Repurposing 5-Azacytidine for the Treatment of Muscle Contractures in Children With Cerebral Palsy

Conditions

• Cerebral Palsy
• Contracture

Eligibility Criteria

Sex

ALL

Age

2 Years - 18 Years

Healthy Volunteers

Not accepted

Interventions

• Placebo for the AZA 10mg/m^2 — DRUG
  Placebo control group for the 10mg/m\^2, one-time subcutaneous injection without the active treatment.
• Placebo for the AZA 20mg/m^2 — DRUG
  Placebo control group for the 20mg/m\^2, one-time subcutaneous injection without the active treatment.
• Placebo for the AZA 35mg/m^2 — DRUG
  Placebo control group for the 35mg/m\^2, one-time subcutaneous injection without the active treatment.
• Placebo for the AZA 75mg/m^2 — DRUG
  Placebo control group for the 75mg/m\^2, one-time subcutaneous injection without the active treatment.
• 5-Azacytidine 10mg/m^2 — DRUG
  5-Azacytidine 10mg/m\^2, one-time subcutaneous injection
• 5-Azacytidine 20mg/m^2 — DRUG
  5-Azacytidine 20mg/m\^2, one-time subcutaneous injection
• 5-Azacytidine 35mg/m^2 — DRUG
  5-Azacytidine 35mg/m\^2, one-time subcutaneous injection
• 5-Azacytidine 75mg/m^2 — DRUG
  5-Azacytidine 75mg/m\^2, one-time subcutaneous injection

Study Details

In this controlled dose-escalation study, we will study the initial safety, biological properties, and potential efficacy of 5-azacytidine (AZA). Our overarching aspiration is for AZA to evolve into an approved pharmacological treatment, fostering muscle growth and enhancing body movement, ultimately contributing to an improved quality of life in children with CP. The main questions this study aims to answer are: 1. What is the optimal dose of AZA injection that can be used safely in children with CP? 2. Can the optimal safe dose of AZA improve the function of muscle-generating stem cells in children with CP? Each participant will have up to five research visits over the course of the study duration, in which they will participate in: blood draws, pregnancy test(s) (if applicable), medical assessments, and a muscle biopsy during a surgery for muscle contractures. Researchers will compare participants with four different dosages of AZA injections to those with four different dosages of placebo injections. A placebo is a look-alike substance that contains no active drug. They will see if a single injection of AZA at a standard concentration currently approved by the FDA to treat myelodysplastic syndromes, can also safely improve muscle growth and function in children with CP.

Key Dates

Start date

Jul 31, 2025

Status verified

Jun 2025

Primary completion

Dec 31, 2026

Completion

Jul 31, 2027

Study Design

Enrollment

27 participants (estimated)

Allocation

NON_RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Arms

• Experimental: AZA 10mg/m^2
  5-Azacytidine, subcutaneous injection, 10mg/m\^2, one subcutaneous injection for duration of study (estimated at 46 - 76 days)
• Experimental: AZA 20mg/m^2
  5-Azacytidine, subcutaneous injection, 20mg/m\^2, one subcutaneous injection for duration of study (estimated at 46 - 76 days)
• Experimental: AZA 35mg/m^2
  5-Azacytidine, subcutaneous injection, 35mg/m\^2, one subcutaneous injection for duration of study (estimated at 46 - 76 days)
• Experimental: AZA 75mg/m^2
  5-Azacytidine, subcutaneous injection, 75mg/m\^2, one subcutaneous injection for duration of study (estimated at 46 - 76 days)
• Placebo Comparator: Placebo for 10mg/m^2
  Placebo, subcutaneous injection, 10mg/m\^2, one subcutaneous injection without the active treatment for duration of study (estimated at 46 - 76 days)
• Placebo Comparator: Placebo for 20mg/m^2
  Placebo, subcutaneous injection, 20mg/m\^2, one subcutaneous injection without the active treatment for duration of study (estimated at 46 - 76 days)
• Placebo Comparator: Placebo for 35mg/m^2
  Placebo, subcutaneous injection, 35mg/m\^2, one subcutaneous injection without the active treatment for duration of study (estimated at 46 - 76 days)
• Placebo Comparator: Placebo for 75mg/m^2
  Placebo, subcutaneous injection, 75mg/m\^2, one subcutaneous injection without the active treatment for duration of study (estimated at 46 - 76 days)

Primary Outcome Measure

Dose-limiting toxicity (DLT). [ Time Frame: Through study completion, an average of 2 years. ]

Locations (1)

Find similar trials in San Diego, CA

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