Phase 1 N=20 Treatment

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)

Congenital Muscular Dystrophy

Bottom Line

View on ClinicalTrials.gov: NCT01805024 ↗

Enrolled (actual)

Serious AEs

0.0%

Results posted

Sep 2019

Primary outcome: Primary: Pharmacokinetic Profile of Omigapil:Maximum Observed Plasma Concentration (Cmax) of Omigapil — 1.05; 4.75; 2.15; 3.27 ng/mL

Study Design & Population

Study type: Interventional
Phase: Phase 1
Interventions: Omigapil (Drug)
Age: Pediatric · 5+ yrs
Sex: All
Sponsor: Santhera Pharmaceuticals
Primary completion: Dec 2017

Outcome Measures

Outcome	Result	p-value
PRIMARY Pharmacokinetic Profile of Omigapil:Maximum Observed Plasma Concentration (Cmax) of Omigapil	1.05; 4.75; 2.15; 3.27; 1.10; 10.5	—
PRIMARY Pharmacokinetic Profile of Omigapil: Time at Which Cmax Was Apparent (Tmax) of Omigapil	0.8; 0.8; 0.8; 0.5; 0.6; 0.5	—
PRIMARY Pharmacokinetic Profile of Omigapil Area Under the Plasma Concentration Versus Time Curve From Time Zero to 8h Post-dose (AUC0-8)	3.37; 15.7; 7.12; 10.8; 3.47; 29.7	—
SECONDARY Summary of All Treatment-emergent Adverse Events (TEAEs)	41; 40; 29; 75; 0; 0	—

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

Eligibility Criteria

Inclusion Criteria

Ambulatory and non-ambulatory patients from age 5 - 16 years (5 years old 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,,

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)

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. 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,

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01805024). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.