Muscular Dystrophy, DuchennePhase 1/2NCT00243789
Study of Daily Pentoxifylline as a Rescue Treatment in Duchenne Muscular Dystrophy
A Phase 1/2 study testing Pentoxifylline in people with Muscular Dystrophy, Duchenne, run by Cooperative International Neuromuscular Research Group at 11 sites in 6 countries.
Trial summary
The purpose of this study is to see if male children with Duchenne muscular dystrophy (DMD) have changes in strength when given the drug Pentoxifylline as a rescue treatment. A total of 64 subjects are expected to participate through all other centers of the Cooperative International Neuromuscular Research Group… Read more about this study
- Who is it for?
- Ages 7 years and older, men only
- Conditions the study is for
- Muscular Dystrophy, Duchenne
- What is being tested?
- Pentoxifylline
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 64 participants
- Where
- 11 sitesin 6 countries
- Study dates
- Sep 2005to Jan 2008
- 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 7 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male
- Age 7 years to 100 years
- Ability to ambulate for 10 meters. Assistive devices are allowed.
- Diagnosis of DMD confirmed by at least one the following:
- On stable dose of prednisone, prednisolone or deflazacort for at least 12 months prior to screening.
- Participants who are on stable dose of any combination of the following compounds (creatine, glutamine, coenzyme Q10, vitamin E, C or D, JUVEN, arginine, calcium) must have taken these medications for at least 2 months prior to screening. Subjects are not required to take these medications to participate in the study.
Show all 9Show fewer
- All other herbs, supplements or green tea (other than those noted above) have been discontinued 3 months prior to screening.
- Ability to provide reproducible QMT bicep score with no more than 15% variation between scores during screening.
- Normal blood clotting ability evidenced by a platelet function assessment (PFA).
Who cannot join
- Currently enrolled in another treatment clinical trial.
- History of significant concomitant illness or significant impairment of renal or hepatic function.
- History of impairment of blood clotting ability (as evidenced by increased PT/PTT or PFA over the upper limit of normal (ULN)).
- Recent cerebral or retinal hemorrhage.
- History of bleeding diathesis or gastric ulcer.
Trial locations and contacts
11 sites in 6 countries.
11 sites in 6 countries
United States5 sites
University of Tennessee
Memphis, Tennessee, 38104
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15213
Mayo Clinic
Rochester, Minnesota, 55905
Washington University, St. Louis
St Louis, Missouri, 63110
Children's National Medical Center
Washington D.C., District of Columbia, 20010
Canada2 sites
Alberta Children's Hospital
Calgary, Alberta, T2T 5C7
University of Alberta
Edmonton, Alberta, T6G 2J3
About this study
Official title: A Double-Blinded Randomized Placebo Controlled Study of Daily Pentoxifylline as a Rescue Treatment in DMD
The purpose of this study is to see if male children with Duchenne muscular dystrophy (DMD) have changes in strength when given the drug Pentoxifylline as a rescue treatment. A total of 64 subjects are expected to participate through all other centers of the Cooperative International Neuromuscular Research Group (CINRG) worldwide.
The primary purpose of this study is to see whether the addition of pentoxifylline to a steroid regimen is effective in treating deteriorating muscle strength by comparing the muscle strength of PTX treated subjects and placebo treated subjects.
Read the detailed description
DMD is the most common and devastating type of muscular dystrophy (incidence 1 in 3500 live born males worldwide). DMD is characterized by a complete loss of dystrophin, leading to progressive muscle weakness and wasting.
No cure is currently available despite our present understanding of the disorder and the discovery and characterization of the causative gene and its protein product dystrophin in 1987. Corticosteroids (prednisone, deflazacort) may delay disease progression and until now it is the only treatment that proved to be beneficial for patients with DMD. Other alternative supplements like creatine and glutamine also delay diseased progression.
