Your session is about to expire
← Back to Search
JAK Inhibitor
Ruxolitinib for Bone Marrow Failure
Phase 1 & 2
Recruiting
Led By Emma M Groarke, M.D.
Research Sponsored by National Heart, Lung, and Blood Institute (NHLBI)
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 3, 12 months, and yearly thereafter
Awards & highlights
Study Summary
This trial tests a drug to treat different types of immune bone marrow failure in adults. Participants will take the drug for up to 6 months & have clinic visits & tests. Those who improve may enter an extension phase.
Who is the study for?
Adults over 18 with immune bone marrow failure, willing to consent and use contraception if of childbearing potential. Excluded are those with certain genetic disorders, recent cancer treatments, uncontrolled infections or severe organ dysfunction, current pregnancy or breastfeeding, inability to swallow pills, or a history of acute thrombosis within the last 6 months.Check my eligibility
What is being tested?
The trial is testing Ruxolitinib tablets taken twice daily for up to six months in patients with various types of immune bone marrow failure. It includes regular blood tests and clinic visits at specified intervals including after treatment completion to monitor effects.See study design
What are the potential side effects?
Potential side effects include risks associated with long-term immunosuppression such as increased susceptibility to infections. Specific side effects related to Ruxolitinib may also occur but are not detailed in the provided information.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 3, 12 months, and yearly thereafter
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~3, 12 months, and yearly thereafter
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Overall Response Rate. Patients who develop a Complete response at 3 months and stop the drug will also be deemed responders even if they subsequently relapse.
The number of patients who complete a full course of ruxolitinib without cessation is required by hematologic toxicity.
Secondary outcome measures
Depth of response
Hematological response
Hematological response of relapse subjects that re-start treatment
+5 moreSide effects data
From 2020 Phase 3 trial • 149 Patients • NCT0203803633%
Anaemia
19%
Hypertension
17%
Nasopharyngitis
16%
Weight increased
14%
Herpes zoster
14%
Constipation
14%
Abdominal pain
14%
Headache
12%
Pruritus
12%
Back pain
12%
Epistaxis
12%
Pyrexia
12%
Dizziness
10%
Asthenia
10%
Fatigue
10%
Cough
10%
Oedema peripheral
10%
Arthralgia
9%
Thrombocytosis
9%
Upper respiratory tract infection
9%
Hypercholesterolaemia
7%
Dyslipidaemia
7%
Pain in extremity
7%
Haematoma
7%
Abdominal discomfort
7%
Diarrhoea
7%
Dyspepsia
7%
Vomiting
7%
Blood lactate dehydrogenase increased
7%
Memory impairment
7%
Dyspnoea
5%
Tinnitus
5%
Osteoarthritis
5%
Leukocytosis
5%
Thrombocytopenia
5%
Flatulence
5%
Nausea
5%
Sinusitis
5%
Basal cell carcinoma
5%
Neuropathy peripheral
5%
Hyperuricaemia
3%
Paraesthesia
3%
Cystitis
3%
Bronchitis
3%
Blood creatine phosphokinase increased
3%
Skin ulcer
3%
Abdominal pain upper
3%
Pulmonary embolism
3%
Pneumonia
3%
Influenza
3%
Myalgia
3%
Urinary tract infection
3%
Depression
2%
Localised infection
2%
Intervertebral disc protrusion
2%
Urethral stenosis
2%
Night sweats
2%
Acute pulmonary oedema
2%
Vertigo
2%
Peripheral artery thrombosis
2%
Ureterolithiasis
2%
Pericardial effusion
2%
Acute myocardial infarction
2%
Syncope
2%
Gastrooesophageal reflux disease
2%
General physical health deterioration
2%
Atrial fibrillation
2%
Cardiac disorder
2%
Mitral valve incompetence
2%
Vertigo positional
2%
Retinal artery occlusion
2%
Visual acuity reduced
2%
Gastrointestinal haemorrhage
2%
Oesophageal varices haemorrhage
2%
Lower respiratory tract infection
2%
Pyelonephritis
2%
Respiratory tract infection
2%
Sepsis
2%
Tendon rupture
2%
Ulna fracture
2%
Weight decreased
2%
Decreased appetite
2%
Hyponatraemia
2%
Blast cell crisis
2%
Bone marrow tumour cell infiltration
2%
Lung adenocarcinoma
2%
Metastases to spine
2%
Myelofibrosis
2%
Prostatic adenoma
2%
Squamous cell carcinoma of skin
2%
Nephrolithiasis
2%
Gamma-glutamyltransferase increased
2%
Haematocrit increased
2%
Musculoskeletal pain
2%
Ischaemic stroke
2%
Diabetes mellitus
100%
80%
60%
40%
20%
0%
Study treatment Arm
All Crossover Patients
Best Available Therapy
Ruxolitinib
Trial Design
5Treatment groups
Experimental Treatment
Group I: Subjects with hypoplastic MDSExperimental Treatment1 Intervention
Subjects are defined as patients with a diagnosis of hMDS clinically confirmed by a licensed physician or an advanced practitioner who meets the inclusion and exclusion criteria and can provide informed consent.
