Congenital Muscular DystrophyPhase 1CALLISTONCT01805024
Congenital Muscular Dystrophy Ascending Multiple Dose Cohort Study Analyzing Pharmacokinetics at Three Dose Levels In Children and Adolescents With Assessment of Safety and Tolerability of Omigapil (CALLISTO)
A Phase 1 study testing Omigapil in people with Congenital Muscular Dystrophy, run by Santhera Pharmaceuticals at 1 site in United States.
Trial summary
The purpose of the study is to establish the pharmacokinetic profile of omigapil in paediatric and adolescent patients with CMD and to evaluate the safety and tolerability of omigapil. Funding source - FDA OOPD Read more about this study
- Who is it for?
- Ages 5 to 16 years
- Conditions the study is for
- Congenital Muscular Dystrophy
- What is being tested?
- Omigapil
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2014to 29 Jan 2018
- Phase
- Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 5 to 16 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Ambulatory and non-ambulatory patients from age 5 - 16 years (5 years old and <17 years old) at time of screening with a clinical picture (see below) consistent with COL6-related dystrophy (COL6-RD) or LAMA2-related dystrophy (LAMA2-RD)
- Under regular review at a neuromuscular center
- On adequate double-barrier contraception (if of child-bearing potential)
- Stable on any allowed concomitant medications for 1 month prior to run in Phase
- Forced Vital Capacity (FVC) 30-80% of the predicted value and confirmed at Screening and Baseline visit(s)
- For patients with Collagen VI-related dystrophy (COL6-RD)- required clinical picture:
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- Muscle weakness: inability to walk or, if patient is still ambulatory, inability to run and > 5 s for 10 m walk
- Genetic and Pathology:
- Molecular diagnosis of COL6-RD, defined by one dominant or two recessive mutation(s) in COL6A1, COL6A2 or COL6A3 known to cause the clinical picture,,
- OR
- Histological diagnosis showing (i) absent or significantly decreased expression of collagen VI in muscle (overall reduction or basal lamina specific) or (ii) absent or significantly abnormal matrix in skin fibroblast culture
- For patients with Laminin alpha 2 related dystrophy (LAMA2-RD) - required clinical picture:
- Muscle weakness: Inability to walk; if patient is still ambulatory, inability to run and > 5 s for 10 m walk.
- Genetics and Pathology:
- Either: 2 identified pathologic or probable pathologic mutations in LAMA2 gene
- OR:
- 1 identified pathologic or probable pathologic mutation in LAMA 2 gene with evidence of decrease in laminin alpha 2 staining on muscle or skin biopsy
- OR:
- Evidence of decrease in laminin alpha 2 staining on muscle or skin biopsy with matching clinical phenotype and no suspicion of alpha dystroglycanopathy (aDG-RD) (clinically or by staining on muscle biopsy)
Who cannot join
- Use of any investigational drug other than the study medication within 12 weeks of study start.
- Recurrent hospitalisation for chest infections in previous 2 years (≥2 per year)
- Patients with respiratory parameters (eg: low pulmonary function test value i.e. <30% or need for brief course of daytime non-invasive ventilation) currently affected by short term medications, or acute illness/ conditions (conduct baseline assessments when the patient has recovered and no longer taking acute medication)
- Any need for surgery (scoliosis, gastrostomy, other) in the preceding 24 weeks or foreseen during the course of the study.
- Patient has an intercurrent significant medical condition or situation which in the opinion of the Investigator or the study Medical Monitor may put the patient at significant risk, confound the study results or interfere significantly with the patient's participation in the study
- Failure to thrive, defined as:
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- Falling 20 percentiles (20/100) in body weight in the 12 weeks preceding Screening/Baseline (based on family report of weight loss and acquiring relevant medical records)
- In patients below the 3rd percentile, any further drop in body weight percentile in the 12 weeks preceding Screening/Baseline (based on family report of weight loss and acquiring relevant medical records)
- Weight less than 17kg at Baseline
- Morbidly obese or grossly overweight (≥86 percentile BMI in children)
- History of epilepsy or on antiepileptic medication at Screening/Baseline
- Diabetes
- On daytime Non Invasive Ventilation (NIV)
- Intake of prohibited medication (as listed in Appendix I)
- Anticipated need for anesthesia during the course of this study
- Patients with renal impairment defined as urinary protein concentration ≥ 0.2 g/L
- Patients with moderate to severe hepatic impairment
- ALT ≥ 8x upper limit of normal (ULN) and total bilirubin 2x ULN (plus >35% 'direct' bilirubin), or
- ALT ≥ 8x ULN and INR >1.5 or ALT >2x baseline levels, and total bilirubin > 2x ULN (plus >35% 'direct' bilirubin), or
- ALT >2x baseline levels, and INR greater than 1.5,
Trial locations and contacts
1 site in 1 country.