- Conditions
- Muscular Dystrophy, Duchenne
- Being tested
- Pentoxifylline
- Keywords
- Duchenne, Genetic, Muscular Dystrophy, DMD
- Medical subject headings
- Muscular Dystrophy, Duchenne, Muscular Dystrophies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PentoxifyllineDrug
Also called Trental
Participants will be randomized to receive either pentoxifylline or placebo in addition to their stable steroid therapy. Active drug and placebo preparations will be supplied as gel capsules of identical size, appearance and taste. Active drug capsules will contain one 400 mg time-release pentoxifylline tablet and inert filler. Placebo capsules will contain inert filler.
Based on weight at screening, <30 mg will receive 1 400 capsule/day; 30-49 kg will receive two 400 capsules/day; 50 kg or greater will receive three 400 mg capsules/day.
| Study group | Role | What they receive |
|---|---|---|
| 1 | Active comparator | Pentoxifylline Receives: Drug: Pentoxifylline |
| 2 | No intervention | Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Quantitative muscle strength will be measured using a CINRG Quantitative Muscle System (CQMS). The highest value of two consecutive maximal efforts will be recorded. The primary strength endpoint will be total CQMS score.
Measured: January 2008
Secondary outcomes (8)
Strength of arm, leg and grip QMT scores Measured Screening and Months 1, 3, 6, 9 & 12
Measured: January 2008
Manual Muscle Testing (MMT) score measured at screening and months 1, 3, 6, 9 & 12 using the Medical Research Council (MRC) scoring system.
Measured: January 2008
Functional evaluations measured at screening and months 1, 3, 6, 9 & 12
Measured: January 2008
Time function assessments, including time rising from the floor, time to climb four standard stairs, and time to walk 10 meters. They will be measured at screening and months 1, 3, 6, 9 & 12.
Measured: January 2008
pulmonary function test (PFA's) measured at screening and months 1, 3, 6, 9 & 12
Measured: January 2008
Pediatric Quality of Life (PQOL) measured at screening and months 1, 3, 6, 9 & 12
Measured: January 2008
Goniometry measured at screening and months 1, 3, 6, 9 & 12
Measured: January 2008
TNF-alpha and TGF-beta measured at screening and months 1, 3, 6, 9 & 12
Measured: February 2008
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Oct 2005 (estimated)
- Study startSep 2005
- Primary completionDec 2007
- Study completionJan 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Muscular Dystrophy, DuchenneStudies 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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 64 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cooperative International Neuromuscular Research Group
- Sponsor type
- Research network
- Investigators
- Diana Escolar, MD, Study chair, Children's National Medical Center, Center for Genetic Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00243789
- Sponsor's protocol ID
- CNMC0705
- Status last verified
- Oct 2011
- First submitted
- 21 Oct 2005
- Last updated
- 27 Oct 2011
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 27 Oct 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Double-Blinded Randomized Placebo Controlled Study of Daily Pentoxifylline as a Rescue Treatment in DMD
- ClinicalTrials.gov ID
- NCT00243789
- Acronym / sponsor ID
- CNMC0705
- Conditions
- Muscular Dystrophy, Duchenne
- Keywords
- Duchenne, Genetic, Muscular Dystrophy, DMD
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 64 participants (actual)
- Lead sponsor
- Cooperative International Neuromuscular Research GroupResearch network
- Study start
- Sep 2005
- Primary completion
- Dec 2007 (actual)
- Study completion
- Jan 2008 (actual)
- First posted
- 25 Oct 2005 (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.
The purpose of this study is to see if male children with Duchenne muscular dystrophy (DMD) have changes in strength when given the drug Pentoxifylline as a rescue treatment. A total of 64 subjects are expected to participate through all other centers of the Cooperative International Neuromuscular Research Group (CINRG) worldwide.
The primary purpose of this study is to see whether the addition of pentoxifylline to a steroid regimen is effective in treating deteriorating muscle strength by comparing the muscle strength of PTX treated subjects and placebo treated subjects.
Detailed description
DMD is the most common and devastating type of muscular dystrophy (incidence 1 in 3500 live born males worldwide). DMD is characterized by a complete loss of dystrophin, leading to progressive muscle weakness and wasting.
