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Romiplostim for Bone Marrow Failure Disorders
Study Summary
This trial will study the safety and effectiveness of a drug called Romiplostim in children with bone marrow failure disorders.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowTimeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Side effects data
From 2010 Phase 3 trial • 313 Patients • NCT00116688Trial Design
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Who is running the clinical trial?
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- My cancer has specific genetic features known to increase cancer risk.I have aplastic anemia.My condition is MDS transforming into a more aggressive form.I do not have severe health issues that would stop me from tolerating the treatment.My child is currently under the care of a pediatric cancer specialist.I am having a stem cell transplant and have low platelets, needing transfusions.My child has low platelet counts due to cancer treatment, causing treatment delays.My liver is working properly.My liver enzymes are high due to hepatitis-induced severe aplastic anemia, but I don't have chronic liver issues.I have a condition like Fanconi anemia that increases my cancer risk.I have severe Aplastic anemia with very low blood cell counts.I have a long-term liver condition, like fibrosis or cirrhosis.I have been diagnosed with a specific type of anemia or bone marrow failure.I have moderate aplastic anemia with specific low blood counts.My child has refractory cytopenia without specific genetic risks for leukemia.I am 21 years old or younger.I have very low blood cell counts due to medication or infection.My organs are working well.My liver enzymes are high, but I don't have a chronic liver condition.I have a condition that increases my risk of blood clots.I am over 21 years old or my pregnancy is less than 32 weeks along.I am 21 years old or younger.I am diagnosed with a condition listed for Arm B of the study.I am not pregnant or nursing.I do not have any ongoing serious infections like sepsis or hepatitis.I agree to use effective birth control during and for 4 months after the study.I have been diagnosed with inherited bone marrow failure without a chromosomal disorder.
- Group 1: Arm A
- Group 2: Arm B
- All Individual Drugs Already Approved - Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
Is the age restriction for this clinical trial in excess of forty years old?
"This clinical trial stipulates that only those aged between 0 and 21 may enroll in the program."
What is the enrollment capacity for this trial?
"Affirmative. Clinicaltrials.gov states that this medical trial is currently recruiting 25 individuals from a single location, with the original post date being August 18th 2020 and most recent update on September 2nd 2022."
Are there currently opportunities to enroll in this trial?
"According to clinicaltrials.gov, this study is in the process of recruitment and was initially advertised on August 18th 2020 with a subsequent update done on September 2nd 2021."
What are the overarching goals of this investigation?
"The primary focus of this medical study, which will last for 24 weeks, is to monitor and document any treatment-related adverse events (AEs) according to NCI CTCAE v5.0. Secondary objectives include assessing the incidence of bleeding as defined by the International Society of Haemostasis and Thrombosis Bleeding Assessment Tool, measuring occurrences of neutropenic fever, and tracking time from Romiplostim initiation to hematological response."
Has this clinical trial been conducted previously, or is it the inaugural one?
"Since 2014, researchers have been studying the effects of Romiplostim. Initially sponsored by Amgen in 2014, the first trial involved 60 participants and led to its Phase 2 drug approval. Currently there are 9 ongoing studies across 23 countries and 62 cities."
What are the prerequisites for taking part in this clinical research?
"In order to be eligible for this trial, patient must have bone marrow failure and are between 0-21 years of age. The maximum number of recruits is 25 individuals."
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