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

NCT ID: NCT03049046

Last Updated: 2017-08-03

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE1
• Total Enrollment: 21 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2017-04-07
• Study Completion Date: 2018-03-30

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

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-∞).

Conditions

Amyotrophic Lateral Sclerosis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

CC100 250 mg

CC100 250 mg once daily by mouth for 7 days

Group Type: ACTIVE_COMPARATOR

CC100

Intervention Type: DRUG

synthetic caffeic acid phenethylester

CC100 500 mg

CC100 500 mg once daily by mouth for 7 days

Group Type: ACTIVE_COMPARATOR

CC100

Intervention Type: DRUG

synthetic caffeic acid phenethylester

CC100 1000 mg

CC100 1000 mg once daily by mouth for 7 days

Group Type: ACTIVE_COMPARATOR

CC100

Intervention Type: DRUG

synthetic caffeic acid phenethylester

Placebo

Placebo once daily by mouth for 7 days

Group Type: PLACEBO_COMPARATOR

Placebos

Intervention Type: DRUG

Diluent

Interventions

CC100

synthetic caffeic acid phenethylester

Intervention Type: DRUG

Placebos

Diluent

Intervention Type: DRUG

Other Intervention Names

synthetic caffeic acid phenethylester; Placebo oral liquid

Eligibility 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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 64 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Chemigen, LLC

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Indiana University, IU Health Physicians Neurology

Indianapolis, Indiana, United States

Site Status: RECRUITING

Countries

United States

Facility Contacts

Sandra Guingrich, LPN, CCRC

Role: primary

sguingri@iu.edu

317-963-7382

Other Identifiers

1R01FD004790-01A2

Identifier Type: FDA

Identifier Source: secondary_id

View Link

CC100B

Identifier Type: -

Identifier Source: org_study_id