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Supporter terminated the study due to no active patients (secondary to travel restrictions due to COVID).
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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.
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:
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Sotatercept | Experimental | 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. |
|
| Sotatercept with prednisone boost | Experimental | 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). |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Sotatercept | Drug | 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | 9 months |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | 9 months |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Adrianna Vlachos, MD | Northwell Health/Feinstein Institute for Medical Research | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| North Shore- LIJ campus of The Feinstein Institute for Medical Research | Manhasset | New York | 11030 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 12351372 | Background | Abkowitz JL, Schaison G, Boulad F, Brown DL, Buchanan GR, Johnson CA, Murray JC, Sabo KM. Response of Diamond-Blackfan anemia to metoclopramide: evidence for a role for prolactin in erythropoiesis. Blood. 2002 Oct 15;100(8):2687-91. doi: 10.1182/blood.V100.8.2687. | |
| 773132 | Background | Diamond LK, Wang WC, Alter BP. Congenital hypoplastic anemia. Adv Pediatr. 1976;22:349-78. |
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| ID | Title | Description |
|---|---|---|
| FG000 | Sotatercept Cohort 1 | Cohort 1: Sotatercept 0.1 mg/kg given as a subcutaneous injection once every 4 weeks - to be completed in 3 patients without untoward events |
| FG001 | Sotatercept Cohort 2 | Cohort 2: Sotatercept 0.3 mg/kg given as a subcutaneous injection once every 4 weeks - to be completed in 3 patients without untoward events |
| FG002 | Sotatercept Cohort 3 | Cohort 3: Sotatercept 0.5 mg/kg given as a subcutaneous injection once every 4 weeks - to be completed in 3 patients without untoward events |
| FG003 | Sotatercept With Prednisone Boost Cohort 4a | Cohort 4a: Sotatercept 0.75 mg/kg given as a subcutaneous injection every 3 weeks with a three week prednisone boost of 1 mg/kg/day (max 60 mg) - to be completed in 3 patients without untoward events |
| FG004 | Sotatercept Cohort 4b | Cohort 4b: Sotatercept 0.75 mg/kg given as a subcutaneous injection every 3 weeks - to be completed in 3 patients without untoward events |
| FG005 | Sotatercept With Prednisone Boost Cohort 5a | Cohort 5a: Sotatercept 1 mg/kg as a subcutaneous injection every 3 weeks with a 3 week prednisone boost of 1 mg/kg/day (max 60 mg) to be completed in 3 patients without untoward events |
| FG006 | Sotatercept Cohort 5b | Cohort 5b: Sotatercept 1 mg/kg given as a subcutaneous injection every 3 weeks - to be completed in 3 patients without untoward events |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
There were no subjects in Cohort 5a at the time of the closure of the study.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Sotatercept Cohort 1 | Cohort 1: Sotatercept 0.1 mg/kg given as a subcutaneous injection once every 4 weeks - to be completed in 3 patients without untoward events |
| BG001 | Sotatercept Cohort 2 |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Three subjects of the original consented subjects did not meet the eligibility criteria and were screen failures. Three subjects met the criteria for enrollment but did not complete the study to be analyzed as the subjects were terminated early. |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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 | Only subjects who completed the trial are analyzed. | Posted | Count of Participants | Participants | 9 months |
|
Adverse events were collected for the time the subject was on study, a total of 9 months.
Cohort 5a had no subjects enrolled.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Sotatercept Cohort 1 | Cohort 1: Sotatercept 0.1 mg/kg given as a subcutaneous injection once every 4 weeks - to be completed in 3 patients without untoward events |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Hospitalization | Infections and infestations | Systematic Assessment | Cellulitis at injection site of non-study drug with episode of neutropenia. |
Not provided
Early termination of 3 subjects lead to small numbers of subjects analyzed, as does the early closure of the study.
