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NCT ID: NCT03773770 Available - Clinical trials for Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

Expanded Access to Triheptanoin

Start date: n/a
Phase:
Study type: Expanded Access

Expanded access may be provided for qualified patients who have limited treatment options and are not eligible for a clinical trial.

NCT ID: NCT03775174 Available - Clinical trials for Mucopolysaccharidosis VII

Expanded Access to Mepsevii

Start date: n/a
Phase:
Study type: Expanded Access

Individual patient expanded access requests may be considered for patients who have no other treatment options

NCT ID: NCT03775187 Available - Clinical trials for X-linked Hypophosphatemia

Expanded Access to Burosumab

Start date: n/a
Phase:
Study type: Expanded Access

Individual patient expanded access requests may be considered for patients who have no other treatment options

NCT ID: NCT03778424 Available - Clinical trials for Lennox Gastaut Syndrome

An Extended Access Program (EAP) for Participants Who Have Completed Rufinamide Study E2080-G000-303

Start date: n/a
Phase:
Study type: Expanded Access

This is an extended access study for participants who have completed Rufinamide Study E2080-G000-303 to continue to have access to rufinamide until it becomes commercially available in Poland or until no participants remain in the EAP.

NCT ID: NCT03786900 Available - Clinical trials for Recurrent Clostridium Difficile Infection

Fecal Microbiota Transplantation (FMT): PRIM-DJ2727

Start date: n/a
Phase:
Study type: Expanded Access

The Fecal Microbiota Transplantation (FMT) product PRIM-DJ2727 is prepared from human stool from a healthy, screened donor. Requestors will contact the study team about the product (PRIM-DJ2727) by email, visit, or phone call. A screening list for donors will be provided to make sure that the list fits the requestor's requirements. A basic fee will be requested to recover the cost of making the product. After an agreement is made, a contract will be signed between the 2 parties. A week before the treatment, requestors will contact the study team for possible FMT product delivery. Delivery method will be confirmed for delivery by personnel (within 10 minutes driving distance) or by using FedEx services. Each delivered product will have an approved delivery form signed and dated by both the person who prepared the delivery and the person who received the package.

NCT ID: NCT03863119 Available - Clinical trials for Duchenne Muscular Dystrophy

Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy

Start date: n/a
Phase:
Study type: Expanded Access

The intent of this protocol is to provide continued access to vamorolone for subjects in the United States who Have Completed the VBP15-LTE, VBP15- 004, or VBP15-006 protocols (and are thereby ineligible to enroll in another trial of vamorolone therapy), during the time a new drug application for vamorolone is under preparation and review.

NCT ID: NCT03865836 Available - Pompe Disease Clinical Trials

Expanded Access for ATB200/AT2221 for the Treatment of Pompe Disease

Start date: n/a
Phase:
Study type: Expanded Access

This is an expanded access program (EAP) for eligible participants designed to provide access to ATB200/AT2221.

NCT ID: NCT03876964 Available - Clinical trials for Aortic Valve Stenosis

J-Valve Compassionate Use

Start date: n/a
Phase:
Study type: Expanded Access

J-Valve TF Compassionate Use cases approved on a case by case basis by the FDA

NCT ID: NCT03886506 Available - Clinical trials for Critical Limb Ischemia (CLI)

Expanded Access Treatment of Subjects With Critical Limb Ischemia (CLI) With Minor Tissue Loss Who Are Unsuitable for Revascularization

Start date: n/a
Phase:
Study type: Expanded Access

A Phase III study of PLX-PAD for CLI patients with minor tissue loss who are unsuitable for revascularization has been initiated (PLX-CLI-03, PACE study). In parallel, this expanded access program (EAP) will be conducted to allow the treatment of patients who are ineligible to be enrolled in the PACE study. The EAP treatment is administered in addition to standard of care of the subjects.PLX-PAD 300×106 cells in a mixture containing 10% DMSO, 5% human serum albumin and Plasma-Lyte, will be administered via 30 IM injections (0.5 mL each) delivered into the leg twice,at 8 weeks interval. The locations of injections of the PLX-PAD are detailed in Appendix 1. Antihistamine treatment should be given at least 1 hour and no more than 1.5 hours prior to PLXPAD administration to ensure coverage for 24 hours, and as long as necessary post PLX-PAD treatment. Consider treatment with second generation H1 inhibitors such as Cetirizine 10 mg once per day.Subjects will be followed-up until 12 months after the 2nd treatment according to the schedule of routine medical visits at the medical institutions. In addition to this routine follow-up, a phone call will be made 12 months after 2nd treatment to inquire on the occurrence of subsequent intervention, amputation, or death.

NCT ID: NCT03905499 Available - Clinical trials for Pseudoparalysis Due to Massive Rotator Cuff Tear

Robot Assisted Physical Therapy for Pseudoparalysis in Massive Rotator Cuff Tear

Start date: n/a
Phase:
Study type: Expanded Access

The primary objective of this study is to assess the efficacy of a robot assisted therapy in a clinical setting and its potential benefit for patients with rotator cuff tear associated with pseudoparalysis. eighty patients with clinical signs of pseudoparalysis will be recruited through doctors and physical therapists based on eligibility criteria. After completing the baseline assessments, patients will be randomized into the robot-assisted physical therapy intervention or control comparison group. Enrolled patients will conduct a specific trainings program with the MJS Tecnobody (Multi Joint System 614P Tecnobody Srl, IT) three times a week over a period of six weeks. Feasibility assessment will be performed six and twelve weeks after baseline and secondary outcomes (Oxford shoulder score, subjective shoulder value, pain level during activity of daily living, active shoulder flexion and abduction and quality of life EQ-5D-5L) will be assessed at baseline, six and twelve weeks post intervention.