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Last Updated: December 6, 2022

CLINICAL TRIALS PROFILE FOR ORFADIN


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All Clinical Trials for Orfadin

Trial ID Title Status Sponsor Phase Start Date Summary
NCT00107783 ↗ Long-Term Study of Nitisinone to Treat Alkaptonuria Completed National Human Genome Research Institute (NHGRI) Phase 2 2005-01-01 This 3-year study will examine the safety and effectiveness of long-term use of nitisinone (Orfadin) for treating joint problems in patients with alkaptonuria, an inherited disease in which a compound called homogentisic acid accumulates. The excess homogentisic acid causes arthritis and limited joint movement. It can also cause heart valve damage and kidney stones. Patients between 30 and 80 years of age with alkaptonuria may be eligible for this study. Patients must have hip involvement, but at least one remaining hip joint. Candidates are recruited from among patients enrolled in protocol 00-HG-0141, "Clinical, Biochemical, and Molecular Investigations into Alkaptonuria." Participants may enter both protocols simultaneously. Participants are randomly assigned to one of two treatment groups: one group takes their regular medicines plus a 2-mg nitisinone capsule daily; the other group takes only their regular medicines. Patients taking nitisinone have blood tests to measure liver function 2 weeks and 6 weeks after starting treatment. Before starting therapy, all patients are admitted to the NIH Clinical Center for 4-5 days to undergo the following procedures: - Medical history and physical examination - 24-hour urine collection to test for sugar, protein, and other molecules - Blood tests for liver and thyroid function, blood counts, and blood chemistries - Blood and urine tests to measure tyrosine and other amino acids and homogentisic acid - Bone x-rays - Spiral CT (computed tomography) of the abdomen to detect kidney stones - Eye examination and evaluations by specialists in rehabilitation medicine and pain, plus other consults in skin, brain, lung, heart, and kidney, as needed All patients, whether or not they receive nitisinone, return to the Clinical Center for a 2-3 day follow-up admission every 4 months for a history and physical examination, blood tests, and two 24-hour urine collections. Every 12 months (12, 24 and 36 months after starting the study), patients also have repeat bone x-rays, spiral CT, kidney ultrasound, echocardiogram, and electrocardiogram. An Magnetic Resonance Imaging (MRI) of the brain is done at the end of the study. Sixteen months after the end of the study enrollment period, the treated and non-treated groups are evaluated. If nitisinone has delayed the progression of joint disease in the treated group, the study continues and all patients receive the drug for the remainder of the study. If not, the study continues for another 20 months, at which time the study ends and the evaluation process is repeated. Patients who develop symptoms such as corneal crystals, pain, or severe liver or nervous system toxicity may be taken off the study.
NCT01682538 ↗ Bioequivalence of Orfadin Suspension Compared to Orfadin Capsules, and the Effect of Food on the Bioavailability of the Suspension Completed Swedish Orphan Biovitrum Phase 1 2012-08-01 The study is primarily being performed in order to demonstrate bioequivalence between the Orfadin (nitisinone) suspension and the marketed capsule formulation. The study will also contain a comparison of the bioavailability of the suspension given with food and on an empty stomach.
NCT01734889 ↗ Taste and Palatability of Orfadin Suspension Completed Swedish Orphan Biovitrum Phase 1 2012-10-01 The purpose of this study is to verify that pediatric patients, especially those who are not old enough to swallow capsules, accept the taste and palatability of a new suspension.
NCT01838655 ↗ Nitisinone for Type 1B Oculocutaneous Albinism Completed National Human Genome Research Institute (NHGRI) Phase 1/Phase 2 2013-04-16 Background: - Oculocutaneous albinism, type 1B (OCA1B) is a genetic disease caused by problems in the gene that makes tyrosine. Tyrosine is an amino acid needed to produce pigment in the skin, hair, and eyes. People with OCA1B have pale skin, white hair, and light-colored eyes. Pigment in the back of the eye helps vision, so people with OCA-1B often have visual problems. Researchers want to see if a drug called nitisinone can help improve eye pigmentation and vision in people with OCA1B. Nitisinone is approved for treating a related genetic disease that causes problems with tyrosine, so it may help people with OCA1B. Objectives: - To see if nitisinone can help improve eye pigmentation and vision in people with OCA1B. Eligibility: - Individuals at least 18 years of age who have OCA1B. Design: - This study will last about 18 months. It requires eight outpatient visits, each about 3 months apart. Each visit will require 1 to 2 days of testing. - Participants will be screened with a physical exam, eye exam, and medical history. They will have additional vision and neurological tests. They will be tested to see how their brain and retinas respond to light. They will also take hair and blood samples, and answer questions about diet. - Participants will receive the study drug. They will take one pill a day for 1 year. They will keep track of the dose in a study diary. - At the outpatient visits, participants will have the following tests: - Medical history and physical exam - Neurological and eye exams - Retina function tests - Tests of the skin and brain's response to light - Blood and urine tests - Dietary consultation - Visual function questionnaire. - After the end of the study, participants will return to the care of their regular eye doctor.
NCT01838655 ↗ Nitisinone for Type 1B Oculocutaneous Albinism Completed National Eye Institute (NEI) Phase 1/Phase 2 2013-04-16 Background: - Oculocutaneous albinism, type 1B (OCA1B) is a genetic disease caused by problems in the gene that makes tyrosine. Tyrosine is an amino acid needed to produce pigment in the skin, hair, and eyes. People with OCA1B have pale skin, white hair, and light-colored eyes. Pigment in the back of the eye helps vision, so people with OCA-1B often have visual problems. Researchers want to see if a drug called nitisinone can help improve eye pigmentation and vision in people with OCA1B. Nitisinone is approved for treating a related genetic disease that causes problems with tyrosine, so it may help people with OCA1B. Objectives: - To see if nitisinone can help improve eye pigmentation and vision in people with OCA1B. Eligibility: - Individuals at least 18 years of age who have OCA1B. Design: - This study will last about 18 months. It requires eight outpatient visits, each about 3 months apart. Each visit will require 1 to 2 days of testing. - Participants will be screened with a physical exam, eye exam, and medical history. They will have additional vision and neurological tests. They will be tested to see how their brain and retinas respond to light. They will also take hair and blood samples, and answer questions about diet. - Participants will receive the study drug. They will take one pill a day for 1 year. They will keep track of the dose in a study diary. - At the outpatient visits, participants will have the following tests: - Medical history and physical exam - Neurological and eye exams - Retina function tests - Tests of the skin and brain's response to light - Blood and urine tests - Dietary consultation - Visual function questionnaire. - After the end of the study, participants will return to the care of their regular eye doctor.
NCT01857362 ↗ Bioequivalence of Orfadin 20 mg Compared to Orfadin 10 mg Capsules. Completed Swedish Orphan Biovitrum Phase 1 2013-05-01 The purpose of this study is to evaluate the bioequivalence between Orfadin 20 mg and 10 mg capsules in healthy volunteers.
NCT01916382 ↗ Suitability of Nitisinone in Alkaptonuria 2 Unknown status University of Liverpool Phase 3 2014-04-01 This is a proposal to develop the orphan designated drug, nitisinone, for the treatment of a rare Mendelian disease, Alkaptonuria (AKU). Thanks to our existing successful fundamental and clinical research (cell models, animal models, natural history studies), we are now ready for this final stage of clinical development of nitisinone for AKU: a phase 3 clinical trial to prove efficacy. The results of DevelopAKUre will allow us to make the case to the European Medicines Agency for marketing authorisation of nitisinone for AKU, thereby contributing to the goal of the International Rare Diseases Research Consortium of developing 200 new therapies by 2020.
>Trial ID >Title >Status >Phase >Start Date >Summary

