Erythropoietic ProtoporphyriaNCT00004831
Study of Cysteine Hydrochloride for Erythropoietic Protoporphyria
A study testing cysteine hydrochloride in people with Erythropoietic Protoporphyria, run by FDA Office of Orphan Products Development.
Trial summary
OBJECTIVES: I. Determine the efficacy of cysteine hydrochloride in preventing or decreasing photosensitivity in patients with erythropoietic protoporphyria. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Erythropoietic Protoporphyria
- What is being tested?
- cysteine hydrochloride
- How many people
- 20 participants
- Where
- No sites listed yet
- Study dates
- Oct 1996to Jul 1998
- 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 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics-- Documented erythropoietic protoporphyria Determination of elevated protoporphyrin within the past year Experiencing photosensitivity --Prior/Concurrent Therapy-- No concurrent use of betacarotene --Patient Characteristics-- Other: Not pregnant or nursing Fertile female patients must use effective contraception while on study and for 3 weeks thereafter
About this study
OBJECTIVES:
- Determine the efficacy of cysteine hydrochloride in preventing or decreasing photosensitivity in patients with erythropoietic protoporphyria.
Read the detailed description
PROTOCOL OUTLINE: This is a randomized, double blind, placebo controlled, crossover study.
Patients are randomly assigned to 1 of 2 groups to receive cysteine hydrochloride orally twice daily, 2 capsules with breakfast and 2 with lunch. Group 1 receives cysteine hydrochloride in drug ingestion period 1 followed by placebo in period 2. Group 2 receives placebo in period 1 followed by cysteine hydrochloride in period 2. Both groups ingest placebo for 1 week between the periods. Each drug ingestion period lasts 8 weeks.
Follow up phone calls are made at the end of months 1 and 3. All patients schedule follow up visits at the end of each drug ingestion period.
- Conditions
- Erythropoietic Protoporphyria
- Being tested
- cysteine hydrochloride
- Keywords
- erythropoietic protoporphyria, inborn errors of metabolism, porphyria, rare disease
- Medical subject headings
- Protoporphyria, Erythropoietic, Metabolism, Inborn Errors, Porphyrias, Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
cysteine hydrochlorideDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Feb 2000 (estimated)
- Study startOct 1996
- Study completionJul 1998
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Erythropoietic ProtoporphyriaStudies 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
- Double blind
- Enrolment
- 20 participants
Sponsor and investigators
- Lead sponsor
- FDA Office of Orphan Products Development
- Sponsor type
- US federal agency
- Collaborators
- St. Luke's-Roosevelt Hospital Center
- Investigators
- Micheline M Mathews-Roth, Study chair, St. Luke's-Roosevelt Hospital Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00004831
- Sponsor's protocol ID
- 199/13413
- Other IDs
- BWH-FDR000996-EF, SLRH-CU-FDR000996-EF
- Status last verified
- Aug 1998
- First submitted
- 24 Feb 2000
- Last updated
- 25 Mar 2015
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Mar 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of Cysteine Hydrochloride for Erythropoietic Protoporphyria
- ClinicalTrials.gov ID
- NCT00004831
- Acronym / sponsor ID
- 199/13413
- Conditions
- Erythropoietic Protoporphyria
- Keywords
- erythropoietic protoporphyria, inborn errors of metabolism, porphyria, rare disease
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants
- Lead sponsor
- FDA Office of Orphan Products DevelopmentUS federal agency
- Collaborators
- St. Luke's-Roosevelt Hospital Center
- Study start
- Oct 1996
- Study completion
- Jul 1998
- First posted
- 25 Feb 2000 (estimated)
- 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.
OBJECTIVES:
- Determine the efficacy of cysteine hydrochloride in preventing or decreasing photosensitivity in patients with erythropoietic protoporphyria.
Detailed description
PROTOCOL OUTLINE: This is a randomized, double blind, placebo controlled, crossover study.
Patients are randomly assigned to 1 of 2 groups to receive cysteine hydrochloride orally twice daily, 2 capsules with breakfast and 2 with lunch. Group 1 receives cysteine hydrochloride in drug ingestion period 1 followed by placebo in period 2. Group 2 receives placebo in period 1 followed by cysteine hydrochloride in period 2. Both groups ingest placebo for 1 week between the periods. Each drug ingestion period lasts 8 weeks.
Follow up phone calls are made at the end of months 1 and 3. All patients schedule follow up visits at the end of each drug ingestion period.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
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 |
|---|---|---|
| cysteine hydrochloride | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Mar 2015.
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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.
Micheline M Mathews-Roth study chair
St. Luke's-Roosevelt Hospital Center
Questions about this trial
What is this clinical trial studying?
A study testing cysteine hydrochloride in people with Erythropoietic Protoporphyria, run by FDA Office of Orphan Products Development.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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: finished, as of the sponsor's last update on 25 Mar 2015. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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 25 Mar 2015.