NCT02047461

Brief Summary

This study will include a screening period, a 6-month treatment period, followed by long-term extension period expected to last approximately 72 months.

Trial Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
8

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Apr 2014

Longer than P75 for phase_2

Geographic Reach
5 countries

6 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

January 9, 2014

Completed
19 days until next milestone

First Posted

Study publicly available on registry

January 28, 2014

Completed
2 months until next milestone

Study Start

First participant enrolled

April 1, 2014

Completed
8.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 1, 2022

Completed
2 months until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2022

Completed
1 year until next milestone

Results Posted

Study results publicly available

October 17, 2023

Completed
Last Updated

October 17, 2023

Status Verified

September 1, 2023

Enrollment Period

8.3 years

First QC Date

January 9, 2014

Results QC Date

August 10, 2023

Last Update Submit

September 22, 2023

Conditions

Molybdenum Cofactor Deficiency, Type A

Keywords

Molybdenum cofactor deficiency (MoCD)molybdenum cofactor (MoCo) biosynthesissulfite oxidase (SO)xanthine dehydrogenasealdehyde oxidaseS sulfocysteine (SSC)

Outcome Measures

Primary Outcomes (1)

  • Safety of ORGN001 (Formerly ALXN1101)

    Treatment Emergent Serious Adverse Events

    Baseline to Month 24 for all patients plus additional follow-up up to Month 90

Secondary Outcomes (4)

  • Pharmacokinetics (Actual Plasma Concentration) of ORGN001 (Formerly ALXN1101)

    First 6 months at each dose level, where available

  • S-sulfocysteine (Umol/L) Normalized to Urine Creatinine (mmol/L) - Change From Baseline Over Time

    Baseline to Month 24 for all patients plus additional follow-up to Month 90

  • Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination

    Baseline to Month 24 for all patients plus additional follow-up until Month 30

  • Long-term Safety of ORGN001 (Formerly ALXN1101)

    Baseline to Month 24 for all patients plus additional follow up until Month 72

Study Arms (1)

ORGN001 (formerly ALXN1101)

EXPERIMENTAL

daily IV infusions

Drug: ORGN001 (formerly ALXN1101)

Interventions

ORGN001 (formerly ALXN1101)DRUG

IV infusion

ORGN001 (formerly ALXN1101)

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Male or female patients with a genetically confirmed diagnosis of MoCD Type A (MOCS1 mutation)
  • Currently treated with rcPMP infusions

You may not qualify if:

  • \- Current or planned treatment with another investigational drug or device, with the exception rcPMP treatment through Day -1.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (6)

Children's Hospital of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

Monash Medical Centre

Melbourne, Australia

Location

Beatrix Children's Hospital

Groningen, Netherlands

Location

Unité des maladies métaboliques

Tunis, Tunisia

Location

Royal Hospital for Sick Children

Glasgow, United Kingdom

Location

Manchester University Hospitals NHS Foundation Trust

Manchester, United Kingdom

Location

Related Links

MeSH Terms

Conditions

Molybdenum Cofactor Deficiency, Complementation Group AMolybdenum cofactor deficiency

Limitations and Caveats

This was an open label study with no comparator arm. Limited statistical analysis were performed. Most data was summarized over time by individual patient, making reportable results in tabular format truncated. Refer to FDA approval package for more information.

Results Point of Contact

Title
Business Development and Operations
Organization
Origin Biosciences (affiliate of BridgeBio)
info@bridgebio.com
650-391-9740

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 9, 2014

First Posted

January 28, 2014

Study Start

April 1, 2014

Primary Completion

August 1, 2022

Study Completion

October 1, 2022

Last Updated

October 17, 2023

Results First Posted

October 17, 2023

Record last verified: 2023-09

Locations

NL(1)US(1)TU(1)GB(2)AU(1)