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New treatment option for Neurofibromatosis 1
Official title Cyclin-Dependent Kinase (CDK)4/6 Inhibitor Abemaciclib for Neurofibromatosis Type I (NF1) Related Atypical Neurofibromas
ClinicalTrials.gov ID: NCT04750928
What this study is testing
What is Abemaciclib?
Abemaciclib is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for neurofibromatosis 1.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- Background: NF1 is a genetic disease that causes tumors called atypical neurofibromas. These tumors, which arise from nerves, can cause serious medical problems.
- Phase 2: a mid-size study of how well it works
- Time commitment: about 2 years
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 12 and older
You may be able to join if
- All Participants
- Participants must have a clinical diagnosis of NF1, i.e., participants must have at least two of the diagnostic criteria for NF1 or a confirmed NF1...
- Six or more caf(SqrRoot)(Copyright)-au-lait macules (\>= 0.5cm in prepubertal participants or \>= 1.5 cm in post pubertal participants)
- Freckling in axilla or groin
- A neurofibroma or plexiform neurofibroma
You likely can't join if
- Pregnant women, or women who intend to become pregnant during the study, are excluded from this study because of the teratogenic effects of...
- May not have a NF1-related tumor such as optic pathway glioma or malignant peripheral nerve sheath tumor, which requires treatment with chemotherapy...
- Serious preexisting medical condition(s) that would preclude participation in this study (for example, interstitial lung disease, severe dyspnea at...
- Uncontrolled intercurrent illness including, but not limited to, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia...
- Personal history of any of the following conditions: syncope of cardiovascular etiology, ventricular arrhythmia of pathological origin (including...
- Active bacterial infection (requiring intravenous [IV] antibiotics at time of initiating study treatment), fungal infection, or detectable viral...
See the full eligibility criteria
Who can join
- All Participants
- Participants must have a clinical diagnosis of NF1, i.e., participants must have at least two of the diagnostic criteria for NF1 or a confirmed NF1 mutation from a CLIA-certified laboratory:
- Six or more caf(SqrRoot)(Copyright)-au-lait macules (\>= 0.5cm in prepubertal participants or \>= 1.5 cm in post pubertal participants)
- Freckling in axilla or groin
- A neurofibroma or plexiform neurofibroma
- Optic glioma
- Two or more Lisch nodules
- A distinctive bony lesion (dysplasia of the sphenoid bone or dysplasia or thinning of long bone cortex)
- A first-degree relative with NF1
- Measurable disease: Participants must have at least one measurable ANF defined as a lesion of at least 3 centimeters (cm) measured in one dimension. Measurability and suitability for volumetric MRI analysis of the...
- Prior Therapies:
- Since there is no standard effective chemotherapy for patients with NF1 and ANF, participants may be treated on this trial without having received prior medical therapy directed at their ANF.
- Investigational agents/biologic therapies (not chemotherapy): Participants who have received previous investigational agents or biologic therapies are eligible for enrollment. At least 30 days or 5 half-lives must have...
- Chemotherapy agents: Participants who received chemotherapy must have recovered (CTCAE Grade \<=1) from the acute effects of chemotherapy except for residual alopecia or Grade 2 peripheral neuropathy prior to...
- Participants who received adjuvant radiotherapy must have completed and fully recovered from the acute effects of radiotherapy. Participants who have received previous radiation therapy are eligible for enrollment. At...
- At least 28 days must have elapsed since any major surgeries, with evidence of good wound healing. Minimally invasive biopsies and central line placements are not considered major surgeries.
- Participants who have received prior treatment with abemaciclib or another specific CDK4/6 inhibitor are not eligible for enrollment
- Adequate performance scale (Lansky/Karnofsky \>=70%).
- Adequate organ function as defined below: --Hematologic Function: Participants must have an absolute neutrophil count \>=1500/microliter, hemoglobin \>=9 g/dL (transfusion independent, defined as not receiving blood...
- Hepatic Function: Participants must have bilirubin within 1.5 x the upper limit of normal for age, with the exception of those with Gilbert syndrome, and AST/ALT within \<= 3 x upper limit of normal.
- Renal Function: Participants must have a creatinine clearance or radioisotope GFR \>=60ml/min/1.73 m2 or a normal serum creatinine based on age, described in the table below.