Time to response was unable to be evaluated as no subjects attained response. Response duration was also not evaluated as no subjects responded.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Adrianna Vlachos, MD | Feinstein Institutes for Medical Research | 516-562-1506 | avlachos@northwell.edu |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Jul 3, 2018 | Oct 4, 2021 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D029503 | Anemia, Diamond-Blackfan |
| D029502 | Anemia, Hypoplastic, Congenital |
| C538442 | Aase Smith syndrome 2 |
| D012010 | Red-Cell Aplasia, Pure |
| D000080983 | Bone Marrow Failure Disorders |
| ID | Term |
|---|---|
| D000741 | Anemia, Aplastic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C542017 | ACE-011 |
| D011241 | Prednisone |
| ID | Term |
|---|---|
| D011244 | Pregnadienediols |
| D011245 | Pregnadienes |
| D011278 | Pregnanes |
| D013256 | Steroids |
| D000072473 |
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|
|
| Sotatercept with prednisone boost | Drug | 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). |
|
|
| 8826887 | Background | Ball SE, McGuckin CP, Jenkins G, Gordon-Smith EC. Diamond-Blackfan anaemia in the U.K.: analysis of 80 cases from a 20-year birth cohort. Br J Haematol. 1996 Sep;94(4):645-53. doi: 10.1046/j.1365-2141.1996.d01-1839.x. |
| 8286756 | Background | Bastion Y, Bordigoni P, Debre M, Girault D, Leblanc T, Tchernia G, Ball S, McGuckin C, Gordon-Smith EC, Bekassy A, et al. Sustained response after recombinant interleukin-3 in diamond blackfan anemia. Blood. 1994 Jan 15;83(2):617-8. No abstract available. |
| 17647292 | Background | Cmejla R, Cmejlova J, Handrkova H, Petrak J, Pospisilova D. Ribosomal protein S17 gene (RPS17) is mutated in Diamond-Blackfan anemia. Hum Mutat. 2007 Dec;28(12):1178-82. doi: 10.1002/humu.20608. |
| 20655265 | Background | Da Costa L, Moniz H, Simansour M, Tchernia G, Mohandas N, Leblanc T. Diamond-Blackfan anemia, ribosome and erythropoiesis. Transfus Clin Biol. 2010 Sep;17(3):112-9. doi: 10.1016/j.tracli.2010.06.001. Epub 2010 Jul 23. |
| 20116044 | Background | Doherty L, Sheen MR, Vlachos A, Choesmel V, O'Donohue MF, Clinton C, Schneider HE, Sieff CA, Newburger PE, Ball SE, Niewiadomska E, Matysiak M, Glader B, Arceci RJ, Farrar JE, Atsidaftos E, Lipton JM, Gleizes PE, Gazda HT. Ribosomal protein genes RPS10 and RPS26 are commonly mutated in Diamond-Blackfan anemia. Am J Hum Genet. 2010 Feb 12;86(2):222-8. doi: 10.1016/j.ajhg.2009.12.015. Epub 2010 Jan 28. |
| 9988267 | Background | Draptchinskaia N, Gustavsson P, Andersson B, Pettersson M, Willig TN, Dianzani I, Ball S, Tchernia G, Klar J, Matsson H, Tentler D, Mohandas N, Carlsson B, Dahl N. The gene encoding ribosomal protein S19 is mutated in Diamond-Blackfan anaemia. Nat Genet. 1999 Feb;21(2):169-75. doi: 10.1038/5951. |
| 18535205 | Background | Farrar JE, Nater M, Caywood E, McDevitt MA, Kowalski J, Takemoto CM, Talbot CC Jr, Meltzer P, Esposito D, Beggs AH, Schneider HE, Grabowska A, Ball SE, Niewiadomska E, Sieff CA, Vlachos A, Atsidaftos E, Ellis SR, Lipton JM, Gazda HT, Arceci RJ. Abnormalities of the large ribosomal subunit protein, Rpl35a, in Diamond-Blackfan anemia. Blood. 2008 Sep 1;112(5):1582-92. doi: 10.1182/blood-2008-02-140012. Epub 2008 Jun 5. |
| 20692036 | Background | Gale RP, Barosi G, Barbui T, Cervantes F, Dohner K, Dupriez B, Gupta V, Harrison C, Hoffman R, Kiladjian JJ, Mesa R, Mc Mullin MF, Passamonti F, Ribrag V, Roboz G, Saglio G, Vannucchi A, Verstovsek S. What are RBC-transfusion-dependence and -independence? Leuk Res. 2011 Jan;35(1):8-11. doi: 10.1016/j.leukres.2010.07.015. Epub 2010 Aug 7. |
