It introduced the following changes: • Increasing the number of patients from 60 to 150; • Increasing the number of research centers from 7 to 20; • Increasing the patient observation duration from 4 to 4-6 months; • Lack of necessity to conduct all the completion procedures with the patients who have recalled their informed consent or refused to continue their participation in the trial; • New conditions for providing the study drug to the patients in terms of providing the centers with the drug at the expense of Novartis Pharma Company during the first 6 months of the therapy. Later if a medical institution isn’t able to provide the drug, the patients continue to be observed within the protocol; • Lowering the cases of returning of the unused drug and giving the next portion of the study drug to the patients once every 3 months instead of once a month; • Optional mode of Visits 7-11 to the patients who have stopped taking the drug after 6 months of the trial; • Control MRI only to the patients who have been taking the drug during the whole period of the investigation; • Optional mode of clinical and biochemical blood and urine analyses and ferrum metabolism variables on Visits 7-11 with the patients who stopped taking the drug after 6 months of the trial; • Optional mode of USC, ECG, EchoCG, an oculist and an otolaryngologist consultations, audiographics on the Final Visit to the patients who stopped taking the drug after 6 months of the trial.

The amendment introduced the following changes: • Change in the expected duration of the observation of the patients from 6 to 4 months after the last center was initiated. The whole period of patients selection was 8 months (from the time when the first center was initiated); • Addition to the inclusion criteria №2 the possibility to include patients with other rare forms of aplastic and hemolytic anemias after the Sponsor’s preliminary approval; • Specification to the inclusion criteria №9 concerning the date when the pregnancy test should be done. Before the amendment fertile women must have done the test 7 days prior to the inclusion in the trial. After the amendment they should do the test 48 hours prior to the inclusion in the trial. Postmenopausal women must have their last menstruation not later than a year ago. • Allowance to conduct EchoCG, X-ray examination of the thoracic cage organs, MRI, audiogram from 2 weeks during the screening examination to 6 months prior to the inclusion in the trial; • Specification to dates when the interim Visits must be conducted (1 month ± 7 days from the date of inclusion in the protocol); • Prolonging the final Visit to 2 weeks; • Lack of necessity to conduct the glucose-resistance test to the patients who suffer from verified diabetes; • Allowance to use the standard methods of the particular institution to calculate the creatinine clearance; • In order to get the accurate results of the pregnancy tests some specification was done concerning the age categories of women patients (teenagers aged ≥ 10 y.o. with fixed menstrual cycle) and the number of negative results (there must be at least 2 negative results).

The amendment introduced the following changes: • In accordance with the Sponsor’s decision the target number of patients must be decreased from 150 to 120; • Shortening of the expected duration of the patients selection from 4 to 2 months after the last center was initiated in order to make it 12 months total; • The patients should stay in the investigation and the Visits should continue even in case of the study drug has stopped being taken. Upon that the reasons for the participation completion weren’t alternated.