AniridiaPhase 2NCT04117880
A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia
A Phase 2 study testing Ataluren in people with Aniridia, run by PTC Therapeutics.
Trial summary
This is a Phase 2, multicenter, open-label study evaluating the overall systemic and ocular safety profile of ataluren in nonsense mutation aniridia as determined by the incidences of treatment-emergent adverse events (TEAEs) as well as abnormal findings on laboratory assessments, vital signs, physical examinations… Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Aniridia
- What is being tested?
- Ataluren
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 31 Dec 2018to 31 Jan 2021
- 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 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Participants who will be selected for this study must meet the following criteria:
- Evidence of signed and dated informed consent document(s) indicating that the study candidate (and/or a parent/legal guardian) has been informed of all pertinent aspects of the study. Note: If the study candidate is considered a child under local regulation, a parent or legal guardian must provide written consent prior to initiation of study screening procedures and the study candidate may be required to provide written assent. The rules of the responsible institutional review board/independent ethics committee (IRB/IEC) regarding whether one or both parents must provide consent and the appropriate ages for obtaining consent and assent from the participant should be followed.
- Must have participated in and exited from Study PTC124-GD-028 ANI
- Age ≥2 years and of either gender.
- Body weight ≥12 kg.
- Willingness and ability to comply with scheduled visits, drug administration plan, study procedures, and study restrictions.
Show all 18Show fewer
- Good general health.
- Female participants of childbearing potential are eligible for the study but must be willing to use adequate (at least 1 form of) contraceptive methods as described below during the study treatment period (starting from the day of first dose of study drug and ending 60 days after the last dose of study drug). Childbearing potential is defined as participants who have experienced menarche and who are neither postmenopausal or have been permanently sterilized.
- Hormonal methods of contraception (including oral and transdermal contraceptives, injectable progesterone, progestin subdermal implants, progesterone-releasing intrauterine contraceptive devices [IUDs]) initiated at least 14 days prior to the first dose of study drug
- Abstinence
- Placement of a copper-containing IUD
- Condom with spermicidal foam/gel/film/cream/suppository
- Postmenopausal at least 12 months prior to first dose of study drug or permanently sterilized (for example, tubal occlusion, hysterectomy, bilateral salpingectomy)
- Male partner who has had a vasectomy for at least 3 months prior to the first dose of study drug
- Male participants with partners of childbearing potential must agree to use the following adequate (at least 1 form of) contraception during the study treatment period (starting from the day of first dose of study drug and ending 60 days after the last dose of study drug):
- Abstinence
- Vasectomy for at least 3 months prior to first dose of study drug or surgically sterile
- Without a vasectomy, must use a condom with spermicidal foam/gel/film/cream suppository
Who cannot join
- The presence of any of the following conditions will exclude a participant from study enrollment:
- General exclusion criteria
- Participation in any drug or device clinical investigation (other than Study PTC124-GD-028 ANI) within 90 days prior to Visit 1 (Screening) or anticipation of participating in any other drug or device clinical investigation during this study.
- Surgery within 30 days prior to enrollment.
- Female participants who are pregnant or breastfeeding. Female participants of childbearing potential must have a negative pregnancy test (beta-human chorionic gonadotropin [β-HCG]) at screening and must use adequate (at least 1 form of) contraceptive methods.
- Active ocular infection or inflammation.
- Prior or ongoing medical condition (for example, concomitant illness, alcoholism, drug abuse, psychiatric condition), medical history, physical 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 study drug administration or follow-up would be completed, or could impair the assessment of study results.
- Participants with a positive result for hepatitis B, hepatitis C, or human immunodeficiency virus at Visit 1 (Screening).
About this study
Official title: Nonsense Mutation Aniridia: An Ataluren (PTC124) Phase 2 Extension Study
This is a Phase 2, multicenter, open-label study evaluating the overall systemic and ocular safety profile of ataluren in nonsense mutation aniridia as determined by the incidences of treatment-emergent adverse events (TEAEs) as well as abnormal findings on laboratory assessments, vital signs, physical examinations, ophthalmoscopy, and slit-lamp examination.
Participants who complete PTC124-GD-028 ANI (NCT02647359) meet all inclusion and none of the exclusion criteria will be enrolled into this study.
Read the detailed description
This study was a planned extension study of PTC124-GD-028 ANI (NCT02647359) and was never initiated.
