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

Status: Recruiting
Location: See all (36) locations...
Intervention Type: Drug, Biological
Study Type: Interventional
Study Phase: Phase 2
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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 12
Healthy Volunteers: f
View:

• Adult participant≥ 18 years with documented diagnosis of A-Thal HbH disease with Transfusion dependence defined as:.

‣ TD participant: ≥ 6 RBC units during the 24 weeks prior to randomization.

⁃ 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:.

‣ 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.

⁃ 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.

⁃ Participant has Karnofsky (age ≥16 years) or Lansky (age \< 16 years) performance status score ≥ 50 at screening.

Locations
Other Locations
Canada
Local Institution - 0008
WITHDRAWN
Halifax
China
The First People's Hospital of Foshan
RECRUITING
Foshan
Nanfang Hospital of Southern Medical University
RECRUITING
Guangzhou
Sun Yat-sen Memorial Hospital, Sun Yat-Sen University
RECRUITING
Guangzhou
Hainan General Hospital
RECRUITING
Haikou
Local Institution - 0011
ACTIVE_NOT_RECRUITING
Haikou
Local Institution - 0012
COMPLETED
Kunming
Liuzhou People's Hospital
RECRUITING
Liuchow
Maoming People's Hospital
RECRUITING
Maoming Shi
People's Liberation Army The 923rd Hospital
RECRUITING
Nanning
The First Affiliated Hospital of Guangxi Medical University
RECRUITING
Nanning
Shenzhen Second People's Hospital
RECRUITING
Shenzhen Shi
Greece
Local Institution - 0006
ACTIVE_NOT_RECRUITING
Athens
Local Institution - 0009
ACTIVE_NOT_RECRUITING
Goudi
Local Institution - 0007
ACTIVE_NOT_RECRUITING
Larissa
Local Institution - 0018
ACTIVE_NOT_RECRUITING
Rio
Local Institution - 0005
WITHDRAWN
Thessaloniki
Hong Kong Special Administrative Region
Local Institution - 0025
COMPLETED
Hong Kong
Local Institution - 0024
WITHDRAWN
Hong Kong Island
Italy
Local Institution - 0022
WITHDRAWN
Cagliari
Local Institution - 0026
ACTIVE_NOT_RECRUITING
Genova
Local Institution - 0028
ACTIVE_NOT_RECRUITING
Napoli
Universita degli Studi della Campania Luigi Vanvitelli - AOU - Clinica Pediatrica
RECRUITING
Napoli
Local Institution - 0020
ACTIVE_NOT_RECRUITING
Orbassano
Malaysia
Hospital Sultanah Aminah
RECRUITING
Johor Bahru
Local Institution - 0032
NOT_YET_RECRUITING
Kuala Lumpur
Saudi Arabia
Local Institution - 0035
NOT_YET_RECRUITING
Al-ahsa
Local Institution - 0034
NOT_YET_RECRUITING
Riyadh
Singapore
Local Institution - 0036
NOT_YET_RECRUITING
Singapore
Taiwan
Kaohsiung Medical University Chung-Ho Memorial Hospital
RECRUITING
Kaohsiung City
National Taiwan University Hospital
RECRUITING
Nan Gang Qu
China Medical University Hospital
RECRUITING
Taichung
Thailand
Siriraj Hospital
RECRUITING
Bangkok
Local Institution - 0031
NOT_YET_RECRUITING
Mueang Phitsanulok
Turkey
Local Institution - 0027
NOT_YET_RECRUITING
Altındağ
Local Institution - 0021
NOT_YET_RECRUITING
Topkapı
Contact Information
Primary
BMS Study Connect Contact Center www.BMSStudyConnect.com
Clinical.Trials@bms.com
855-907-3286
Backup
First line of the email MUST contain the NCT# and Site #.
Time Frame
Start Date: 2022-12-09
Estimated Completion Date: 2034-08-14
Participants
Target number of participants: 249
Treatments
Experimental: Transfusion Dependent (TD): Luspatercept + Best supportive care (BSC)
Placebo_comparator: Adult TD Cohort: Placebo + BSC
Experimental: Non-transfusion Dependent (NTD): Luspatercept + BSC
Placebo_comparator: Adult NTD Cohort: Placebo + BSC
Related Therapeutic Areas
Sponsors
Leads: Bristol-Myers Squibb

This content was sourced from clinicaltrials.gov