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Trial record 1 of 1 for:    sotatercept (ACE-011) myelofibrosis
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Study of Sotatercept (ACE-011) in Subjects With Myeloproliferative Neoplasm (MPN)-Associated Myelofibrosis (MF) and Anemia

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ClinicalTrials.gov Identifier: NCT01712308
Recruitment Status : Recruiting
First Posted : October 23, 2012
Last Update Posted : September 14, 2018
Sponsor:
Collaborator:
Celgene
Information provided by (Responsible Party):
M.D. Anderson Cancer Center

October 18, 2012
October 23, 2012
September 14, 2018
February 2013
February 2020   (Final data collection date for primary outcome measure)
Anemia Response [ Time Frame: 15 weeks ]
Primary analysis of response for each dose cohort performed once all 20 patients have been accrued to that dose cohort and treated for at least 5 cycles. Clinical efficacy assessed as anemia response with a target rate of greater than or equal to 30% at either dose level. Anemia response is a composite endpoint defined as an increase in hemoglobin in a subject with anemia or becoming RBC-transfusion-independent in a subject who is RBC-transfusion-dependent.
Same as current
Complete list of historical versions of study NCT01712308 on ClinicalTrials.gov Archive Site
Duration of Response [ Time Frame: 15 weeks ]
Duration of response defined as date at which subject's objective status is first noted to be a response, to date progression is documented (if one has occurred) or to date of last follow-up (for those subjects who have not progressed).
Same as current
Not Provided
Not Provided
 
Study of Sotatercept (ACE-011) in Subjects With Myeloproliferative Neoplasm (MPN)-Associated Myelofibrosis (MF) and Anemia
A Phase-2, Prospective, Open-Label Study to Determine the Safety and Efficacy of Sotatercept (ACE-011) in Subjects With Myeloproliferative Neoplasm (MPN) -Associated Myelofibrosis and Anemia

The goal of this clinical research study is to learn if sotatercept can help to control MPN-associated myelofibrosis and anemia. The safety of this drug will also be studied.

This is an investigational study. Sotatercept is not FDA approved or commercially available. It is currently being used for research purposes only.

Up to 60 patients will take part in this study. All will be enrolled at MD Anderson.

Study Groups:

If you are found to be eligible to take part in this study, you will be assigned to 1 of 3 study groups, that will receive different doses of the study drug, based on when you join this study. Patients who are already on treatment with ruxolitinib will be assigned to Group 3.

  • Group 1 will receive sotatercept 1 time every 3 weeks (at a lower dose).
  • Group 2 will receive will receive sotatercept 1 time every 3 weeks (at a higher dose).
  • Group 3 will receive sotatercept 1 time every 3 weeks (at a lower dose).

Study Drug Administration:

Each cycle is 21 days.

You will receive sotatercept at the clinic as an injection under the skin on Day 1 of every cycle. The injections will be given in the upper arm, abdomen, or thigh.

Study Visits:

On Day 1 of every cycle:

  • You will have a physical exam, including measurement of your weight and vital signs (blood pressure, breathing rate, temperature, and heart rate).
  • Your spleen and/or liver will be measured by feeling your abdomen.
  • You will be asked about any drugs you are taking and about recent blood transfusions you may have had.
  • You will also be asked about any new side effects or medical conditions you may have experienced.
  • Your performance status will be recorded.
  • Blood (about 4 teaspoons) will be drawn for routine tests
  • Urine will be collected for kidney function tests.
  • You will complete a questionnaire that asks you about quality of life (for the first 6 cycles only and then 1 time every 3 cycles). It should take about 10 minutes to complete the questionnaire.

Weekly during Cycle 1-5 only:

  • Blood (about 2 teaspoons) will be drawn for routine tests.
  • You will have vital signs measured on Day 1 of Week 3 (around Day 15 of Cycle 1).

On Day 1 of Cycle 6, and then every 6-24 cycles after that if the disease responds to treatment, a bone marrow biopsy will be performed to check the status of the disease.

On Day 1 of Cycles 1-5, 7, 9, and 11, then every 6 cycles after that:

°Blood (about 2 teaspoons) will be drawn for antibody testing and pharmacokinetic (PK) testing. Antibodies are created by the immune system and may attack foreign cells or substances, such as the study drug. PK testing measures the amount of study drug in the body at different time points.

Length of Study:

You may continue taking the study drug for as long as the doctor thinks it is in your best interest. You will no longer be able to take the study drug if the disease gets worse, if intolerable side effects occur, or if you are unable to follow study directions.

Your participation on the study will be over after the follow-up visits.

End-of-Treatment Visit:

After your last dose of study drug, you will have an end-of-study visit and the following tests and procedures will be performed:

  • You will have a physical exam, including measurement of your weight and vital signs (blood pressure, heart rate, breathing rate, and temperature) and your spleen and/or liver will be measured by feeling your abdomen.
  • Your performance status will be recorded.
  • Blood (about 4 teaspoons) and urine will be collected for routine tests.
  • You will be asked about any drugs you are taking and about recent blood transfusions you may have had.
  • You will also be asked about any new side effects or medical conditions you may have experienced.
  • You will complete the quality of life questionnaire.

