Today, we will be looking into the latest research and clinical trials focused on thalassemia in adults.
The following list has been carefully curated by evaluating the ongoing phase 3 trials for thalassemia, specifically targeting adults in the United States. Please note that the dates provided are approximate and subject to change. This compilation primarily features studies that have released updates within the past 12 months.
This series aims to offer a glimpse into upcoming innovations in the field and how the outcomes of these studies could potentially influence clinical guidelines related to the topic.
Without further ado, let us explore the list of Thalassemia Clinical Trials!
Quick View Table of Thalassemia Clinical Trials
Phase 3 Clinical Trials:
A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia
- Sponsor: Novo Nordisk A/S
- Etavopivat is a new medicine under development for treating blood disorders like sickle cell disease and thalassaemia. Sickle cell disease and thalassaemia are inherited blood disorders that affect haemoglobin. Haemoglobin is the protein that carries oxygen through the body. This study is looking into how safe treatment with etavopivat is and how well it works over a long period of time. The study will last for up to 264 weeks, but it will end earlier if etavopivat is approved in the participant’s country.
- Interventions: DRUG: Etavopivat A | DRUG: Etavopivat B
- Primary Outcomes Measures: Number of treatment emergent adverse events (TEAEs), reported for each indication and age group separately, Measured as number of events., Baseline (week 0 of FLORAL) to end of study (week 264, or earlier)|Number of adverse reactions, reported for each indication and age group separately, Measured as number of adverse reactions., Baseline (week 0 of FLORAL) to end of study (week 264, or earlier)
Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
- Sponsor: Vertex Pharmaceuticals Incorporated
- This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) using CTX001.
- Interventions: BIOLOGICAL: CTX001
- Primary Outcomes Measures: Fetal Hemoglobin (HbF) Concentration Over Time, Up to 12 Months After CTX001 Infusion|Total Hemoglobin (Hb) Concentration Over Time, Up to 12 Months After CTX001 Infusion
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
- Sponsor: Agios Pharmaceuticals, Inc.
- The primary purpose of this study is to compare the effect of mitapivat versus placebo on transfusion burden in participants with transfusion-dependent alpha- or beta-thalassemia (TDT).
- Interventions: DRUG: Placebo Matching Mitapivat | DRUG: Mitapivat
- Primary Outcomes Measures: Percentage of Participants With Transfusion Reduction Response (TRR), TRR is defined as ≥50% reduction in transfused red blood cells (RBC) units with a reduction of ≥2 units of transfused RBCs in any consecutive 12-week period through Week 48 compared with baseline., Baseline up to Week 48
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)
- Sponsor: Pfizer
- The purpose of this open-label extension (OLE) study is to evaluate the safety and efficacy of etrasimod in participants with moderately to severely active ulcerative colitis (UC) who previously received double-blind treatment (either etrasimod 2 mg per day or placebo) during participation in one of the qualified Phase 3 or Phase 2 double-blind, placebo-controlled parent studies including but not limited to: (APD334-301 [NCT03945188] or APD334-302 [NCT03996369] or APD334-210 [NCT04607837]).
- Interventions: DRUG: Etrasimod
- Primary Outcomes Measures: Number and Severity of Safety Measures, Safety as assessed by the evaluation of adverse events, Up to approximately 8 years
A Long-term Follow-up Study in Participants Who Received CTX001
- Sponsor: Vertex Pharmaceuticals Incorporated
- This is a multi-site, open- label rollover study to evaluate the long-term safety and efficacy of CTX001 in pediatric and adult participants who received CTX001 in parent studies 111 (NCT03655678) 141 (NCT05356195) or 161 (NCT05477563) (transfusion-dependent β-thalassemia \[TDT\] studies) or Study 121 (NCT03745287) or 151 (NCT05329649), 161(NCT05477563),171 (NCT05951205) (severe sickle cell disease \[SCD\] studies).
- Interventions: BIOLOGICAL: CTX001
- Primary Outcomes Measures: New malignancies, Signing of informed consent up to 15 years post CTX001 infusion|New or worsening hematologic disorders, Signing of informed consent up to 15 years post CTX001 infusion|All-cause mortality, Signing of informed consent up to 15 years post CTX001 infusion|Serious adverse events (SAEs), Signing of informed consent up to 15 years post CTX001 infusion|CTX001-related adverse events (AEs), Signing of informed consent up to 15 years post CTX001 infusion
A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials
- Sponsor: Celgene
- A Phase 3b, open-label, single-arm, rollover study to evaluate the long-term safety of luspatercept, to the following participants:
- * Participants 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 from continuing treatment with luspatercept
- * Participants 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
- Interventions: BIOLOGICAL: CTX001
- Primary Outcomes Measures: New malignancies, Signing of informed consent up to 15 years post CTX001 infusion|New or worsening hematologic disorders, Signing of informed consent up to 15 years post CTX001 infusion|All-cause mortality, Signing of informed consent up to 15 years post CTX001 infusion|Serious adverse events (SAEs), Signing of informed consent up to 15 years post CTX001 infusion|CTX001-related adverse events (AEs), Signing of informed consent up to 15 years post CTX001 infusion
A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion-Dependent β-Thalassemia
- Sponsor: Vertex Pharmaceuticals Incorporated
- This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in subjects with transfusion-dependent β-thalassemia (TDT). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using CTX001.
- Interventions: BIOLOGICAL: CTX001
- Primary Outcomes Measures: Proportion of subjects achieving transfusion independence for at least 12 consecutive months (TI12), From 60 days after last RBC transfusion up to 24 months post-CTX001 infusion]|Proportion of subjects with engraftment (first day of 3 consecutive measurements of absolute neutrophil count [ANC] ≥500/µL on three different days), Within 42 days after CTX001 infusion|Time to neutrophil and platelet engraftment, Days post-infusion to engraftment|Frequency and severity of collected adverse events (AEs), Signing of informed consent through Month 24 visit|Incidence of transplant-related mortality (TRM), Baseline (pre-transfusion) to 100 days and 1 year post-CTX001 infusion|All-cause mortality, Signing of informed consent through Month 24 visit
A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia
- Sponsor: bluebird bio
- This is a single-arm, multi-site, single-dose, Phase 3 study in approximately 18 participants less than or equal to (\<=) 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.
- Interventions: GENETIC: LentiGlobin BB305 Drug Product
- Primary Outcomes Measures: Percentage of Participants Who Have Achieved Transfusion Independence (TI), TI was defined as a weighted average hemoglobin (Hb) \>= 9 grams per deciliter (g/dL) without any packed red blood cell (pRBC) transfusions for a continuous period of \>= 12 months at any time during the study after drug product infusion., From 12 to 24 months post-transplant
Potential Guideline That May Be Affected Includes:
- Guidelines for the Management of α-thalassaemia
- Thalassaemia International Federation
- 2023
- Guidelines for NTD Beta-Thalassaemia
- Thalassaemia International Federation
- 2023
There you have it – a list of phase 3 Clinical Trials for Thalassemia as of December 2024. Stay tuned, for our next Guidelines+ Trials Rundown. Sign up for alerts and stay informed on the latest published guidelines and articles.
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