Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular Dystrophy

Processa Pharmaceuticals

Status and phase: Terminated; Phase 2; Phase 1

Conditions: Duchenne Muscular Dystrophy

Treatments: Drug: HT-100

Study type: Interventional

Funder types: Industry

Identifiers

NCT01847573

HALO-DMD-01

HALO (Other Identifier)

Details and patient eligibility

About

The main purpose of this study is to test the safety and tolerability of different, increasing doses of an experimental medication called HT-100 in boys and young men with Duchenne muscular dystrophy (DMD). The study medication, HT-100, is a medicine that may help promote healthy muscle regeneration, diminish inflammation and the resulting damage to muscle, and decrease the scar tissue that forms in the muscles of children with DMD. In this study, pharmacokinetic sampling, or measurements of the amount of HT-100 in the bloodstream will also be taken.

Enrollment: 17 patients

Sex: Male

Ages: 6 to 20 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Main Inclusion Criteria:

• Ambulatory or non-ambulatory
• Diagnosis of DMD with confirmation of minimal to no dystrophin
• Corticosteroid naive or on therapy for at least 12 months (stable dose and regimen)

Main Exclusion Criteria:

• Recent, substantial change in use of cardiac medications or medications affecting muscle function
• Inability to undergo magnetic resonance imaging (MRI)
• Significantly compromised cardio-respiratory function
• Prior treatment with another investigational product in past 6 months

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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17 participants in 6 patient groups

Cohort 1: HT-100 tablet, Dose 1

Experimental group

Description:

* Single dose administration: Dose 1 * Multiple dose administration: Dose 1

Treatment:

Drug: HT-100

Cohort 2: HT-100 tablet, Dose 2

Experimental group

Description:

* Single dose administration: Dose 2 * Multiple dose administration: Dose 2

Treatment:

Drug: HT-100

Cohort 3: HT-100 tablet, Dose 3

Experimental group

Description:

* Single dose administration: Dose 3 * Multiple dose administration: Dose 3

Treatment:

Drug: HT-100

Cohort 4a: HT-100 tablet, Dose 4

Experimental group

Description:

* Single dose administration: Dose 4 * Multiple dose administration: Dose 4

Treatment:

Drug: HT-100

Cohort 4b: HT-100 tablet, Dose 5

Experimental group

Description:

\* Multiple dose administration: Dose 5

Treatment:

Drug: HT-100

Cohort 5: HT-100 tablet, Dose 6

Experimental group

Description:

\* Multiple dose administration: Dose 5

Treatment:

Drug: HT-100