Cystic FibrosisPhase 2NCT00237380
Safety and Efficacy of Ataluren (PTC124) for Cystic Fibrosis
A Phase 2 study testing Ataluren in people with Cystic Fibrosis, run by PTC Therapeutics at 1 site in Israel.
Trial summary
In some participants with cystic fibrosis (CF), the disease is caused by a nonsense mutation (premature stop codon) in the gene that makes the cystic fibrosis transmembrane regulator (CFTR) protein. Ataluren has been shown to partially restore CFTR production in animals with CF due to a nonsense mutation. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Ataluren
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 1 siteIsrael
- Study dates
- 30 Nov 2005to 31 May 2006
- Phase
- Phase 2
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of CF based on documented evidence of a conclusively abnormal sweat test (sweat chloride >60 milliequivalents/litre [mEq/liter]).
- Abnormal chloride secretion as measured by TEPD (a less than -5 mV TEPD assessment of chloride secretion with chloride-free amiloride and isoproterenol).
- Presence of a nonsense mutation in one of the alleles of the CFTR gene.
- Age ≥18 years.
- Body weight ≥40 kg.
- Forced expiratory volume in 1 second (FEV1) ≥40% of predicted for age, gender, and height (Knudson standards).
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- Oxygen saturation (as measured by pulse oximetry) ≥92% on room air.
- Willingness of male and female participants, if not surgically sterile, to abstain from sexual intercourse or employ a barrier or medical method of contraception during the study drug administration and follow-up periods.
- Negative pregnancy test (for females of childbearing potential).
- Willingness and ability to comply with scheduled visits, drug administration plan, study procedures, and study restrictions.
- Ability to provide written informed consent.
Who cannot join
- Prior or ongoing medical condition, medical history, physical findings, ECG findings, or laboratory abnormality that, in the Investigator's opinion, could adversely affect the safety of the participant, makes it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results.
- Ongoing acute illness including acute upper or lower respiratory infections within 2 weeks before start of study treatment.
- History of major complications of lung disease within 2 months prior to start of study treatment.
- Abnormalities on screening chest x-ray suggesting clinically significant active pulmonary disease other than CF, or new, significant abnormalities that may be indicative of clinically significant active pulmonary involvement secondary to CF.
- Positive hepatitis B surface antigen, hepatitis C antibody test, or human immunodeficiency virus (HIV) test.
- Hemoglobin <10 grams per deciliter (g/dL).
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- Serum albumin <2.5 g/dL.
- Abnormal liver function (serum alanine aminotransferase [ALT], aspartate aminotransferase [AST], gamma-glutamyl transferase [GGT], alkaline phosphatase, lactate dehydrogenase [LDH], or total bilirubin > upper limit of normal).
- Abnormal renal function (serum creatinine >1.5 times upper limit of normal).
- Pregnancy or breast-feeding.
- History of solid organ or hematological transplantation.
- Exposure to another investigational drug within 14 days prior to start of study treatment.
- Ongoing participation in any other therapeutic clinical trial.
- Ongoing use of thiazolidinedione peroxisome proliferator-activated receptor gamma (PPAR γ) agonists, eg, rosiglitazone (Avandia® or equivalent) or pioglitazone (Actos® or equivalent)
- Change in intranasal medications (including use of corticosteroids, cromolyn, ipratropium bromide, phenylephrine, or oxymetazoline) within 14 days prior to start of study treatment.
- Change in treatment with systemic or inhaled corticosteroids within 14 days prior to start of study treatment.
- Use or requirement for inhaled gentamicin or amikacin within 14 days prior to start of study treatment or during study treatment.
- Requirement for systemic aminoglycoside antibiotics within 14 days prior to start of study treatment.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 2 Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis
In some participants with cystic fibrosis (CF), the disease is caused by a nonsense mutation (premature stop codon) in the gene that makes the cystic fibrosis transmembrane regulator (CFTR) protein. Ataluren has been shown to partially restore CFTR production in animals with CF due to a nonsense mutation. The main purpose of this study is to understand whether ataluren can safely increase functional CFTR protein in the cells of participants with CF due to a nonsense mutation.
