Cockayne SyndromePhase 1/2NCT01142154
Pharmacokinetics and Safety Study of Single and Multiple Oral Doses Prodarsan™ in Patients With Cockayne Syndrome
A Phase 1/2 study testing Prodarsan in people with Cockayne Syndrome, run by DNage B.V. at 1 site in United States.
Trial summary
This study is to compare the exposure of orally administered Prodarsan to the intravenous dosed Osmitrol (10% solution) in Cockayne Syndrome (CS) patients. Also the pharmacokinetics of single and multiple orally dosed Prodarsan will be evaluated and compared to intravenous dose of Osmitrol in CS patients. Read more about this study
- Who is it for?
- Ages 2 to 10 years
- Conditions the study is for
- Cockayne Syndrome
- What is being tested?
- Prodarsan
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2010to Feb 2011
- Phase
- Phase 1/2
- 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 2 to 10 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Parents or legal guardian(s) of the pediatric patient with CS must be willing and able to give written Informed Consent. Informed Assent will be offered to children who can understand and participate in the Informed Assent process.
- Diagnosis of CS confirmed by one of the following laboratory diagnostic test results:
- Demonstration by molecular diagnostic analyses of two mutations in either the ERCC6 gene or the ERCC8 gene, wherein both mutations are either known to be pathogenic or are obviously detrimental (including nonsense or frameshift mutations, mutations of "invariant" splice site consensus signals, or large deletions/rearrangements); OR
- A pattern of DNA repair responses in patient's cultured skin fibroblast cells indicative of a specific deficiency of transcription-coupled DNA nucleotide excision repair after irradiation with ultraviolet light, namely a significant deficiency of cellular survival (and/or "recovery of ribonucleic acid [RNA] synthesis," if that has been specifically measured) coupled with a normal test for "unscheduled DNA synthesis" OR
- Decreased cell survival and/or "recovery of RNA synthesis" in UV-irradiated patient's skin fibroblast cultures and rescue of these parameters by fusion to reference cell lines with known NER defects (functional complementation analysis) OR
- Quantitative RT-PCR to quantify mRNA levels of CS-A and CS-B transcripts.
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- Weight inclusive of 10 kg to 25 kg.
- Male or female, inclusive of two (2) to ten (10) years of age.
- Clinically acceptable hematocrit as judged by the Principal Investigator (PI).
- The investigator has the opinion that the patient and caregiver are willing and able to comply with protocol requirements.
Who cannot join
- Any concurrent illness (other than related to CS), disability or clinically significant abnormality, including laboratory tests, that may affect the interpretation of the PK or safety data or prevent the patient from safely completing the assessments required by the protocol as judged by the investigator. Such conditions include, but are not limited to:
- Ascites or generalized edema.
- Nephrotic syndrome or history of abnormal kidney function.
- Clinically significant thyrotoxicosis.
- Known history of hyperprolinemia.
- Clinically significant dehydration as judged by the investigator
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- Severely compromised venous access.
- Presence of an external ventricular, abdominal, or chest drain.
- Subjects due to receive radioiodine therapy, two (2) weeks before or two (2) weeks following the study period.
- Participation in another PK or treatment clinical study within thirty (30) days prior to signing and dating of Informed Consent/Assent Form for this study.
- As judged by the investigator, clinical features present at the time of initial screening, that are associated with the terminal phases of the natural progression of CS, indicating that safe travel and completion of the study and its assessments are unlikely, including any of the following:
- Continuous or intermittent dependence on supplemental oxygen at home during the six (6) months prior to enrollment in this study; OR
- Two or more hospitalizations due to pneumonia, during the twelve (12) months prior to enrollment in this study; OR
- A documented, net weight loss of at least 10%, which has not been recovered, and which includes a significant net weight loss (beyond the estimated error of the measurement) over the most recent 6 months despite intensive nutritional support including the use of gastrostomy tube feedings.
- Known hypersensitivity to any of the components found in Prodarsan, D-mannitol, iohexol or iodine compounds.
- History of clinically significant drug sensitivity or allergic reaction such as anaphylaxis.
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Hospital Boston
Boston, Massachusetts, 02115
About this study
Official title: A Phase I/II Crossover Study To Evaluate and Compare the Pharmacokinetics of a Single IV Dose of D-Mannitol (Osmitrol®10%) to Single and Multiple, Escalating Doses of Liquid, Oral Prodarsan™ in Patients With Cockayne Syndrome
This study is to compare the exposure of orally administered Prodarsan to the intravenous dosed Osmitrol (10% solution) in Cockayne Syndrome (CS) patients. Also the pharmacokinetics of single and multiple orally dosed Prodarsan will be evaluated and compared to intravenous dose of Osmitrol in CS patients.
