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CX-8998

Phase 2

Epilepsy | Small molecule | Neurology |Jazz Pharmaceuticals plc|Last Updated: Sep 8, 2022

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment7

FDA Designations

No designations recorded

Clinical trial landscape

CX-8998 · 2 trials · 2 indications

Phase 2 2
NCT03406702CX-8998 for Absence SeizuresEpilepsy
COMPLETED7 Analytics
NCT03101241A Phase 2 RCT Study of CX-8998 for Essential TremorEssential Tremor
COMPLETED95 Analytics
PHASE2COMPLETED
CX-8998 for Absence Seizures
EpilepsyUnlock trial analytics
PHASE2COMPLETED
A Phase 2 RCT Study of CX-8998 for Essential Tremor
Essential TremorUnlock trial analytics

Study Endpoints

Primary Endpoints

Change From Baseline to End of Treatment in QT Interval Corrected for Heart Rate Using Fridericia's Formula (QTcF)
Baseline (Day 1) to end of treatment 1-2 hours post-dose, up to 4 weeks post-dose.

Fridericia's Correction Formula (QTCF) is a formula which takes into account the physiologic shortening of the QT interval which occurs as the heart rate increases, permitting comparison of the QT interval across a range of rates.

Change From Baseline to End of Treatment in Clinical Alanine Aminotransferase Serum Chemistry Concentration
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory assessment in alanine aminotransferase serum chemistry concentration.

Change From Baseline to End of Treatment in Clinical Albumin Serum Chemistry Concentration
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory assessment in albumin serum chemistry.

Change From Baseline to End of Treatment in Clinical Albumin/Globulin Serum Chemistry Concentration
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory assessment in albumin/globulin serum chemistry.

Change From Baseline to End of Treatment in Clinical Alkaline Phosphatase Serum Chemistry Concentration
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory assessment in alkaline phosphatase serum chemistry.

Change From Baseline to End of Treatment in Clinical Aspartate Aminotransferase Serum Chemistry Concentration
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory assessment in aspartate aminotransferase serum chemistry.

Baseline Clinical Blood Urea Nitrogen/Creatinine Serum Chemistry Concentration
Baseline (Day 1)

Clinical safety laboratory assessment in BUN/Creatinine serum chemistry.

Change From Baseline to End of Treatment in Clinical Bilirubin Serum Chemistry Concentration
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory assessment in bilirubin serum chemistry.

Change From Baseline to End of Treatment in Clinical Blood Urea Nitrogen Serum Chemistry Concentration
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory assessment in blood urea nitrogen serum chemistry.

Change From Baseline to End of Treatment in Clinical Carbon Dioxide Serum Chemistry Concentration
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory assessment in carbon dioxide serum chemistry.

Change From Baseline to End of Treatment in Clinical Chloride Serum Chemistry Concentration
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory assessment in chloride serum chemistry.

Change From Baseline to End of Treatment in Clinical Calcium Serum Chemistry Concentration
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory assessment in calcium serum chemistry.

Change From Baseline to End of Treatment in Clinical Cholesterol Serum Chemistry Concentration
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory assessment in cholesterol serum chemistry.

Baseline Clinical Cholesterol/HDL-Cholesterol Serum Chemistry Concentration
Baseline (Day 1)

Clinical safety laboratory assessment in cholesterol/HDL-cholesterol serum chemistry.

Change From Baseline to End of Treatment in Clinical Creatine Kinase Serum Chemistry Concentration
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory assessment in creatine kinase serum chemistry.

Change From Baseline to End of Treatment in Clinical Creatinine Serum Chemistry Concentration
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory assessment in creatinine serum chemistry.

Change From Baseline to End of Treatment in Clinical Globulin Serum Chemistry Concentration
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory assessment in globulin serum chemistry.

Baseline Clinical Glomerular Filtration Rate (GFR) Adjusted for Body Surface Area (BSA) Serum Chemistry Concentration
Baseline (Day 1)

Clinical safety laboratory assessment in glomerular filtration rate adjusted for BSA chemistry.

Change From Baseline to End of Treatment in Clinical Estimated Glomerular Filtration Rate (GFR) Serum Chemistry Concentration
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory assessment in GFR serum chemistry.

Change From Baseline to End of Treatment in Clinical Glucose Serum Chemistry Concentration
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory assessment in glucose serum chemistry.

Baseline Clinical HDL Cholesterol Serum Chemistry Concentration
Baseline (Day 1)

Clinical safety laboratory assessment in HDL cholesterol serum chemistry.

