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NCT05664737 | RECRUITING | Anemia

A Study to Determine the Efficacy and Safety of Luspatercept in Adult Participants and to Evaluate the Safety and Pharmacokinetics in and Adolescent Participants With Alpha (α)-Thalassemia
Sponsor:

Bristol-Myers Squibb

Brief Summary:

The purpose of the study is to evaluate the efficacy and safety of luspatercept plus best supportive care (BSC) vs placebo plus BSC on anemia in adult participants with α-thalassemia hemoglobin H (HbH) disease and determine the safety and drug levels in adolescent participants.

Condition or disease

Anemia

Intervention/treatment

Luspatercept

Placebo

Phase

PHASE2

Study Type : INTERVENTIONAL
Estimated Enrollment : 249 participants
Masking : QUADRUPLE
Primary Purpose : TREATMENT
Official Title : A Phase 2, Study for the Treatment of Anemia With Alpha (α)-Thalassemia to Determine the Efficacy and Safety of Luspatercept (BMS-986346/ACE-536) in Adults and Evaluate the Safety and Pharmacokinetics in Adolescents
Actual Study Start Date : 2022-12-09
Estimated Primary Completion Date : 2027-07-16
Estimated Study Completion Date : 2034-08-14

Information not available for Arms and Intervention/treatment

Ages Eligible for Study: 12 Years
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers:
Criteria
Key Inclusion Criteria
  • * Adult participant≥ 18 years with documented diagnosis of A-Thal HbH disease with Transfusion dependence defined as:.
  • 1. TD participant: ≥ 6 RBC units during the 24 weeks prior to randomization.
  • 2. NTD participant:\< 6 RBC units during the 24 weeks prior to randomization(transfusion due to conditions other than A-Thal will not be considered)and, RBC transfusion-free during at least 8 weeks prior to randomization(unless transfusion was required to treat an acute medical condition other than A-Thal) and, mean baseline Hb ≤ 10 g/dL, based on a minimum of 2 measurements ≥ 1 week apart within 4 weeks prior to randomization; hemoglobin values within 21 days post-transfusion will be excluded.
  • * Adult participant has Eastern Cooperative Oncology Group (ECOG) 34 score of 0 or 1.
  • * Adolescent participant 12 years to \< 18 years with documented diagnosis of A-Thal HbH disease with transfusion dependence defined as:.
  • 1. TD participant: ≥ 4 RBC events during the 24 weeks prior to enrollment and, no transfusion-free period for \> 56 days during the 24 weeks prior to enrollment. Participants must have a history of regular transfusions for at least 2 years.
  • 2. NTD participant:\< 4 RBC events during the 24 weeks prior to enrollment and RBC transfusion-free during at least 8 weeks prior to enrollment and, mean baseline Hb ≤ 10 g/dL, based on a minimum of 2 measurements ≥ 1 week apart within 4 weeks prior to enrollment, hemoglobin values within 21 days post-transfusion will be excluded.
  • 3. Participant has Karnofsky (age ≥16 years) or Lansky (age \< 16 years) performance status score ≥ 50 at screening.
  • Key Exclusion Criteria
    • * Medical Conditions: Diagnosis of A-ThalTrait, Hb Bart hydrops, ATRx A-Thal, hemoglobin S/β-thalassemia, myelodysplasia subtype anemia, or with HbE homozygous beta gene mutation. Anemia related to nutritional deficiency, anemia of chronic disease, autoimmune hemolytic anemia, or any other hemolytic anemias. Undergone episodes of hemolysis not related to A-Thal within the 8 weeks prior to randomization.
    • * Participant has deep vein thrombosis (DVT), stroke or other thromboembolic event(s) (except clogged indwelling catheter) requiring medical intervention ≤ 24weeks prior to randomization.
    • * Participant has uncontrolled hypertension. Controlled hypertension for this protocol is considered: blood pressure value corresponding to ≤Grade 1 according to NCI CTCAE Version 5.0. with or without pharmacological treatment.
    • * Reproductive Status: Women who are pregnant, plan to get pregnant during the study, or who are breastfeeding.
    • * Prior/Concomitant: Undergone HSCTs or gene therapy (candidates for HSCT or gene therapy with waiting period of ≥ 12 months are eligible).
    • * Use of hydroxyurea treatment ≤ 12 weeks prior to enrollment for NTD participants and ≤ 24 weeks for TD participants.
    • * Participant who has extramedullary hematopoiesis (EMH) complications requiring treatment to control the growth of EMH mass(es) during the screening period.
    • * Any medical or psychiatric condition (including active infections, recent surgery, sequelae of diseases or interventions, clinically significant laboratory abnormalities or concurrent treatment) that in the opinion of the investigator would put the participant at unacceptable risk of participating in the study or that could affect interpretability of data.
    • * Other protocol-defined inclusion/exclusion criteria apply.
A Study to Determine the Efficacy and Safety of Luspatercept in Adult Participants and to Evaluate the Safety and Pharmacokinetics in and Adolescent Participants With Alpha (α)-Thalassemia

Location Details

NCT05664737

Please Choose a site

How to Participate

Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.

