Wilson DiseaseNCT05686564
Early Access Program for ALXN1840 in Patients With Wilson Disease
A study testing ALXN1840 in people with Wilson Disease, run by Alexion Pharmaceuticals, Inc..
Trial summary
This is an open-label, single-arm, multi-center EAP, designed to provide early access to ALXN1840 for eligible patients with WD. Read more about this study
- Who is it for?
- Ages 3 years and older
- Conditions the study is for
- Wilson Disease
- What is being tested?
- ALXN1840
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 years and older
- All sexes
- Child, Adult, Older adult
Who may be able to join
- ≥ 3 years of age or older at the time of providing informed consent/assent and expressed desire to continue treatment with ALXN1840.
- Confirmed diagnosis of WD.
- Has completed one of the following ALXN1840 clinical studies:
- ALXN1840-WD-205
- WTX101-301
- ALXN1840-WD-302
Show all 10Show fewer
- Other ALXN1840 studies
- In the Treating Physician's medical opinion, the potential benefits of treatment with ALXN1840 outweigh the potential risks for the participant.
- Participant or legal representative provided informed consent/assent to be treated with ALXN1840 through this EAP.
- Female participants of childbearing potential and male participants must follow protocol-specified-contraception guidance.
Who cannot join
- Eligible for and able to participate in an Alexion-sponsored study of ALXN1840.
- Unwilling or unable to comply with the Treating Physician's treatment plan related to this EAP for any reason.
- Has decompensated hepatic cirrhosis.
- Model for End-Stage Liver Disease (MELD) score > 13.
- Modified Nazer Score > 7.
- End-stage renal disease on dialysis (chronic kidney disease stage 5 [CKD 5]) or creatinine clearance < 30 mL/min.
- Known hypersensitivity to ALXN1840, ALXN1840 excipients, or any of the ingredients contained in ALXN1840.
- Women who are nursing or pregnant (or women who are planning to become pregnant during treatment with ALXN1840).
About this study
This is an open-label, single-arm, multi-center EAP, designed to provide early access to ALXN1840 for eligible patients with WD.
Read the detailed description
To provide access to ALXN1840 treatment, an investigational medicinal product (IMP) that has not yet been granted marketing authorization, to participants who complete Studies ALXN1840-WD-205, WTX101-301, or ALXN1840-WD-302, or other studies with ALXN1840 who, in the opinion of the Treating Physician, may benefit from continued treatment with ALXN1840, and who meet the eligibility criteria described in this protocol.
- Conditions
- Wilson Disease
- Being tested
- ALXN1840
- Keywords
- Wilson Disease, ALXN1840
- Medical subject headings
- Hepatolenticular Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ALXN1840Drug
Also called INN: tiomolibdic acid; USAN/JAN: tiomolibdate choline (WTX101)
bis-choline tetrathiomolybdate
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jan 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Wilson DiseaseStudies of the same condition, open and under way.
- More trials run by Alexion Pharmaceuticals, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05686564
- Sponsor's protocol ID
- D9430R00001
- Status last verified
- May 2023
- First submitted
- 6 Jan 2023
- Last updated
- 17 May 2023
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 May 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Early Access Program for ALXN1840 in Patients With Wilson Disease
- ClinicalTrials.gov ID
- NCT05686564
- Acronym / sponsor ID
- D9430R00001
- Conditions
- Wilson Disease
- Keywords
- Wilson Disease, ALXN1840
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- First posted
- 17 Jan 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is an open-label, single-arm, multi-center EAP, designed to provide early access to ALXN1840 for eligible patients with WD.
Detailed description
To provide access to ALXN1840 treatment, an investigational medicinal product (IMP) that has not yet been granted marketing authorization, to participants who complete Studies ALXN1840-WD-205, WTX101-301, or ALXN1840-WD-302, or other studies with ALXN1840 who, in the opinion of the Treating Physician, may benefit from continued treatment with ALXN1840, and who meet the eligibility criteria described in this protocol.
Study design
Who may qualify
- Ages
- 3 years and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (10 listed)
- ≥ 3 years of age or older at the time of providing informed consent/assent and expressed desire to continue treatment with ALXN1840.
- Confirmed diagnosis of WD.
- Has completed one of the following ALXN1840 clinical studies:
- In the Treating Physician's medical opinion, the potential benefits of treatment with ALXN1840 outweigh the potential risks for the participant.
- Participant or legal representative provided informed consent/assent to be treated with ALXN1840 through this EAP.
Key exclusion criteria (8 listed)
- Eligible for and able to participate in an Alexion-sponsored study of ALXN1840.
- Unwilling or unable to comply with the Treating Physician's treatment plan related to this EAP for any reason.
- Has decompensated hepatic cirrhosis.
- Model for End-Stage Liver Disease (MELD) score > 13.
- Modified Nazer Score > 7.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ALXN1840Also: INN: tiomolibdic acid; USAN/JAN: tiomolibdate choline (WTX101)bis-choline tetrathiomolybdate | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 May 2023.
Related trials for Wilson Disease
RecruitingObservational study
Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.
Inherited Disease, Rare Diseases
RecruitingPhase 3
Trientine Tetrahydrochloride Administered Once a Day for the First Line Treatment of Wilson's Disease Patients.
Wilson's Disease
RecruitingObservational study
Oral Health and Wilson's Disease: SOMAWI
Wilson Disease
Recruiting
Description of the Copper Concentration in Breast Milk in Women Treated for Wilson's Disease
Wilson's Disease
Recruiting
Cardiac Involvement in Wilson's Disease
Wilson's Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A study testing ALXN1840 in people with Wilson Disease, run by Alexion Pharmaceuticals, Inc..
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 17 May 2023. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 May 2023.