Baseline Clinical LDL Cholesterol Serum Chemistry Concentration
Baseline (Day 1)

Clinical safety laboratory assessment in LDL cholesterol serum chemistry.

Change From Baseline to End of Treatment in Clinical Lactate Dehydrogenase Serum Chemistry Concentration
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory assessment in lactate dehydrogenase serum chemistry.

Change From Baseline to End of Treatment in Clinical Magnesium Serum Chemistry Concentration
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory assessment in magnesium serum chemistry.

Baseline Clinical Non-HDL Cholesterol Serum Chemistry Concentration
Baseline (Day 1)

Clinical safety laboratory assessment in non-HDL cholesterol serum chemistry.

Change From Baseline to End of Treatment in Clinical Phosphate Serum Chemistry Concentration
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory assessment in phosphate serum chemistry.

Change From Baseline to End of Treatment in Clinical Potassium Serum Chemistry Concentration
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory assessment in potassium serum chemistry.

Change From Baseline to End of Treatment in Clinical Protein Serum Chemistry Concentration
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory assessment in protein serum chemistry.

Change From Baseline to End of Treatment in Clinical Sodium Serum Chemistry Concentration
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory assessment in sodium serum chemistry.

Change From Baseline to End of Treatment in Clinical Triglycerides Serum Chemistry Concentration
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory assessment in triglycerides serum chemistry.

Change From Baseline to End of Treatment in Clinical Urate Serum Chemistry Concentration
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory assessment in urate serum chemistry.

Change From Baseline to End of Treatment in Basophils Hematology Assessment
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory basophils hematology assessment.

Change From Baseline to End of Treatment in Basophils/Leukocytes Hematology Assessment
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory basophils/leukocytes hematology assessment.

Change From Baseline to End of Treatment in Eosinophils Hematology Assessment
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory eosinophils hematology assessment.

Change From Baseline to End of Treatment in Eosinophils/Leukocytes Hematology Assessment
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory eosinophils/leukocytes hematology assessment.

Change From Baseline to End of Treatment in Mean Corpuscular Hemoglobin (HGB) Concentration Hematology Assessment
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory mean corpuscular HGB concentration hematology assessment.

Change From Baseline to End of Treatment in Mean Corpuscular Hemoglobin (HGB) Hematology Assessment
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory mean corpuscular HGB hematology assessment.

Change From Baseline to End of Treatment in Mean Corpuscular Volume Hematology Assessment
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory mean corpuscular volume hematology assessment.

Change From Baseline to End of Treatment in Erythrocytes Hematology Assessment
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory erythrocytes hematology assessment.

Change From Baseline to End of Treatment in Erythrocytes Distribution Width Hematology Assessment
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory erythrocytes distribution width hematology assessment.

Change From Baseline to End of Treatment in Hematocrit Hematology Assessment
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory hematocrit hematology assessment.

Change From Baseline to End of Treatment in Hemaglobin Hematology Assessment
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory hemaglobin hematology assessment.

Change From Baseline to End of Treatment in Leukocytes Hematology Assessment
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory leukocytes hematology assessment.

Change From Baseline to End of Treatment in Lymphocytes Hematology Assessment
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory lymphocytes hematology assessment.

Change From Baseline to End of Treatment in Lymphocytes/Leukocytes Hematology Assessment
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory lymphocytes/leukocytes hematology assessment.

Change From Baseline to End of Treatment in Mean Platelet Volume Hematology Assessment
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory mean platelet volume hematology assessment.

Change From Baseline to End of Treatment in Monocytes Hematology Assessment
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory monocytes hematology assessment.

Change From Baseline to End of Treatment in Monocytes/Leukocytes Hematology Assessment
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory monocytes/leukocytes hematology assessment.

Change From Baseline to End of Treatment in Neutrophils Hematology Assessment
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory neutrophils hematology assessment.

Change From Baseline to End of Treatment in Neutrophils/Leukocytes Hematology Assessment
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory neutrophils/leukocytes hematology assessment.

Change From Baseline to End of Treatment in Platelets Hematology Assessment
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory platelets hematology assessment.

Baseline to End of Treatment in Bacteria Urinalysis Assessment
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory bacteria urinalysis assessment.

Baseline to End of Treatment in Urine Bilirubin Urinalysis Assessment
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory urine bilirubin urinalysis assessment.

Baseline to End of Treatment in Epithelial Cells Urinalysis Assessment
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory epithelial cells urinalysis assessment. Shifts from baseline to normal/abnormal status were assessed. A normal range is 0-10 epithelial cells/high power field (hpf). A worse outcome is \>10 epithelial cells.

