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)

This study is currently recruiting participants. (see Contacts and Locations)
Verified March 2015 by Santhera Pharmaceuticals
Sponsor:
Information provided by (Responsible Party):
Santhera Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT01805024
First received: March 4, 2013
Last updated: March 26, 2015
Last verified: March 2015
  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 (CALLISTO)

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: December 2014
Estimated Primary Completion Date: September 2016 (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 (5 years old or more and less than 17 years old) at time of screening 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

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)
  • FVC 30 - 80% of the predicted value and confirmed at Screening and Baseline visit(s)

Genetic 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.
  • FVC 30 - 80% of the predicted value and confirmed at Screening and Baseline visit(s)

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)

Exclusion Criteria:

  • 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:
  • 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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01805024

Locations
United States, Maryland
NINDS Recruiting
Bethesda, Maryland, United States, 20892
Contact: Carsten Bonnemann, MD       carsten.bonnemann@nih.gov   
Contact: Gilberto (Mike) Averion, BSN, RN    3015942760    averion.gilberto@nih.gov   
Sponsors and Collaborators
Santhera Pharmaceuticals
  More Information

No publications provided

Responsible Party: Santhera Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01805024     History of Changes
Obsolete Identifiers: NCT02326831
Other Study ID Numbers: SNT-I-015
Study First Received: March 4, 2013
Last Updated: March 26, 2015
Health Authority: United States: Food and Drug Administration

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

ClinicalTrials.gov processed this record on June 29, 2015