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CC100: Phase 1 Multiple-Dose Safety and Tolerability in Subjects With ALS (CC100B)

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2017 by Chemigen, LLC
Sponsor:
Information provided by (Responsible Party):
Chemigen, LLC
ClinicalTrials.gov Identifier:
NCT03049046
First received: February 3, 2017
Last updated: February 7, 2017
Last verified: February 2017
  Purpose
Approximately 21 subjects with amyotrophic lateral sclerosis (ALS) will be randomized (6 to 1) to receive by mouth seven morning doses of CC100 or placebo for 7 days. Subjects are required to stay in the Clinic for approximately 9 hours following the first and last dose. Subjects will also have a mid-week clinic visit and will be contacted by phone within 3 to 5 days after the last dose.

Condition Intervention Phase
Amyotrophic Lateral Sclerosis
Drug: CC100
Drug: Placebos
Phase 1

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Participant, Investigator
Primary Purpose: Treatment
Official Title: Protocol CC100B. CC100: Phase 1 Multiple-Dose Safety and Tolerability in Subjects With ALS

Resource links provided by NLM:


Further study details as provided by Chemigen, LLC:

Primary Outcome Measures:
  • Safety and Tolerability: Adverse events, safety labs, vital signs, and ECGs [ Time Frame: From start of first dose to a minimum of 3 days after last dose ]
    Safety and tolerability assessed by group/dose measured by number of unsolicited adverse events (MedDRA), and changes in blood chemistry, hematology, urinalysis, vital signs, and 12-lead ECGs from baseline (prior to dosing).


Secondary Outcome Measures:
  • Pharmacokinetics (PK)--Peak plasma concentration (Cmax) [ Time Frame: 0.5, 1, 2, 4, and 8 hours after first and last dose ]
    Cmax after first (single) and last (multiple) CC100 doses

  • Pharmacokinetics (PK)--Area under the plasma concentration versus time curve (AUC) [ Time Frame: 0.5, 1, 2, 4, and 8 hours after first and last dose ]
    AUC after first (single) and last (multiple) CC100 doses

  • Pharmacokinetics (PK)--Half life (T 1/2) [ Time Frame: 0.5, 1, 2, 4, and 8 hours after first and last dose ]
    Estimated half-life after first (single) and last (multiple) CC100 doses

  • Pharmacodynamics (PD)--Monocyte chemotactic protein 1 (MCP-1) [ Time Frame: Pretreatment and 8 hours post last dose ]
    Short-term effects of CC100 on potential ALS inflammation biomarker MCP-1

  • Pharmacodynamics (PD)--Excitotoxicity/oxidative stress biomarkers [ Time Frame: Pretreatment and 8 hours post last dose ]
    Short-term effects of CC100 on potential ALS excitotoxicity/oxidative stress biomarkers: Heme oxygenase-1 (HMOX-1)/thioredoxin (TRX)/heat-shock protein 70 (HSP-70)


Estimated Enrollment: 21
Anticipated Study Start Date: February 2017
Estimated Study Completion Date: September 30, 2017
Estimated Primary Completion Date: July 31, 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: CC100 250 mg
CC100 250 mg once daily by mouth for 7 days
Drug: CC100
synthetic caffeic acid phenethylester
Other Name: synthetic caffeic acid phenethylester
Active Comparator: CC100 500 mg
CC100 500 mg once daily by mouth for 7 days
Drug: CC100
synthetic caffeic acid phenethylester
Other Name: synthetic caffeic acid phenethylester
Active Comparator: CC100 1000 mg
CC100 1000 mg once daily by mouth for 7 days
Drug: CC100
synthetic caffeic acid phenethylester
Other Name: synthetic caffeic acid phenethylester
Placebo Comparator: Placebo
Placebo once daily by mouth for 7 days
Drug: Placebos
Diluent
Other Name: Placebo oral liquid

Detailed Description:

Primary objective: to assess the safety and tolerability of multiple doses of orally administered CC100 in subjects with amyotrophic lateral sclerosis (ALS). Secondary objectives: to determine pharmacokinetics and pharmacodynamics of CC100 in plasma after single and after multiple doses; and to determine short-term effects of CC100 on potential blood-cell ALS biomarkers.

Study Design: Phase 1 double-blind, randomized, placebo-controlled multiple-dose of three CC100-dose cohorts. Approximately 18 subjects will receive CC100. Approximately 3 subjects will be randomized to placebo (across 3 cohorts). Periodic Assessment Committee safety reviews. Note: Participation will not exclude subjects from future CC100 studies Criteria for Evaluation: Safety Endpoints: Adverse events, blood chemistry, hematology, urinalysis, vital signs, 12-lead ECGs. Pharmacokinetic (PK)/Pharmacodynamic (PD): Plasma for CC100 concentrations (PK). Blood collected at baseline and after each subject's last dose will be assayed for potential biomarker(s). Stored specimens will be de-identified or combined for validating diagnostic tools/assays related to ALS. Statistical Methods: A minimum of 6 subjects per CC100 dose group and 3 placebo-dosed subjects (total across cohorts) are considered sufficient to evaluate initial safety and tolerability for the cohorts. Pharmacokinetic parameter estimates will be calculated by standard noncompartmental methods of analysis. Absolute bioavailability of administration will be estimated based on the total area under the time- concentration curve (AUC0-∞).

  Eligibility

Ages Eligible for Study:   18 Years to 64 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Have definite or probable ALS with a forced vital capacity of >60% predicted.
  • Men must practice a reliable method of birth control during study and for 2 weeks following study. Women must be non-fertile or post-menopausal.
  • Riluzole is allowed if dose has been stable for at least 30 days. Other allowed medications: lipid-lowering drugs, anti-hypertensives, anti-depressants, oral medications for type II diabetes, estrogen replacement therapy, thyroid replacement therapy, antihistamines, antacids, nonsteroidal anti-inflammatory drugs (except indomethacin), histamine H2-receptor antagonists, proton-pump inhibitors, calcium supplements, topical eye medications, and topical antibiotics.

Exclusion Criteria:

  • Greater than 250 pounds
  • Have serious or unstable illnesses as determine by the investigator.
  • Have current or a history of asthma or severe drug allergies or pollen allergy.
  • Have had serious infectious disease affecting the brain within the preceding 5 years; or have existing evidence of serious infection.
  • Have laboratory test values that are considered clinically significant as determined by the investigators.
  • Have ECG abnormalities that are clinically significant.
  • Have donated blood (a pint or more) or received an experimental drug within 30 days prior to dosing.
  • Have a history of chronic alcohol or drug abuse within the past 2 years.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT03049046

Locations
United States, Indiana
Indiana University, IU Health Physicians Neurology Recruiting
Indianapolis, Indiana, United States, 46202
Contact: Sandra Guingrich, LPN, CCRC    317-963-7382    sguingri@iu.edu   
Principal Investigator: Robert Pascuzzi, MD         
Sponsors and Collaborators
Chemigen, LLC
  More Information

Responsible Party: Chemigen, LLC
ClinicalTrials.gov Identifier: NCT03049046     History of Changes
Other Study ID Numbers: CC100B
1R01FD004790-01A2 ( US NIH Grant/Contract Award Number )
Study First Received: February 3, 2017
Last Updated: February 7, 2017
Individual Participant Data  
Plan to Share IPD: No
Plan Description: Single-site study

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases
Caffeic acid
Antioxidants
Molecular Mechanisms of Pharmacological Action
Protective Agents
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on March 24, 2017