Read the detailed description
In this study, participants with CF due to a nonsense mutation will be treated with a new investigational drug called ataluren. Evaluation procedures to determine if a participant qualifies for the study will be performed within 21 days prior to the start of treatment. Eligible participants who elect to enroll in the study will then participate in two 28-day treatment and follow-up periods (56 days total). There will be a 2-night stay at the clinical research center at the beginning and at the end of each 14 days of ataluren treatment, which means that there will be four 2-night stays at the clinical research center during the study.
One of the measurements for the study is transepithelial potential difference (TEPD), which is also known as nasal potential difference and provides a sensitive evaluation of sodium and chloride transport directly in secretory epithelial cells. TEPD assessments are made on the nasal epithelium cells lining the inferior turbinate because these cells are easier to access than the respiratory epithelial cells lining the lower airways and have been shown to have the same ion transport characteristics. As an endpoint, TEPD has the advantage that it can detect chloride transport changes that are a quantitative integration of the presence, functional activity, and apical location of the CFTR in airway cells. Furthermore, it is a direct measure of CFTR activity that is not likely to be affected by supportive or palliative treatments for CF (with the possible exception of systemically administered aminoglycoside antibiotics).
- Conditions
- Cystic Fibrosis
- Being tested
- Ataluren
- Keywords
- Cystic fibrosis, Nonsense mutation, Premature stop codon
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AtalurenDrug
Also called PTC124
Ataluren will be provided as a vanilla-flavored powder to be mixed with water.
| Study group | Role | What they receive |
|---|---|---|
| Ataluren | Experimental | Cycle 1: Within the first 28-day period, ataluren treatment will be taken 3 times per day with meals for 14 days at doses of 4 milligrams/kilogram (mg/kg) (breakfast), 4 mg/kg (lunch), and 8 mg/kg (dinner); there will then be an interval of 14 days without treatment.
Cycle 2: Within the second 28-day period, ataluren treatment will be taken 3 times per day with meals for 14 days at doses of 10 mg/kg (breakfast), 10 mg/kg (lunch), and 20 mg/kg (dinner); there will then be an interval of 14 days without treatment. Receives: Drug: Ataluren |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline to End of Treatment in Total Chloride Transport
Measured: Baseline of Cycle 1 and Cycle 2, Day 14 and Day 28 of Cycle 1 and Cycle 2 (1 cycle=28 days)
Number of Participants With a Chloride Transport Response
Measured: Baseline of Cycle 1 and Cycle 2 and Day 28 of Cycle 1 and Cycle 2 (1 cycle=28 days)
Number of Participants With a Chloride Transport Normalization Between Baseline and End of Treatment
Measured: Baseline of Cycle 1 and Cycle 2 and Day 28 of Cycle 1 and Cycle 2 (1 cycle=28 days)
Secondary outcomes (12)
Change From Baseline in Nasal Chloride Secretion as Assessed by Transepithelial Potential Difference (TEPD)
Measured: Baseline of Cycle 1 and Cycle 2, Day 14 and Day 28 of Cycle 1 and Cycle 2 (1 cycle=28 days)
Change From Baseline in Sweat Chloride Concentration as Determined by Pilocarpine Iontophoresis
Measured: Baseline, Day 41
Change From Baseline in CFTR Protein in Nasal Mucosa as Determined by Immunofluorescence
Measured: Baseline of Cycle 1 and Cycle 2, Day 14 and Day 28 of Cycle 1 and Cycle 2 (1 cycle=28 days)
Change From Baseline in Nonsense Mutation CFTR mRNA in Nasal Mucosa as Determined by Quantitative Polymerase Chain Reaction (PCR) Assay
