Trial · NCT00107783

Long-Term Study of Nitisinone to Treat Alkaptonuria

NCT00107783, Phase 2, Completed, National Human Genome Research Institute (NHGRI); results first posted 2011-01-19

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Long-Term Study of Nitisinone to Treat Alkaptonuria. Trials, record NCT00107783. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00107783

On this page

Trial

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

Title: Long-Term Study of Nitisinone to Treat Alkaptonuria
NCT number: NCT00107783
Status: Completed
Phases: Phase 2
Enrollment: 40 participants, actual
Lead sponsor: National Human Genome Research Institute (NHGRI)
Conditions: Alkaptonuria
Interventions: Nitisinone (NTBC)
First posted: 2005-04-08
Last updated: 2021-08-26
Results first posted: 2011-01-19
Limitations and caveats: Small number of patients involved in study and several patients dropped out; primary outcome parameter selection possibly inappropriate because hip joint damage that is already present may be irreversible.

Source ClinicalTrials.gov

Registered outcomes5

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

List	Outcome	Time frame	Description
Primary	Change in Total ROM Worse Hip	Measured at baseline and at 36 months	Change from baseline in the total (external + internal) hip range of motion (ROM) in the worse hip at 36 months. The patient lies on exam table in the supine position. The patient flexes his/her hip and knee to 90 degrees. The examiner measures the patient's hip external rotation and hip internal rotation range of motion with a goniometer.
Secondary	Change in Schober's Test	Measured at baseline and at 36 months	Change from baseline of Schober's test at 36 months. Schober's test measures a patient's ability to flex his/her lower back. The examiner makes a mark at L5 (fifth lumbar vertebra) and places one finger 5 cm below and another finger 10 cm above this mark. The patient is asked to touch his/her toes. The examiner measures the increase in distance between the two fingers.
Secondary	Change in Functional Reach Assessment	Measured at baseline and at 36 months	Change from baseline of functional reach assessment at 36 months. Functional reach assessment measures the difference between the length of a person's outstretched arm and their maximal reach forward, while maintaining balance.
Secondary	Change in Timed Get up and go	Measured at baseline and at 36 months	Change from baseline of timed get up and go at 36 months. In timed get up and go, the patient is asked to stand up from a standard chair and walk a distance of 3 meters, turn around and walk back to the chair and sit down. The examiner measures the time it takes for the patient to perform this series of tasks.
Secondary	Change in 6 Minute Walk Test (6MWT)	Measured at baseline and at 36 months	Change from baseline of the 6MWT at 36 months. The 6MWT measures the distance that a patient can quickly walk on a flat hard surface in a period of six minutes.

Source ClinicalTrials.gov

Outcomes5

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

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Change in Total ROM Worse Hip	Measured at baseline and at 36 months	Intention to treat.	degrees	MEAN
SECONDARY	Change in Schober's Test	Measured at baseline and at 36 months	In the control group, two patients who dropped out for personal reasons were not included in this analysis, and in the treated group one patient who died was not included in this analysis.	cm	MEAN
SECONDARY	Change in Functional Reach Assessment	Measured at baseline and at 36 months	In the control group, two patients who dropped out for personal reasons were not included in this analysis, and in the treated group one patient who died was not included in this analysis.	cm	MEAN
SECONDARY	Change in Timed Get up and go	Measured at baseline and at 36 months	In the control group, two patients who dropped out for personal reasons were not included in this analysis, and in the treated group one patient who died was not included in this analysis.	seconds	MEAN
SECONDARY	Change in 6 Minute Walk Test (6MWT)	Measured at baseline and at 36 months	In the control group, two patients who dropped out for personal reasons were not included in this analysis, and in the treated group one patient who died was not included in this analysis.	ft	MEAN

Source ClinicalTrials.gov

Results by group10

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

Outcome	Group	Value	Spread	Units
Change in Total ROM Worse Hip	Control	-9.1	Standard Deviation 24.7	degrees
Change in Total ROM Worse Hip	Nitisinone-treated	1.6	Standard Deviation 21.8	degrees
Change in Schober's Test	Control	-0.06	Standard Deviation 1.33	cm
Change in Schober's Test	Nitisinone-treated	0.12	Standard Deviation 1.05	cm
Change in Functional Reach Assessment	Control	-1.21	Standard Deviation 3.81	cm
Change in Functional Reach Assessment	Nitisinone-treated	-1.86	Standard Deviation 3.59	cm
Change in Timed Get up and go	Control	-0.54	Standard Deviation 1.84	seconds
Change in Timed Get up and go	Nitisinone-treated	-1.33	Standard Deviation 5.04	seconds
Change in 6 Minute Walk Test (6MWT)	Control	22	Standard Deviation 356	ft
Change in 6 Minute Walk Test (6MWT)	Nitisinone-treated	169	Standard Deviation 591	ft

Source ClinicalTrials.gov

Participant flow6

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

Period	Milestone	Group	Participants
Overall Study	STARTED	Control	20
Overall Study	STARTED	Nitisinone-treated	20
Overall Study	COMPLETED	Control	17
Overall Study	COMPLETED	Nitisinone-treated	16
Overall Study	NOT COMPLETED	Control	3
Overall Study	NOT COMPLETED	Nitisinone-treated	4

Source ClinicalTrials.gov

Adverse events26

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	elevated liver enzymes	Hepatobiliary disorders	Control	6	20	8
other	elevated liver enzymes	Hepatobiliary disorders	Nitisinone-treated	6	20	8
other	depression	Psychiatric disorders	Control	2	20	2
other	depression	Psychiatric disorders	Nitisinone-treated	0	20	0
other	upper respiratory infection	Infections and infestations	Control	5	20	6
other	upper respiratory infection	Infections and infestations	Nitisinone-treated	2	20	2
other	skin rash	Skin and subcutaneous tissue disorders	Control	3	20	3
other	skin rash	Skin and subcutaneous tissue disorders	Nitisinone-treated	0	20	0
other	sensorineural hearing loss	Ear and labyrinth disorders	Control	0	0	0
other	sensorineural hearing loss	Ear and labyrinth disorders	Nitisinone-treated	3	20	3

1 to 10 of 26Source ClinicalTrials.gov

See all 26 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Control
Total, all-cause mortality	Nitisinone-treated
Total, other adverse events	Control	10	20
Total, other adverse events	Nitisinone-treated	10	20
Total, serious adverse events	Control	0	20
Total, serious adverse events	Nitisinone-treated	6	20

Source ClinicalTrials.gov

Papers3

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
8188241	BACKGROUND	PubMed
12359141	BACKGROUND	PubMed
12501223	BACKGROUND	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 comparisons1

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

Comparison
Nitisinone-treated vs Control: serious adverse events

Source Trial comparisons

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/NCT00107783

GET https://conductscience.org/api/trials/v1/NCT00107783/events?page=2

API reference For AI assistants