Found 15 clinical trials
Efficacy, Safety and Pharmacokinetics of Rilzabrutinib in Patients With Warm Autoimmune Hemolytic Anemia (wAIHA)
This is a single group treatment, Phase 2, open-label, study to evaluate the efficacy, safety and pharmacokinetics of rilzabrutinib in adult patients with wAIHA. All participants will receive rilzabrutinib orally. The screening period is up to 28 days, followed by a treatment period of 24 weeks for Part A. Participants …
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- 17 Nov, 2021
- 3 locations
The TAPS Trial - Fetoscopic Laser Surgery for Twin Anemia Polycythemia Sequence
This multicenter open-label randomized controlled trial is set up to evaluate the effect of fetoscopic laser surgery on the gestational age at birth for monochorionic twin pregnancies diagnosed with twin anemia-polycythemia sequence. Half op the patients will be treated with fetoscopic laser surgery, while the other half will be managed …
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- 16 Feb, 2024
- 5 locations
Study to Evaluate the Effect of GBT440 on TCD in Pediatrics With Sickle Cell Disease
This study is a Phase 3, randomized, double-blind, placebo-controlled study of voxelotor in pediatric participants, aged 2 to < 15 years old, with Sickle Cell Disease. The primary objective is to evaluate the effect of voxelotor on the TCD (Transcranial Doppler Ultrasound) measurements in SCD participants in this age range.
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- 16 Feb, 2024
- 35 locations
Randomised Evaluation of COVID-19 Therapy (RECOVERY)
RECOVERY is a randomised trial investigating whether treatment with Lopinavir-Ritonavir, Hydroxychloroquine, Corticosteroids, Azithromycin, Colchicine, IV Immunoglobulin (children only), Convalescent plasma, Casirivimab+Imdevimab, Tocilizumab, Aspirin, Baricitinib, Infliximab, Empagliflozin, Sotrovimab, Molnupiravir, Paxlovid or Anakinra (children only) prevents death in patients with COVID-19.
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- 14 Sep, 2022
- 6 locations
Gene Therapy for Fanconi Anemia Complementation Group A
The objective of this study is to assess the therapeutic efficacy of a hematopoietic cell-based gene therapy for patients with Fanconi anemia, subtype A (FA-A). Hematopoietic stem cells from mobilized peripheral blood of patients with FA-A will be transduced ex vivo (outside the body) with a lentiviral vector carrying the …
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- 16 Feb, 2024
- 1 location
Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag
Background Diamond-Blackfan anemia (DBA) is treated with steroids. But some people cannot take steroids, or steroids don t work. Other patients must get blood transfusions regularly which are time consuming and can have significant side effects.
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- 16 Feb, 2024
- 1 location
The Safety and Efficacy of Ibrutinib in Refractory/Relapsed Autoimmune Hemolytic Anemia
This is an open-label, single-arm study to evaluate the safety and efficacy of Ibrutinib in subjects with refractory/relapsed autoimmune hemolytic anemia.
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- 05 Aug, 2020
A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease
This is a non-randomized, open-label, multi-site, single-dose, Phase 3 study in approximately 35 adults and pediatric subjects 2 and 50 years of age with sickle cell disease (SCD). The study will evaluate hematopoietic stem cell (HSC) transplantation (HSCT) with LentiGlobin BB305 Drug Product for SCD.
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- 16 Feb, 2024
- 2 locations
Telitacicept in Primary APS Patients
Telitacicept will be added on traditional antithrombotic and immunosuppressive treatment in primary antiphospholipid syndrome (APS) patients with three positive antiphospholipid antibodies (aPL) and at least one extra-criteria manifestations, including thrombocytopenia, autoimmune hemolytic anemia, aPL associated nephropathy, heart valvular disease, non-stroke neurological manifestations. This study aims to evaluate the efficacy of …
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- 15 Oct, 2021
- 1 location
Randomised Evaluation of COVID-19 Therapy
RECOVERY is a randomised trial investigating whether treatment with either Lopinavir-Ritonavir, Hydroxychloroquine, Corticosteroids, Azithromycin, Convalescent plasma or Tocilizumab prevents death in patients with COVID-19.
- 0 views
- 05 Aug, 2020
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