Measured: Baseline of Cycle 1 and Cycle 2, Day 14 and Day 28 of Cycle 1 and Cycle 2 (1 cycle=28 days)
Change From Baseline in Number of Neutrophils in Blood
Measured: Baseline Up to Day 56
Change From Baseline in Pulmonary Function as Measured by Spirometry
Measured: Baseline of Cycle 1 and Cycle 2, Day 14 of Cycle 1 and Cycle 2 (1 cycle=28 days)
Change From Baseline in Body Weight
Measured: Baseline of Cycle 1 and Cycle 2, Day 28 of Cycle 1 and Cycle 2 (1 cycle=28 days)
Compliance with Study Treatment
Measured: Baseline Up to Day 56
Pharmacokinetics (PK): Time to Maximum Concentration (Tmax) of Ataluren
Measured: 0 (predose), 1, 2, 3, and 4 hours (hrs) postdose of the midday dose; 0 (predose), 1, 2, 3, 4, and 12 hrs postdose of the evening dose on Day 1 and Day 27
PK: Maximum Plasma Concentration (Cmax) of Ataluren
Measured: 0 (predose), 1, 2, 3, and 4 hrs postdose of the morning dose; 0 (predose), 1, 2, 3, and 4 hrs postdose of the midday dose; and 0 hrs (predose), 1, 2, 3, 4, and 12 hrs postdose of the evening dose on Days 1 and 13 of Cycles 1 and 2 (1 cycle=28 days)
PK: Area Under the Plasma Concentration Time Curve (AUC) of Ataluren
Measured: 0 (predose), 1, 2, 3, and 4 hrs postdose of the morning dose; 0 (predose), 1, 2, 3, and 4 hrs postdose of the midday dose; and 0 hrs (predose), 1, 2, 3, 4, and 12 hrs postdose of the evening dose on Days 1 and 13 of Cycles 1 and 2 (1 cycle=28 days)
PK: Terminal Elimination Half Life (T1/2) of Ataluren
Measured: 0 (predose), 1, 2, 3, and 4 hrs postdose of the morning dose; 0 (predose), 1, 2, 3, and 4 hrs postdose of the midday dose; and 0 hrs (predose), 1, 2, 3, 4, and 12 hrs postdose of the evening dose on Days 1 and 13 of Cycles 1 and 2 (1 cycle=28 days)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Oct 2005 (estimated)
- Study start30 Nov 2005
- Primary completion31 May 2006
- Study completion31 May 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- PTC Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Eiten Kerem, MD, Principal investigator, Hadassah University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00237380
- Sponsor's protocol ID
- PTC124-GD-005-CF
- Other IDs
- 20050188
- Status last verified
- Jun 2020
- First submitted
- 7 Oct 2005
- Last updated
- 11 Jun 2020
- Results posted
- No
Publications
Kerem E, Hirawat S, Armoni S, Yaakov Y, Shoseyov D, Cohen M, Nissim-Rafinia M, Blau H, Rivlin J, Aviram M, Elfring GL, Northcutt VJ, Miller LL, Kerem B, Wilschanski M. Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial. Lancet. 2008 Aug 30;372(9640):719-27. doi: 10.1016/S0140-6736(08)61168-X. Epub 2008 Aug 20.
PubMed 18722008
Study at a glance
From the registry record, last updated by the sponsor on 11 Jun 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT00237380
- Acronym / sponsor ID
- PTC124-GD-005-CF
- Conditions
- Cystic Fibrosis
- Keywords
- Cystic fibrosis, Nonsense mutation, Premature stop codon
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- PTC TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 30 Nov 2005 (actual)
- Primary completion
- 31 May 2006 (actual)
- Study completion
- 31 May 2006 (actual)
- First posted
- 12 Oct 2005 (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.
In some participants with cystic fibrosis (CF), the disease is caused by a nonsense mutation (premature stop codon) in the gene that makes the cystic fibrosis transmembrane regulator (CFTR) protein. Ataluren has been shown to partially restore CFTR production in animals with CF due to a nonsense mutation. The main purpose of this study is to understand whether ataluren can safely increase functional CFTR protein in the cells of participants with CF due to a nonsense mutation.
