← Back to Search

Enzyme Therapy

Pegtibatinase for Homocystinuria

Phase 1 & 2
Waitlist Available
Research Sponsored by Orphan Technologies Ltd
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Subjects born biologically as female who are of child-bearing potential must have a negative pregnancy test at screening and be willing to have additional pregnancy tests during the study. Subjects born biologically as male who identify as female and are not of childbearing potential are not required to undergo pregnancy tests
Sexually active subjects who have childbearing potential or those who have partners of childbearing potential must be willing to use acceptable methods of contraception while on the study and for 4 weeks after the end of study
Timeline
Screening 3 weeks
Treatment Varies
Follow Up through double-blind study completion, approximately 10 months per patient
Awards & highlights

Study Summary

This trial is to test the efficacy and safety of Pegtibatinase in adult patients with CBS Deficiency-associated homocystinuria.

Who is the study for?
This trial is for individuals with confirmed homocystinuria, evidenced by specific blood test levels. Participants must be able to consent and follow study procedures, including maintaining a stable diet and using contraception if they have childbearing potential. Exclusions include recent use of investigational products, exposure to pegtibatinase, pregnancy or breastfeeding, certain health risks as assessed by the investigator, use of PEG-containing injectables (except some vaccines), and positive tests for HIV or hepatitis.Check my eligibility
What is being tested?
The COMPOSE trial is testing Pegtibatinase as an enzyme therapy against a placebo in patients with Homocystinuria caused by Cystathionine Beta-Synthase Deficiency. The aim is to introduce CBS enzyme into circulation to reduce harmful homocysteine levels and improve related symptoms affecting eyes, central nervous system, skeleton, and vascular system.See study design
What are the potential side effects?
Potential side effects are not explicitly listed but may relate to hypersensitivity reactions due to PEG (polyethylene glycol) content in pegtibatinase. Side effects could also mimic those associated with other enzyme replacement therapies such as infusion reactions or immune responses.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I can become pregnant and will take pregnancy tests during the study.
Select...
I am willing to use birth control during and 4 weeks after the study.
Select...
I have a genetic diagnosis of homocystinuria with high homocysteine levels.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~through double-blind study completion, approximately 10 months per patient
This trial's timeline: 3 weeks for screening, Varies for treatment, and through double-blind study completion, approximately 10 months per patient for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Anti-PEG antibodies
Anti-pegtibatinase antibodies
Incidence of AEs
Secondary outcome measures
Bone densitometry using dual-energy X-ray absorptionmetry (DEXA) scans
Changes in Met cycle metabolites levels - Cth
Changes in Met cycle metabolites levels - Met
+9 more

Trial Design

2Treatment groups
Active Control
Placebo Group
Group I: PegtibatinaseActive Control1 Intervention
Group II: PlaceboPlacebo Group1 Intervention

Find a Location

Who is running the clinical trial?

Orphan Technologies LtdLead Sponsor
1 Previous Clinical Trials
150 Total Patients Enrolled
1 Trials studying Homocystinuria
150 Patients Enrolled for Homocystinuria
Travere Therapeutics, Inc.Lead Sponsor
20 Previous Clinical Trials
177,594 Total Patients Enrolled
2 Trials studying Homocystinuria
220 Patients Enrolled for Homocystinuria
Feriandas GreblikasStudy DirectorTravere Therapeutics, Inc.
1 Previous Clinical Trials
150 Total Patients Enrolled
1 Trials studying Homocystinuria
150 Patients Enrolled for Homocystinuria

Media Library

Pegtibatinase (Enzyme Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03406611 — Phase 1 & 2
Homocystinuria Research Study Groups: Placebo, Pegtibatinase
Homocystinuria Clinical Trial 2023: Pegtibatinase Highlights & Side Effects. Trial Name: NCT03406611 — Phase 1 & 2
Pegtibatinase (Enzyme Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03406611 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Would this experiment include individuals who are elderly?

"The age range for this clinical trial is 12 to 65. There are 2 other trials available for minors and another 2 trials open to seniors."

Answered by AI

Are there any available openings for patients who want to enroll in this research project?

"Yes, this trial is currently looking for participants. The clinicaltrials.gov website reports that the study was originally posted on 1/22/2019 and updated as recently as 7/11/2022."

Answered by AI

How many research subjects will be enrolled in this trial?

"That is correct, the online information does show that this study is recruiting patients. The trial was first posted on 1/22/2019 and has since been edited on 7/11/2022. They are looking for a total of 32 patients that will be recruited from 7 sites."

Answered by AI

In how many different locations is this trial being run today?

"Presently, this experiment is being conducted at 7 sites. The locations are situated in Miami, Philadelphia and Indianapolis with 4 other cities also hosting a location. If you want to limit travel, it would be best to select a site near your residence."

Answered by AI

Can I join this clinical trial?

"This study is looking for 32 participants with homocystinuria who fall between the ages of 12 and 65. The most important criteria that candidates must meet are as follows: They must be able to provide written consent, they must be willing to comply with all procedures related to the study, they must be sexually active or have sexually active partners, and they must be willing to use contraception while on the study. Additionally, subjects who identify as female but were born biologically male do not need to undergo pregnancy tests. Lastly, all participants must be willing to maintain a stable diet and complete a daily diet diary."

Answered by AI
~9 spots leftby Jun 2026