Pegtibatinase as a Treatment for Patients With Classical Homocystinuria (HCU) (Also Known as the COMPOSE Study)

Conditions

• Homocystinuria

Eligibility Criteria

Sex

ALL

Age

5 Years - 65 Years

Healthy Volunteers

Not accepted

Interventions

• Pegtibatinase — DRUG
  Pegtibatinase sterile solution for subcutaneous injection
• Placebo — DRUG
  Normal saline for subcutaneous injection

Study Details

Researchers are looking for a better way to treat people who have classical homocystinuria (HCU), a rare condition that is passed down by parents (or "genetic condition"). It is caused by changes in the cystathionine beta-synthase (or "CBS") gene and prevents an enzyme from working correctly in the body. This enzyme breaks down a substance called homocysteine (from dietary methionine found in protein) and keeps both homocysteine and methionine at normal levels. When this enzyme is not working, homocysteine and methionine build up in the blood, which spreads into different tissues of the body and stops these body tissues from working normally. People with HCU can experience problems with vision, bones, blood vessels, and cognitive function (the ability to think, learn, and remember). Treatments available for HCU, such as a low protein diet and betaine (Cystadane®), help reduce homocysteine levels. The diet is a low methionine diet and a methionine-free protein supplement (a product that provides extra protein to help meet daily protein needs). These treatments are either not sufficient or are hard to take for many patients. Pegtibatinase was developed by scientists to be a version of the CBS enzyme that can be given to people with HCU. Researchers believe that giving pegtibatinase to people with HCU already getting medical treatment (or "standard of care") may reduce their homocysteine levels. This study is split into 7 different groups getting different amounts of drug. The first 6 groups have already finished the study. Group 7 plans to enroll participants from the US (virtual and in-person), France, and Qatar.

Key Dates

First listed

Jan 23, 2018

Start date

Jan 22, 2019

Status verified

Jul 2026

Primary completion

Jul 31, 2027

Completion

Jul 31, 2027

Study Design

Enrollment

39 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Active Comparator: Pegtibatinase (Cohort 1-6)
  Double-Blind Treatment Cohorts (≥12 to ≤65 years)
• Placebo Comparator: Placebo (Cohort 1-6)
  Double-Blind Treatment Cohorts (≥12 to ≤65 years)
• Experimental: Pegtibatinase (Cohort 7)
  Pediatric Open-label Treatment Cohort (≥5 to \<12 years)

Primary Outcome Measure

Incidence of AEs [ Time Frame: • Cohorts 1-6: Through double-blind study completion, approximately 10 months per patient • Cohort 7: First dose through End of Treatment (up to 32 weeks) ]

Central Contacts

• Travere Call Center
  1-877-659-5518
  [email protected]

Locations (10)

Find similar trials in Aurora, CO

Related Studies

• A Study to Investigate Efficacy and Safety of Pegtibatinase Compared With Placebo in Participants ≥12 to ≤65 Years of Age With Classical Homocystinuria (HCU) Due to Cystathionine Beta Synthase Deficiency Receiving Standard of Care Treatment
  PHASE3 · Recruiting · Travere Therapeutics, Inc. · Phoenix, Arizona
• A Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants ≥5 to ≤65 Years of Age With Classical Homocystinuria (HCU) (ENSEMBLE)
  PHASE3 · Enrolling By Invitation · Travere Therapeutics, Inc. · Phoenix, Arizona