Myotonic DystrophyPhase 1/2NCT00233519
Effects of SomatoKine (Iplex)Recombinant Human Insulin-like Growth Factor-1/Recombinant Human Insulin-like Growth Factor-binding Protein-3 (rhIGF-I/rhIGFBP-3) in Myotonic Dystrophy Type 1 (DM1)
A Phase 1/2 study testing SomatoKine/IPLEX in people with Myotonic Dystrophy, run by University of Rochester at 1 site in United States.
Trial summary
The aim of this study is to investigate the safety and feasibility of daily subcutaneous injections of recombinant IGF1 complexed with IGF binding protein 3 (SomatoKine-INSMED) as a treatment for muscle wasting and weakness in myotonic dystrophy type 1. Read more about this study
- Who is it for?
- Ages 21 to 60 yearsHealthy volunteers can take part
- Conditions the study is for
- Myotonic Dystrophy
- What is being tested?
- SomatoKine/IPLEX
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 17 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2005to May 2008
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 21 to 60 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- A clinical diagnosis of DM-1 according to accepted clinical research criteria.23 The clinical research criteria require each of the following: (1) clinically evident myotonia; (2) muscle weakness in a characteristic distribution (distal predominant); and (3) similar findings in a first degree relative.
- Age 21 to 60 years (inclusive).
- Ability to walk 30 feet without assistance (cane and leg bracing is permitted).
- Weakness of sufficient severity to justify treatment and provide a reasonable opportunity to observe a therapeutic effect. At the eligibility evaluation, eligible patients must show both of the following:
- muscle strength in a distal muscle group (ankle dorsiflexors or deep flexors of the fingers) which is less than or equal to grade 4 (Medical Research Council grade).
- muscle strength in a proximal or mid-limb muscle group (flexors or extensors of the knee, elbow, shoulder, or hip) which is which is greater than or equal to 4- (Medical Research Council grade).
Show all 9Show fewer
- For patients that are not within driving distance to Rochester, a local health care provider in their area must be able to complete their home visits.
- Competent, willing, and able to give informed consent.
- Able to self-administer study medication by subcutaneous injection or caregiver available to administer study medication.
Who cannot join
- Congenital DM-1. Congenital disease constitutes ~10% of all cases of DM-1. Early in life, the weakness in individuals with congenital DM-1 derives from maldevelopment of skeletal muscle rather than muscle degeneration. Later in life, these individuals are also subject to the added effects of a wasting process similar to classical DM-1. However, it is difficult to determine which of these phenomena are mainly to blame for weakness in a particular patient. Furthermore, more than 75% of patients with congenital DM-1 have mental retardation.
- Prior treatment with glucocorticoids, anabolic steroids, testosterone, growth hormone, or IGF-I within 1 year of entry; or any investigational agent within 60 days of entry.
- Any history of malignancy except for surgically cured skin cancer or pilomatricoma (benign tumor of the hair follicle that is associated with DM-1).
- Women of childbearing potential who are not using effective birth control; women who are pregnant or lactating.
- Medical illness which would prevent assessment of muscle strength or function. This exclusion would include individuals with orthopedic, cardiac, or pulmonary disorders which preclude proper positioning on the myometry testing table, or restrict their ability to tolerate repeated maximum muscle contractions.
- Known allergy to tetracycline.
Show all 18Show fewer
- Diaphragmatic weakness such that patients are unable to tolerate supine position, or swallowing impairment such that patients are unable to maintain nutrition without use of gastrostomy.
- Symptomatic liver or kidney disease, insulin requiring diabetes or type 2 diabetes requiring oral anti-diabetic agents.
- Untreated thyroid disease (hypo or hyperthyroidism)
- Major psychiatric illness (major depression, bipolar disorder, or schizophrenia) within twelve months of entry.
- History of non-compliance with other therapies.
- Drug or alcohol abuse within 12 months of enrollment.
- In men, evidence of a mass lesion on clinical examination by their primary care physician within twelve months prior to entry into the study (specifically prostate or testicular mass on clinical exam or other signs of mass lesion) or evidence of mass lesion on chest x-ray. In men 50 years of age or older, prostate specific antigen (PSA) elevation above normal.
- In women, evidence for mass lesion on clinical examination by their primary care physician or gynecologist (specifically breast \& pelvic exam) within 12 months of entry into the study or evidence of mass lesion on chest x-ray. In women 40 years of age or older, evidence of mass lesion on mammogram. Women with Gail Scores > 1.7 will be excluded due to their increased risk of developing cancer.
- Atrial fibrillation/flutter; 2nd or 3rd degree heart block without pacemaker treatment
- Weight greater than 100 kilograms(kg).
- Body Mass Index greater than 30.