Clinical Trial Conditions for Orfadin

Condition Name

Condition Name for Orfadin
Intervention Trials
Hereditary Tyrosinemia, Type I 6
Alkaptonuria 2
Healthy Volunteers 1
Vision Loss 1
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Condition MeSH

Condition MeSH for Orfadin
Intervention Trials
Tyrosinemias 6
Alkaptonuria 2
Ochronosis 2
Albinism, Oculocutaneous 1
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Clinical Trial Locations for Orfadin

Trials by Country

Trials by Country for Orfadin
Location Trials
Germany 4
Netherlands 3
United Kingdom 3
France 3
United States 2
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Trials by US State

Trials by US State for Orfadin
Location Trials
Maryland 2
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Clinical Trial Progress for Orfadin

Clinical Trial Phase

Clinical Trial Phase for Orfadin
Clinical Trial Phase Trials
Phase 3 2
Phase 2 1
Phase 1/Phase 2 1
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Clinical Trial Status

Clinical Trial Status for Orfadin
Clinical Trial Phase Trials
Completed 10
Unknown status 2
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Clinical Trial Sponsors for Orfadin

Sponsor Name

Sponsor Name for Orfadin
Sponsor Trials
Swedish Orphan Biovitrum 6
Parexel 3
National Human Genome Research Institute (NHGRI) 2
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Sponsor Type

Sponsor Type for Orfadin
Sponsor Trials
Industry 12
NIH 3
Other 1
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