An open single center study investigating the effects and side-effects of CRD007 in children with Duchenne Muscular Dystrophy (DMD -an inherited disorder which results in muscle degeneration) or Becker Muscular Dystrophy (BMD- an inherited disorder characterized by slowly progressive muscle weakness of the legs and pelvis) or children being symptomatic carriers for DMD or BMD.

An open-label, un-controlled, single-centre trial investigating the efficacy and safety of CRD007 tablets administered twice daily for 12 weeks in children with Duchenne Muscular Dystrophy (DMD) or Becker Muscular Dystrophy (BMD) or children being symptomatic carriers for DMD or BMD. - CRD007 for the treatment of DMD, BMD and symptomatic carriers

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2011-004667-76-SE

Enrollment

Unknown

Registered

2011-10-21

Start date

2011-12-19

Completion date

Unknown

Last updated

2013-05-13

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy (BMD) and symptomatic carriers for DMD or BMD MedDRA version: 14.1 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 14.1 Level: PT Classification code 10059117 Term: Becker's muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 14.1 Level: PT Classification code 100

Interventions

Product Name: pemirolast Product Code: CRD007 Pharmaceutical Form: Tablet INN or Proposed INN: PEMIROLAST CAS Number: 69372-19-6 Current Sponsor code: CRD007 Concentration unit: mg milligram(s) Conce

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1.Documented diagnosis of dystrophinopathy in the form of mutation analysis in the dystrophin gene 2.Clinically documented delayed motor skills and muscle weakness 3.Prior documented elevated creatine kinase (>5 x ULN) 4.Age =65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: 1.Severe functional impairment (e.g. non-walkers) resulting in inability to perform meaningful repeated assessment of functional capabilities, according to the investigator’s judgment 2.Moderate or severe hepatic impairment or severe renal impairment, according to the investigator’s judgment 3.Prior or ongoing medical condition or laboratory abnormality that in the investigator’s opinion could adversely affect the safety of the subject. 4.Subject or parent(s) / legal guardian(s) not willing and able to comply with the requirements of the study 5.Subject not able to follow trial procedures, according to the investigator’s judgement 6.Known individual hypersensitivity to any of the ingredients/excipients of the study medication (see Section 7.1.1) 7. Applicable for part B, only: Systemic glucocorticoid therapy within 30 days prior to start of IMP treatment 8.Planned systemic glucocorticoid therapy during the trial period 9.Previous exposure to idebenone 10.Participation in any other therapeutic trial and/or intake of any investigational drug within 90 days prior to Visit 1

Design outcomes

Primary

Measure	Time frame
Main Objective: To assess the ability of CRD007 to decrease creatine kinase activity levels in subjects with DMD or BMD and in subjects being carriers for DMD or BMD;Secondary Objective: -To assess the ability of CRD007 to decrease the level of a range of other disease related biomarkers -To assess the ability of CRD007 to improve the outcome of functional tests -To assess the safety of CRD007 - To assess the safety margins of CRD007 in relation to plasma concentrations of pemirolast observed in clinical and toxicological studies ;Primary end point(s): The relative change in creatine kinase from enrolment (Visit 1) to 12 weeks of treatment (Visit 5) ;Timepoint(s) of evaluation of this end point: From enrolment (Visit 1) to 12 weeks of treatment (Visit 5)	—

Measure

Time frame

Main Objective: To assess the ability of CRD007 to decrease creatine kinase activity levels in subjects with DMD or BMD and in subjects being carriers for DMD or BMD;Secondary Objective: -To assess the ability of CRD007 to decrease the level of a range of other disease related biomarkers -To assess the ability of CRD007 to improve the outcome of functional tests -To assess the safety of CRD007 - To assess the safety margins of CRD007 in relation to plasma concentrations of pemirolast observed in clinical and toxicological studies ;Primary end point(s): The relative change in creatine kinase from enrolment (Visit 1) to 12 weeks of treatment (Visit 5) ;Timepoint(s) of evaluation of this end point: From enrolment (Visit 1) to 12 weeks of treatment (Visit 5)

—

Secondary

Measure	Time frame
Secondary end point(s): •The relative change in other disease related biomarkers between baseline and 12 weeks of treatment a. Myoglobin b. miR-1, miR-133, miR-206 •The relative change in the total North Star Ambulatory Assessment score •The relative change in the time needed to perform "Rise from floor", "Run (10 m)", "Climb 4 steps" Change in safety related assessments between baseline and 12 weeks a. Blood pressure (diastolic and systolic) and pulse b. Electrocardiogram c. Biochemistry laboratory values d. Hematology laboratory values •Incidence of adverse events by type and severity •Cpl(CRD007) and derived pharmacokinetic parameters ;Timepoint(s) of evaluation of this end point: •Myoglobin: at baseline and 12 weeks of treatment •miR-1, miR-133, miR-206:at baseline and 12 weeks of treatment •The relative change in the total North Star Ambulatory Assessment score:at baseline and 12 weeks of treatment •The relative change in the time needed to perform "Rise from floor", "Run (10 m)", "Climb 4 steps": at baseline and 12 weeks of treatment •Change in safety related assessments : at baseline, after 6 and 12 weeks of treatment •Incidence of adverse events: at baseline and once every week and 2 weeks after end of treatment	—

Measure

Time frame

Secondary end point(s): •The relative change in other disease related biomarkers between baseline and 12 weeks of treatment a. Myoglobin b. miR-1, miR-133, miR-206 •The relative change in the total North Star Ambulatory Assessment score •The relative change in the time needed to perform "Rise from floor", "Run (10 m)", "Climb 4 steps" Change in safety related assessments between baseline and 12 weeks a. Blood pressure (diastolic and systolic) and pulse b. Electrocardiogram c. Biochemistry laboratory values d. Hematology laboratory values •Incidence of adverse events by type and severity •Cpl(CRD007) and derived pharmacokinetic parameters ;Timepoint(s) of evaluation of this end point: •Myoglobin: at baseline and 12 weeks of treatment •miR-1, miR-133, miR-206:at baseline and 12 weeks of treatment •The relative change in the total North Star Ambulatory Assessment score:at baseline and 12 weeks of treatment •The relative change in the time needed to perform "Rise from floor", "Run (10 m)", "Climb 4 steps": at baseline and 12 weeks of treatment •Change in safety related assessments : at baseline, after 6 and 12 weeks of treatment •Incidence of adverse events: at baseline and once every week and 2 weeks after end of treatment

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Countries

Sweden

Contacts

Public ContactClinical trial information desk

Cardoz AB

carl-johan.dalsgaard@ofco.se+46 70 975 98 63

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026

Conditions

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Interventions

Sponsors

Eligibility

Inclusion criteria

Exclusion criteria

Design outcomes

Primary

Secondary

Countries

Contacts

Outcome results