Familial Mediterranean FeverPhase 1NCT01075906
Pharmacokinetics Study of Colchicine in Familial Mediterranean Fever (FMF) Patients
A Phase 1 study testing colchicine sprinkle capsules in people with Familial Mediterranean Fever, run by Mutual Pharmaceutical Company, Inc. at 10 sites in 4 countries.
Trial summary
Colchicine is widely recognized as safe and effective treatment of Familial Mediterranean Fever (FMF) in children and adults. Colchicine is currently used to treat FMF in younger patients by inexact dosing through breaking or crushing adult-dose tablets. Read more about this study
- Who is it for?
- Ages 2 to 65 yearsHealthy volunteers can take part
- Conditions the study is for
- Familial Mediterranean Fever
- What is being tested?
- colchicine sprinkle capsules
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 75 participants
- Where
- 10 sitesin 4 countries
- Study dates
- Aug 2010to Dec 2011
- 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 2 to 65 years
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Who may be able to join
- Patients age 2-65 years with a confirmed clinical diagnosis of FMF,
- Non-pregnant, and
- If of child-bearing potential, using effective contraceptive measures.
Who cannot join
- Recent participation (within 30 days) in other research studies,
- Pregnant or lactating,
- History or current infection of human immunodeficiency virus (HIV), hepatitis A, B or C,
- Current or recent use of any drugs/drug classes or combinations thereof that may affect the absorption or metabolism of colchicine,
- Clinically relevant abnormal clinical laboratories at screening,
- Current or recent (<6 months) history of severe, unstable or uncontrolled neurological, cardiovascular, gastrointestinal, hematological, moderate or severe hepatic and/or renal disease, or evidence of other diseases at the physical examination conducted at the screening.
Trial locations and contacts
10 sites in 4 countries.
10 sites in 4 countries
Israel6 sites
Soroka Medical Center
Beersheba, Beer Sheba, 84141
Rambam Medical Center
Haifa, Haifa District, 24035
Hadassah Medical Center
Jerusalem, Israel, 91120
Pediatric Rheumatology Unit - Shaare Zedek Medical Center
Jerusalem, Jerusalem, 91031
Safra Children's Hospital
Tel HaShomer, Tel Hashomer, 52621
Sheba Medical Center
Tel HaShomer, Tel Hashomer, 52621
Turkey (Türkiye)2 sites
Hacettepe University
Ankara, Ankara, 06100
Cerrahpasa Medical Facility
Istanbul, Istanbul, 34303
United States1 site
Childrens Hospital Los Angeles
Los Angeles, California, 90027
Armenia1 site
Center of Medical Genetics and Primary Health Care
Yerevan, Yerevan, 0010
About this study
Official title: An Open-label, Parallel-group, Multiple-Dose, Pharmacokinetic and Safety Study of Colchicine Pediatric Formulation in Pediatric and Adult Patients With FMF
Colchicine is widely recognized as safe and effective treatment of Familial Mediterranean Fever (FMF) in children and adults. Colchicine is currently used to treat FMF in younger patients by inexact dosing through breaking or crushing adult-dose tablets. An age-appropriate sprinkle formulation will allow for more accurate dosing in pediatric patients. The primary objective of this study is to evaluate and compare the steady-state pharmacokinetics of multiple oral doses of colchicine sprinkle capsules administered to pediatric and adult FMF patients.
Secondary objectives include evaluation of the safety and tolerability of this regimen in pediatric and adult FMF patients and measurement of the levels of acute phase reactants (i.e, serum amyloid A [SAA], erythrocyte sedimentation rate [ESR], C-reactive protein [CRP]) at baseline and after dosing.
Read the detailed description
FMF patients who have not been taking colchicine (colchicine-naïve patients) will be enrolled into a 1 week dose-titration period (Days -7 to -1). Beginning on Day -7, a pre-dose blood sample will be collected from the colchicine-naïve patient population for determination of pharmacodynamic markers. Patients will then be administered a low starting dose of colchicine (as determined by the principal investigator) titrated up to the study colchicine dose which is 0.6 mg (2 capsules) in children ≥2 to < 6 years old, 0.9 mg (3 capsules) in children ≥6 to < 12, 1.2 mg (4 capsules) in children ≥12 to < 16 and adults ≥16 and < 65. On Day 2, patients will return to the clinic for collection of a pre-dose blood sample followed by administration of the study dose of colchicine. On Days 3-7, patients (parent/guardian) will self-medicate with the study dose of colchicine recording the time of dosing and any adverse events. On Days 8-14, patients (parent/guardian) will self-medicate with the study dose of colchicine recording the time of dosing and any adverse events. On the morning of Day 15, patients will return to the clinic for collection of a pre-dose blood sample followed by administration of the study dose of colchicine. Blood samples will be collected post-dose at times sufficient to adequately define the pharmacokinetics of colchicine and its metabolites. Safety and tolerability of this dosing regimen will be determined by evaluation of vital signs and adverse events during the study and upon completion of the study. All adverse events will be evaluated by the investigator and reported in the subject's case report form.
