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CDK4/6 Inhibitor

Abemaciclib for Neurofibroma

Phase 1 & 2
Recruiting
Led By Andrea M Gross, M.D.
Research Sponsored by National Cancer Institute (NCI)
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
A neurofibroma or plexiform neurofibroma
Adequate performance scale (Lansky/Karnofsky >=70%)
Must not have
Patients with interstitial lung disease
Personal history of syncope of cardiovascular etiology, ventricular arrhythmia of pathological origin, or sudden cardiac arrest
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 2 years of treatment or until disease progression or unacceptable treatment-related toxicity
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing a drug, abemaciclib, to see if it can help treat atypical neurofibromas, which are tumors that arise from nerves and can cause serious medical problems. The drug is taken orally, twice daily, in 28-day cycles.

Who is the study for?
This trial is for individuals aged 12 or older with Neurofibromatosis Type I (NF1) and at least one inoperable tumor measuring over 3 cm. Participants must not be pregnant, agree to use effective contraception, have no severe allergies to abemaciclib, and should not require certain other medications that could interfere with the study drug.
What is being tested?
The trial is testing the safety and tolerability of a drug called abemaciclib for treating atypical neurofibromas associated with NF1. Patients will take this medication orally twice daily in cycles of 28 days for up to two years, possibly longer.
What are the potential side effects?
Potential side effects of abemaciclib may include digestive issues like nausea and vomiting, possible infections due to lowered immune response, liver problems indicated by blood tests, fatigue, and reactions related to heart function.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have a neurofibroma or plexiform neurofibroma.
Select...
I can take care of myself but may not be able to do active work.
Select...
I have been diagnosed with NF1 based on symptoms or a genetic test.
Select...
I have a parent, sibling, or child with NF1.
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I have been diagnosed with optic glioma.
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I have freckles under my arms or in my groin area.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I have interstitial lung disease.
Select...
I have a history of fainting due to heart issues, irregular heartbeats, or sudden cardiac arrest.
Select...
I need medication that strongly affects liver enzymes.
Select...
I cannot swallow pills.
Select...
I do not have any unmanaged ongoing illnesses.
Select...
I have severe nausea and vomiting that could interfere with taking medication.
Select...
I do not have any active infections.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 2 years of treatment or until disease progression or unacceptable treatment-related toxicity
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 2 years of treatment or until disease progression or unacceptable treatment-related toxicity for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
safety of abemaciclib in patients with NF1 and a measurable ANF
Secondary study objectives
Abemaciclib pharmacokinetics and pharmacodynamic
Effect of abemaciclib on ANF related pain and quality of life
Effect of abemaciclib on CDK4/6 target inhibition
+3 more

Side effects data

From 2018 Phase 2 trial • 132 Patients • NCT02102490
91%
Diarrhoea
67%
Nausea
48%
Fatigue
45%
Decreased appetite
35%
Vomiting
27%
Anaemia
26%
Abdominal pain
23%
Asthenia
23%
Neutrophil count decreased
21%
Cough
20%
Constipation
20%
Headache
19%
Arthralgia
18%
White blood cell count decreased
18%
Neutropenia
15%
Alopecia
14%
Dry mouth
14%
Platelet count decreased
14%
Weight decreased
14%
Dysgeusia
13%
Dyspnoea
12%
Abdominal pain upper
12%
Back pain
12%
Dizziness
11%
Oedema peripheral
11%
Dyspepsia
11%
Pyrexia
11%
Blood creatinine increased
10%
Pain
9%
Stomatitis
9%
Aspartate aminotransferase increased
8%
Thrombocytopenia
8%
Lacrimation increased
8%
Dehydration
8%
Dry skin
8%
Pruritus
8%
Alanine aminotransferase increased
7%
Flatulence
7%
Upper respiratory tract infection
7%
Urinary tract infection
7%
Hypokalaemia
6%
Chills
6%
Musculoskeletal chest pain
6%
Musculoskeletal pain
6%
Anxiety
5%
Gastrooesophageal reflux disease
5%
Rash
5%
Myalgia
2%
Cellulitis
2%
Pleural effusion
1%
Atypical pneumonia
1%
Gastroenteritis viral
1%
Lung infection
1%
Sepsis
1%
Fall
1%
Hip fracture
1%
Pneumonitis
1%
Pneumothorax
1%
Febrile neutropenia
1%
Respiratory tract infection
1%
Haematotoxicity
1%
Sinus bradycardia
1%
Tachycardia
1%
Large intestinal obstruction
1%
Pancreatic enzyme abnormality
1%
Pancreatitis
1%
Varices oesophageal
1%
Electrocardiogram abnormal
1%
Liver function test abnormal
1%
Renal function test abnormal
1%
Bone pain
1%
Muscular weakness
1%
Acute kidney injury
1%
Pulmonary embolism
1%
Arterial thrombosis
1%
Epilepsy
100%
80%
60%
40%
20%
0%
Study treatment Arm
Abemaciclib

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups
Experimental Treatment
Group I: 2/ Phase II Objective Response RateExperimental Treatment1 Intervention
Abemaciclib orally twice daily at the RP2D
Group II: 1/ Phase I Dose EscalationExperimental Treatment1 Intervention
Abemaciclib orally twice daily at escalating doses to determine the MTD/RP2D
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Abemaciclib
2019
Completed Phase 2
~1890

Find a Location

Who is running the clinical trial?

National Cancer Institute (NCI)Lead Sponsor
13,928 Previous Clinical Trials
41,017,983 Total Patients Enrolled
3 Trials studying Neurofibromatosis
290 Patients Enrolled for Neurofibromatosis
Andrea M Gross, M.D.Principal InvestigatorNational Cancer Institute (NCI)
1 Previous Clinical Trials
52 Total Patients Enrolled

Media Library

Abemaciclib (CDK4/6 Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT04750928 — Phase 1 & 2
Neurofibromatosis Research Study Groups: 1/ Phase I Dose Escalation, 2/ Phase II Objective Response Rate
Neurofibromatosis Clinical Trial 2023: Abemaciclib Highlights & Side Effects. Trial Name: NCT04750928 — Phase 1 & 2
Abemaciclib (CDK4/6 Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04750928 — Phase 1 & 2
~32 spots leftby Dec 2028
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