| 17186470 | Background | Gazda HT, Grabowska A, Merida-Long LB, Latawiec E, Schneider HE, Lipton JM, Vlachos A, Atsidaftos E, Ball SE, Orfali KA, Niewiadomska E, Da Costa L, Tchernia G, Niemeyer C, Meerpohl JJ, Stahl J, Schratt G, Glader B, Backer K, Wong C, Nathan DG, Beggs AH, Sieff CA. Ribosomal protein S24 gene is mutated in Diamond-Blackfan anemia. Am J Hum Genet. 2006 Dec;79(6):1110-8. doi: 10.1086/510020. Epub 2006 Nov 2. |
| 19061985 | Background | Gazda HT, Sheen MR, Vlachos A, Choesmel V, O'Donohue MF, Schneider H, Darras N, Hasman C, Sieff CA, Newburger PE, Ball SE, Niewiadomska E, Matysiak M, Zaucha JM, Glader B, Niemeyer C, Meerpohl JJ, Atsidaftos E, Lipton JM, Gleizes PE, Beggs AH. Ribosomal protein L5 and L11 mutations are associated with cleft palate and abnormal thumbs in Diamond-Blackfan anemia patients. Am J Hum Genet. 2008 Dec;83(6):769-80. doi: 10.1016/j.ajhg.2008.11.004. |
| 6646173 | Background | Glader BE, Backer K, Diamond LK. Elevated erythrocyte adenosine deaminase activity in congenital hypoplastic anemia. N Engl J Med. 1983 Dec 15;309(24):1486-90. doi: 10.1056/NEJM198312153092404. |
| 9321770 | Background | Gustavsson P, Skeppner G, Johansson B, Berg T, Gordon L, Kreuger A, Dahl N. Diamond-Blackfan anaemia in a girl with a de novo balanced reciprocal X;19 translocation. J Med Genet. 1997 Sep;34(9):779-82. doi: 10.1136/jmg.34.9.779. |
| 17312003 | Background | Leblanc TM, Da Costa L, Marie I, Demolis P, Tchernia G. Metoclopramide treatment in DBA patients: no complete response in a French prospective study. Blood. 2007 Mar 1;109(5):2266-7. doi: 10.1182/blood-2006-08-039545. No abstract available. |
| 16317735 | Background | Lipton JM, Atsidaftos E, Zyskind I, Vlachos A. Improving clinical care and elucidating the pathophysiology of Diamond Blackfan anemia: an update from the Diamond Blackfan Anemia Registry. Pediatr Blood Cancer. 2006 May 1;46(5):558-64. doi: 10.1002/pbc.20642. |
| 3955239 | Background | Lipton JM, Kudisch M, Gross R, Nathan DG. Defective erythroid progenitor differentiation system in congenital hypoplastic (Diamond-Blackfan) anemia. Blood. 1986 Apr;67(4):962-8. |
| 16507776 | Background | Liu JM, Ellis SR. Ribosomes and marrow failure: coincidental association or molecular paradigm? Blood. 2006 Jun 15;107(12):4583-8. doi: 10.1182/blood-2005-12-4831. Epub 2006 Feb 28. |
| 3348775 | Background | Murata M, Onomichi K, Eto Y, Shibai H, Muramatsu M. Expression of erythroid differentiation factor (EDF) in Chinese hamster ovary cells. Biochem Biophys Res Commun. 1988 Feb 29;151(1):230-5. doi: 10.1016/0006-291x(88)90583-9. |
| 21435508 | Background | Narla A, Vlachos A, Nathan DG. Diamond Blackfan anemia treatment: past, present, and future. Semin Hematol. 2011 Apr;48(2):117-23. doi: 10.1053/j.seminhematol.2011.01.004. |
| 15543555 | Background | Nishiura H, Tanase S, Shibuya Y, Futa N, Sakamoto T, Higginbottom A, Monk P, Zwirner J, Yamamoto T. S19 ribosomal protein dimer augments metal-induced apoptosis in a mouse fibroblastic cell line by ligation of the C5a receptor. J Cell Biochem. 2005 Feb 15;94(3):540-53. doi: 10.1002/jcb.20318. |
| 15238419 | Background | Ohene-Abuakwa Y, Orfali KA, Marius C, Ball SE. Two-phase culture in Diamond Blackfan anemia: localization of erythroid defect. Blood. 2005 Jan 15;105(2):838-46. doi: 10.1182/blood-2004-03-1016. Epub 2004 Jul 6. |
| 17194739 | Background | Perrien DS, Akel NS, Edwards PK, Carver AA, Bendre MS, Swain FL, Skinner RA, Hogue WR, Nicks KM, Pierson TM, Suva LJ, Gaddy D. Inhibin A is an endocrine stimulator of bone mass and strength. Endocrinology. 2007 Apr;148(4):1654-65. doi: 10.1210/en.2006-0848. Epub 2006 Dec 28. |