- History of bleeding diathesis or use of anticoagulant medications. Patients taking nonsteroidal anti-inflammatory agents will be asked to discontinue these medications 3 days prior to muscle biopsy.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Rochester Medical Center
Rochester, New York, 14642
About this study
Official title: Effects of SomatoKine (Iplex) (rhIGF-I/rhIGFBP-3) in Myotonic Dystrophy Type 1 (DM1)
The aim of this study is to investigate the safety and feasibility of daily subcutaneous injections of recombinant IGF1 complexed with IGF binding protein 3 (SomatoKine-INSMED) as a treatment for muscle wasting and weakness in myotonic dystrophy type 1.
- Conditions
- Myotonic Dystrophy
- Being tested
- SomatoKine/IPLEX
- Medical subject headings
- Myotonic Dystrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SomatoKine/IPLEXDrug
Cohort 1: self-administered subcuteanous injections of 0.5 mg/kg/day of iPlex for 8 weeks, followed by 1.0 mg/kg/day of iPlex for 16 weeks.
Cohort 2: consecutive 8 week treatments of 0.5, 1.0, and 2.0 mg/kg/day of iPlex for a total of 24 weeks by self-administered subcuteanous injection.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1- Two Escalating Doses of Iplex | Experimental | 0.5 and 1.0 mg/kg/day Receives: Drug: SomatoKine/IPLEX |
| Cohort 2 - Three Escalating Doses of Iplex | Active comparator | 0.5, 1.0, and 2.0 mg/kg/day Receives: Drug: SomatoKine/IPLEX |
What is being measured
The primary outcome is the main question the study is designed to answer.
The Number of Study Participants Who Safely Tolerated Somatokine
Safety and tolerability was measured via interval laboratory studies,electrocardiograms, echocardiograms, ultrasounds of the abdomen and pelvis, dual energy x-ray absorptiometry (DEXA) studies, chest and neck x-rays, and serial physical examinations. The participants had six inpatient evaluations at the University of Rochester General Clinical Research Center (Weeks 0, 8, 16, 24, 28, and 40) and nine outpatient evaluations. Patients also completed side effects diaries to record any adverse events in the interval time between inpatient and outpatient evaluations.
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Oct 2005 (estimated)
- Study startNov 2005
- Primary completionMay 2008
- Study completionMay 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Myotonic DystrophyStudies of the same condition, open and under way.
- More trials run by University of RochesterThe sponsor's other registered studies.
- 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
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 17 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Rochester
- Sponsor type
- University, hospital or other
- Collaborators
- National Institute of Neurological Disorders and Stroke (NINDS), Imsmed Incorporated
- Responsible party
- Principal investigator, Richard T Moxley, University of Rochester
- Investigators
- Richard T. Moxley, III, M.D., Principal investigator, University of Rochester
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00233519
- Sponsor's protocol ID
- 5 U54 NS048843-03 A
- Other IDs
- 5U54NS048843-03
- Status last verified
- Jun 2012
- First submitted
- 3 Oct 2005
- Last updated
- 25 Jun 2012
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Heatwole CR, Eichinger KJ, Friedman DI, Hilbert JE, Jackson CE, Logigian EL, Martens WB, McDermott MP, Pandya SK, Quinn C, Smirnow AM, Thornton CA, Moxley RT 3rd. Open-label trial of recombinant human insulin-like growth factor 1/recombinant human insulin-like growth factor binding protein 3 in myotonic dystrophy type 1. Arch Neurol. 2011 Jan;68(1):37-44. doi: 10.1001/archneurol.2010.227. Epub 2010 Sep 13.
PubMed 20837825
Study at a glance
From the registry record, last updated by the sponsor on 25 Jun 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effects of SomatoKine (Iplex) (rhIGF-I/rhIGFBP-3) in Myotonic Dystrophy Type 1 (DM1)
- ClinicalTrials.gov ID
- NCT00233519
- Acronym / sponsor ID
- 5 U54 NS048843-03 A
- Conditions
- Myotonic Dystrophy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 17 participants (actual)
- Lead sponsor
- University of RochesterUniversity, hospital or other
- Collaborators
- National Institute of Neurological Disorders and Stroke (NINDS), Imsmed Incorporated
- Study start
- Nov 2005
- Primary completion
- May 2008 (actual)
- Study completion
- May 2008 (actual)
- First posted
- 5 Oct 2005 (estimated)
- Results
- Posted 1 Feb 2010
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The aim of this study is to investigate the safety and feasibility of daily subcutaneous injections of recombinant IGF1 complexed with IGF binding protein 3 (SomatoKine-INSMED) as a treatment for muscle wasting and weakness in myotonic dystrophy type 1.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort 1- Two Escalating Doses of Iplex | Experimental | Drug: SomatoKine/IPLEX0.5 and 1.0 mg/kg/day |
| Cohort 2 - Three Escalating Doses of Iplex | Active comparator | Drug: SomatoKine/IPLEX0.5, 1.0, and 2.0 mg/kg/day |
Who may qualify
- Ages
- 21 to 60 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (9 listed)
- A clinical diagnosis of DM-1 according to accepted clinical research criteria.23 The clinical research criteria require each of the following: (1) clinically evident myotonia; (2) muscle weakness in a characteristic distribution (distal predominant); and (3) similar findings in a first degree relative.
