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Proteasome Inhibitor

Marizomib + Panobinostat for DIPG (DIPG Trial)

Phase 1
Waitlist Available
Led By Katherine Warren, MD
Research Sponsored by Dana-Farber Cancer Institute
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Patients must have DIPG, as defined in the study protocol
Patients may enroll at any point in their disease course provided they have received standard radiation therapy and meet all other eligibility requirements
Timeline
Screening 3 weeks
Treatment Varies
Follow Up time from enrollment until death due to any cause up to 60 months
Awards & highlights

DIPG Trial Summary

This trial is looking at the safety and effectiveness of these drugs in children with a type of brain cancer called diffuse intrinsic pontine glioma (DIPG).

Who is the study for?
This trial is for children with a brain cancer called diffuse intrinsic pontine glioma (DIPG). They must be able to swallow pills, have finished standard radiation therapy, and recovered from any previous treatments. Participants need proper organ function and no ongoing brain hemorrhage. They should not have severe GI issues or be on other cancer drugs.Check my eligibility
What is being tested?
The safety and initial effectiveness of two drugs, Marizomib and Panobinostat, are being tested in pediatric patients with DIPG. This phase I study aims to determine how well these children tolerate the drug combination and its potential benefits.See study design
What are the potential side effects?
While specific side effects aren't listed here, common ones for cancer treatments like Marizomib and Panobinostat may include nausea, fatigue, blood count changes leading to infection risk increase or bleeding problems, liver function changes, diarrhea or constipation.

DIPG Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have been diagnosed with DIPG.
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I have had standard radiation therapy for my condition.
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I am under 22 years old.
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I can swallow pills without any issues.
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I can do most activities but may need help, based on a recent health score.
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I have recovered from side effects of my previous cancer treatments.
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I finished my radiation therapy at least 2 weeks ago.
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I received my last strong chemotherapy dose more than 3 weeks ago, or 6 weeks if it was a specific type.
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I have no ongoing side effects from previous treatments and it's been over a week since my last dose.
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It's been over 4 weeks since my last CED procedure, I don't have a permanent CED device, and I don't have current brain bleeding.
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It's been over 4 weeks since my last artery treatment, and I don't have any current bleeding in my tumor.
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My organs and bone marrow are working well.

DIPG Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~time from enrollment until death due to any cause up to 60 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and time from enrollment until death due to any cause up to 60 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Area under the plasma concentration time curve (AUC)
Dose-limiting toxicity (DLT) of marizomib as a single agent
Dose-limiting toxicity (DLT) of marizomib in combination with panobinostat
+6 more
Secondary outcome measures
Clinical Benefit Score
Overall survival (OS)
Radiographic progression-free survival (rPFS)

