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Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan Anemia (ACE-011-DBA)

Primary Purpose

Diamond Blackfan Anemia

Status
Terminated
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Sotatercept
Sotatercept with prednisone boost
Sponsored by
Northwell Health
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Diamond Blackfan Anemia focused on measuring Diamond Blackfan anemia, DBA, Blackfan Diamond anemia, Congenital pure red cell aplasia, Aase-Smith II Syndrome, Aase Syndrome, Anemia, Congenital Pure Red Cell, Aplasia, Congenital Pure Red Cell, BDA, Congenital Hypoplastic Anemia, Erythrogenesis Imperfecta, Hypoplastic Congenital Anemia, Inherited Erythroblastopenia, pure red cell aplasia, bone marrow failure syndrome

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • >/= 18 years of age
  • DBA diagnosed
  • RBC transfusion- dependence (defined as >/= 10 cc/kg of RBC per 28 days average)
  • Karnofsky performance scale >/= 70
  • Females of childbearing potential are to use birth control during study participation and for 112 days following the last dose of sotatercept
  • Males must agree to use a latex condom during any sexual contact with females of childbearing potential while participating in the study and for 112 days following the last dose of sotatercept
  • Agreement to adhere to the study visit schedule, understand and comply with all protocol requirements
  • Understand and sign a written informed consent

Exclusion Criteria:

  • Creatinine clearance < 30 ml/min
  • SGOT > 3x upper limit of normal, SGPT > 3x upper limit normal, or bilirubin >3x upper limit normal
  • Heart disease (NY Heart Association classification of >/= 3
  • History of hypertension
  • Subjects currently responsive to corticosteroids for treatment of Diamond Blackfan anemia
  • Treatment with another investigational drug or device <56 days pre-study entry
  • Pregnant or lactating females
  • Cancer

Sites / Locations

  • North Shore- LIJ campus of The Feinstein Institute for Medical Research

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

Sotatercept

Sotatercept with prednisone boost

Arm Description

Sotatercept to be given as a subcutaneous injection once a month for 4 consecutive months, using a dose escalation scale among 3 cohorts. *Protocol Amendment: Two additional cohorts will be given Sotatercept 0.75mg/kg and 1 mg/kg as a subcutaneous injection once every 3 weeks.

Protocol Amendment: Two additional cohorts will be given Sotatercept 0.75mg/kg and 1 mg/kg as a subcutaneous injection once every 3 weeks along with a prednisone boost of 1 mg/kg daily for 3 weeks (max of 60 mg).

Outcomes

Primary Outcome Measures

Number of Participants With Complete Response and Partial Response
Complete response is transfusion independence with hemoglobin >9 gm/dl; partial response is transfusion dependence with hemoglobin < 9gm/dl with an increase in reticulocyte count over baseline

Secondary Outcome Measures

Severe Adverse Events Attributable to Study Drug
Assess severity of adverse events and relationship to sotatercept according to the currently active minor version of the NCI Common Terminology for Adverse Events version 4.0

Full Information

First Posted
October 31, 2011
Last Updated
February 27, 2023
Sponsor
Northwell Health
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1. Study Identification

Unique Protocol Identification Number
NCT01464164
Brief Title
Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan Anemia
Acronym
ACE-011-DBA
Official Title
Phase I/II, Open-Label Study to Determine Safety and Efficacy of Sotatercept (ACE-011) in Adults With Red Blood Cell Transfusion- Dependent Diamond Blackfan Anemia
Study Type
Interventional

2. Study Status

Record Verification Date
February 2023
Overall Recruitment Status
Terminated
Why Stopped
Supporter terminated the study due to no active patients (secondary to travel restrictions due to COVID).
Study Start Date
January 2012 (Actual)
Primary Completion Date
April 13, 2021 (Actual)
Study Completion Date
April 13, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Northwell Health

