A study to investigate efficacy and safety with pegtibatinase compared to placebo in participants ≥12 to ≤65 years of age with classical homocystinuria (HCU) due to cystathionine beta synthase deficiency receiving standard of care treatment

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Travere Therapeutics Inc.

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nutritional and Metabolic Diseases [C18]

Decision date (initial)

2026-04-29

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Travere Therapeutics, Inc. 3611 Valley Centre Drive, Suite 300 San Diego, CA 92130 USA

External identifiers

EU CT number

2023-504135-40-00

ClinicalTrials.gov

NCT06247085

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety, Pharmacodynamic, Pharmacokinetic

Efficacy: To determine the effect of pegtibatinase administered subcutaneously on plasma tHcy and methionine (Met) as compared with placebo in participants with HCU receiving standard of care
Safety: To determine the safety and tolerability of pegtibatinase administered subcutaneously in participants with HCU

Secondary objectives 3

1. To characterize the pharmacokinetics (PK) of pegtibatinase
2. To determine the immunogenicity of pegtibatinase administered subcutaneously in participants with HCU
3. To assess the impact of pegtibatinase administered subcutaneously in participants with HCU on the daily lives of patients living with HCU

Conditions and MedDRA coding

Classical Homocystinuria

Regulatory references

Scientific advice from competent authorities

Food And Drug Administration, European Medicines Agency

Plan to share IPD

Yes

IPD plan description

Requests for clinical trial data, including language stating its intended use, should be directed to [email protected]. If approved, the requested information will be provided to the requestor after signing a data access agreement. Requests can be made following completion of the study and full publication of the study data in a peer reviewed journal for up to 36 months following its publication. Travere reserves the right to decline or recommend modifications to a request if it does not comply with the data sharing policy or if it is determined that the request is made by a biased source.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 2

1. Individuals eligible to be enrolled into this study must have a diagnosis of HCU based on clinical, biochemical, and/or molecular genetic testing. Approximately 25% of participants may be enrolled with a Screening plasma tHcy ≥50 to <80 µM, and the remaining majority (approximately 75%) of participants will be enrolled with a Screening plasma tHcy ≥80 µM.
2. Individuals must also be ≥12 to ≤65 years of age at Screening, and be willing to maintain a stable diet with consistent levels of DIPI, and HCU-related therapies such as betaine, pyridoxine, and medical food (eg, metabolic formula), if applicable, as part of pre-study treatment

Exclusion criteria 4

1. Diagnosis of Marfan syndrome, methylenetetrahydrofolate reductase (MTHFR) deficiency, or disorders of cobalamin metabolism; body weight ≥160 kg (measured from screening through the PND)
2. Any prior exposure to pegtibatinase or pegtarviliase
3. Use or planned use of any injectable drug containing PEG (other than pegtibatinase or PEG containing vaccines)
4. History of a severe immune reaction to a PEG containing product; organ transplant; or intensive chronic immunosuppressive treatment within the 6 months prior to screening

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The change from baseline averaged over Week -3, Week -1, and Day 1 pre-dose in plasma tHcy levels averaged over Weeks 6 through 12 (6, 8, 10, and 12) in participants receiving standard of care and treated with pegtibatinase as compared to placebo

Secondary endpoints 10

1. The change from baseline in plasma tHcy levels averaged post Week 12 (Weeks 16, 20, and 24) in participants receiving standard of care and treated with pegtibatinase as compared to placebo
2. The change from baseline in plasma Met levels averaged over Weeks 6 through 12 in participants receiving standard of care and treated with pegtibatinase as compared to placebo
3. The change from baseline in plasma Met levels averaged post Week 12 in participants receiving standard of care and treated with pegtibatinase as compared to placebo
4. The proportion of participants achieving tHcy <100 µM averaged over Weeks 6 through 12, among participants with tHcy ≥100 µM at baseline
5. The proportion of participants achieving tHcy <100 µM averaged post Week 12 among participants with tHcy ≥100 µM at baseline
6. The proportion of participants with tHcy <50 µM averaged over Weeks 6 through 12
7. The proportion of participants with tHcy <50 µM averaged post Week 12
8. The proportion of participants achieving tHcy reduction ≥35% from baseline averaged over Weeks 6 through 12, and averaged post Week 12
9. The proportion of participants achieving tHcy <100 µM and a ≥35% reduction from baseline averaged over Weeks 6 through 12, and averaged post Week 12 among participants with tHcy ≥100 µM at baseline
10. The proportion of participants with tHcy ≤15 µM averaged over Weeks 6 through 12, and averaged post Week 12

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Travere Therapeutics Inc.

Sponsor organisation

Travere Therapeutics Inc.

Address

3611 Valley Centre Drive Suite 300

City

San Diego

Postcode

92130-3331

Country

United States

Scientific contact point

Organisation

Travere Therapeutics Inc.

Contact name

Travere Call Center

Public contact point

Organisation

Travere Therapeutics Inc.

Contact name

Travere Call Center

Third parties 11

Locations

8 EU/EEA countries · 18 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 100 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

2 submissions — initial application plus substantial / non-substantial modifications