Side effects data

From 2023 Phase 3 trial • 749 Patients • NCT03345095
67%
Nausea
62%
Fatigue
53%
Headache
52%
Vomiting
41%
Constipation
38%
Hallucination
29%
Insomnia
24%
Gait Disturbance
24%
Alopecia
23%
Dizziness
23%
Ataxia
21%
Decreased Appetite
20%
Confusional State
16%
Aphasia
16%
Weight Decreased
15%
Diarrhoea
15%
Dysarthria
14%
Balance Disorder
11%
Anxiety
11%
Pyrexia
11%
Seizure
11%
Asthenia
11%
Fall
10%
Cough
10%
Muscular Weakness
9%
Thrombocytopenia
8%
Hemiparesis
8%
Vertigo
8%
Platelet Count Decreased
7%
Hallucination, Visual
7%
Diplopia
6%
Memory Impairment
6%
Back Pain
6%
Tremor
6%
Disturbance In Attention
6%
Somnolence
6%
Vision Blurred
6%
Pruritus
6%
Oedema Peripheral
6%
Alanine Aminotransferase Increased
6%
Lymphopenia
6%
Arthralgia
6%
Cognitive Disorder
6%
Agitation
6%
Depression
6%
Radiation Skin Injury
6%
Rash
5%
Nightmare
5%
Brain Oedema
5%
Dysgeusia
5%
Hypertension
5%
Dyspepsia
5%
Lymphocyte Count Decreased
5%
Anaemia
4%
Myalgia
4%
Delirium
4%
Injection Site Pain
4%
Dry Mouth
4%
Paraesthesia
4%
Influenza Like Illness
4%
Neutropenia
4%
Infusion Related Reaction
4%
Neck Pain
4%
Urinary Tract Infection
4%
Hyperglycaemia
4%
Tinnitus
4%
Abdominal Pain Upper
4%
Neutrophil Count Decreased
4%
Weight Increased
3%
Urinary Incontinence
3%
Speech Disorder
3%
Dry Skin
3%
Lacrimation Increased
3%
Gamma-Glutamyltransferase Increased
3%
Sleep Disorder
3%
Injection Site Reaction
3%
Lethargy
3%
Abdominal Pain
3%
Malignant Neoplasm Progression
3%
Leukopenia
3%
Face Oedema
3%
Infusion Site Pain
3%
Aspartate Aminotransferase Increased
3%
Hypersomnia
3%
Amnesia
3%
Dysphonia
3%
Bronchitis
3%
Dermatitis
3%
Herpes Zoster
3%
Erythema
3%
Deep Vein Thrombosis
3%
Dyspnoea
3%
Blood Creatinine Increased
3%
Dehydration
3%
Hypoaesthesia
2%
Irritability
2%
Nervous System Disorder
2%
Nystagmus
2%
Pain
2%
Malaise
2%
Pneumonia
2%
Neurological Decompensation
2%
Muscle Spasms
2%
Hypotension
2%
Hot Flush
2%
Epilepsy
2%
Personality Change
2%
Rash Maculo-Papular
2%
Thrombophlebitis Superficial
2%
Oral Candidiasis
2%
Palpitations
2%
Chills
2%
Photophobia
2%
General Physical Health Deterioration
2%
Gastrooesophageal Reflux Disease
2%
Hypokalaemia
2%
Pain In Extremity
2%
Pain In Jaw
2%
Oropharyngeal Pain
2%
Candida Infection
2%
Abnormal Dreams
2%
Hypoacusis
2%
Photopsia
2%
Visual Impairment
2%
Ear Pain
2%
Depressed Mood
2%
Depressed Level Of Consciousness
2%
Cushingoid
2%
Pulmonary Embolism
2%
Nasopharyngitis
2%
Dysphagia
2%
Upper Respiratory Tract Infection
2%
Motor Dysfunction
2%
Phlebitis
2%
Thrombophlebitis
2%
Blood Bilirubin Increased
2%
White Blood Cell Count Decreased
1%
Bradyphrenia
1%
Joint Swelling
1%
Proteinuria
1%
Generalised Tonic-Clonic Seizure
1%
Epistaxis
1%
Rhinorrhoea
1%
Micturition Urgency
1%
Lower Respiratory Tract Infection
1%
Hydrocephalus
1%
Skin Toxicity
1%
Cerebral Haemorrhage
1%
Mania
1%
Hallucination, Auditory
1%
Injection Site Bruising
1%
Oral Fungal Infection
1%
Neuropathy Peripheral
1%
Syncope
1%
Sepsis
1%
Sinusitis
1%
Dyspraxia
1%
Visual Field Defect
1%
Paranoia
1%
Renal Failure
1%
Myopathy
1%
Psychomotor Retardation
1%
Dry Eye
1%
Eye Irritation
1%
Eye Pain
1%
Dysaesthesia
1%
Haematoma
1%
Pneumothorax
1%
Haemorrhoids
1%
Hyponatraemia
1%
Encephalopathy
1%
Nervousness
1%
Perseveration
1%
Skin Irritation
1%
Embolism
1%
Abdominal Distension
1%
Haematemesis
1%
Hypoaesthesia Oral
1%
Toothache
1%
Rectal Haemorrhage
1%
Catheter Site Thrombosis
1%
Intracranial Pressure Increased
1%
Oedema
1%
Device Related Infection
1%
Stomatitis
1%
Gastroenteritis
1%
Gastroenteritis Viral
1%
Urticaria
1%
Pharyngitis
1%
Rhinitis
1%
Contusion
1%
Sinus Tachycardia
1%
Catheter Site Pain
1%
Akathisia
1%
Nasal Congestion
1%
Lumbar Vertebral Fracture
1%
Chest Pain
1%
Anosmia
1%
Feeling Cold
1%
Peripheral Swelling
1%
Respiratory Tract Infection
1%
Infusion Site Phlebitis
1%
Oral Herpes
1%
Mucosal Inflammation
1%
Taste Disorder
1%
Head Injury
1%
Vitamin B12 Deficiency
1%
Muscle Twitching
1%
Monoparesis
1%
Neuralgia
1%
Slow Speech
1%
Glioblastoma
1%
Partial Seizures
1%
Peripheral Sensory Neuropathy
1%
Post Herpetic Neuralgia
1%
Presyncope
1%
Vasogenic Cerebral Oedema
1%
Hiccups
1%
Coordination Abnormal
1%
Vestibular Disorder
1%
Flatulence
1%
Gingival Bleeding
1%
Cystitis
1%
Folliculitis
1%
Delusion
1%
Blood Potassium Decreased
1%
Transaminases Increased
1%
Hypomagnesaemia
1%
Infection
1%
Affect Lability
1%
Disorientation
1%
Hallucinations, Mixed
1%
Haematuria
1%
Pancytopenia
1%
Ear Discomfort
1%
Dermatitis Acneiform
1%
Eczema
1%
Mouth Ulceration
1%
Tachycardia
1%
Scar Pain
1%
Vascular Pain
1%
Hyperkalaemia
1%
Hypophosphataemia
1%
Facial Asymmetry
1%
Musculoskeletal Chest Pain
1%
Facial Paralysis
1%
Psychomotor Hyperactivity
1%
Restless Legs Syndrome
1%
Pollakiuria
1%
Flushing
1%
Gastritis
1%
Procedural Pain
1%
Radiation Necrosis
1%
Blood Alkaline Phosphatase Increased
1%
Blood Lactate Dehydrogenase Increased
1%
C-Reactive Protein Increased
1%
White Blood Cell Count Increased
1%
Productive Cough
100%
80%
60%
40%
20%
0%
Study treatment Arm
Experimental Arm
Standard Arm