No cure is currently available despite our present understanding of the disorder and the discovery and characterization of the causative gene and its protein product dystrophin in 1987. Corticosteroids (prednisone, deflazacort) may delay disease progression and until now it is the only treatment that proved to be beneficial for patients with DMD. Other alternative supplements like creatine and glutamine also delay diseased progression.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1 | Active comparator | Drug: PentoxifyllinePentoxifylline |
| 2 | No intervention | –Placebo |
Who may qualify
- Ages
- 7 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- Male
- Age 7 years to 100 years
- Ability to ambulate for 10 meters. Assistive devices are allowed.
- Diagnosis of DMD confirmed by at least one the following:
- On stable dose of prednisone, prednisolone or deflazacort for at least 12 months prior to screening.
Key exclusion criteria (5 listed)
- Currently enrolled in another treatment clinical trial.
- History of significant concomitant illness or significant impairment of renal or hepatic function.
- History of impairment of blood clotting ability (as evidenced by increased PT/PTT or PFA over the upper limit of normal (ULN)).
- Recent cerebral or retinal hemorrhage.
- History of bleeding diathesis or gastric ulcer.
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 |
|---|---|---|
| PentoxifyllineAlso: TrentalParticipants will be randomized to receive either pentoxifylline or placebo in addition to their stable steroid therapy. Active drug and placebo preparations will be supplied as gel capsules of identical size, appearance and taste. Active drug capsules will contain one 400 mg time-release pentoxifylline tablet and inert filler. Placebo capsules will contain inert filler. Based on weight at screening, <30 mg will receive 1 400 capsule/day; 30-49 kg will receive two 400 capsules/day; 50 kg or greater will receive three 400 mg capsules/day. | Drug | 1 |
Primary outcome
Quantitative muscle strength will be measured using a CINRG Quantitative Muscle System (CQMS). The highest value of two consecutive maximal efforts will be recorded. The primary strength endpoint will be total CQMS score.
Time frame: January 2008
8 secondary outcomes
Strength of arm, leg and grip QMT scores Measured Screening and Months 1, 3, 6, 9 & 12
Time frame: January 2008
Manual Muscle Testing (MMT) score measured at screening and months 1, 3, 6, 9 & 12 using the Medical Research Council (MRC) scoring system.
Time frame: January 2008
Functional evaluations measured at screening and months 1, 3, 6, 9 & 12
Time frame: January 2008
Time function assessments, including time rising from the floor, time to climb four standard stairs, and time to walk 10 meters. They will be measured at screening and months 1, 3, 6, 9 & 12.
Time frame: January 2008
pulmonary function test (PFA's) measured at screening and months 1, 3, 6, 9 & 12
Time frame: January 2008
Pediatric Quality of Life (PQOL) measured at screening and months 1, 3, 6, 9 & 12
Time frame: January 2008
Goniometry measured at screening and months 1, 3, 6, 9 & 12
Time frame: January 2008
TNF-alpha and TGF-beta measured at screening and months 1, 3, 6, 9 & 12
Time frame: February 2008
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 27 Oct 2011.
- Related InfoLink provided by the sponsor.
Related trials for Muscular Dystrophy, Duchenne
RecruitingEarly Phase 1
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
Duchenne Muscular Dystrophy (DMD)
RecruitingPhase 2
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Not yet recruitingPhase 2
A Phase 2 Trial to Investigate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tegacorat in Male Participants 4 to <7 Years of Age With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD)
Not yet recruitingObservational study
Multimodal Study About Absence of Dp140 in Becker Muscular Dystrophy: Brain-muscle Imaging, Cognition and Muscle Function
Becker Muscular Dystrophy
Enrolling by invitationObservational study
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
Duchenne Muscular Dystrophy
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.
Diana Escolar, MD study chair
Children's National Medical Center, Center for Genetic Medicine
11 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Pentoxifylline in people with Muscular Dystrophy, Duchenne, run by Cooperative International Neuromuscular Research Group at 11 sites in 6 countries.
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 9 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: finished, as of the sponsor's last update on 27 Oct 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in United States, Canada, Argentina, Australia, Israel and 1 more country. 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?
Quantitative muscle strength will be measured using a CINRG Quantitative Muscle System (CQMS). The highest value of two consecutive maximal efforts will be recorded. The primary strength endpoint will be total CQMS score. (time frame: January 2008). 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 27 Oct 2011.