Detailed description
In this study, participants with CF due to a nonsense mutation will be treated with a new investigational drug called ataluren. Evaluation procedures to determine if a participant qualifies for the study will be performed within 21 days prior to the start of treatment. Eligible participants who elect to enroll in the study will then participate in two 28-day treatment and follow-up periods (56 days total). There will be a 2-night stay at the clinical research center at the beginning and at the end of each 14 days of ataluren treatment, which means that there will be four 2-night stays at the clinical research center during the study.
One of the measurements for the study is transepithelial potential difference (TEPD), which is also known as nasal potential difference and provides a sensitive evaluation of sodium and chloride transport directly in secretory epithelial cells. TEPD assessments are made on the nasal epithelium cells lining the inferior turbinate because these cells are easier to access than the respiratory epithelial cells lining the lower airways and have been shown to have the same ion transport characteristics. As an endpoint, TEPD has the advantage that it can detect chloride transport changes that are a quantitative integration of the presence, functional activity, and apical location of the CFTR in airway cells. Furthermore, it is a direct measure of CFTR activity that is not likely to be affected by supportive or palliative treatments for CF (with the possible exception of systemically administered aminoglycoside antibiotics).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Ataluren | Experimental | Drug: AtalurenCycle 1: Within the first 28-day period, ataluren treatment will be taken 3 times per day with meals for 14 days at doses of 4 milligrams/kilogram (mg/kg) (breakfast), 4 mg/kg (lunch), and 8 mg/kg (dinner); there will then be an interval of 14 days without treatment. Cycle 2: Within the second 28-day period, ataluren treatment will be taken 3 times per day with meals for 14 days at doses of 10 mg/kg (breakfast), 10 mg/kg (lunch), and 20 mg/kg (dinner); there will then be an interval of 14 days without treatment. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Diagnosis of CF based on documented evidence of a conclusively abnormal sweat test (sweat chloride >60 milliequivalents/litre [mEq/liter]).
- Abnormal chloride secretion as measured by TEPD (a less than -5 mV TEPD assessment of chloride secretion with chloride-free amiloride and isoproterenol).
- Presence of a nonsense mutation in one of the alleles of the CFTR gene.
- Age ≥18 years.
- Body weight ≥40 kg.
Key exclusion criteria (18 listed)
- Prior or ongoing medical condition, medical history, physical findings, ECG findings, or laboratory abnormality that, in the Investigator's opinion, could adversely affect the safety of the participant, makes it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results.
- Ongoing acute illness including acute upper or lower respiratory infections within 2 weeks before start of study treatment.
- History of major complications of lung disease within 2 months prior to start of study treatment.
- Abnormalities on screening chest x-ray suggesting clinically significant active pulmonary disease other than CF, or new, significant abnormalities that may be indicative of clinically significant active pulmonary involvement secondary to CF.
- Positive hepatitis B surface antigen, hepatitis C antibody test, or human immunodeficiency virus (HIV) test.