- Conditions
- Cockayne Syndrome
- Being tested
- Prodarsan
- Keywords
- Cockayne Syndrome, Ageing, pediatrics, Pharmacokinetics, Prodarsan, Dwarfism, Genetic disease, inborn, DNA Repair-Deficiency Disorders, Tolerability, Safety
- Medical subject headings
- Cockayne Syndrome, Dwarfism, Genetic Diseases, Inborn, DNA Repair-Deficiency Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ProdarsanDrug
Prodarsan TID, oral solution, 6-8 days
| Study group | Role | What they receive |
|---|---|---|
| oral, liquid solution | Experimental | Not described Receives: Drug: Prodarsan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluate and compare the pharmacokinetics of D-mannitol following a single IV dose of Osmitrol to single and multiple oral doses of Prodarsan in pediatric patients with Cockayne Syndrome
Measured: 6 months
Secondary outcomes (1)
Evaluate the safety and tolerability of administering oral Prodarsan in CS patients over a six (6) to eight (8) day period, including dose escalation to reach a Target Dose
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jun 2010 (estimated)
- Study startJun 2010
- Primary completionSep 2010
- Study completionFeb 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cockayne SyndromeStudies 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 1/2
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- DNage B.V.
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Edward Neilan, MD, Principal investigator, Boston Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01142154
- Sponsor's protocol ID
- MP1104-02
- Status last verified
- Jun 2011
- First submitted
- 20 May 2010
- Last updated
- 23 Jun 2011
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 23 Jun 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Crossover Study To Evaluate and Compare the Pharmacokinetics of a Single IV Dose of D-Mannitol (Osmitrol®10%) to Single and Multiple, Escalating Doses of Liquid, Oral Prodarsan™ in Patients With Cockayne Syndrome
- ClinicalTrials.gov ID
- NCT01142154
- Acronym / sponsor ID
- MP1104-02
- Conditions
- Cockayne Syndrome
- Keywords
- Cockayne Syndrome, Ageing, pediatrics, Pharmacokinetics, Prodarsan, Dwarfism, Genetic disease, inborn, DNA Repair-Deficiency Disorders
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- DNage B.V.Industry (pharma, biotech, devices)
- Study start
- Jun 2010
- Primary completion
- Sep 2010 (actual)
- Study completion
- Feb 2011 (actual)
- First posted
- 11 Jun 2010 (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.
This study is to compare the exposure of orally administered Prodarsan to the intravenous dosed Osmitrol (10% solution) in Cockayne Syndrome (CS) patients. Also the pharmacokinetics of single and multiple orally dosed Prodarsan will be evaluated and compared to intravenous dose of Osmitrol in CS patients.
Study design
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| oral, liquid solution | Experimental | Drug: Prodarsan |
Who may qualify
- Ages
- 2 to 10 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (10 listed)
- Parents or legal guardian(s) of the pediatric patient with CS must be willing and able to give written Informed Consent. Informed Assent will be offered to children who can understand and participate in the Informed Assent process.
- Diagnosis of CS confirmed by one of the following laboratory diagnostic test results:
- Weight inclusive of 10 kg to 25 kg.
- Male or female, inclusive of two (2) to ten (10) years of age.
- Clinically acceptable hematocrit as judged by the Principal Investigator (PI).
Key exclusion criteria (16 listed)
- Any concurrent illness (other than related to CS), disability or clinically significant abnormality, including laboratory tests, that may affect the interpretation of the PK or safety data or prevent the patient from safely completing the assessments required by the protocol as judged by the investigator. Such conditions include, but are not limited to:
- Severely compromised venous access.
- Presence of an external ventricular, abdominal, or chest drain.
- Subjects due to receive radioiodine therapy, two (2) weeks before or two (2) weeks following the study period.
- Participation in another PK or treatment clinical study within thirty (30) days prior to signing and dating of Informed Consent/Assent Form for this study.
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 |
|---|---|---|
| ProdarsanProdarsan TID, oral solution, 6-8 days | Drug | oral, liquid solution |
Primary outcome
Evaluate and compare the pharmacokinetics of D-mannitol following a single IV dose of Osmitrol to single and multiple oral doses of Prodarsan in pediatric patients with Cockayne Syndrome
Time frame: 6 months
1 secondary outcome
Evaluate the safety and tolerability of administering oral Prodarsan in CS patients over a six (6) to eight (8) day period, including dose escalation to reach a Target Dose
Time frame: 6 months
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 23 Jun 2011.
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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.
Edward Neilan, MD principal investigator
Boston Children's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Prodarsan in people with Cockayne Syndrome, run by DNage B.V. at 1 site in United States.
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 10 inclusion and 16 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: finished, as of the sponsor's last update on 23 Jun 2011. 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 United States. 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.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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.
What does the primary outcome measure?
Evaluate and compare the pharmacokinetics of D-mannitol following a single IV dose of Osmitrol to single and multiple oral doses of Prodarsan in pediatric patients with Cockayne Syndrome (time frame: 6 months). 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 23 Jun 2011.