Hereditary Tyrosinemia, Type IOrfadinNCT04113772
Bio Equivalency 20 Mgm Orfadin and 20 Mgm of Nitisonine
A study testing Nitisinone and Orfadin in people with Hereditary Tyrosinemia, Type I, run by Sutphin Drugs at 1 site in India.
Trial summary
he purpose of this study is to determine whether Nitisinone 10 mg Tablets (Test Product are bioequivalent to the reference product Orfadin 10 mg Read more about this study
- Who is it for?
- Ages 18 to 50 years
- Conditions the study is for
- Hereditary Tyrosinemia, Type I
- What is being tested?
- Nitisinone and Orfadin
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteIndia
- Study dates
- 1 Nov 2019to 31 Mar 2021
- Phase
- Interventional
- 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 18 to 50 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- All HT-1 patients receiving Orfadin treatment are eligible for entry.
- Male and female patients of all ages diagnosed with HT-1.
- Stable lab values, including liver values <2 ULN (ALP, ALT, AST, bilirubin, INR).
- Women of childbearing potential willing to use adequate contraception
- Signed informed consent/assent.
Who cannot join
- Any medical condition which in the opinion of the investigator makes the patient unsuitable for inclusion.
- Enrollment in another concurrent clinical interventional study within three months prior to inclusion in this study.
- Pregnant women.
- Lactating women. .Known hepatitis B, hepatitis C or HIV infection.
- Foreseeable inability to cooperate with given instructions or study procedures.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Orfadin and Nitinosine Study
he purpose of this study is to determine whether Nitisinone 10 mg Tablets (Test Product are bioequivalent to the reference product Orfadin 10 mg
Read the detailed description
Detailed Description:
The specific aim is to conduct a randomized, single dose, three-period crossover bioequivalence study in at least 18 healthy male and female subjects at a single study center to evaluate the in vivo performance of two formulations of Nitisinone 10 mg and the reference product Orfadin under fasting.
A total of 4 healthy female and male volunteers (age 18 to 55 years old) will be entered into the study. Volunteers will be determined to be free of significant medical conditions as assessed by medical history, physical examination, and blood and urine tests. Volunteers will be randomly allocated to a treatment sequence, before administration of investigational nitinosine
Determination of succinylacetone (SA) in blood (serum/plasma) and/or urine will be performed. Results from samples analyzed at the central laboratory, including determination of nitisinone, will be used in the evaluation of pharmacokinetics, efficacy and safety during the two treatment periods.
- Conditions
- Hereditary Tyrosinemia, Type I
- Being tested
- Nitisinone, Orfadin
- Medical subject headings
- Tyrosinemias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NitisinoneDrug
Measure bio equivalency/efficacy of nitinosine and orfadin
OrfadinDrug
Orfadin
| Study group | Role | What they receive |
|---|---|---|
| Orfandin .5 mgm/kg mgm bid | Active comparator | Two participant will receive .5 mgm/kg mgm of orfadin Receives: Drug: Orfadin |
| Nitinosine .5 mgm/kg bid | Experimental | Two participant will receive .5 mgm/kg of nitinosine Receives: Drug: Nitisinone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Succinylacetone level
Succinylacetone level will be measured every 2 weeks for eight weeks
Measured: 8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Oct 2019
- Study start1 Nov 2019 (estimated)
- Primary completion31 Dec 2020 (estimated)
- Study completion31 Mar 2021 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hereditary Tyrosinemia, Type IStudies of the same condition, open and under way.
- 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 4 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sutphin Drugs
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04113772
- Sponsor's protocol ID
- Global CRO Orfadin
- Acronym
- Orfadin
- Status last verified
- Oct 2019
- First submitted
- 1 Oct 2019
- Last updated
- 3 Oct 2019
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Oct 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Orfadin and Nitinosine Study
- ClinicalTrials.gov ID
- NCT04113772
- Acronym / sponsor ID
- Orfadin / Global CRO Orfadin
- Conditions
- Hereditary Tyrosinemia, Type I
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 4 participants (estimated)
- Lead sponsor
- Sutphin DrugsIndustry (pharma, biotech, devices)
- Study start
- 1 Nov 2019 (estimated)
- Primary completion
- 31 Dec 2020 (estimated)
- Study completion
- 31 Mar 2021 (estimated)
- First posted
- 3 Oct 2019 (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.
he purpose of this study is to determine whether Nitisinone 10 mg Tablets (Test Product are bioequivalent to the reference product Orfadin 10 mg
Detailed description
Detailed Description:
The specific aim is to conduct a randomized, single dose, three-period crossover bioequivalence study in at least 18 healthy male and female subjects at a single study center to evaluate the in vivo performance of two formulations of Nitisinone 10 mg and the reference product Orfadin under fasting.
A total of 4 healthy female and male volunteers (age 18 to 55 years old) will be entered into the study. Volunteers will be determined to be free of significant medical conditions as assessed by medical history, physical examination, and blood and urine tests. Volunteers will be randomly allocated to a treatment sequence, before administration of investigational nitinosine
Determination of succinylacetone (SA) in blood (serum/plasma) and/or urine will be performed. Results from samples analyzed at the central laboratory, including determination of nitisinone, will be used in the evaluation of pharmacokinetics, efficacy and safety during the two treatment periods.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Single blind (participant)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Orfandin .5 mgm/kg mgm bid | Active comparator | Drug: OrfadinTwo participant will receive .5 mgm/kg mgm of orfadin |
| Nitinosine .5 mgm/kg bid | Experimental | Drug: NitisinoneTwo participant will receive .5 mgm/kg of nitinosine |
Who may qualify
- Ages
- 18 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (5 listed)
- All HT-1 patients receiving Orfadin treatment are eligible for entry.
- Male and female patients of all ages diagnosed with HT-1.
- Stable lab values, including liver values <2 ULN (ALP, ALT, AST, bilirubin, INR).
- Women of childbearing potential willing to use adequate contraception
- Signed informed consent/assent.
Key exclusion criteria (5 listed)
- Any medical condition which in the opinion of the investigator makes the patient unsuitable for inclusion.
- Enrollment in another concurrent clinical interventional study within three months prior to inclusion in this study.
- Pregnant women.
- Lactating women. .Known hepatitis B, hepatitis C or HIV infection.
- Foreseeable inability to cooperate with given instructions or study procedures.
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 |
|---|---|---|
| NitisinoneMeasure bio equivalency/efficacy of nitinosine and orfadin | Drug | Nitinosine .5 mgm/kg bid |
| OrfadinOrfadin | Drug | Orfandin .5 mgm/kg mgm bid |
Primary outcome
Succinylacetone level
Time frame: 8 weeks
Succinylacetone level will be measured every 2 weeks for eight weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Oct 2019.
- Individual participant dataSharing planned: No
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Nitisinone and Orfadin in people with Hereditary Tyrosinemia, Type I, run by Sutphin Drugs at 1 site in India.
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 5 inclusion and 5 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 3 Oct 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in India. Whether a site is taking part right now should be confirmed with that site. See every site.
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?
Contact Ajai Prakash (7185260310, ajaiprakashny@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Succinylacetone level (time frame: 8 weeks). It is what the study was designed to measure, not a finding.
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 3 Oct 2019.