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 |
|---|---|---|
| AtalurenAlso: PTC124Ataluren will be provided as a vanilla-flavored powder to be mixed with water. | Drug | Ataluren |
Primary outcome
Change From Baseline to End of Treatment in Total Chloride Transport
Time frame: Baseline of Cycle 1 and Cycle 2, Day 14 and Day 28 of Cycle 1 and Cycle 2 (1 cycle=28 days)
Number of Participants With a Chloride Transport Response
Time frame: Baseline of Cycle 1 and Cycle 2 and Day 28 of Cycle 1 and Cycle 2 (1 cycle=28 days)
Number of Participants With a Chloride Transport Normalization Between Baseline and End of Treatment
Time frame: Baseline of Cycle 1 and Cycle 2 and Day 28 of Cycle 1 and Cycle 2 (1 cycle=28 days)
12 secondary outcomes
Change From Baseline in Nasal Chloride Secretion as Assessed by Transepithelial Potential Difference (TEPD)
Time frame: Baseline of Cycle 1 and Cycle 2, Day 14 and Day 28 of Cycle 1 and Cycle 2 (1 cycle=28 days)
Change From Baseline in Sweat Chloride Concentration as Determined by Pilocarpine Iontophoresis
Time frame: Baseline, Day 41
Change From Baseline in CFTR Protein in Nasal Mucosa as Determined by Immunofluorescence
Time frame: Baseline of Cycle 1 and Cycle 2, Day 14 and Day 28 of Cycle 1 and Cycle 2 (1 cycle=28 days)
Change From Baseline in Nonsense Mutation CFTR mRNA in Nasal Mucosa as Determined by Quantitative Polymerase Chain Reaction (PCR) Assay
Time frame: Baseline of Cycle 1 and Cycle 2, Day 14 and Day 28 of Cycle 1 and Cycle 2 (1 cycle=28 days)
Change From Baseline in Number of Neutrophils in Blood
Time frame: Baseline Up to Day 56
Change From Baseline in Pulmonary Function as Measured by Spirometry
Time frame: Baseline of Cycle 1 and Cycle 2, Day 14 of Cycle 1 and Cycle 2 (1 cycle=28 days)
Change From Baseline in Body Weight
Time frame: Baseline of Cycle 1 and Cycle 2, Day 28 of Cycle 1 and Cycle 2 (1 cycle=28 days)
Compliance with Study Treatment
Time frame: Baseline Up to Day 56
Pharmacokinetics (PK): Time to Maximum Concentration (Tmax) of Ataluren
Time frame: 0 (predose), 1, 2, 3, and 4 hours (hrs) postdose of the midday dose; 0 (predose), 1, 2, 3, 4, and 12 hrs postdose of the evening dose on Day 1 and Day 27
PK: Maximum Plasma Concentration (Cmax) of Ataluren
Time frame: 0 (predose), 1, 2, 3, and 4 hrs postdose of the morning dose; 0 (predose), 1, 2, 3, and 4 hrs postdose of the midday dose; and 0 hrs (predose), 1, 2, 3, 4, and 12 hrs postdose of the evening dose on Days 1 and 13 of Cycles 1 and 2 (1 cycle=28 days)
PK: Area Under the Plasma Concentration Time Curve (AUC) of Ataluren
Time frame: 0 (predose), 1, 2, 3, and 4 hrs postdose of the morning dose; 0 (predose), 1, 2, 3, and 4 hrs postdose of the midday dose; and 0 hrs (predose), 1, 2, 3, 4, and 12 hrs postdose of the evening dose on Days 1 and 13 of Cycles 1 and 2 (1 cycle=28 days)
PK: Terminal Elimination Half Life (T1/2) of Ataluren
Time frame: 0 (predose), 1, 2, 3, and 4 hrs postdose of the morning dose; 0 (predose), 1, 2, 3, and 4 hrs postdose of the midday dose; and 0 hrs (predose), 1, 2, 3, 4, and 12 hrs postdose of the evening dose on Days 1 and 13 of Cycles 1 and 2 (1 cycle=28 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 11 Jun 2020.
- Derived from this study: PubMed 18722008Kerem E, Hirawat S, Armoni S, Yaakov Y, Shoseyov D, Cohen M, Nissim-Rafinia M, Blau H, Rivlin J, Aviram M, Elfring GL, Northcutt VJ, Miller LL, Kerem B, Wilschanski M. Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial. Lancet. 2008 Aug 30;372(9640):719-27. doi: 10.1016/S0140-6736(08)61168-X. Epub 2008 Aug 20.
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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.
Eiten Kerem, MD principal investigator
Hadassah University Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Ataluren in people with Cystic Fibrosis, run by PTC Therapeutics at 1 site in Israel.
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 11 inclusion and 18 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 11 Jun 2020. 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 Israel. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Change From Baseline to End of Treatment in Total Chloride Transport (time frame: Baseline of Cycle 1 and Cycle 2, Day 14 and Day 28 of Cycle 1 and Cycle 2 (1 cycle=28 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?
On the ClinicalTrials.gov record for NCT00237380, which also lists 1 linked publication.
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 11 Jun 2020.