A clinical study to learn about the effects of a virus that transfers the gene for human Ornithine Transcarbamylase (OTC) in adults with late-onset OTC deficiency in the long term

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Ultragenyx Pharmaceutical Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Trial duration

17 Sep 2018 → ongoing

Decision date (initial)

2022-12-16

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Ultragenyx Pharmaceutical, Inc.

External identifiers

EU CT number

2022-501146-30-00

EudraCT number

2018-000156-18

ClinicalTrials.gov

NCT03636438

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Dose response, Efficacy, Therapy, Safety

To determine the long-term safety of DTX301 following a single IV dose in adults with late-onset OTC deficiency.

Secondary objectives 2

1. To evaluate the long-term efficacy of DTX301 on AUC0-24 for plasma ammonia following a single IV dose in adults with late-onset OTC deficiency.
2. To evaluate the long-term effects of DTX301 on the rate of ureagenesis in adults with late-onset OTC deficiency.

Conditions and MedDRA coding

Ornithine transcarbamylase deficiency

Study design 1 period

Regulatory references

Scientific advice from competent authorities

European Medicines Agency, Paul Ehrlich Institute, Medicines And Healthcare Products Regulatory Agency

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. Completed the Week 52 visit in Study 301OTC01. Note that the Day 0 visit of Study 301OTC02 may coincide with the Week 52 visit of Study 301OTC01.
2. Willing and able to provide written informed consent.
3. Willing, able, and committed to comply with scheduled study site visits, study procedures, and requirements.

Exclusion criteria 2

1. Planned or current participation in another interventional clinical study that may confound the efficacy or safety evaluation of DTX301 during the duration of this study
2. Any clinically significant medical condition that, in the opinion of the investigator, would pose a risk to subject safety or would impede the study.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The incidence of AEs and SAEs for each dosing cohort assessed by severity and relationship to study product.

Secondary endpoints 2

1. The change from baseline (Day 0 of Study 301OTC01) in AUC0-24 for plasma ammonia over time to 208 weeks following IV administration of DTX301
2. The change from baseline (the baseline of each subject before DTX301 administration in Study 301OTC01) in the rate of ureagenesis (as measured by the generation of [13 C]urea over 4 hours) as determined by gas chromatography mass spectrometry over time to 416 weeks following IV administration of DTX301 The rate of ureagenesis was assessed prior to Amendment 3; ureagenesis data collected will be analyzed as a secondary efficacy endpoint.

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Ultragenyx Pharmaceutical Inc.

Sponsor organisation

Ultragenyx Pharmaceutical Inc.

Address

840 Memorial Drive

City

Cambridge

Postcode

02139-3789

Country

United States

Scientific contact point

Organisation

Ultragenyx Pharmaceutical Inc.

Contact name

Medical information

Public contact point

Organisation

Ultragenyx Pharmaceutical Inc.

Contact name

Ultragenyx trial information group

Third parties 9

Locations

2 EU/EEA countries · 3 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

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

Documents 12 files

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

Application history

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