| 19773262 | Background | Quarello P, Garelli E, Carando A, Brusco A, Calabrese R, Dufour C, Longoni D, Misuraca A, Vinti L, Aspesi A, Biondini L, Loreni F, Dianzani I, Ramenghi U. Diamond-Blackfan anemia: genotype-phenotype correlations in Italian patients with RPL5 and RPL11 mutations. Haematologica. 2010 Feb;95(2):206-13. doi: 10.3324/haematol.2009.011783. Epub 2009 Sep 22. |
| 4074357 | Background | Rivier J, Spiess J, McClintock R, Vaughan J, Vale W. Purification and partial characterization of inhibin from porcine follicular fluid. Biochem Biophys Res Commun. 1985 Nov 27;133(1):120-7. doi: 10.1016/0006-291x(85)91849-2. |
| 19049340 | Background | Ruckle J, Jacobs M, Kramer W, Pearsall AE, Kumar R, Underwood KW, Seehra J, Yang Y, Condon CH, Sherman ML. Single-dose, randomized, double-blind, placebo-controlled study of ACE-011 (ActRIIA-IgG1) in postmenopausal women. J Bone Miner Res. 2009 Apr;24(4):744-52. doi: 10.1359/jbmr.081208. |
| 18671700 | Background | Vlachos A, Ball S, Dahl N, Alter BP, Sheth S, Ramenghi U, Meerpohl J, Karlsson S, Liu JM, Leblanc T, Paley C, Kang EM, Leder EJ, Atsidaftos E, Shimamura A, Bessler M, Glader B, Lipton JM; Participants of Sixth Annual Daniella Maria Arturi International Consensus Conference. Diagnosing and treating Diamond Blackfan anaemia: results of an international clinical consensus conference. Br J Haematol. 2008 Sep;142(6):859-76. doi: 10.1111/j.1365-2141.2008.07269.x. Epub 2008 Jul 30. |
| 20651069 | Background | Vlachos A, Muir E. How I treat Diamond-Blackfan anemia. Blood. 2010 Nov 11;116(19):3715-23. doi: 10.1182/blood-2010-02-251090. Epub 2010 Jul 22. |
| 11313667 | Background | Vlachos A, Federman N, Reyes-Haley C, Abramson J, Lipton JM. Hematopoietic stem cell transplantation for Diamond Blackfan anemia: a report from the Diamond Blackfan Anemia Registry. Bone Marrow Transplant. 2001 Feb;27(4):381-6. doi: 10.1038/sj.bmt.1702784. |
| 11563775 | Background | Vlachos A, Klein GW, Lipton JM. The Diamond Blackfan Anemia Registry: tool for investigating the epidemiology and biology of Diamond-Blackfan anemia. J Pediatr Hematol Oncol. 2001 Aug-Sep;23(6):377-82. doi: 10.1097/00043426-200108000-00015. |
| Physician Decision |
|
Cohort 2: Sotatercept 0.3 mg/kg given as a subcutaneous injection once every 4 weeks - to be completed in 3 patients without untoward events
| BG002 | Sotatercept Cohort 3 | Cohort 3: Sotatercept 0.5 mg/kg given as a subcutaneous injection once every 4 weeks - to be completed in 3 patients without untoward events |
| BG003 | Sotatercept With Prednisone Boost Cohort 4a | Cohort 4a: Sotatercept 0.75 mg/kg given as a subcutaneous injection every 3 weeks with a three week prednisone boost of 1 mg/kg/day (max 60 mg) - to be completed in 3 patients without untoward events |
| BG004 | Sotatercept Cohort 4b | Cohort 4b: Sotatercept 0.75 mg/kg given as a subcutaneous injection every 3 weeks - to be completed in 3 patients without untoward events |
| BG005 | Sotatercept With Prednisone Boost Cohort 5a | Cohort 5a: Sotatercept 1 mg/kg as a subcutaneous injection every 3 weeks with a 3 week prednisone boost of 1 mg/kg/day (max 60 mg) to be completed in 3 patients without untoward events |
| BG006 | Sotatercept Cohort 5b | Cohort 5b: Sotatercept 1 mg/kg given as a subcutaneous injection every 3 weeks - to be completed in 3 patients without untoward events |
| BG007 | Total | Total of all reporting groups |
| Median |
| Full Range |
| years |
|