Baseline to End of Treatment in Urine Erythrocytes Urinalysis Assessment
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory urine erythrocytes urinalysis assessment. Shifts from baseline to normal/abnormal status were assessed. A normal range is 0-2 erythrocytes/high power field (hpf). A better outcome is 0 or "none seen."

Baseline to End of Treatment in Urine Glucose Urinalysis Assessment
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory urine glucose urinalysis assessment.

Baseline to End of Treatment in Ketones Urinalysis Assessment
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory ketones urinalysis assessment.

Baseline to End of Treatment in Leukocyte Esterase Urinalysis Assessment
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory leukocyte esterase urinalysis assessment. Shifts from baseline to normal/abnormal status were assessed. A normal assessment or better outcome is "negative." An abnormal assessment or worse outcome is a positive assessment (i.e., 2+).

Baseline to End of Treatment in Urine Leukocytes Urinalysis Assessment
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory urine leukocytes urinalysis assessment. Shifts from baseline to normal/abnormal status were assessed. A normal range is 0-5 leukocytes/high power field (hpf). An abnormal assessment or worse outcome is \>5 leukocytes/hpf (i.e., 11-30).

Baseline to End of Treatment in Mucous Threads Urinalysis Assessment
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory mucous threads urinalysis assessment.

Baseline to End of Treatment in Nitrite Urinalysis Assessment
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory nitrite urinalysis assessment.

Baseline to End of Treatment in Occult Blood Urinalysis Assessment
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory occult blood urinalysis assessment. Shifts from baseline to normal/abnormal status were assessed. A normal assessment or better outcome is "negative." An abnormal assessment or worse outcome is a positive assessment (i.e., 2+).

Baseline to End of Treatment in Protein Urinalysis Assessment
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory protein urinalysis assessment.

Baseline to End of Treatment in Specific Gravity Urinalysis Assessment
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory specific gravity urinalysis assessment. Shifts from baseline to normal/abnormal status were assessed. A normal assessment or better outcome is 1.005-1.030. An abnormal assessment or worse outcome is a value outside of this range.

Baseline to End of Treatment in Specimen Appearance Urinalysis Assessment
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory specimen appearance urinalysis assessment.

Change From Baseline to End of Treatment in Urobilinogen Urinalysis Assessment
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory urobilinogen urinalysis assessment.

Baseline to End of Treatment in pH Urinalysis Assessment
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory pH urinalysis assessment. Shifts from baseline to normal/abnormal status were assessed.

Baseline to End of Treatment in Urine Color Urinalysis Assessment
Baseline (Day 1) to end of treatment, or up to 4 weeks post-dose.

Clinical safety laboratory urine color urinalysis assessment.

Number (%) of Participants Who Did Not Complete The Study Due to Treatment-Emergent Adverse Events
Baseline (Day 1) up to Day 26 post-dose, or up to 1 year 3 weeks.

Treatment-emergent adverse events are all adverse events occurring during the treatment period or a pretreatment event that worsens in intensity during the treatment period as assessed by CTCAE v4.0.

Number (%) of Participants With Adverse Events of Special Interest
Baseline (Day 1) up to Day 26 post-dose, or up to 1 year 3 weeks.

An adverse event of special interest is a serious adverse event as defined in Outcome 6. This includes, however is not limited to, increased seizure frequency, new seizure types, worsening of EEG parameters, systemic adverse events based on safety profile as assessed by CTCAE v4.0.

Change From Baseline to End of Treatment in Respiration Rate
Baseline (Day 1) to end of treatment 1-2 hours post-dose, up to 4 weeks post-dose.
Change From Baseline to End of Treatment in Temperature
Baseline (Day 1) to end of treatment 1-2 hours post-dose, up to 4 weeks post-dose.
Baseline Weight
Baseline (Day 1)
Change From Baseline to End of Treatment in Pulse
Baseline (Day 1) to end of treatment 1-2 hours post-dose, up to 4 weeks post-dose.

The change from baseline to end of treatment in participants' pulses was assessed. The changes in recumbent pulse, standing pulse, and the change from recumbent to standing pulse are reported. Change from recumbent to standing pulse was measured by the difference in recumbent pulse change and standing pulse change.

Change From Baseline to End of Treatment in Systolic Blood Pressure
Baseline (Day 1) to end of treatment 1-2 hours post-dose, up to 4 weeks post-dose.

The change from baseline to end of treatment in participants' systolic blood pressure (sbp) was assessed. The changes in recumbent sbp, standing sbp, and the change from recumbent to standing sbp are reported. Change from recumbent to standing sbp was measured by the difference in recumbent sbp change and standing sbp change.