- Age 21 to 60 years (inclusive).
- Ability to walk 30 feet without assistance (cane and leg bracing is permitted).
- Weakness of sufficient severity to justify treatment and provide a reasonable opportunity to observe a therapeutic effect. At the eligibility evaluation, eligible patients must show both of the following:
- For patients that are not within driving distance to Rochester, a local health care provider in their area must be able to complete their home visits.
Key exclusion criteria (18 listed)
- Congenital DM-1. Congenital disease constitutes ~10% of all cases of DM-1. Early in life, the weakness in individuals with congenital DM-1 derives from maldevelopment of skeletal muscle rather than muscle degeneration. Later in life, these individuals are also subject to the added effects of a wasting process similar to classical DM-1. However, it is difficult to determine which of these phenomena are mainly to blame for weakness in a particular patient. Furthermore, more than 75% of patients with congenital DM-1 have mental retardation.
- Prior treatment with glucocorticoids, anabolic steroids, testosterone, growth hormone, or IGF-I within 1 year of entry; or any investigational agent within 60 days of entry.
- Any history of malignancy except for surgically cured skin cancer or pilomatricoma (benign tumor of the hair follicle that is associated with DM-1).
- Women of childbearing potential who are not using effective birth control; women who are pregnant or lactating.
- Medical illness which would prevent assessment of muscle strength or function. This exclusion would include individuals with orthopedic, cardiac, or pulmonary disorders which preclude proper positioning on the myometry testing table, or restrict their ability to tolerate repeated maximum muscle contractions.
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 |
|---|---|---|
| SomatoKine/IPLEXCohort 1: self-administered subcuteanous injections of 0.5 mg/kg/day of iPlex for 8 weeks, followed by 1.0 mg/kg/day of iPlex for 16 weeks. Cohort 2: consecutive 8 week treatments of 0.5, 1.0, and 2.0 mg/kg/day of iPlex for a total of 24 weeks by self-administered subcuteanous injection. | Drug | Cohort 1- Two Escalating Doses of Iplex, Cohort 2 - Three Escalating Doses of Iplex |
Primary outcome
The Number of Study Participants Who Safely Tolerated Somatokine
Time frame: 24 weeks
Safety and tolerability was measured via interval laboratory studies,electrocardiograms, echocardiograms, ultrasounds of the abdomen and pelvis, dual energy x-ray absorptiometry (DEXA) studies, chest and neck x-rays, and serial physical examinations. The participants had six inpatient evaluations at the University of Rochester General Clinical Research Center (Weeks 0, 8, 16, 24, 28, and 40) and nine outpatient evaluations. Patients also completed side effects diaries to record any adverse events in the interval time between inpatient and outpatient evaluations.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Jun 2012.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 20837825Heatwole CR, Eichinger KJ, Friedman DI, Hilbert JE, Jackson CE, Logigian EL, Martens WB, McDermott MP, Pandya SK, Quinn C, Smirnow AM, Thornton CA, Moxley RT 3rd. Open-label trial of recombinant human insulin-like growth factor 1/recombinant human insulin-like growth factor binding protein 3 in myotonic dystrophy type 1. Arch Neurol. 2011 Jan;68(1):37-44. doi: 10.1001/archneurol.2010.227. Epub 2010 Sep 13.
- Webpage for the National Registry of Myotonic Dystrophy and Facioscapulohumeral Dystrophy patients and their family members.Link provided by the sponsor.
Related trials for Myotonic Dystrophy
RecruitingPhase 3
Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1
Myotonic Dystrophy Type 1 (DM1), DM1
RecruitingObservational study
DMCRN-02-001: Assessing Pediatric Endpoints in DM1
Congenital Myotonic Dystrophy, CDM
RecruitingObservational study
Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)
Myotonic Dystrophy 1, DM1
RecruitingObservational study
The Spanish National Registry for Myotonic Dystrophy Type 1
Myotonic Dystrophy 1, DM1
Enrolling by invitationObservational study
Personalized Disease Modeling for Rare Genetic Myopathies
Congenital Myopathies, 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.
Richard T. Moxley, III, M.D. principal investigator
University of Rochester
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 SomatoKine/IPLEX in people with Myotonic Dystrophy, run by University of Rochester 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 9 inclusion and 18 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 25 Jun 2012. 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?
Yes. Results are posted in the registry (first posted 1 Feb 2010). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
The Number of Study Participants Who Safely Tolerated Somatokine (time frame: 24 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00233519, which also lists 1 linked publication.
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 Jun 2012.