About this study
The purpose of the study is to establish the pharmacokinetic profile of omigapil in paediatric and adolescent patients with CMD and to evaluate the safety and tolerability of omigapil.
Funding source - FDA OOPD
- Conditions
- Congenital Muscular Dystrophy
- Being tested
- Omigapil
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OmigapilDrug
Cohort 1 0.02 mg/kg/day Cohort 2 0.08 mg/kg/day Cohort 3a 0.04 mg/kg/day Cohort 3b 0.06 mg/kg/day
| Study group | Role | What they receive |
|---|---|---|
| Omigapil | Experimental | Omigapil treatment oral administration once per day after breakfast Cohort 1 Cohort 1 0.02 mg/kg/day Cohort 2 0.08 mg/kg/day Cohort 3a 0.04 mg/kg/day Cohort 3b 0.06 mg/kg/day Receives: Drug: Omigapil |
What is being measured
The primary outcome is the main question the study is designed to answer.
Pharmacokinetic Profile of Omigapil:Maximum Observed Plasma Concentration (Cmax) of Omigapil
Measured: 0.5, 1, 1.5, 2, 4 and 8 hours post-dose on Day 1, Week 4 and Week 12
Pharmacokinetic Profile of Omigapil: Time at Which Cmax Was Apparent (Tmax) of Omigapil
Measured: 0.5, 1, 1.5, 2, 4 and 8 hours post-dose on Day 1, Week 4 and Week 12
Pharmacokinetic Profile of Omigapil Area Under the Plasma Concentration Versus Time Curve From Time Zero to 8h Post-dose (AUC0-8)
Measured: 0 to 8 hours post-dose on Day 1, Week 4, Week 12
Secondary outcomes (1)
Summary of All Treatment-emergent Adverse Events (TEAEs)
TEAEs reported during the treatment period
Measured: 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Mar 2013 (estimated)
- Study startDec 2014
- Primary completion5 Dec 2017
- Study completion29 Jan 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Congenital Muscular DystrophyStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Santhera Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01805024
- Sponsor's protocol ID
- SNT-I-015
- Other IDs
- FDA-OPD 5076
- Acronym
- CALLISTO
- Status last verified
- Sep 2021
- First submitted
- 4 Mar 2013
- Last updated
- 24 Sep 2021
- Results posted
- Yes, on ClinicalTrials.gov
Publications
Foley AR, Yun P, Leach ME, Neuhaus SB, Averion GV, Hu Y, Hayes LH, Donkervoort S, Jain MS, Waite M, Parks R, Bharucha-Goebel DX, Mayer OH, Zou Y, Fink M, DeCoster J, Mendoza C, Arevalo C, Hausmann R, Petraki D, Cheung K, Bonnemann CG. Phase 1 Open-Label Study of Omigapil in Patients With LAMA2- or COL6-Related Dystrophy. Neurol Genet. 2024 May 29;10(3):e200148. doi: 10.1212/NXG.0000000000200148. eCollection 2024 Jun.
PubMed 38915423
Study at a glance
From the registry record, last updated by the sponsor on 24 Sep 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Congenital Muscular Dystrophy Ascending Multiple Dose Cohort Study Analyzing Pharmacokinetics at Three Dose Levels In Children and Adolescents With Assessment of Safety and Tolerability of Omigapil (CALLISTO)
- ClinicalTrials.gov ID
- NCT01805024
- Acronym / sponsor ID
- CALLISTO / SNT-I-015
- Conditions
- Congenital Muscular Dystrophy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Santhera PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Dec 2014
- Primary completion
- 5 Dec 2017 (actual)
- Study completion
- 29 Jan 2018 (actual)
- First posted
- 5 Mar 2013 (estimated)
- Results
- Posted 20 Sep 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of the study is to establish the pharmacokinetic profile of omigapil in paediatric and adolescent patients with CMD and to evaluate the safety and tolerability of omigapil.