Change From Baseline to End of Treatment in Diastolic Blood Pressure
Baseline (Day 1) to end of treatment 1-2 hours post-dose, up to 4 weeks post-dose.

The change from baseline to end of treatment in participants' diastolic blood pressure (dbp) was assessed. The changes in recumbent dbp, standing dbp, and the change from recumbent to standing dbp are reported. Change from recumbent to standing dbp was measured by the difference in recumbent dbp change and standing dbp change.

Change From Baseline to End of Treatment in QT Interval
Baseline (Day 1) to end of treatment 1-2 hours post-dose, up to 4 weeks post-dose.
Change From Baseline to End of Treatment in QRS Interval
Baseline (Day 1) to end of treatment 1-2 hours post-dose, up to 4 weeks post-dose.
Change From Baseline to End of Treatment in PR Interval
Baseline (Day 1) to end of treatment 1-2 hours post-dose, up to 4 weeks post-dose.
Change From Baseline to End of Treatment in Heart Rate
Baseline (Day 1) to end of treatment 1-2 hours post-dose, up to 4 weeks post-dose.
Baseline and Day 28 on The Essential Tremor Rating Assessment Scale Performance Subscale (TETRAS-PS) Total Score
Baseline and Day 28 post-dose.

The Essential Tremor Rating Assessment Scale Performance Subscale (TETRAS-PS) was used to assess the efficacy of CX-8998 in reducing essential tremor as scored by the independent video raters. TETRAS-PS quantifies tremor in the head, face, voice, limbs, and trunk. The overall subscale score ranges from a minimum of 0 to a maximum of 64. A lower score is indicative of improvement and a better outcome.

Change From Baseline to Day 28 on The Essential Tremor Rating Assessment Scale Performance Subscale (TETRAS-PS)
Baseline up to Day 28 post-dose.

The Essential Tremor Rating Assessment Scale Performance Subscale (TETRAS-PS) was used to assess the efficacy of CX-8998 in reducing essential tremor as scored by the independent video raters. TETRAS-PS quantifies tremor in the head, face, voice, limbs, and trunk. The overall subscale score ranges from a minimum of 0 to a maximum of 64. A decrease or negative change in the score is indicative of improvement in outcome.

Secondary Endpoints

Baseline and Day 28 on The Essential Tremor Rating Assessment Scale Activities of Daily Living (TETRAS-ADL) Subscale Score
Baseline and Day 28 post-dose.
Change From Baseline to Day 28 on The Essential Tremor Rating Assessment Scale Activities of Daily Living (TETRAS-ADL) Subscale Score
Baseline up to Day 28 post-dose.
Baseline and Day 28 in the Kinesia ONE Score
Baseline and Day 28 post-dose.
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
CX-8998EXPERIMENTALT-type calcium channel blocker
PlaceboPLACEBO_COMPARATOR -

Interventions

NameTypeDescription
CX-8998DRUGT-type calcium channel blocker
PlaceboDRUGPlacebo comparator
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Eligibility Criteria

Age Range16 Years to 55 Years
SexALL
Healthy VolunteersNo
Study Sites7

Inclusion Criteria: 1. Signed informed consent form (ICF) indicating that the subject has been informed of the procedures to be followed, the experimental nature of the therapy, alternatives, potential benefits, side effects, risks, and discomforts. 2. Men or non-pregnant, non-breastfeeding women 1...

Countries:United States
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Frequently asked questions about CX-8998

What is CX-8998 used for?

CX-8998 is an investigational small molecule being developed for the treatment of epilepsy and essential tremor. It is in Phase 2 clinical development for these neurological conditions.

How does CX-8998 work?

CX-8998 is a small molecule that modulates ion channels involved in neuronal excitability, which may help reduce abnormal electrical activity in the brain associated with epilepsy and essential tremor.

Who is developing CX-8998?

CX-8998 is being developed by Jazz Pharmaceuticals plc, a biopharmaceutical company traded on NASDAQ under the ticker JAZZ.

What phase is CX-8998 in?

CX-8998 is in Phase 2 clinical development. It is investigational and has not been approved by regulatory authorities for any indication.

What clinical trials has CX-8998 been in?

CX-8998 has been studied in two completed Phase 2 trials: NCT03101241 for essential tremor with 95 participants, and NCT03406702 for absence seizures in epilepsy with 7 participants. Both trials were conducted in the United States.

Is CX-8998 the same as any other drug?

CX-8998 is also known by its chemical name, and it is not the same as any other marketed drug. It is a distinct investigational compound being developed by Jazz Pharmaceuticals.