- Conditions
- Aniridia
- Being tested
- Ataluren
- Medical subject headings
- Aniridia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AtalurenDrug
Also called PTC124
Oral
| Study group | Role | What they receive |
|---|---|---|
| Ataluren | Experimental | Ataluren Oral suspension taken 3 times per day (10 mg/kg in the morning, 10 mg/kg at mid-day, and 20 mg/kg in the evening). Receives: Drug: Ataluren |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Measured: 104 weeks
Secondary outcomes (2)
Change From Baseline in Visual Acuity at Week 104
Measured: 104 weeks
Change From Baseline in Severity of Corneal Keratopathy at Week 104
Measured: 104 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Oct 2019
- Study start31 Dec 2018 (estimated)
- Primary completion31 Jan 2021 (estimated)
- Study completion31 Jan 2021 (estimated)
Why it stopped: Study cancelled before protocol was implemented.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AniridiaStudies 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
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- PTC Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04117880
- Sponsor's protocol ID
- PTC124-GD-028e- ANI
- Status last verified
- Sep 2019
- First submitted
- 19 Sep 2019
- Last updated
- 7 Oct 2019
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Oct 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Nonsense Mutation Aniridia: An Ataluren (PTC124) Phase 2 Extension Study
- ClinicalTrials.gov ID
- NCT04117880
- Acronym / sponsor ID
- PTC124-GD-028e- ANI
- Conditions
- Aniridia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Study cancelled before protocol was implemented.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- PTC TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 31 Dec 2018 (estimated)
- Primary completion
- 31 Jan 2021 (estimated)
- Study completion
- 31 Jan 2021 (estimated)
- First posted
- 7 Oct 2019 (actual)
- 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.
This is a Phase 2, multicenter, open-label study evaluating the overall systemic and ocular safety profile of ataluren in nonsense mutation aniridia as determined by the incidences of treatment-emergent adverse events (TEAEs) as well as abnormal findings on laboratory assessments, vital signs, physical examinations, ophthalmoscopy, and slit-lamp examination.
Participants who complete PTC124-GD-028 ANI (NCT02647359) meet all inclusion and none of the exclusion criteria will be enrolled into this study.
Detailed description
This study was a planned extension study of PTC124-GD-028 ANI (NCT02647359) and was never initiated.
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: AtalurenAtaluren Oral suspension taken 3 times per day (10 mg/kg in the morning, 10 mg/kg at mid-day, and 20 mg/kg in the evening). |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (18 listed)
- Evidence of signed and dated informed consent document(s) indicating that the study candidate (and/or a parent/legal guardian) has been informed of all pertinent aspects of the study. Note: If the study candidate is considered a child under local regulation, a parent or legal guardian must provide written consent prior to initiation of study screening procedures and the study candidate may be required to provide written assent. The rules of the responsible institutional review board/independent ethics committee (IRB/IEC) regarding whether one or both parents must provide consent and the appropriate ages for obtaining consent and assent from the participant should be followed.
- Must have participated in and exited from Study PTC124-GD-028 ANI
- Age ≥2 years and of either gender.
- Body weight ≥12 kg.
- Willingness and ability to comply with scheduled visits, drug administration plan, study procedures, and study restrictions.
Key exclusion criteria (8 listed)
- The presence of any of the following conditions will exclude a participant from study enrollment:
- General exclusion criteria
- Participation in any drug or device clinical investigation (other than Study PTC124-GD-028 ANI) within 90 days prior to Visit 1 (Screening) or anticipation of participating in any other drug or device clinical investigation during this study.
- Surgery within 30 days prior to enrollment.
- Female participants who are pregnant or breastfeeding. Female participants of childbearing potential must have a negative pregnancy test (beta-human chorionic gonadotropin [β-HCG]) at screening and must use adequate (at least 1 form of) contraceptive methods.
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: PTC124Oral | Drug | Ataluren |
Primary outcome
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Time frame: 104 weeks
2 secondary outcomes
Change From Baseline in Visual Acuity at Week 104
Time frame: 104 weeks
Change From Baseline in Severity of Corneal Keratopathy at Week 104
Time frame: 104 weeks
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Oct 2019.
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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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Ataluren in people with Aniridia, run by PTC Therapeutics.
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 18 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: withdrawn, as of the sponsor's last update on 7 Oct 2019. Status can change between updates, so confirm with the study contact before making plans.
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 was withdrawn before enrolling participants, so there are no results.
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?
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) (time frame: 104 weeks). 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 7 Oct 2019.