Homocystinuria Advocacy Hub | UniteRare

At a Glance

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Regulatory Watchboard

8 events

Jan 2025Health Related Quality of Life (HrQoL) in Classical Homocystinuria (CBS Deficiency)

University Children's Hospital, Zurich

TrialRECRUITING

Dec 2024The Effect of Dietary Management and Cysteine Supplementation on Growth Parameters and Biochemical Control for Pediatric Qatari Patients Affected with Classical B6 Non-responsive Homocystinuria.

Hamad Medical Corporation — NA

TrialNOT YET RECRUITING

Aug 2024Proof of Concept Creatine Supplementation for Homocystinuria Study

University of British Columbia — NA

TrialACTIVE NOT RECRUITING

Apr 2024A Phase 3 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)

Travere Therapeutics, Inc. — PHASE3

TrialENROLLING BY INVITATION

Dec 2023A 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

Travere Therapeutics, Inc. — PHASE3

TrialACTIVE NOT RECRUITING

Jan 2019Pegtibatinase As an Enzyme Therapy for Patients with Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (COMPOSE)

Travere Therapeutics, Inc. — PHASE1, PHASE2

TrialACTIVE NOT RECRUITING

Jan 2017Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA)

Travere Therapeutics, Inc.

TrialRECRUITING

Oct 1996Cystadane: FDA approved

Treatment of homocystinuria to decrease elevated homocysteine blood levels.

FDAcompleted

Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Financial Assistance

4 programs

FINANCIAL LANDSCAPE SUMMARY

Total programs

Open now

Copay cards

Travel grants

Copay Assistance2

Cystadane

Recordati

OpenContact for detailsApply ↗

Betaine

Recordati

OpenContact for detailsApply ↗

Travel Grants2

Travere Patient Support Programs

Travere Therapeutics

Contact for detailsApply ↗

Travere Therapeutics Patient Support Programs

Contact for detailsApply ↗

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Approved Treatments

2 FDA-approved

Cystadane

(BETAINE)Orphan drug

Recordati Rare Diseases

Methylating Agent [EPC]

Approved Oct 1996FDA label ↗

Betaine Anhydrous

(BETAINE ANHYDROUS)Orphan drugstandard

Eton Pharmaceuticals, Inc.

Methylating Agent [EPC]

FDA label ↗

View full treatment details on disease page →

Clinical Trial Landscape

6 active trials

2Phase 3

1N/A

2Unknown

1PHASE1, PHASE2

6Total recruiting

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Recent News & Research

No recent news articles indexed yet for Homocystinuria.
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Specialist Network

Top 6 by expertise

Rajavel Elango, PhD

BC Children's Hospital Research Institute, University of British Columbia

📍 Vancouver, British Columbia

Score 65 trials

Anita MacDonald

Birmingham Women's and Children's NHS Foundation Trust

📍 Birmingham

Score 610 trials

Johan VanHove

University of Colorado, Denver

Score 01 trial

Carlo Dionisi-Vici

Bambino Gesù Hospital and Research Institute

Score 01 trial

Brian M GILFIX

McGill University Health Centre/Research Institute of the McGill University Health Centre

Score 01 trial

Michael Imperiale

Travere Therapeutics, Inc.

Score 04 trials

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