Trial · NCT03539952

Trientine Tetrahydrochloride (TETA 4HCL) for the Treatment of Wilson's Disease

NCT03539952, Phase 3, Completed, Orphalan; results first posted 2023-04-20

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Trientine Tetrahydrochloride (TETA 4HCL) for the Treatment of Wilson's Disease. Trials, record NCT03539952. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03539952

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Trientine Tetrahydrochloride (TETA 4HCL) for the Treatment of Wilson's Disease
NCT number: NCT03539952
Status: Completed
Phases: Phase 3
Enrollment: 77 participants, actual
Lead sponsor: Orphalan
Conditions: Wilson Disease
Interventions: Penicillamine (D1-W12), TETA 4HCL (W12-60), Penicillamine (W12-W60), TETA 4HCL (60-<W108)
First posted: 2018-05-30
Last updated: 2025-07-02
Results first posted: 2023-04-20

Source ClinicalTrials.gov

Registered outcomes3

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Serum NCC Concentration	Week 36	The primary outcome of efficacy was serum NCC by speciation assay (μg/L), with comparative analysis of mean difference between the two groups 24 weeks after randomization. The non-inferiority margin was set at -50 μg/L.
Secondary	24-hour Urinary Copper Excretion (UCE)	Week 36	24-hour urinary copper excretion (μg/ 24 hr) from urine collected by the patient over a 24-hour period.
Secondary	Clinical Global Impression of Change (CGIC) Rating Scale	Week 36	The clinician will rate the change in the patient's Wilson's disease relative to the prior study clinic visit using a 7-point scale to a specific statement: 'Please rate the change in the overall severity of the patients Wilson's disease compared to the previous study clinic visit". Available options were (1) very much improved; (2) much improved; (3) minimally improved; (4) no change; (5) minimally worse; (6), much worse; or (7) very much worse.

Source ClinicalTrials.gov

Outcomes3

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Serum NCC Concentration	Week 36	ITT	µg/L	MEAN
SECONDARY	24-hour Urinary Copper Excretion (UCE)	Week 36		μg/24 hours	MEAN
SECONDARY	Clinical Global Impression of Change (CGIC) Rating Scale	Week 36		score on a scale	MEAN

Source ClinicalTrials.gov

Results by group6

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Serum NCC Concentration	Penicillamine Arm	46.5	Standard Error 5.69	µg/L
Serum NCC Concentration	TETA 4HCL Arm	58.7	Standard Error 5.54	µg/L
24-hour Urinary Copper Excretion (UCE)	TETA 4HCL	274.5	Standard Error 45.59	μg/24 hours
24-hour Urinary Copper Excretion (UCE)	Penicillamine	510.8	Standard Error 47.77	μg/24 hours
Clinical Global Impression of Change (CGIC) Rating Scale	Penicillamine	4.1	Standard Error 0.10	score on a scale
Clinical Global Impression of Change (CGIC) Rating Scale	TETA 4HCL	3.9	Standard Error 0.05	score on a scale

Source ClinicalTrials.gov

Participant flow24

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Penicillamine Baseline Period (Day1-W12)	STARTED	Penicillamine	77
Penicillamine Baseline Period (Day1-W12)	STARTED	TETA 4HCl	0
Penicillamine Baseline Period (Day1-W12)	COMPLETED	Penicillamine	53
Penicillamine Baseline Period (Day1-W12)	COMPLETED	TETA 4HCl	0
Penicillamine Baseline Period (Day1-W12)	NOT COMPLETED	Penicillamine	24
Penicillamine Baseline Period (Day1-W12)	NOT COMPLETED	TETA 4HCl	0
Post-randomization Period (Week 12-36)	STARTED	Penicillamine	27
Post-randomization Period (Week 12-36)	STARTED	TETA 4HCl	26
Post-randomization Period (Week 12-36)	COMPLETED	Penicillamine	26
Post-randomization Period (Week 12-36)	COMPLETED	TETA 4HCl	26

1 to 10 of 24Source ClinicalTrials.gov

See all 24 participant flow

Adverse events240

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Abdominal pain	Gastrointestinal disorders	D1-W12 Penicillamine Arm	0	27	0
other	Abdominal pain	Gastrointestinal disorders	D1-W12 TETA 4HCl Arm	0	26	0
other	Abdominal pain	Gastrointestinal disorders	W12-W36 Penicllamine Arm	1	27	1
other	Abdominal pain	Gastrointestinal disorders	W12-W36 TETA4HCl Arm	2	26	12
other	Abdominal pain	Gastrointestinal disorders	W36-60 Pencillamine Arm	0	26	0
other	Abdominal pain	Gastrointestinal disorders	W36-60 TETA4HCl Arm	0	26	0
other	Abdominal pain	Gastrointestinal disorders	W60-108 Pencillamine Arm	2	19	4
other	Abdominal pain	Gastrointestinal disorders	W60-108 TETA4HCl Arm	0	23	0
other	Abnormal faeces	Gastrointestinal disorders	D1-W12 Penicillamine Arm	0	27	0
other	Abnormal faeces	Gastrointestinal disorders	D1-W12 TETA 4HCl Arm	1	26	1

1 to 10 of 240Source ClinicalTrials.gov

See all 240 adverse events

Adverse event totals24

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	D1-W12 Penicillamine Arm	0	27
Total, all-cause mortality	D1-W12 TETA 4HCl Arm	0	26
Total, all-cause mortality	W12-W36 Penicllamine Arm	0	27
Total, all-cause mortality	W12-W36 TETA4HCl Arm	0	26
Total, all-cause mortality	W36-60 Pencillamine Arm	0	26
Total, all-cause mortality	W36-60 TETA4HCl Arm	0	26
Total, all-cause mortality	W60-108 Pencillamine Arm	0	19
Total, all-cause mortality	W60-108 TETA4HCl Arm	1	23
Total, other adverse events	D1-W12 Penicillamine Arm	11	27
Total, other adverse events	D1-W12 TETA 4HCl Arm	10	26

1 to 10 of 24Source ClinicalTrials.gov

See all 24 adverse event totals

Papers3

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
36183738	RESULT	PubMed
39139457	DERIVED	PubMed
41831608	DERIVED	PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT03539952

GET https://conductscience.org/api/trials/v1/NCT03539952/flow?page=2

API reference For AI assistants