Pharmacokinetic Study With Assessment of Safety and Tolerability of Omigapil in Children and Adolescents With Congenital Muscular Dystrophy (CALLISTO)

This study is not yet open for participant recruitment.
Verified December 2013 by Santhera Pharmaceuticals
Sponsor:
Information provided by (Responsible Party):
Santhera Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT01805024
First received: March 4, 2013
Last updated: December 6, 2013
Last verified: December 2013
  Purpose

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.


Condition Intervention Phase
Congenital Muscular Dystrophy
Drug: Omigapil
Phase 1

Study Type: Interventional
Study Design: Endpoint Classification: Pharmacokinetics Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Santhera Pharmaceuticals:

Primary Outcome Measures:
  • Primary objective: to establish the pharmacokinetic profile of omigapil at a range of doses in paediatric and adolescent patients with CMD. [ Time Frame: Week 4 and Week 8 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Secondary objective: to evaluate the safety and tolerability of omigapil at a range of doses in paediatric and adolescent patients. [ Time Frame: Baseline, Week 4, 8 and 12 ] [ Designated as safety issue: Yes ]

Other Outcome Measures:
  • Tertiary objective: to establish the feasibility of conducting disease-relevant clinical assessments in paediatric and adolescent patients with CMD to aid in the design of future studies [ Time Frame: Baseline, Week 12 and 16 ] [ Designated as safety issue: No ]

Estimated Enrollment: 24
Study Start Date: May 2014
Estimated Primary Completion Date: December 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Omigapil Drug: Omigapil

  Eligibility

Ages Eligible for Study:   5 Years to 16 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Ambulatory and non-ambulatory patients from age 5 - 16 years with a clinical picture (see below) consistent with Ullrich CMD or MDC1A (LAMA2/merosin deficient CMD)
  • Under regular review at a neuromuscular centre
  • On adequate double-barrier contraception (if of child-bearing potential)
  • Stable on any allowed concomitant medications for 1 month prior to run in phase
  • FVC < 80% of the predicted value at Screening and Baseline visit(s)

For patients with Ullrich CMD - required clinical picture:

• Muscle weakness: inability to walk or, if patient is still ambulatory, inability to run and > 5 s for 10 m walk

Genetics and Pathology:

  • Molecular diagnosis of collagen VI related myopathy, defined by one dominant or two recessive COL6A1, COL6A2 or COL6A3 mutation(s) 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 LAMA2 deficient CMD (MDC1A) - 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 aDG-RD

Exclusion Criteria:

  • Use of any investigational drug other than the study medication within 3 months of study start.
  • Recurrent hospitalisation for chest infections in previous 2 years (≥2 per year)
  • Patients with respiratory parameters currently affected by concomitant medications or 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 6 months or foreseen during the course of the study.
  • Any intercurrent significant medical condition which in the opinion of the Investigator or the study Medical Monitor is suspected of potentially affecting the patient's safety or compliance with the study procedures
  • Failure to thrive, defined as:

    • Falling 20 percentiles (20/100) in body weight in the 3 months preceding Screening/Baseline
    • In patients below the 3rd percentile, any further drop in body weight percentile in the 3 months preceding Screening/Baseline
  • 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
  • FVC% predicted <30%
  • On Non Invasive Ventilation (NIV) Expectation of non-compliance with study procedures (i.e. study medication intake and/or PK blood sampling) excluding exploratory efficacy assessments
  • Intake of prohibited medication (as listed in Appendix A of the protocol)
  • Patients with renal impairment defined as urinary protein concentration ≥ 0.2 g/L
  • Patients with moderate to severe hepatic impairment
  Contacts and Locations
No Contacts or Locations Provided
  More Information

No publications provided

Responsible Party: Santhera Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01805024     History of Changes
Other Study ID Numbers: SNT-I-015
Study First Received: March 4, 2013
Last Updated: December 6, 2013
Health Authority: United States: Food and Drug Administration
United Kingdom: Medicines and Healthcare Products Regulatory Agency

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on April 23, 2014