
-
A Double-Blind, Placebo-Controlled Study With Open-Label Follow-Up To Determine the Safety and Efficacy of r-HuEPO in AIDS and Advanced ARC Patients With Anemia
NCT00002042 ·Status: COMPLETED ·Phase: NA
-
A Study to Evaluate Whether Correction of Anemia Using Recombinant Human Erythropoietin Reduces the Progression of Atherosclerosis and Cardiac Hypertrophy in Pre-dialysis Chronic Kidney Disease Patients
NCT00563355 ·Status: COMPLETED ·Phase: NA
-
Open-Label Extension Safety Study of PROCRIT in Patients With Anemia of Chronic Disease Due to Rheumatoid Arthritis
NCT00123149 ·Status: WITHDRAWN ·Phase: PHASE2
-
An Efficacy and Safety Study of Luspatercept (ACE-536) for the Treatment of Anemia Due to IPSS-R Very Low, Low or Intermediate Risk Myelodysplastic Syndromes (MDS) in Japanese Subjects Who Are Not Requiring Red Blood Cell Transfusion
NCT03900715 ·Status: COMPLETED ·Phase: PHASE2
-
Study to Assess the Efficacy and Safety of HX575 in the Treatment of Chemotherapy Associated Anemia in Cancer Patients
NCT00711958 ·Status: COMPLETED ·Phase: PHASE3
-
Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria
NCT03520036 ·Status: COMPLETED ·Phase: PHASE2
-
Treatment of the Anemia of Myelodysplastic Syndromes by the Association of Epoetin Beta and All Trans Retinoic Acid
NCT00437450 ·Status: UNKNOWN ·Phase: PHASE2
-
Erythropoietin in the Treatment of Anemia After Autologous Hematopoietic Stem Cell Transplantation
NCT03010579 ·Status: UNKNOWN ·Phase: PHASE4
-
Study of the Safety and Efficacy of Roxadustat in the Treatment of Heart Failure With Chronic Kidney Disease and Anemia
NCT05691257 ·Status: UNKNOWN ·Phase: PHASE4
-
A Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) Versus Epoetin Alfa for the Treatment of Anemia Due to IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Participants Who Require Red Blood Cell Transfusions and Are ESA Naïve
NCT03682536 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE3
-
Autoantibodies Against Human Recombinant Erythropoietin in Myelodysplatic Syndrome Patients
NCT01548495 ·Status: UNKNOWN
-
A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)
NCT03559699 ·Status: COMPLETED ·Phase: PHASE3
-
The Benefits of a Preoperative Anemia Management Program
NCT01888003 ·Status: TERMINATED ·Phase: NA
-
Herombopag Added to Cyclosporine in Non Severe Aplastic Anemia
NCT05660785 ·Status: COMPLETED ·Phase: PHASE2
-
Safety and Efficacy of Sustained Erythropoietin Therapy
NCT00542568 ·Status: COMPLETED ·Phase: PHASE1/PHASE2
-
A Study of Elritercept to Treat Anemia in Adults With Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Who Need Regular Blood Transfusions
NCT06499285 ·Status: RECRUITING ·Phase: PHASE3
-
Study of Epoetin Alfa to Treat Anemia After Kidney Transplantation
NCT01290328 ·Status: TERMINATED ·Phase: NA
-
Efficacy and Safety of Peginesatide (AF37702) in the Treatment of Anemia in Participants With Chronic Kidney Disease
NCT00314795 ·Status: COMPLETED ·Phase: PHASE2
-
Erythropoietin Therapy in Patients With Chronic Renal Failure: A Study of Time Dependent Activity
NCT00744445 ·Status: COMPLETED ·Phase: PHASE2