Amyotrophic Lateral SclerosisPhase 1CC100BNCT03049046
CC100: Phase 1 Multiple-Dose Safety and Tolerability in Subjects With ALS
A Phase 1 study testing CC100 in people with Amyotrophic Lateral Sclerosis, run by Chemigen, LLC at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 64 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- CC100
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 21 participants
- Where
- 1 siteUnited States
- Study dates
- 7 Apr 2017to 30 Mar 2018
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 64 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site, 1 recruiting
- Recruiting
Indiana University, IU Health Physicians Neurology
Indianapolis, Indiana, 46202
Sandra Guingrich, LPN, CCRC, 317-963-7382, sguingri@iu.edu
Robert Pascuzzi, MD
About this study
Official title: Protocol CC100B. CC100: Phase 1 Multiple-Dose Safety and Tolerability in Subjects With ALS
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
Read the 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
- Being tested
- CC100, Placebos
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CC100Drug
Also called synthetic caffeic acid phenethylester
synthetic caffeic acid phenethylester
PlacebosDrug
Also called Placebo oral liquid
Diluent
| Study group | Role | What they receive |
|---|---|---|
| CC100 250 mg | Active comparator | CC100 250 mg once daily by mouth for 7 days Receives: Drug: CC100 |
| CC100 500 mg | Active comparator | CC100 500 mg once daily by mouth for 7 days Receives: Drug: CC100 |
| CC100 1000 mg | Active comparator | CC100 1000 mg once daily by mouth for 7 days Receives: Drug: CC100 |
| Placebo | Placebo comparator | Placebo once daily by mouth for 7 days Receives: Drug: Placebos |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: From start of first dose to a minimum of 3 days after last dose
Secondary outcomes (5)
Pharmacokinetics (PK)--Peak plasma concentration (Cmax)
Cmax after first (single) and last (multiple) CC100 doses
Measured: 0.5, 1, 2, 4, and 8 hours after first and last dose
Pharmacokinetics (PK)--Area under the plasma concentration versus time curve (AUC)
AUC after first (single) and last (multiple) CC100 doses
Measured: 0.5, 1, 2, 4, and 8 hours after first and last dose
Pharmacokinetics (PK)--Half life (T 1/2)
Estimated half-life after first (single) and last (multiple) CC100 doses
Measured: 0.5, 1, 2, 4, and 8 hours after first and last dose
Pharmacodynamics (PD)--Monocyte chemotactic protein 1 (MCP-1)
Short-term effects of CC100 on potential ALS inflammation biomarker MCP-1
Measured: Pretreatment and 8 hours post last dose
Pharmacodynamics (PD)--Excitotoxicity/oxidative stress biomarkers
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)
Measured: Pretreatment and 8 hours post last dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Feb 2017 (estimated)
- Study start7 Apr 2017
- Primary completion30 Jan 2018 (estimated)
- Study completion30 Mar 2018 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 21 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Chemigen, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03049046
- Sponsor's protocol ID
- CC100B
- Other IDs
- 1R01FD004790-01A2
- Acronym
- CC100B
- Status last verified
- Aug 2017
- First submitted
- 3 Feb 2017
- Last updated
- 3 Aug 2017
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: Single-site study
Study at a glance
From the registry record, last updated by the sponsor on 3 Aug 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Protocol CC100B. CC100: Phase 1 Multiple-Dose Safety and Tolerability in Subjects With ALS
- ClinicalTrials.gov ID
- NCT03049046
- Acronym / sponsor ID
- CC100B / CC100B
- Conditions
- Amyotrophic Lateral Sclerosis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 21 participants (estimated)
- Lead sponsor
- Chemigen, LLCIndustry (pharma, biotech, devices)
- Study start
- 7 Apr 2017 (actual)
- Primary completion
- 30 Jan 2018 (estimated)
- Study completion
- 30 Mar 2018 (estimated)
- First posted
- 9 Feb 2017 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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-∞).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| CC100 250 mg | Active comparator | Drug: CC100CC100 250 mg once daily by mouth for 7 days |
| CC100 500 mg | Active comparator | Drug: CC100CC100 500 mg once daily by mouth for 7 days |
| CC100 1000 mg | Active comparator | Drug: CC100CC100 1000 mg once daily by mouth for 7 days |
| Placebo | Placebo comparator | Drug: PlacebosPlacebo once daily by mouth for 7 days |
Who may qualify
- Ages
- 18 to 64 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CC100Also: synthetic caffeic acid phenethylestersynthetic caffeic acid phenethylester | Drug | CC100 1000 mg, CC100 250 mg, CC100 500 mg |
| PlacebosAlso: Placebo oral liquidDiluent | Drug | Placebo |
Primary outcome
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).
5 secondary outcomes
Pharmacokinetics (PK)--Peak plasma concentration (Cmax)
Time frame: 0.5, 1, 2, 4, and 8 hours after first and last dose
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
Pharmacokinetics (PK)--Half life (T 1/2)
Time frame: 0.5, 1, 2, 4, and 8 hours after first and last dose
Pharmacodynamics (PD)--Monocyte chemotactic protein 1 (MCP-1)
Time frame: Pretreatment and 8 hours post last dose
Pharmacodynamics (PD)--Excitotoxicity/oxidative stress biomarkers
Time frame: Pretreatment and 8 hours post last dose
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Aug 2017.
- Individual participant dataSharing planned: No. Single-site study
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Indiana University, IU Health Physicians Neurology
Indianapolis, United States
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing CC100 in people with Amyotrophic Lateral Sclerosis, run by Chemigen, LLC at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 3 Aug 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Aug 2017.