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The purpose of this study is to determine the safety and dosing of drug Sotatercept, as a subcutaneous injection, to stimulate production of red blood cell production. To be given every 28 days for up to four doses.
Detailed Description
This study is for adult patients with Diamond Blackfan Anemia who are currently being transfused every 3- 4 weeks. The drug will be given as a subcutaneous injection within one of three dose levels. The first dose level (0.1 mg/kg) will include 3 subjects who will receive the same dose of the drug monthly for a total of 4 doses, as long as there are no dose- limiting toxicities (DLTs). A Dose limiting toxicity is defined as inability to deliver the scheduled doses because of toxicity >/= Grade 3, according to NCI Toxicity Grading Scale. Once all 3 subjects have received and tolerated the low dose level, the next level will open (0.3 mg/kg)to 3 new subjects. Subjects will be monitored closely for response and side effects. If more than 2 subjects have a dose limiting toxicity in the first dose level, then dose level -1 (0.05 mg/kg)will open, enrolling 3 more subjects. If more than 2 subjects at dose level -1, have dose limiting toxicities, the study will be discontinued. If there are no additional dose limiting toxicities, dose level -1 will be the maximum tolerated dose. There are a total of 3 dose levels, not including dose level -1. Once the maximum tolerated dose has been reached, up to a total of 10 additional subjects will be enrolled. Protocol Amendment: The protocol has been amended to include an additional enrollment of 20 subjects with two additional dose levels of Sotatercept (0.075 mg/kg and 0.100 mg/kg ) to be given with or without a prednisone boost. Efficacy will be measured by response. A complete response will be determined if the subject no longer requires transfusion, while on study drug. A partial response will be measured by a reduction by 50% in need for transfusion. Treatment modifications will be made based on evidence of side effects. Dose- escalation will be performed only if no side effects are reported and no efficacy is evidenced. Treatment will be stopped if hemoglobin is >12 gm/dl and/ or any >/+ grade 3 adverse event is related to sotatercept. Study assessments will include: Physical exam (height, weight, vital signs and blood pressure)at screening, day 1 of each cycle and monthly for 3 months of follow up period as well as at study discontinuation. Additional blood pressure monitoring at day 1 of each cycle and weekly there after through cycle 4, monthly in follow up period and at study discontinuation. Karnofsky performance status at screening, day 1 of cycle 2 and day 1 of monthly follow up period for 3 months and study discontinuation. CBC reticulocyte count and ANC will be collected at day 1 of each cycle and weekly there after through cycle 4, monthly in follow up period and at study discontinuation. Blood chemistry, liver and kidney function will be assessed at screening, day 1 and 15 of cycle 1, day 1 of cycle 2, 3, 4 month 1 in follow up and at study discontinuation. Blood for iron status (serum iron, transferrin and %, ferritin) will be collected during the screening period, and at month 1 of the follow-up period as well as study discontinuation. Folate and B12 levels will be assessed during the screening period at day 1 of cycle 3 and again at study discontinuation. Urinalysis (creatinine, protein),at screening, day 15 of cycle 1, day 1 of cycles 2, 3, and 4 monthly during follow-up and at study discontinuation. Serum erythropoietin at screening, day 15 of cycle 1, day 1 of cycle 3, monthly during follow-up and at study discontinuation. FSH & LH (everyone), DHEA & testosterone (males only), estrogen & estradiol (females only)at screening, day 1 of cycles 2, 3, and 4, monthly during follow- up period and at study discontinuation. EKG & ECHO at screening, day 1 of cycle 3 first month of follow-up period and at study discontinuation Dexa Scan at screening, day 1 of monthly follow-up period and at study discontinuation Transfusion assessment at screening, day 1, 8, 15 and 22 of cycle 1, day 1 and 15 of cycles 2, 3, and 4, monthly during follow-up period and at study discontinuation. Response assessment at day 8, 15, 22 of cycle 1, day 1 and 15 of cycles2, 3, and 4, monthly for 3 months during follow-up period and at study discontinuation Adverse events, concomitant therapies to be assessed at day 1, 8, 15, and 22 of cycle 1, day 1 and 15 of cycles 2, 3, and 4, monthly during follow-up period and at study discontinuation Drug administration day 1 of cycles 1, 2, 3, and 4.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Diamond Blackfan Anemia
Keywords
Diamond Blackfan anemia, DBA, Blackfan Diamond anemia, Congenital pure red cell aplasia, Aase-Smith II Syndrome, Aase Syndrome, Anemia, Congenital Pure Red Cell, Aplasia, Congenital Pure Red Cell, BDA, Congenital Hypoplastic Anemia, Erythrogenesis Imperfecta, Hypoplastic Congenital Anemia, Inherited Erythroblastopenia, pure red cell aplasia, bone marrow failure syndrome

