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

NCT03049046 | Unknown Phase 1 FDA Drug

• 2 other identifiers: CC100B1R01FD004790-01A2
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 1

Brief Summary

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. Funding Source - FDA OOPD

Conditions

Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (1)

• Safety and Tolerability: Adverse events, safety labs, vital signs, and ECGs

  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).

  From start of first dose to a minimum of 3 days after last dose

Secondary Outcomes (5)

• Pharmacokinetics (PK)--Peak plasma concentration (Cmax)

  0.5, 1, 2, 4, and 8 hours after first and last dose

• Pharmacokinetics (PK)--Area under the plasma concentration versus time curve (AUC)

  0.5, 1, 2, 4, and 8 hours after first and last dose

• Pharmacokinetics (PK)--Half life (T 1/2)

  0.5, 1, 2, 4, and 8 hours after first and last dose

• Pharmacodynamics (PD)--Monocyte chemotactic protein 1 (MCP-1)

  Pretreatment and 8 hours post last dose

• Pharmacodynamics (PD)--Excitotoxicity/oxidative stress biomarkers

  Pretreatment and 8 hours post last dose

Study Arms (4)

CC100 250 mg

ACTIVE COMPARATOR

CC100 250 mg once daily by mouth for 7 days

Drug: CC100

CC100 500 mg

ACTIVE COMPARATOR

CC100 500 mg once daily by mouth for 7 days

Drug: CC100

CC100 1000 mg

ACTIVE COMPARATOR

CC100 1000 mg once daily by mouth for 7 days

Drug: CC100

Placebo

PLACEBO COMPARATOR

Placebo once daily by mouth for 7 days

Drug: Placebos

Interventions

CC100 DRUG

synthetic caffeic acid phenethylester

Also known as: synthetic caffeic acid phenethylester

CC100 1000 mg; CC100 250 mg; CC100 500 mg

Placebos DRUG

Diluent

Also known as: Placebo oral liquid

Placebo

Eligibility Criteria

• Age: 18 Years - 64 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Chemigen, LLC: lead

Study Sites (1)

Indiana University, IU Health Physicians Neurology

Indianapolis, Indiana, 46202, United States

RECRUITING

Related Publications (1)

• Chang GJ, Yeh YH, Chen WJ, Hsu SC, Lai YJ, Chang CJ, Lee HY. Caffeic Acid Phenethyl Ester Improves Right Ventricular Function and Reduces Arrhythmogenesis by Attenuating Structural and Electromechanical Remodeling in Pulmonary Arterial Hypertensive Rats. Phytother Res. 2026 Jan 26. doi: 10.1002/ptr.70219. Online ahead of print.

  PMID: 41587816 DERIVED

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 3, 2017
• First Posted: February 9, 2017
• Study Start: April 7, 2017
• Primary Completion: January 30, 2018
• Study Completion: March 30, 2018
• Last Updated: August 3, 2017

Record last verified: 2017-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)