Group II: Subjects with TLGLExperimental Treatment1 Intervention
Subjects are defined as patients with a diagnosis of TLGL clinically confirmed by a licensed physician or advanced practitioner who meets the inclusion and exclusion criteria and can provide informed consent.
Group III: Subjects with SAAExperimental Treatment1 Intervention
Subjects are defined as patients with a diagnosis of SAA clinically confirmed by a licensed physician oran advanced practitioner who meets the inclusion andexclusion criteria and can provide informed consent.
Group IV: Subjects with PRCAExperimental Treatment1 Intervention
Subjects are defined as patients with a diagnosis of PRCA clinically confirmed by a licensed physician or advanced practitioners who meet the inclusion and exclusion criteria and can provide informed consent.
Group V: Subjects with MAAExperimental Treatment1 Intervention
Subjects are defined as patients with a diagnosis of MAA clinically confirmed by a licensed physician or an advanced practitioner who meets the inclusion andexclusion criteria and can provide informed consent.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Ruxolitinib
2018
Completed Phase 3
~1140
Find a Location
Who is running the clinical trial?
National Heart, Lung, and Blood Institute (NHLBI)Lead Sponsor
3,840 Previous Clinical Trials
47,851,979 Total Patients Enrolled
11 Trials studying Aplastic Anemia
922 Patients Enrolled for Aplastic Anemia
Emma M Groarke, M.D.Principal InvestigatorNational Heart, Lung, and Blood Institute (NHLBI)
5 Previous Clinical Trials
1,557 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I am currently receiving treatment for cancer.I have recently been treated with specific medications.I do not have any severe illnesses that prevent me from daily activities.I have a specific blood disorder.I do not have untreated or poorly controlled HIV, Hepatitis B, or C.I am not pregnant and willing to use contraception.I cannot swallow pills.I am able to understand and give informed consent.My kidneys do not work well.I have had a brain infection called PML.I have an active skin cancer that is not melanoma.I am 18 years old or older.I have T-LGL leukemia with low blood counts.My condition is hypoplastic MDS.I have been diagnosed with immune-related bone marrow failure.My condition is considered high-risk based on specific genetic tests.I have had a recent blood clot.I am of any gender.I have a high PNH clone count and am not on blood thinners.I have been diagnosed with severe aplastic anemia.I have or might have Fanconi anemia or a similar bone marrow failure condition.I have been diagnosed with moderate aplastic anemia.My condition is a type of bone marrow failure affecting one cell line.
Research Study Groups:
This trial has the following groups:- Group 1: Subjects with hypoplastic MDS
- Group 2: Subjects with SAA
- Group 3: Subjects with MAA
- Group 4: Subjects with TLGL
- Group 5: Subjects with PRCA
Awards:
This trial has 1 awards, including:- 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.
Does this study accept participants aged over thirty-five?
"Eligible applicants for this research must be in the age bracket of 12 and 99 years."
Answered by AI
Am I eligible to partake in this experimentation?
"Individuals with aplastic anemia who are between the ages of 12 and 99 may be eligible for this medical study. The team is aiming to recruit 145 patients in total."
Answered by AI
Are there any enrollments available for this medical trial currently?
"According to clinicaltrials.gov, this trial has concluded its recruitment phase and is thus no longer accepting participants; however, 84 other trials are actively enrolling patients at the present time. This trial was posted on October 3rd 2023 and last updated August 17th of the same year."
Answered by AI
Recent research and studies
Other Trials to Consider
Popular Categories
Share this study with friends
Copy Link
Messenger