Funding source - FDA OOPD
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Omigapil | Experimental | Drug: OmigapilOmigapil treatment oral administration once per day after breakfast Cohort 1 Cohort 1 0.02 mg/kg/day Cohort 2 0.08 mg/kg/day Cohort 3a 0.04 mg/kg/day Cohort 3b 0.06 mg/kg/day |
Who may qualify
- Ages
- 5 to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (19 listed)
- Ambulatory and non-ambulatory patients from age 5 - 16 years (5 years old and <17 years old) at time of screening with a clinical picture (see below) consistent with COL6-related dystrophy (COL6-RD) or LAMA2-related dystrophy (LAMA2-RD)
- Under regular review at a neuromuscular center
- On adequate double-barrier contraception (if of child-bearing potential)
- Stable on any allowed concomitant medications for 1 month prior to run in Phase
- Forced Vital Capacity (FVC) 30-80% of the predicted value and confirmed at Screening and Baseline visit(s)
Key exclusion criteria (20 listed)
- Use of any investigational drug other than the study medication within 12 weeks of study start.
- Recurrent hospitalisation for chest infections in previous 2 years (≥2 per year)
- Patients with respiratory parameters (eg: low pulmonary function test value i.e. <30% or need for brief course of daytime non-invasive ventilation) currently affected by short term medications, or acute illness/ conditions (conduct baseline assessments when the patient has recovered and no longer taking acute medication)
- Any need for surgery (scoliosis, gastrostomy, other) in the preceding 24 weeks or foreseen during the course of the study.
- Patient has an intercurrent significant medical condition or situation which in the opinion of the Investigator or the study Medical Monitor may put the patient at significant risk, confound the study results or interfere significantly with the patient's participation in the study
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 |
|---|---|---|
| OmigapilCohort 1 0.02 mg/kg/day Cohort 2 0.08 mg/kg/day Cohort 3a 0.04 mg/kg/day Cohort 3b 0.06 mg/kg/day | Drug | Omigapil |
Primary outcome
Pharmacokinetic Profile of Omigapil:Maximum Observed Plasma Concentration (Cmax) of Omigapil
Time frame: 0.5, 1, 1.5, 2, 4 and 8 hours post-dose on Day 1, Week 4 and Week 12
Pharmacokinetic Profile of Omigapil: Time at Which Cmax Was Apparent (Tmax) of Omigapil
Time frame: 0.5, 1, 1.5, 2, 4 and 8 hours post-dose on Day 1, Week 4 and Week 12
Pharmacokinetic Profile of Omigapil Area Under the Plasma Concentration Versus Time Curve From Time Zero to 8h Post-dose (AUC0-8)
Time frame: 0 to 8 hours post-dose on Day 1, Week 4, Week 12
1 secondary outcome
Summary of All Treatment-emergent Adverse Events (TEAEs)
Time frame: 12 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Sep 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 38915423Foley AR, Yun P, Leach ME, Neuhaus SB, Averion GV, Hu Y, Hayes LH, Donkervoort S, Jain MS, Waite M, Parks R, Bharucha-Goebel DX, Mayer OH, Zou Y, Fink M, DeCoster J, Mendoza C, Arevalo C, Hausmann R, Petraki D, Cheung K, Bonnemann CG. Phase 1 Open-Label Study of Omigapil in Patients With LAMA2- or COL6-Related Dystrophy. Neurol Genet. 2024 May 29;10(3):e200148. doi: 10.1212/NXG.0000000000200148. eCollection 2024 Jun.
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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.
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 Omigapil in people with Congenital Muscular Dystrophy, run by Santhera Pharmaceuticals 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 19 inclusion and 20 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 24 Sep 2021. 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. 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?
Yes. Results are posted in the registry (first posted 20 Sep 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Pharmacokinetic Profile of Omigapil:Maximum Observed Plasma Concentration (Cmax) of Omigapil (time frame: 0.5, 1, 1.5, 2, 4 and 8 hours post-dose on Day 1, Week 4 and Week 12). 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 NCT01805024, 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 24 Sep 2021.