A study evaluating Romiplostim in Non- Asian Adults with Severe Aplastic Anaemia
Research type
Research Study
Full title
Two Arm Bridging Study to Evaluate the Efficacy of Romiplostim in the Treatment of Non Asian Adult Severe Aplastic Anemia (SAA) Subjects who are Either Previously Untreated With Immunosuppressive Therapy (IST) or Refractory to IST
IRAS ID
1005707
Contact name
Matthew Rodaway
Contact email
Sponsor organisation
Amgen, Inc.
Eudract number
2022-001257-22
Clinicaltrials.gov Identifier
Research summary
We believe this study may be of importance to patients with severe aplastic anaemia (SAA) or very severe aplastic anaemia (VSAA). SAA is a rare and very serious blood disorder in which the bone marrow drastically decreases producing the cells which make up blood; red blood cells, white blood cells, and platelets. This study is being done to learn more about romiplostim (a molecule that activates blood cell receptors, resulting in an increased amount of blood cells) in Non-Asian patients with SAA, who are either previously untreated with immunosuppressive therapy (IST) (a type of medication that suppresses the body’s immune response and is used to treat this disease), or not responsive (or resistant) to IST. Romiplostim has previously been tested in the Asian aplastic anaemia population. Therefore this study will look to see if romiplostim can raise platelet count, red blood cell count, haemoglobin concentration, neutrophil (a type of white blood cell) count and whether it causes any side effects in Non-Asian participants.
This study is divided into two arms. Participants of both arms will receive romiplostim. Arm 1 is for participants who are previously untreated for SAA with IST and will be treated with romiplostim and IST (Horse or Rabbit antithymocyte globulin or Cyclosporine A) for 14 weeks. Arm 2 is for participants who are not responsive (or resistant) to IST. These participants will be treated with romiplostim for 14 weeks.
There is a Screening Period, followed by a 14-week Treatment Period and a 4-week Follow-up Period. At week 24 participants who completed all study assessments will undergo the end of study/safety follow-up examination and complete the study. Approximately 16 people are expected to take part in this study out of which approximately 4 will be from the UK.
REC name
North West - Greater Manchester South Research Ethics Committee
REC reference
22/NW/0231
Date of REC Opinion
12 Sep 2022
REC opinion
Further Information Favourable Opinion