Clinical trial NCT01190644: Study to Evaluate Effect of a Single Dose of Sotatercept (ACE-011) on Red Blood Cell Mass and Plasma Volume in Participants With Solid Tumors
Official title: A Phase 2, Open-Label, Pharmacodynamic Study to Evaluate the Effect of Sotatercept (ACE-011) on Red Blood Cell Mass and Plasma Volume in Subjects With Solid Tumors
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Solid Tumors
Keywords: Red Blood Cell Mass
Plasma Volume
Solid Tumors
Lead sponsor: Merck Sharp & Dohme LLC
Sponsor class: INDUSTRY
First posted: 2010-08-27
Enrollment: 5
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: SUPPORTIVE_CARE

Brief summary: What hematopoietic precursor compartments as well as hemoglobin subtypes are affected by dosing with sotatercept (ACE-011)? Based upon a similar prior study with Procrit, Celgene has determined that all of these goals could be obtained by an intense 10-patient sotatercept (ACE-011) pharmacodynamic study, completed by two well-known experts in the red cell production field.


Eligibility criteria: Inclusion Criteria:

* Men and women ≥18 years of age.
* Histologically confirmed diagnosis of a solid tumor malignancy documented by cytology or biopsy.
* Presence of metastatic disease.
* Hemoglobin value between ≥8.0 to \<11.0 g/dL (≥80 to \<110 g/L).
* ≥28 days must have elapsed (prior to pre-dose red blood cell mass / plasma volume test) since previous treatment with erythropoiesis-stimulating agent (including concurrent treatment with intravenous \[IV\] iron).
* ≥28 days must have elapsed (prior to Day 1) since the last red blood cell transfusion and receipt of ≤2 units of blood in the past 56 days (prior to Day 1).
* Eastern Cooperative Oncology Group (ECOG) Performance status of 0 - 1.

Exclusion Criteria:

At the time of screening, participants who have any grade ≥3 toxicity (according to the currently active minor version of NCI CTCAE v4.0) except for the following disease-related toxicities:

* Hematological events - anemia, thrombocytopenia, neutropenia
* Non-hematological events - nausea, vomiting, fatigue, muscle or bone/joint pain

Primary outcomes:
measure: Change From Baseline in Red Blood Cell Mass Following a Single Dose of Sotatercept
timeFrame: Baseline (Day 1, pre-dose) and one timepoint between Day 14 and Day 28
Secondary outcomes:
measure: Change From Baseline in Plasma Volume Following a Single Dose of Sotatercept
timeFrame: Baseline (Day 1, pre-dose) and one timepoint between Day 14 and Day 28
measure: Change From Baseline in Absolute Reticulocyte Count
timeFrame: Baseline (Day 1, pre-dose) and Day 211
measure: Change From Baseline in Erythropoietin Levels
timeFrame: Baseline (Day 1, pre-dose) and Day 211
measure: Change From Baseline in Hemoglobin Subtype A Following a Single Dose of Sotatercept
timeFrame: Baseline (Day 1, pre-dose) and Day 29
measure: Change From Baseline in Hemoglobin Subtype A2 Following a Single Dose of Sotatercept
timeFrame: Baseline (Day 1, pre-dose) and Day 29
measure: Change From Baseline in Hemoglobin Subtype C Following a Single Dose of Sotatercept
timeFrame: Baseline (Day 1, pre-dose) and Day 29
measure: Number of Participants Who Experienced One or More Adverse Events (AEs)
timeFrame: Up to approximately 7 months
Other outcomes:
Locations:
city: Baltimore
country: United States
facility: Saint Agnes Healthcare
state: Maryland
city: Baltimore
country: United States
facility: Weinberg Cancer Institution at Franklin Square
state: Maryland
city: Philadelphia
country: United States
facility: Pennsylvania Oncology
state: Pennsylvania
Study officials:
affiliation: Merck Sharp & Dohme LLC
name: Medical Director
role: STUDY_DIRECTOR
References:
citation: Cappellini MD, Porter J, Origa R, Forni GL, Voskaridou E, Galacteros F, Taher AT, Arlet JB, Ribeil JA, Garbowski M, Graziadei G, Brouzes C, Semeraro M, Laadem A, Miteva D, Zou J, Sung V, Zinger T, Attie KM, Hermine O. Sotatercept, a novel transforming growth factor beta ligand trap, improves anemia in beta-thalassemia: a phase II, open-label, dose-finding study. Haematologica. 2019 Mar;104(3):477-484. doi: 10.3324/haematol.2018.198887. Epub 2018 Oct 18.