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
19 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Sotatercept
Arm Type
Experimental
Arm Description
Sotatercept to be given as a subcutaneous injection once a month for 4 consecutive months, using a dose escalation scale among 3 cohorts. *Protocol Amendment: Two additional cohorts will be given Sotatercept 0.75mg/kg and 1 mg/kg as a subcutaneous injection once every 3 weeks.
Arm Title
Sotatercept with prednisone boost
Arm Type
Experimental
Arm Description
Protocol Amendment: Two additional cohorts will be given Sotatercept 0.75mg/kg and 1 mg/kg as a subcutaneous injection once every 3 weeks along with a prednisone boost of 1 mg/kg daily for 3 weeks (max of 60 mg).
Intervention Type
Drug
Intervention Name(s)
Sotatercept
Other Intervention Name(s)
ACE-011
Intervention Description
Cohort 4a: 3 patients on Sotatercept 0.75 mg/kg every 3 weeks. If the patients have no untoward events in the Patients in Dose Level 4 by the end of 3 of the 6 doses, then additional patients (Dose Level 5) will be enrolled at the next dose level: Cohort 5a: 3 patients on Sotatercept 1 mg/kg every 3 weeks
Intervention Type
Drug
Intervention Name(s)
Sotatercept with prednisone boost
Other Intervention Name(s)
ACE-011 and Prednisone
Intervention Description
Cohort 4b: 3 patients on Sotatercept 0.75 mg/kg every 3 weeks with a three week prednisone boost of 1 mg/kg/day (max 60 mg). If the patients have no untoward events in the Patients in Dose Level 4 by the end of 3 of the 6 doses, then additional patients (Dose Level 5) will be enrolled at the next dose level: Cohort 5b: 3 patients on Sotatercept 1 mg/kg every 3 weeks with a 3 week prednisone boost of 1 mg/kg/day (max 60 mg).
Primary Outcome Measure Information:
Title
Number of Participants With Complete Response and Partial Response
Description
Complete response is transfusion independence with hemoglobin >9 gm/dl; partial response is transfusion dependence with hemoglobin < 9gm/dl with an increase in reticulocyte count over baseline
Time Frame
9 months
Secondary Outcome Measure Information:
Title
Severe Adverse Events Attributable to Study Drug
Description
Assess severity of adverse events and relationship to sotatercept according to the currently active minor version of the NCI Common Terminology for Adverse Events version 4.0
Time Frame
9 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: >/= 18 years of age DBA diagnosed RBC transfusion- dependence (defined as >/= 10 cc/kg of RBC per 28 days average) Karnofsky performance scale >/= 70 Females of childbearing potential are to use birth control during study participation and for 112 days following the last dose of sotatercept Males must agree to use a latex condom during any sexual contact with females of childbearing potential while participating in the study and for 112 days following the last dose of sotatercept Agreement to adhere to the study visit schedule, understand and comply with all protocol requirements Understand and sign a written informed consent Exclusion Criteria: Creatinine clearance < 30 ml/min SGOT > 3x upper limit of normal, SGPT > 3x upper limit normal, or bilirubin >3x upper limit normal Heart disease (NY Heart Association classification of >/= 3 History of hypertension Subjects currently responsive to corticosteroids for treatment of Diamond Blackfan anemia Treatment with another investigational drug or device <56 days pre-study entry Pregnant or lactating females Cancer
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Adrianna Vlachos, MD
Organizational Affiliation
Northwell Health/Feinstein Institute for Medical Research
Official's Role
Principal Investigator
Facility Information:
Facility Name
North Shore- LIJ campus of The Feinstein Institute for Medical Research
City
Manhasset
State/Province
New York
ZIP/Postal Code
11030
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No
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Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan Anemia

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