DIPG Trial Design

2Treatment groups
Experimental Treatment
Group I: Marizomib + PanobinostatExperimental Treatment2 Interventions
If tolerated,combination of Marizomib: and panobinostat on subsequent cycles. The dose escalation and de-escalation for single agent and combination will be guided using the Bayesian optimal interval (BOIN) design. Intravenously initially given every other week over a 28 day course but may go to weekly x 3 and weekly x 4 as the dose levels increase. Up to 26 courses. Panobinostat: Oral dosage is given 3 times weekly, every other week over a 28 day course
Group II: MarizomibExperimental Treatment1 Intervention
All patients will initially receive marizomib (MRZ) alone (Course A1) The dose escalation and de-escalation for single agent and combination will be guided using the Bayesian optimal interval (BOIN) design -Intravenously initially given every other week over a 28 day course but may go to weekly x 3 and weekly x 4 as the dose levels increase. Up to 26 courses.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Marizomib
2018
Completed Phase 3
~750
Panobinostat
2011
Completed Phase 3
~1560

Find a Location

Who is running the clinical trial?

CelgeneIndustry Sponsor
632 Previous Clinical Trials
127,916 Total Patients Enrolled
Secura Bio, Inc.Industry Sponsor
8 Previous Clinical Trials
260 Total Patients Enrolled
Dana-Farber Cancer InstituteLead Sponsor
1,072 Previous Clinical Trials
340,402 Total Patients Enrolled

Media Library

Marizomib (Proteasome Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT04341311 — Phase 1
Brain Tumor Research Study Groups: Marizomib, Marizomib + Panobinostat
Brain Tumor Clinical Trial 2023: Marizomib Highlights & Side Effects. Trial Name: NCT04341311 — Phase 1
Marizomib (Proteasome Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04341311 — Phase 1

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Has the FDA sanctioned Marizomib for medical usage?

"Marizomib has only been tested in a limited capacity, so its safety is rated as a 1. There is minimal evidence that suggests it may be effective and safe for clinical use."

Answered by AI

Is enrollment for this clinical experiment open at present?

"It appears that this investigation, initially posted on August 10th 2020 and last updated May 3rd 2022, is not currently admitting participants. Nevertheless, 815 other clinical trials are open to potential patients at the present moment."

Answered by AI

Does this investigation introduce any novel therapeutic approaches?

"Marizomib has been in the spotlight since 2010, when Secura Bio, Inc. sponsored its first study involving 18 patients. This Phase 1 drug approval was granted following a successful trial and now 16 active studies are being conducted across 35 countries and 108 cities around the world."

Answered by AI

How many participants are being enlisted in this experiment?

"At this juncture, no new candidates are being sought for the trial that was initially announced on August 10th 2020 and last modified on May 3rd 2022. Nonetheless, those seeking trials related to diffuse intrinsic pontine glioma can explore 799 active studies while 16 clinical trials utilizing Marizomib currently have open enrollment."

Answered by AI

What research has been conducted on Marizomib in the past?

"Currently, there are 16 studies being conducted on Marizomib with 1 of those trials in its final phase. Although the bulk of these experiments take place at New york-based sites, a total of 285 locations have been utilised for examining this therapy."

Answered by AI
~1 spots leftby Mar 2025