Beta Thalassemia clinical trials at UC Health
6 in progress, 3 open to eligible people
A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Subjects With Transfusion-Dependent β-Thalassemia
open to eligible people ages up to 50 years
This is a single-arm, multi-site, single-dose, Phase 3 study in approximately 18 subjects ≤50 years of age with transfusion-dependent β-thalassemia (TDT), who have a β0/β0, β0/IVS-I-110, or IVS-I-110/IVS-I-110 genotype. The study will evaluate the efficacy and safety of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin BB305 Drug Product.
A Study to Evaluate Long-term Safety in Subjects Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials
open to eligible people ages 18 years and up
A Phase 3b, open-label, single-arm, rollover study to evaluate the long-term safety of luspatercept, to the following subjects: - Subjects receiving luspatercept on a parent protocol at the time of their transition to the rollover study, who tolerate the protocol-prescribed regimen in the parent trial and, in the opinion of the investigator, may derive clinical benefit in the opinion of the investigator from continuing treatment with luspatercept. - Placebo arm subjects from parent protocol (at the time of unblinding or in follow-up) crossing over to luspatercept treatment (provided subjects have met all requirements for entering the rollover study as per the parent protocol). - Subjects in the follow-up phase previously treated with luspatercept or placebo in the parent protocol will continue into long-term post-treatment follow-up in the rollover study until the follow-up commitments are met (unless they meet requirements as per parent protocol to cross-over to luspatercept treatment). The study design is divided into the Transition Phase, Treatment Phase and Follow-up Phase. Subjects will enter transition phase and depending on their background will enter either the treatment phase or the Long-term Post-treatment Follow-up (LTPTFU) phase. - Transition Phase (Screening): up to 21 days prior to enrollment - Treatment Phase: For subjects in luspatercept treatment the dose and schedule of luspatercept in this study will be the same as the last dose and schedule in the parent luspatercept study. For placebo arm subjects from parent protocol (at the time of unblinding or in follow-up) crossing over to luspatercept treatment (provided subjects have met all requirements for entering the rollover study as per the parent protocol) will start at a luspatercept dose of 1.0 mg/kg every 3 weeks (Q3W). This does not apply to subjects that are in long-term follow-up from the parent protocol. - Follow-up Phase: - 42 Day Safety Follow-up Phase: subjects will be followed for 42 days after the last dose of luspatercept, for the assessment of safety-related parameters and adverse event (AE) reporting. - Long-term Post-treatment Follow-up (LTPTFU) Phase: All subjects who are continuing in the LTPTFU Phase, will continue to be followed for 5 years from Dose 1 of the parent protocol, or 3 years of post-treatment from last dose of the parent protocol, whichever occurs later. Subjects will be followed every 6 months until death, withdrawal of consent, study termination, or until a subject is lost to follow-up. Subjects will also be monitored for progression to AML or any malignancies/pre- malignancies. New anticancer or disease related therapies should be collected at the same time schedule. Subjects transitioning from a parent luspatercept study in post-treatment follow-up (safety or LTPTFU) will continue from the same equivalent point in this rollover study. The rollover study will be terminated, and relevant subjects will discontinue from the study when all subjects fulfill 5 years from Dose 1 of the parent protocol, or 3 years of post-treatment from last dose of the parent protocol, whichever occurs later. The shift to commercial drug is an alternative way to stop the study.
open to eligible people ages 12-65
In this study will investigate long term safety and use of the PTG-300 in Beta Thalassemia patients.
A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)
Sorry, in progress, not accepting new patients
This is a single-arm, multi-site, single-dose, Phase 1/2 study to assess ST-400 in 6 subjects with transfusion-dependent β-thalassemia (TDT) who are ≥18 and ≤40 years of age. ST-400 is a type of investigational therapy that consists of gene edited cells. ST-400 is composed of the patient's own blood stem cells which are genetically modified in the laboratory using Sangamo's zinc finger nuclease (ZFN) technology to disrupt a precise and specific sequence of the enhancer of the BCL11A gene (which normally suppresses fetal hemoglobin production in erythrocytes). This process is intended to boost fetal hemoglobin (HbF), which can substitute for reduced or absent adult (defective) hemoglobin. ST-400 is then infused back into the patient after receiving conditioning chemotherapy to make room for the new cells in the bone marrow, with the aim of producing new erythrocytes with increased amounts of HbF. The primary objective is to understand safety and tolerability of ST-400, and secondary objectives are to assess the effects on HbF levels and transfusion requirements.
at UCLA UCSF
An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Adults Who Require Regular Red Blood Cell Transfusions Due to Beta (β) Thalassemia
Sorry, in progress, not accepting new patients
This is a Phase 3, double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety of luspatercept (ACE-536) plus Best supportive care (BSC) versus placebo plus BSC in adults who require regular red blood cell transfusion due to (β)-thalassemia. The study is divided into the following periods: - Historical Period, - Screening/Run-in Period, - Double-blind Treatment Period (48 weeks), - Double-blind Long-term Treatment Period, (at the investigator's discretion an additional 48 weeks), - Open-Label Phase post unblinding and upon Data Monitoring Committee positive recommendation - Post-treatment Follow-up Period
Sorry, not yet accepting patients
Beta-thalassemias and hemoglobinopathies are serious inherited blood diseases caused by abnormal or deficiency of beta A chains of hemoglobin, the protein in red blood cells which delivers oxygen throughout the body.The diseases are characterized by hemolytic anemia, organ damage, and early mortality. Increases in another type of (normal) hemoglobin, fetal globin (HbF), which is normally silenced in infancy, reduces the severity of both conditions. Even incremental augmentation of fetal globin is established to reduce red blood cell pathology, anemia, certain complications, and to improve survival. This trial will evaluate an oral drug discovered in a high throughput screen, which increases fetal globin protein and messenger ribonucleic acid (mRNA) to high levels in anemic nonhuman primates and in transgenic mice. The study drug acts by suppressing 4 repressors of the fetal globin gene promoter in progenitor cells from thalassemia patients. The drug has been used for many years in a combination product for different actions - to enhance half-life and reduce side effects of an active drug- and is considered safe for chronic use. This trial will first evaluate 3 dose levels in small cohorts of nontransfused patients with beta thalassemia intermedia. The most active dose will then be evaluated in larger subject groups with beta thalassemia and other hemoglobinopathies.