| Sex: Female, Male | Three subjects of the original consented subjects did not meet the eligibility criteria and were screen failures. Three subjects met the criteria for enrollment but did not complete the study to be analyzed as the subjects were terminated early. | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Three subjects of the original consented subjects did not meet the eligibility criteria and were screen failures. Three subjects met the criteria for enrollment but did not complete the study to be analyzed as the subjects were terminated early. | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| OG002 | Sotatercept Cohort 3 | Cohort 3: Sotatercept 0.5 mg/kg given as a subcutaneous injection once every 4 weeks - to be completed in 3 patients without untoward events |
| OG003 | Sotatercept With Prednisone Boost Cohort 4a | Cohort 4a: Sotatercept 0.75 mg/kg given as a subcutaneous injection every 3 weeks with a three week prednisone boost of 1 mg/kg/day (max 60 mg) - to be completed in 3 patients without untoward events |
| OG004 | Sotatercept Cohort 4b | Cohort 4b: Sotatercept 0.75 mg/kg given as a subcutaneous injection every 3 weeks - to be completed in 3 patients without untoward events |
| OG005 | Sotatercept With Prednisone Boost Cohort 5a | Cohort 5a: Sotatercept 1 mg/kg as a subcutaneous injection every 3 weeks with a 3 week prednisone boost of 1 mg/kg/day (max 60 mg) to be completed in 3 patients without untoward events |
| OG006 | Sotatercept Cohort 5b | Cohort 5b: Sotatercept 1 mg/kg given as a subcutaneous injection every 3 weeks - to be completed in 3 patients without untoward events |
|
|
| Secondary | 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 | Only subjects who completed the trial are analyzed. | Posted | Count of Participants | Participants | 9 months |
|
|
|
| 0 |
| 3 |
| 0 |
| 3 |
| 0 |
| 3 |
| EG001 | Sotatercept Cohort 2 | Cohort 2: Sotatercept 0.3 mg/kg given as a subcutaneous injection once every 4 weeks - to be completed in 3 patients without untoward events | 0 | 2 | 2 | 2 | 0 | 2 |
| EG002 | Sotatercept Cohort 3 | Cohort 3: Sotatercept 0.5 mg/kg given as a subcutaneous injection once every 4 weeks - to be completed in 3 patients without untoward events | 0 | 2 | 0 | 2 | 0 | 2 |
| EG003 | Sotatercept With Prednisone Boost Cohort 4a | Cohort 4a: Sotatercept 0.75 mg/kg given as a subcutaneous injection every 3 weeks with a three week prednisone boost of 1 mg/kg/day (max 60 mg) - to be completed in 3 patients without untoward events | 0 | 2 | 0 | 2 | 0 | 2 |
| EG004 | Sotatercept Cohort 4b | Cohort 4b: Sotatercept 0.75 mg/kg given as a subcutaneous injection every 3 weeks - to be completed in 3 patients without untoward events | 0 | 3 | 1 | 3 | 0 | 3 |
| EG005 | Sotatercept With Prednisone Boost Cohort 5a | Cohort 5a: Sotatercept 1 mg/kg as a subcutaneous injection every 3 weeks with a 3 week prednisone boost of 1 mg/kg/day (max 60 mg) to be completed in 3 patients without untoward events | 0 | 0 | 0 | 0 | 0 | 0 |
| EG006 | Sotatercept Cohort 5b | Cohort 5b: Sotatercept 1 mg/kg given as a subcutaneous injection every 3 weeks - to be completed in 3 patients without untoward events | 0 | 1 | 0 | 1 | 0 | 1 |
|
| Hospitalization | Endocrine disorders | Systematic Assessment | Shortness of breath and fatigue noted with hypothyroidism |
|
| Elevated liver function tests | Infections and infestations | Systematic Assessment | Subject with elevated LFTs found to have EBV infection . |
|
Not provided
Not provided
| D000080984 |
| Congenital Bone Marrow Failure Syndromes |
| D001855 | Bone Marrow Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| Fused-Ring Compounds |
| D011083 | Polycyclic Compounds |
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|