Follow-up Visit:

About 1 month after your last dose of study drug, you will receive a telephone call from someone on the study team. You will be asked about any drugs you are taking, any recent blood transfusions you may have had, any new side effects or medical conditions you may have experienced. This phone call should last about 10 minutes.

Interventional
Phase 2
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Leukemia
Drug: Sotatercept

Higher Dose Group: 1.0 mg/kg dose subcutaneously on Day 1 every 3 weeks.

Lower Dose Group: 0.75 mg/kg dose subcutaneously on Day 1 every 3 weeks.

Myelofibrosis on Treatment with Ruxolitinib Group: 0.75 mg/kg dose subcutaneously on Day 1 every 3 weeks.

Other Name: ACE-011
  • Experimental: Sotatercept (higher dose) Group
    Patients treated with 1.0 mg/kg dose subcutaneously on Day 1 every 3 weeks. If there is at least one responder out of 5 treated patients, this cohort will be expanded with an additional 15 subjects.
    Intervention: Drug: Sotatercept
  • Experimental: Sotatercept (lower dose) Group
    Patients treated with 0.75 mg/kg dose subcutaneously on Day 1 every 3 weeks. If there is at least one responder out of 5 treated patients, this cohort will be expanded with an additional 15 subjects.
    Intervention: Drug: Sotatercept
  • Experimental: Myelofibrosis on Treatment with Ruxolitinib Group
    Participants already on therapy with ruxolitinib (for at least for 6 months, and on stable dose for last 2 months, receive 0.75 mg/kg dose of sotatercept subcutaneously every 3 weeks. If there is at least one responder out of 5 treated patients, this cohort will be expanded with an additional 15 subjects.
    Intervention: Drug: Sotatercept
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
60
40
February 2021
February 2020   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  1. MPN-associated myelofibrosis
  2. Anemic patient OR RBC-transfusion-dependent patient
  3. Equal to or greater than 18 years of age
  4. ALT (SGPT) and AST (SGOT) equal to or less than 2.5x upper limit of normal (ULN), or equal to or less than 4x ULN (if upon judgment of the treating physician, it is believed to be due to extramedullary hematopoiesis [EMH] related to MF)
  5. Direct bilirubin equal to or less than 1.5 x ULN; or equal to or less than 2x ULN (if upon judgment of the treating physician, it is believed to be due to extramedullary hematopoiesis related to MF)
  6. Creatinine clearance equal to or more than 50 mL/min
  7. Treatment-related toxicities from prior therapies must have resolved to Grade equal to or less than 1
  8. Women of childbearing potential and men must agree to using medically approved (i.e., mechanical or pharmacological) contraceptive measure for at least 112 days following the last dose of sotatercept (ACE-011), Males must agree to use a latex condom or non-latex condom NOT made of natural (animal) membrane during any sexual contact with females of childbearing potential or a pregnant female while participating in the study and for at least 112 days following the last dose of sotatercept (ACE-011), even if he has a vasectomy.
  9. For cohort of patients that are already on ruxolitinib therapy: on therapy with ruxolitinib for at least for 6 months, and on stable dose for last 2 months, before starting therapy with sotatercept

Exclusion Criteria:

  1. Serious medical condition or psychiatric illness that would prevent, (as judged by the treating physician) the subject from signing the informed consent form or any condition including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study
  2. Pregnant or lactating female
  3. Known positive for human immunodeficiency virus-1 (HIV-1), or active infection with hepatitis-B or -C
  4. Use of any MPN-associated myelofibrosis-directed therapy within 2 weeks prior to study Day 1
  5. Symptomatic congestive heart failure, unstable angina, or unstable cardiac arrhythmia
  6. Prior sotatercept
  7. Major surgery within 4 weeks prior to Day 1
  8. Severe allergic or anaphylactic reactions or hypersensitivity to recombinant proteins or excipients in the investigational product
  9. Uncontrolled hypertension (systolic blood pressure [SBP] equal to or more than 140 or diastolic blood pressure [DBP] equal to or more than 90).
Sexes Eligible for Study: All
18 Years and older   (Adult, Older Adult)
No
Contact: Prithviraj Bose, MD 713-792-7305 CR_Study_Registration@mdanderson.org
United States
 
 
NCT01712308
2012-0534
NCI-2012-03139 ( Registry Identifier: NCI CTRP )
No
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Not Provided
M.D. Anderson Cancer Center
M.D. Anderson Cancer Center
Celgene
Principal Investigator: Prithviraj Bose, MD M.D. Anderson Cancer Center
M.D. Anderson Cancer Center
September 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP