Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia

Part of paid clinical trials in Los Angeles, California.

Sponsor
Celgene
Study ID
NCT04143724
Phase
PHASE2
Status
Recruiting
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Conditions

  • Beta-Thalassemia

Eligibility Criteria

Sex
ALL
Age
6 Years - 17 Years
Healthy Volunteers
Not accepted

Interventions

  • ACE-536 — DRUG
    Specified dose on specified days

Study Details

This is a Phase 2a study to evaluate the safety and pharmacokinetics (PK) of luspatercept in pediatric participants with β-thalassemia. The study will be conducted in 2 parts for both transfusion-dependent (TD) and non-transfusion-dependent (NTD) β-thalassemia participants: TD Part A will be in adolescent participants aged 12 to \<18 years with two dose escalation cohorts, followed by a dose expansion cohorts. NTD Part A will be conducted in the same age group participants as TD Part A with dose confirmation and expansion cohorts. After Part A TD participants have completed at least one year of treatment, all available safety data from Part A adolescent participants will be evaluated before initiating TD and NTD Part B in the age group from 6 to \<12 years old. Part B will consist of two dose escalation cohorts for TD and two dose escalation cohorts for NTD. Upon completion of the Treatment Period, participants of any cohort who are benefiting from the study treatment, will be offered the opportunity to continue luspatercept treatment in the Long-term Treatment Period for up to 5 years from their first dose. Participants who discontinue study treatment at any time will continue in the Posttreatment Follow-up Period for at least 5 years from their first dose of luspatercept, or 3 years from their last dose, whichever occurs later, or until they withdraw consent/assent, are lost to follow-up, or the End of Trial, whichever occurs first.

Key Dates

Start date
Nov 7, 2019
Status verified
Jan 2026
Primary completion
Jul 2, 2027
Completion
Jun 11, 2035

Study Design

Enrollment
99 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Cohort 1: TD Dose Escalation Cohort: 12 to < 18 years Luspatercept 0.75 mg/kg
  • Experimental: Cohort 2: TD Dose Escalation Cohort: 12 to < 18 years: Luspatercept 1.0 mg/kg
  • Experimental: Cohort 3: TD Dose Expansion Cohort: 12 to <18 years Luspatercept 1.0 mg/kg
  • Experimental: Cohort 4: TD Dose Escalation Cohort: 6 to < 12 years Luspatercept 1.0 mg/kg
  • Experimental: Cohort 5: TD Dose Escalation Cohort: 6 to <12 years Luspatercept 1.2 mg/kg
  • Experimental: Cohort 6: NTD Dose Confirmation Cohort: 12 to < 18 years Luspatercept 1.0 mg/kg
  • Experimental: Cohort 7: NTD Dose Expansion Cohort: NTD 12 to < 18 years
  • Experimental: Cohort 8: NTD Dose Escalation Cohort: 6 to < 12 years Luspatercept 1.0 mg/kg
  • Experimental: Cohort 9: NTD Dose Escalation Cohort: 6 to < 12 years Luspatercept 1.2 mg/kg

Primary Outcome Measure

Determination of the Recommended Dose (RD [ Time Frame: Cycle 1 up to the day before Cycle 2 Day 1 or Study Day 22 if not receiving the second treatment cycle ]

Central Contacts

  • BMS Study Connect Contact Center www.BMSStudyConnect.com
    855-907-3286
    [email protected]
  • First line of email MUST contain NCT # and Site #.

Locations (2)

FacilityCityStateZIPSite coordinators
Local Institution - 601Los AngelesCalifornia90027-
New York Presbyterian HospitalNew YorkNew York10065-4870
Sujit Sheth, Site 602
212-746-3400

Find similar trials in Los Angeles, CA

By research site
Local Institution - 601· Los Angeles, CANew York Presbyterian Hospital· New York, NY

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