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TPO Mimetic
Romiplostim for Bone Marrow Failure Disorders
Phase < 1
Waitlist Available
Led By Anjali A. Sharathkumar, MBBS, MD, MS
Research Sponsored by Anjali Sharathkumar
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Adequate organ function within 7 days of enrollment defined as: Creatinine: ≤ 2.0 mg/dL, Hepatic function: For arm A, elevation of liver enzymes is acceptable for patients with hepatitis induced SAA as long as patient does not have history of chronic liver problem. If necessary, liver biopsy will be performed. For Arm B, elevation of liver enzymes will be accepted as long as no chronic liver problem. Liver biopsy will be performed if necessary., Females of childbearing potential agree to use effective contraception during the study period and for 4 months after completion of therapy, Must be able to provide written and voluntary informed consent.
- aplastic anemia:
Timeline
Screening 3 weeks
Treatment Varies
Follow Up at 3 to 6 months after treatment initiation; within 4 to 6 weeks after discontinuation of treatment or any other time-point if deemed clinically indicated
Awards & highlights
Study Summary
This trial will study the safety and effectiveness of a drug called Romiplostim in children with bone marrow failure disorders.
Who is the study for?
This trial is for children and young adults (0 to 21 years) with various bone marrow failure disorders, including aplastic anemia and myelodysplastic syndrome. Participants must have specific blood count levels indicating severe or moderate conditions, be under the care of pediatric hematology/oncology providers, and have functioning major organs. Females able to bear children must agree to use contraception during the study.Check my eligibility
What is being tested?
The trial tests Romiplostim's safety and effectiveness in treating bone marrow failure in kids as a first-line therapy alongside standard treatments. It's an open-label pilot study where all participants know they're receiving Romiplostim.See study design
What are the potential side effects?
Romiplostim may cause side effects like headaches, dizziness, insomnia, muscle or joint pain, nausea or vomiting, shortness of breath, and increased risk of blood clots. Reactions at the injection site are also possible.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have aplastic anemia.
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My child is currently under the care of a pediatric cancer specialist.
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I have severe Aplastic anemia with very low blood cell counts.
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I have been diagnosed with a specific type of anemia or bone marrow failure.
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I have moderate aplastic anemia with specific low blood counts.
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My child has refractory cytopenia without specific genetic risks for leukemia.
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I am 21 years old or younger.
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I have very low blood cell counts due to medication or infection.
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I am 21 years old or younger.
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I have been diagnosed with inherited bone marrow failure without a chromosomal disorder.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ at 3 to 6 months after treatment initiation; within 4 to 6 weeks after discontinuation of treatment or any other time-point if deemed clinically indicated
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~at 3 to 6 months after treatment initiation; within 4 to 6 weeks after discontinuation of treatment or any other time-point if deemed clinically indicated
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Occurrence of treatment-related adverse events (AEs) according to NCI CTCAE v5.0
To estimate preliminary efficacy of Romiplostim as measured by improvement in hematopoiesis
Secondary outcome measures
To assess development of bone marrow myelofibrosis
To assess longitudinal changes in blood counts
To assess the incidence of bleeding (including muco-cutaneous bleeding)
+4 moreSide effects data
From 2010 Phase 3 trial • 313 Patients • NCT0011668850%
Upper respiratory tract infection
45%
Cough
45%
Headache
45%
Pyrexia
45%
Petechiae
40%
Contusion
35%
Fatigue
35%
Vomiting
30%
Oropharyngeal pain
30%
Nasal congestion
25%
Gingival bleeding
25%
Epistaxis
25%
Rhinorrhoea
25%
Rash
25%
Nasopharyngitis
25%
Arthralgia
20%
Abdominal pain upper
20%
Pain
20%
Mouth haemorrhage
20%
Nausea
15%
Abdominal pain
15%
Excoriation
15%
Myalgia
15%
Viral upper respiratory tract infection
10%
Ear infection
10%
Chills
10%
Idiopathic thrombocytopenic purpura
10%
Pain in extremity
10%
Dyspnoea
10%
Diarrhoea
10%
Joint sprain
10%
Gastroenteritis
10%
Scab
10%
Dizziness
10%
Mouth ulceration
10%
Viral infection
10%
Animal bite
10%
Arthropod bite
10%
Procedural pain
5%
Constipation
5%
Migraine
5%
Anaemia
5%
Muscle spasms
5%
Haematoma
5%
Musculoskeletal pain
5%
Abdominal discomfort
5%
Pharyngitis streptococcal
5%
Seasonal allergy
5%
Influenza
5%
Bronchitis
5%
Ecchymosis
5%
Skin lesion
5%
Toothache
5%
Chest pain
5%
Sinusitis
5%
Back pain
100%
80%
60%
40%
20%
0%
Study treatment Arm
Romiplostim in Pediatric Population
Romiplostim in Adults
Trial Design
2Treatment groups
Experimental Treatment
Group I: Arm BExperimental Treatment1 Intervention
Arm B will include children with chemo and or radiotherapy induced thrombocytopenia/cytopenia and children undergoing stem cell transplantation (SCT).
Arm B: Starting dose 2 microgram/kg/dose per week with increments at 1 microgram/kg/dose (Maximum: 10 micrograms/kg/dose) depending on the laboratory response.
Group II: Arm AExperimental Treatment1 Intervention
Arm A will include acquired bone marrow failure (BMF) disorders including aplastic anemia, refractory cytopenia of childhood/Myelodysplastic Syndrome(MDS) without monosomy 7 and 5q deletion abnormalities, toxin induced myelosuppression due to infection and inherited cytopenia with or without involvement of other cell lines who are transfusion dependent and or showing progression to bone marrow failure.
Arm A: Start at 5 microgram/kg/dose per week along with standard of care and escalate with 2.5 microgram/kg/dose increments (per week at physician's discretion depending on the clinical and laboratory response) (Maximum: 20 microgram/kg/dose) based on response for at least 24 weeks or until hematopoietic response is seen, whichever comes first. If patient shows response, therapy will be continued for a total of 52 weeks.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Romiplostim
FDA approved
Find a Location
Who is running the clinical trial?
Anjali SharathkumarLead Sponsor
AmgenIndustry Sponsor
1,370 Previous Clinical Trials
1,377,673 Total Patients Enrolled
Anjali A. Sharathkumar, MBBS, MD, MSPrincipal InvestigatorUniversity of Iowa
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- 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.
Research Study Groups:
This trial has the following groups:- Group 1: Arm A
- Group 2: Arm B
Awards:
This trial has 2 awards, including:- 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.
Timeline:
This trial has the following timeline:- 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
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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."
Answered by AI
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."
Answered by AI
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."
Answered by AI
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."
Answered by AI
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."
Answered by AI
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."
Answered by AI
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