Locations

WITHDRAWN

Canada, Nova Scotia

Local Institution - 0008

Halifax, Nova Scotia, Canada, Knock knock

RECRUITING

China, GD

Sun Yat-sen Memorial Hospital, Sun Yat-Sen University

Guangzhou, GD, China, 510120

RECRUITING

China, GD

Nanfang Hospital of Southern Medical University

Guangzhou, GD, China, 510515

RECRUITING

China, Guangdong

The First People's Hospital of Foshan

Foshan, Guangdong, China, 528000

RECRUITING

China, Guangdong

Maoming People's Hospital

Mao Mingshi, Guangdong, China, 525000

RECRUITING

China, Guangdong

Shenzhen Second People's Hospital

she N townshi, Guangdong, China, 518025

RECRUITING

China, Guangxi

Liuzhou People's Hospital

Liuzhou, Guangxi, China, 545006

RECRUITING

China, GX

People's Liberation Army The 923rd Hospital

Nanning, GX, China, 530021

ACTIVE NOT RECRUITING

China, Hainan

Local Institution - 0011

Haikou, Hainan, China, 570203

COMPLETED

China, Yunnan

Local Institution - 0012

Kunming, Yunnan, China, 650032

RECRUITING

China,

Hainan General Hospital

Haikou, China, 570311

RECRUITING

China,

The First Affiliated Hospital of Guangxi Medical University

Nanning, China, 530021

WITHDRAWN

Greece, B

Local Institution - 0005

Thessaloniki, B, Greece, 546 42

ACTIVE NOT RECRUITING

Greece, E

Local Institution - 0007

Larissa, E, Greece, 412 21

ACTIVE NOT RECRUITING

Greece, G

Local Institution - 0018

Rio, G, Greece, 265 04

ACTIVE NOT RECRUITING

Greece,

Local Institution - 0006

Athens, Greece, 115 27

ACTIVE NOT RECRUITING

Greece,

Local Institution - 0009

Mortar, Greece, 11527

COMPLETED

Hong Kong, HK

Local Institution - 0025

Hong Kong, HK, Hong Kong,

WITHDRAWN

Hong Kong,

Local Institution - 0024

Hong Kong Island, Hong Kong,

WITHDRAWN

Italy, CA

Local Institution - 0022

Cagliari, CA, Italy, 09121

ACTIVE NOT RECRUITING

Italy, GE

Local Institution - 0026

Genova, GE, Italy, 16128

ACTIVE NOT RECRUITING

Italy, TO

Local Institution - 0020

Orbassano, TO, Italy, 10043

ACTIVE NOT RECRUITING

Italy,

Local Institution - 0028

Napoli, Italy, 80131

RECRUITING

Italy,

"University of Campania ""Luigi Vanvitelli"" - AOU - Pediatric Clinic"

Napoli, Italy, 80138

RECRUITING

Malaysia,

Sultanah Aminah Hospital

Johor Bahru, Malaysia, 80100

NOT YET RECRUITING

Malaysia,

Local Institution - 0032

Kuala Lumpur, Malaysia, 50586

NOT YET RECRUITING

Saudi Arabia,

Local Institution - 0035

The share, Saudi Arabia, 31982

NOT YET RECRUITING

Saudi Arabia,

Local Institution - 0034

Riyadh, Saudi Arabia, 11411

NOT YET RECRUITING

Singapore,

Local Institution - 0036

Singapore, Singapore, 229899

RECRUITING

Taiwan, KHH

Medical University Chunng-Hospital

City fast, KHH, Taiwan, 807

RECRUITING

Taiwan, TPE

National Taiwan University Hospital

NaN gang Q U, TPE, Taiwan, 10002

RECRUITING

Taiwan, TXG

China Medical University Hospital

Taichung, TXT, Taiwan, 40447

RECRUITING

Thailand,

Siriraj Hospital

Bangkok, Thailand, 10700

NOT YET RECRUITING

Thailand,

Local Institution - 0031

Mueang Phitsanulok, Thailand, 65000

NOT YET RECRUITING

Turkey (Türkiye),

Local Institution - 0027

Altindag, Turkey (Türkiye), 06230

NOT YET RECRUITING

Turkey (Türkiye),

Local Institution - 0021

Topkapi, Turkey (Türkiye), 34093