- Conditions
- Familial Mediterranean Fever
- Being tested
- colchicine sprinkle capsules
- Keywords
- pharmacokinetics
- Medical subject headings
- Familial Mediterranean Fever
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
colchicine sprinkle capsulesDrug
0.3 mg
colchicine sprinkle capsulesDrug
0.3 mg
| Study group | Role | What they receive |
|---|---|---|
| Colchicine | Experimental | Colchicine Sprinkle Capsules, 0.3 mg - dose administered according to age range on Day 1 Receives: Drug: colchicine sprinkle capsules |
| colchicine at steady state | Experimental | colchicine sprinkle capsules 0.3 mg - dose administered according to age range on Day 15 following once daily dosing of colchicine on Days 2 - 14 Receives: Drug: colchicine sprinkle capsules |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum Plasma Concentration
pharmacokinetic samples collected pre-dose on Days 1, 2 and 15 and at 0.25 - 0.5 hours, 1.5-2.5 hours and at 5-8 hours post-dose on Days 1 and 15
Measured: 15 days
Area Under the Concentration Time Curve from Time Zero to the Time of Last Measured Concentration (AUC 0-t)
Pharmacokinetic samples collected pre-dose on Days 1,2 and 15 and at 0.25-0.5 hours, 1.5-2.5 hours and 5-8 hours post-dose on Days 1 and 15.
Measured: 15 days
Area Under the Concentration Time Curve from Zero through Infinity
Pharmacokinetic samples collected pre-dose on Days 1, 2 and 15 and at 0.25-0.5 hours, 1.5-2.5 hours and at 5-8 hours post-dose on Days 1 and 15
Measured: 15 days
Secondary outcomes (1)
Acute Phase Reactant (ESR, CRP, SAA) Levels
Pharmacodynamic samples collected pre-dose on Days 7, 1 and 15
Measured: 15 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Feb 2010 (estimated)
- Study startAug 2010
- Primary completionDec 2011
- Study completionDec 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Familial Mediterranean FeverStudies 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
- Basic science
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 75 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mutual Pharmaceutical Company, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Matthew Davis, MD, Study chair, Mutual Pharmaceutical Company, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01075906
- Sponsor's protocol ID
- MPC-006-09-1001
- Status last verified
- Jan 2012
- First submitted
- 24 Feb 2010
- Last updated
- 10 Jan 2012
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 10 Jan 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Parallel-group, Multiple-Dose, Pharmacokinetic and Safety Study of Colchicine Pediatric Formulation in Pediatric and Adult Patients With FMF
- ClinicalTrials.gov ID
- NCT01075906
- Acronym / sponsor ID
- MPC-006-09-1001
- Conditions
- Familial Mediterranean Fever
- Keywords
- pharmacokinetics
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 75 participants (actual)
- Lead sponsor
- Mutual Pharmaceutical Company, Inc.Industry (pharma, biotech, devices)
- Study start
- Aug 2010
- Primary completion
- Dec 2011 (actual)
- Study completion
- Dec 2011 (actual)
- First posted
- 25 Feb 2010 (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.
Colchicine is widely recognized as safe and effective treatment of Familial Mediterranean Fever (FMF) in children and adults. Colchicine is currently used to treat FMF in younger patients by inexact dosing through breaking or crushing adult-dose tablets. An age-appropriate sprinkle formulation will allow for more accurate dosing in pediatric patients. The primary objective of this study is to evaluate and compare the steady-state pharmacokinetics of multiple oral doses of colchicine sprinkle capsules administered to pediatric and adult FMF patients.