- Age (years) is \>=12 and \<=15 then Maximum Serum Creatinine (mg/dL) = 1.2
- Age (years) \>15 Maximum Serum Creatinine (mg/dL) = 1.5
- Cardiac Function: Normal ejection fraction (ECHO or cardiac MRI) \>= 53% (or the institutional normal; if a range is given then the upper value of the range will be used); QTC or QTcF \<= 450 msec.
- Willingness to avoid grapefruit or grapefruit juice during abemaciclib administration
- Informed Consent: Ability of participant or Legally Authorized Representative (LAR) to understand and the willingness to sign a written informed consent document. All participants or their legal guardians (if the...
- Based on animal studies, the effects of abemaciclib can cause fetal harm. For these reasons, women of child-bearing potential and men must agree to use a highly effective contraceptive method during treatment and for at...
- Woman participants of childbearing potential (WOCBP) must have a negative serum pregnancy test within 7 days of the first dose of abemaciclib.
- A woman is considered to be of childbearing potential if she is postmenarcheal, has not reached a postmenopausal state (\>=12 continuous months of amenorrhea with no identified cause other than menopause), and has not...
- Contraceptive methods may include intrauterine devices (IUD), or barrier method, a spermicidal agent should be added as a double barrier protection; abstinence is also acceptable.
- Cases of pregnancy that occur during maternal exposures to abemaciclib should be reported. If a participant or spouse/partner is determined to be pregnant following abemaciclib initiation she must discontinue treatment...
- Phase 0 Participants
- Presence of \>= 1 measurable ANF (biopsy confirmed) for which surgical removal would not likely cause significant morbidity and is clinically indicated NOTES: Definition of ANF. In addition, there will not be a...
- Age \>= 18 years old
- Phase I/II Participants
- Presence of \>= 1 measurable ANF (biopsy confirmed) for whom surgical removal could cause significant clinical morbidity OR for which participant is unwilling to undergo surgical resection OR the presence of more than...
- Age \>= 12 years old (no maximum age) with associated age-related requirements as follows:
- Age \>= 12 years old with BSA \>= 0.71 m2 and able to swallow whole tablets
- Willingness of participants \>= 12 years old and \<18 years old to undergo pretreatment percutaneous biopsy of ANF if deemed feasible with minimal morbidity
- Willingness of participants \>= 18 years old to undergo pre-treatment and on-treatment percutaneous biopsy of ANF if deemed feasible with minimal morbidity NOTE: For participants of all ages with ANF that cannot be...
What rules you out
- Pregnant women, or women who intend to become pregnant during the study, are excluded from this study because of the teratogenic effects of abemaciclib. Because there is an unknown but potential risk for adverse events...
- May not have a NF1-related tumor such as optic pathway glioma or malignant peripheral nerve sheath tumor, which requires treatment with chemotherapy or surgery.
- Serious preexisting medical condition(s) that would preclude participation in this study (for example, interstitial lung disease, severe dyspnea at rest requiring oxygen therapy, history of major surgical resection...
- Uncontrolled intercurrent illness including, but not limited to, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, active bleeding diatheses or renal transplant, or psychiatric...
- Personal history of any of the following conditions: syncope of cardiovascular etiology, ventricular arrhythmia of pathological origin (including, but not limited to, ventricular tachycardia and ventricular...
- Active bacterial infection (requiring intravenous [IV] antibiotics at time of initiating study treatment), fungal infection, or detectable viral infection (such as known human immunodeficiency virus positivity or with...
- Participants with interstitial lung disease
- Requires treatment with strong CYP3A inhibitors or inducers
- Inability to swallow tablets, since tablets cannot be crushed or broken.
- Inability to undergo MRI and/or contraindication for MRI examinations following the MRI protocol. Prosthesis or orthopedic or dental braces that would interfere with volumetric analysis of target ANF on MRI.
- Refractory nausea and vomiting that would limit drug administration in the opinion of the Principal Investigator
- Known severe hypersensitivity to abemaciclib or any excipient of abemaciclib or history of allergic reactions attributed to compounds of similar chemical or biologic composition to abemaciclib
- Clinical judgment by the investigator that the participant should not participate in the study
The study team makes the final eligibility decision.
Where it's taking place
- Bethesda, Maryland, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 2 years per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 12 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Bethesda, Maryland, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.