HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02525302
• Date of registration: 18/07/2015
• Prospective Registration: No
• Primary sponsor: Akashi Therapeutics
• Public title: HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02
• Scientific title: HT-100 Long-term Safety and Pharmacodynamics in Patients With DMD Who Have Completed Protocols HALO-DMD-01 and HALO-DMD-02
• Date of first enrolment: May 2015
• Target sample size: 10
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT02525302
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Diana M Escolar, MD
• Affiliation: Askashi Therapeutics

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Completed both previous studies HALO-DMD-01 and HALO-DMD-02

2. Ability to provide written informed consent

3. Ability to understand and follow site and protocol instruction for the entire duration
of the study

Exclusion Criteria:

Answering yes to any of the following make the subject NOT eligible to participate in the
study.

1. Clinically significant major disease not related to DMD that would make it not safe to
be in the study or affect ability to follow the protocol

2. History of severe allergic or anaphylactic reactions

3. Recent report of drug/alcohol abuse



Age minimum: 6 Years
Age maximum: 20 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Duchenne Muscular Dystrophy

Intervention(s)

Drug: HT-100

Primary Outcome(s)

Number of adverse events by severity and relationship [Time Frame: Every 6 months from enrollment for up to 3 years]

Laboratory values (Number of subjects with clinically significant changes) [Time Frame: Every 6 months from enrollment for up to 3 years]

Vital signs (Number of subjects with clinically significant changes) [Time Frame: Every 6 months from enrollment for up to 3 years]

Cardiovascular Magnetic Resonance [Time Frame: Every 6 months from enrollment for up to 3 years]

Dose reduction or modification due to upper GI or other adverse events [Time Frame: Every 6 months from enrollment for up to 3 years]

Electrocardiograms [Time Frame: Every 6 months from enrollment for up to 3 years]

Trial discontinuations due to upper GI or other AEs [Time Frame: Every 6 months from enrollment for up to 3 years]

Echocardiograms [Time Frame: Every 6 months from enrollment for up to 3 years]

Secondary Outcome(s)

Pulmonary function testing (Number of subjects with clinically significant changes) [Time Frame: Every 6 months from enrollment for up to 3 years]

Tip pinch and key pinch tests (Number of subjects with clinically significant changes) [Time Frame: Every 6 months from enrollment for up to 3 years]

Biomarkers of extracellular matrix turnover (Number of subjects with clinically significant changes) [Time Frame: Every 6 months from enrollment for up to 3 years]

Cardiovascular Magnetic Resonance [Time Frame: Every 6 months from enrollment for up to 3 years]

Motor Function Measure (MFM) [Time Frame: Every 6 months from enrollment for up to 3 years]

Electrical impedance myography (EIM) score [Time Frame: Every 6 months from enrollment for up to 3 years]

Quantitative muscle testing (QMT) scores [Time Frame: Every 6 months from enrollment for up to 3 years]

Performance of upper limb (PUL) scale [Time Frame: Every 6 months from enrollment for up to 3 years]

9-hole peg test [Time Frame: Every 6 months from enrollment for up to 3 years]

Motor function measure (MFM) scale [Time Frame: Every 6 months from enrollment for up to 3 years]

Timed function tests (TFTs) [Time Frame: Every 6 months from enrollment for up to 3 years]

Upper extremity function (proximal, mid-range, and distal) by Performance of Upper Limb (PUL) [Time Frame: Every 6 months from enrollment for up to 3 years]

Secondary ID(s)

HALO-DMD-03

Source(s) of Monetary Support

Please refer to primary and secondary sponsors