Secondary objectives include evaluation of the safety and tolerability of this regimen in pediatric and adult FMF patients and measurement of the levels of acute phase reactants (i.e, serum amyloid A [SAA], erythrocyte sedimentation rate [ESR], C-reactive protein [CRP]) at baseline and after dosing.
Detailed description
FMF patients who have not been taking colchicine (colchicine-naïve patients) will be enrolled into a 1 week dose-titration period (Days -7 to -1). Beginning on Day -7, a pre-dose blood sample will be collected from the colchicine-naïve patient population for determination of pharmacodynamic markers. Patients will then be administered a low starting dose of colchicine (as determined by the principal investigator) titrated up to the study colchicine dose which is 0.6 mg (2 capsules) in children ≥2 to < 6 years old, 0.9 mg (3 capsules) in children ≥6 to < 12, 1.2 mg (4 capsules) in children ≥12 to < 16 and adults ≥16 and < 65. On Day 2, patients will return to the clinic for collection of a pre-dose blood sample followed by administration of the study dose of colchicine. On Days 3-7, patients (parent/guardian) will self-medicate with the study dose of colchicine recording the time of dosing and any adverse events. On Days 8-14, patients (parent/guardian) will self-medicate with the study dose of colchicine recording the time of dosing and any adverse events. On the morning of Day 15, patients will return to the clinic for collection of a pre-dose blood sample followed by administration of the study dose of colchicine. Blood samples will be collected post-dose at times sufficient to adequately define the pharmacokinetics of colchicine and its metabolites. Safety and tolerability of this dosing regimen will be determined by evaluation of vital signs and adverse events during the study and upon completion of the study. All adverse events will be evaluated by the investigator and reported in the subject's case report form.
Study design
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Colchicine | Experimental | Drug: colchicine sprinkle capsulesColchicine Sprinkle Capsules, 0.3 mg - dose administered according to age range on Day 1 |
| colchicine at steady state | Experimental | Drug: colchicine sprinkle capsulescolchicine sprinkle capsules 0.3 mg - dose administered according to age range on Day 15 following once daily dosing of colchicine on Days 2 - 14 |
Who may qualify
- Ages
- 2 to 65 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Patients age 2-65 years with a confirmed clinical diagnosis of FMF,
- Non-pregnant, and
- If of child-bearing potential, using effective contraceptive measures.
Key exclusion criteria (6 listed)
- Recent participation (within 30 days) in other research studies,
- Pregnant or lactating,
- History or current infection of human immunodeficiency virus (HIV), hepatitis A, B or C,
- Current or recent use of any drugs/drug classes or combinations thereof that may affect the absorption or metabolism of colchicine,
- Clinically relevant abnormal clinical laboratories at screening,
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 |
|---|---|---|
| colchicine sprinkle capsules0.3 mg | Drug | Colchicine |
| colchicine sprinkle capsules0.3 mg | Drug | colchicine at steady state |
Primary outcome
Maximum Plasma Concentration
Time frame: 15 days
pharmacokinetic samples collected pre-dose on Days 1, 2 and 15 and at 0.25 - 0.5 hours, 1.5-2.5 hours and at 5-8 hours post-dose on Days 1 and 15
Area Under the Concentration Time Curve from Time Zero to the Time of Last Measured Concentration (AUC 0-t)
Time frame: 15 days
Pharmacokinetic samples collected pre-dose on Days 1,2 and 15 and at 0.25-0.5 hours, 1.5-2.5 hours and 5-8 hours post-dose on Days 1 and 15.
Area Under the Concentration Time Curve from Zero through Infinity
Time frame: 15 days
Pharmacokinetic samples collected pre-dose on Days 1, 2 and 15 and at 0.25-0.5 hours, 1.5-2.5 hours and at 5-8 hours post-dose on Days 1 and 15
1 secondary outcome
Acute Phase Reactant (ESR, CRP, SAA) Levels
Time frame: 15 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jan 2012.
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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.
Matthew Davis, MD study chair
Mutual Pharmaceutical Company, Inc.
10 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing colchicine sprinkle capsules in people with Familial Mediterranean Fever, run by Mutual Pharmaceutical Company, Inc. at 10 sites in 4 countries.
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 6 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: finished, as of the sponsor's last update on 10 Jan 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in Israel, Turkey (Türkiye), United States, Armenia. 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. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maximum Plasma Concentration (time frame: 15 days). The study lists 3 primary outcomes in all. 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 10 Jan 2012.