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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


Phase 2
18 Years
N/A
Open (Enrolling)
Both
Leukemia

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Trial Information

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


Study Groups:

If you are found to be eligible to take part in this study, you will be assigned to 1 of 2
study groups, that will receive different doses of the study drug, based on when you join
this study.

- 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).

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.

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 6-12 cycles after that if the disease responds to
treatment, a bone marrow biopsy will be performed to check the status of the disease.

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) 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.

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.

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

Up to 40 patients will take part in this study. All will be enrolled at MD Anderson.


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 2.5* upper limit of normal (ULN), or
equal to or less than 4 * 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 * ULN; or equal to or less than 2 * ULN
(if upon judgment of the treating physician, it is believed to be due to
extramedullary hematopoiesis related to MF)

6. Serum creatinine equal to or less than 2.5 mg/dL

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.

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 human immunodeficiency virus-1 (HIV-1), or hepatitis-B or -C

4. Use of any MPN-associated myelofibrosis-directed therapy within 4 weeks prior to
study Day 1

5. Symptomatic congestive heart failure, unstable angina, or unstable cardiac arrhythmia

6. Uncontrolled hypertension. If hypertension is considered clinically stable, SBP must
be <150 mmHg and DBP must be <100 mmHg

7. Prior sotatercept

8. Major surgery within 4 weeks prior to Day 1

9. Severe allergic or anaphylactic reactions or hypersensitivity to recombinant proteins
or excipients in the investigational product

10. Any patient who is being treated for hypertension

Type of Study:

Interventional

Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Anemia Response

Outcome Description:

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.

Outcome Time Frame:

15 weeks

Safety Issue:

Yes

Principal Investigator

Srdan Verstovsek, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

UT MD Anderson Cancer Center

Authority:

United States: Food and Drug Administration

Study ID:

2012-0534

NCT ID:

NCT01712308

Start Date:

February 2013

Completion Date:

February 2017

Related Keywords:

  • Leukemia
  • Leukemia
  • Myeloproliferative Neoplasm
  • MPN
  • Associated Myelofibrosis
  • Anemia
  • Sotatercept
  • ACE-011
  • Primary Myelofibrosis
  • Anemia
  • Neoplasms
  • Leukemia
  • Myeloproliferative Disorders

Name

Location

UT MD Anderson Cancer CenterHouston, Texas  77030